Trial Outcomes & Findings for Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease) (NCT NCT04209634)
NCT ID: NCT04209634
Last Updated: 2025-08-29
Results Overview
Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of \<2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
10 participants
Primary outcome timeframe
At Week 24
Results posted on
2025-08-29
Participant Flow
A total of 11 participants were screened for this study. Of these, 1 participant did not meet inclusion/exclusion criteria, and 10 were assigned to study treatment.
Participant milestones
| Measure |
Pozelimab Injection
Participants received a single loading dose of pozelimab 30 milligrams per kilogram (mg/kg) intravenous (IV) injection on day 1, followed by weekly subcutaneous (SC) injection as maintenance dosing of 125 mg for body weight less than (\<) 10 kilograms (kg), 200 mg for greater than or equal to (\>=) 10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)
Baseline characteristics by cohort
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
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|---|---|
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Age, Continuous
|
9.3 years
STANDARD_DEVIATION 4.88 • n=39 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: At Week 24Population: The FAS included all enrolled participants who received the study drug.
Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of \<2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
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|---|---|
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Percentage of Participants With Active Disease at Baseline Who Achieved Normalization of Serum Albumin and Improvement in Prespecified Clinical Outcomes at Week 24
|
100 percentage of participants
Interval 74.1 to 100.0
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SECONDARY outcome
Timeframe: From start of study drug administration up to approximately 144 weeksPopulation: The safety analysis set included all enrolled participants who received the study drug.
TEAEs are defined as AEs that developed or worsened during the on-treatment period. The on-treatment period is defined as the time from first dose of investigational product up to 21 weeks after the last dose of investigational product. Severity of TEAEs was graded according to the following scale: Mild: Does not interfere in a significant manner with the patient's normal functioning level, Moderate: Produces some impairment of functioning but is not hazardous to health and Severe: Produces significant impairment of functioning or incapacitation and is a definite hazard to the participants health.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
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|---|---|
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Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs
Participants with TEAEs
|
10 Participants
|
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Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs
Participants with mild severity
|
3 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs
Participants with moderate severity
|
5 Participants
|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Severity of TEAEs
Participants with severe severity
|
2 Participants
|
SECONDARY outcome
Timeframe: At Week 24Population: FAS included all enrolled participants who received the study drug.
Improvement in most bothersome sign/symptom determined using semi-structured concept elicitation interview, from 'core' clinical endpoints of frequency of bowel movements, peripheral edema, facial edema, abdominal pain frequency, nausea, vomiting, stool consistency.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
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|---|---|
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Number of Participants With Improvement in Most Bothersome Signs and Symptoms at Week 24
|
10 Participants
|
SECONDARY outcome
Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24Population: FAS included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
Daily bowel movements captured by e-diary. The number of bowel movements per day was calculated each week of the study. It was based on a 1-week average and calculated as the sum of the number of bowel movements in a given week divided by the number of days with non-missing bowel movement frequency data. If more than 3 days of bowel movement data was missing in a given week, bowel movement frequency data was considered missing for that week.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
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|---|---|
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Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Baseline
|
1.64 Bowel movements per day
Interval 0.5 to 3.4
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 1
|
1.13 Bowel movements per day
Interval 0.3 to 3.0
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 2
|
1.36 Bowel movements per day
Interval 0.7 to 3.4
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 3
|
1.43 Bowel movements per day
Interval 0.7 to 2.4
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 4
|
1.50 Bowel movements per day
Interval 0.8 to 2.6
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 5
|
1.57 Bowel movements per day
Interval 0.5 to 2.3
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 6
|
1.21 Bowel movements per day
Interval 0.6 to 3.0
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 7
|
1.21 Bowel movements per day
Interval 0.3 to 2.5
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 8
|
1.07 Bowel movements per day
Interval 0.2 to 2.3
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 9
|
1.00 Bowel movements per day
Interval 0.3 to 2.0
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 10
|
1.00 Bowel movements per day
Interval 0.5 to 1.7
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 11
|
1.00 Bowel movements per day
Interval 0.2 to 2.0
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 12
|
0.79 Bowel movements per day
Interval 0.0 to 1.9
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 13
|
0.93 Bowel movements per day
Interval 0.1 to 2.3
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 14
|
0.93 Bowel movements per day
Interval 0.3 to 2.1
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 15
|
1.07 Bowel movements per day
Interval 0.3 to 2.3
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 16
|
1.10 Bowel movements per day
Interval 0.4 to 2.0
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 17
|
1.13 Bowel movements per day
Interval 0.3 to 2.0
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 18
|
1.00 Bowel movements per day
Interval 0.0 to 1.9
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 19
|
1.00 Bowel movements per day
Interval 0.3 to 1.9
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 20
|
0.83 Bowel movements per day
Interval 0.2 to 2.1
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 21
|
1.00 Bowel movements per day
Interval 0.5 to 1.7
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 22
|
0.83 Bowel movements per day
Interval 0.4 to 1.7
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 23
|
0.86 Bowel movements per day
Interval 0.5 to 1.8
|
|
Number of Bowel Movements Per Day Based on a 1-week Average up to Week 24
Week 24
|
0.90 Bowel movements per day
Interval 0.3 to 2.0
|
SECONDARY outcome
Timeframe: Baseline, Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11,12,13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23 and 24Population: FAS included all enrolled participants who received any study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
The number of days per week with \>=1 loose/watery bowel movement, is calculated each week of the study as the sum of the number of days with \>=1 loose/watery bowel movement in a given week divided by the number of days with non-missing stool consistency data and then multiplied by 7, is presented. If more than 3 days of stool consistency data was missing in a given week, stool consistency data was considered missing for that week.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
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|---|---|
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Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 15
|
1.00 Days per week
Interval 0.0 to 7.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 16
|
0.00 Days per week
Interval 0.0 to 5.8
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 17
|
1.28 Days per week
Interval 0.0 to 5.6
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 18
|
0.50 Days per week
Interval 0.0 to 7.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 19
|
0.00 Days per week
Interval 0.0 to 5.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 20
|
0.00 Days per week
Interval 0.0 to 5.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 21
|
0.00 Days per week
Interval 0.0 to 6.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 22
|
0.00 Days per week
Interval 0.0 to 6.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 23
|
0.00 Days per week
Interval 0.0 to 6.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 24
|
0.00 Days per week
Interval 0.0 to 5.6
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Baseline
|
2.00 Days per week
Interval 0.0 to 7.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 1
|
0.50 Days per week
Interval 0.0 to 5.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 2
|
0.00 Days per week
Interval 0.0 to 2.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 3
|
0.00 Days per week
Interval 0.0 to 3.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 4
|
0.00 Days per week
Interval 0.0 to 4.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 5
|
0.00 Days per week
Interval 0.0 to 5.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 6
|
1.20 Days per week
Interval 0.0 to 5.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 7
|
0.50 Days per week
Interval 0.0 to 2.8
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 8
|
0.50 Days per week
Interval 0.0 to 3.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 9
|
0.50 Days per week
Interval 0.0 to 3.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 10
|
0.00 Days per week
Interval 0.0 to 4.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 11
|
1.00 Days per week
Interval 0.0 to 2.3
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 12
|
0.00 Days per week
Interval 0.0 to 2.3
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 13
|
0.00 Days per week
Interval 0.0 to 4.0
|
|
Number of Days Per Week With >=1 Bowel Movement of Loose/Watery Stool Consistency at Week 24
Week 14
|
1.58 Days per week
Interval 0.0 to 4.7
|
SECONDARY outcome
Timeframe: Baseline up to Week 24Population: FAS included all enrolled participants who received any study drug.
The measurement of abdominal ascites (excess abdominal fluid) was based on abdominal circumference. Abdominal circumference was measured regardless of the physician's assessment of the presence or absence of ascites.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Number of Participants With Abdominal Ascites at Week 24
|
1 Participants
|
SECONDARY outcome
Timeframe: Baseline, Day 2, Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22 and 24Population: FAS included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
Blood samples were collected from participants at defined time points for the assessment of albumin. Absolute value of albumin at specified timepoints was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Baseline
|
2.160 g/dL
Standard Deviation 0.5296
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Day 2
|
2.267 g/dL
Standard Deviation 0.4000
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 1
|
3.311 g/dL
Standard Deviation 0.4485
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 2
|
3.589 g/dL
Standard Deviation 0.4428
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 3
|
4.025 g/dL
Standard Deviation 0.3955
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 4
|
4.167 g/dL
Standard Deviation 0.3841
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 6
|
4.411 g/dL
Standard Deviation 0.7026
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 8
|
4.188 g/dL
Standard Deviation 0.3137
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 10
|
4.322 g/dL
Standard Deviation 0.2279
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 12
|
4.288 g/dL
Standard Deviation 0.2748
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 14
|
4.363 g/dL
Standard Deviation 0.2615
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 16
|
4.367 g/dL
Standard Deviation 0.3674
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 18
|
4.344 g/dL
Standard Deviation 0.2555
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 20
|
4.449 g/dL
Standard Deviation 0.2539
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 22
|
4.422 g/dL
Standard Deviation 0.2906
|
|
Absolute Value of Albumin at Specified Timepoints up to Week 24
Week 24
|
4.544 g/dL
Standard Deviation 0.2603
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
Blood samples were collected from participants at defined time points for the assessment of protein and IgG. Absolutes values of protein and IgG measured as g/L at baseline and Week 24 was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24
Protein at Baseline
|
37.50 g/L
Standard Deviation 6.996
|
|
Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24
Protein at Week 24
|
72.22 g/L
Standard Deviation 4.969
|
|
Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24
IgG at Baseline
|
2.183 g/L
Standard Deviation 0.9103
|
|
Absolute Values of Protein, and Immunoglobulin G (IgG) at Baseline and Week 24
IgG at Week 24
|
11.301 g/L
Standard Deviation 2.0503
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
Blood samples were collected from participants at defined time points for the assessment of Ig, IgM and IgA. Absolute value of Ig, IgM and IgA measured as mg/dL at baseline and Week 24 was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24
Ig at Baseline
|
318.2 mg/dL
Standard Deviation 113.36
|
|
Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24
Ig at Week 24
|
1430.1 mg/dL
Standard Deviation 263.36
|
|
Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24
IgM at Baseline
|
52.9 mg/dL
Standard Deviation 28.36
|
|
Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24
IgM at Week 24
|
180.3 mg/dL
Standard Deviation 74.81
|
|
Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24
IgA at Baseline
|
47.0 mg/dL
Standard Deviation 18.51
|
|
Absolute Values of Immunoglobulin (Ig), Immunoglobulin M (IgM), and Immunoglobulin A (IgA) at Baseline and Week 24
IgA at Week 24
|
119.8 mg/dL
Standard Deviation 52.46
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.
Blood samples were collected from participants at defined time points for the assessment of vitamin B12. Absolute values of vitamin B12 at baseline and Week 24 was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Vitamin B12 at Baseline and Week 24
Baseline
|
158.5 picomoles per liter (pmol/L)
Standard Deviation 52.32
|
|
Absolute Values of Vitamin B12 at Baseline and Week 24
Week 24
|
383.1 picomoles per liter (pmol/L)
Standard Deviation 102.73
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety analysis set included all enrolled participants who received the study drug. Here, "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of unsaturated iron and unsaturated iron binding capacity measured as micromoles per liter (mcmol/L) at baseline and Week 24 was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24
Iron at Baseline
|
3.40 mcmol/L
Standard Deviation 1.478
|
|
Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24
Iron at Week 24
|
7.38 mcmol/L
Standard Deviation 5.036
|
|
Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24
Unsaturated Binding Capacity at Baseline
|
38.24 mcmol/L
Standard Deviation 12.357
|
|
Absolute Values of Iron and Unsaturated Iron Binding Capacity at Baseline and Week 24
Unsaturated Iron Binding Capacity at Week 24
|
73.95 mcmol/L
Standard Deviation 12.049
|
SECONDARY outcome
Timeframe: Baseline up to Week 24Population: Number of participants based on the total in the safety analysis set (SAF)
Absolute Values of Vitamin B9 - Central Lab
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Vitamin B9 up to Week 24
Week 1
|
17.80 nanomoles per liter (nmol/L)
Standard Deviation 15.017
|
|
Absolute Values of Vitamin B9 up to Week 24
Week 4
|
22.07 nanomoles per liter (nmol/L)
Standard Deviation 14.342
|
|
Absolute Values of Vitamin B9 up to Week 24
Week 8
|
17.32 nanomoles per liter (nmol/L)
Standard Deviation 8.042
|
|
Absolute Values of Vitamin B9 up to Week 24
Week 12
|
17.89 nanomoles per liter (nmol/L)
Standard Deviation 9.646
|
|
Absolute Values of Vitamin B9 up to Week 24
Week 16
|
18.01 nanomoles per liter (nmol/L)
Standard Deviation 10.610
|
|
Absolute Values of Vitamin B9 up to Week 24
Week 20
|
19.91 nanomoles per liter (nmol/L)
Standard Deviation 15.436
|
|
Absolute Values of Vitamin B9 up to Week 24
Week 24
|
18.93 nanomoles per liter (nmol/L)
Standard Deviation 6.538
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure.
Blood samples were collected from participants at defined time points for the assessment of iron indices. Absolute values of ferritin was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Ferritin at Baseline and Week 24
Baseline
|
10.0 microgram per liter (mcg/L)
Standard Deviation 5.90
|
|
Absolute Values of Ferritin at Baseline and Week 24
Week 24
|
12.0 microgram per liter (mcg/L)
Standard Deviation 7.86
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: The safety analysis set included all enrolled participants who received the study drug. Here, "overall number of participants analyzed" signifies those participants who were evaluable for this outcome measure and "number analyzed" signifies those participants who were evaluable for specified timepoints in categories.
Blood samples were collected from participants at defined time points for the assessment of magnesium, total cholesterol, and triglycerides. Absolute values of magnesium, total cholesterol, and triglycerides measured as mmol/L at baseline and Week 24 was reported.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24
Magnesium at Baseline
|
0.793 mmol/L
Standard Deviation 0.0381
|
|
Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24
Magnesium at Week 24
|
0.858 mmol/L
Standard Deviation 0.0245
|
|
Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24
Total Cholesterol at Baseline
|
3.819 mmol/L
Standard Deviation 0.8786
|
|
Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24
Total Cholesterol at Week 24
|
3.604 mmol/L
Standard Deviation 0.3118
|
|
Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24
Triglycerides at Baseline
|
2.033 mmol/L
Standard Deviation 0.7946
|
|
Absolute Values of Magnesium, Total Cholesterol, and Triglycerides at Week 24
Triglycerides at Week 24
|
1.094 mmol/L
Standard Deviation 0.3745
|
SECONDARY outcome
Timeframe: Week 12, Week 24Population: The FAS included all enrolled participants who received the study drug.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Alpha-1 Antitrypsin Levels in Stool at Week 12 and Week 24
Change from Baseline to Week 12
|
-527.80 milligrams per deciliter (mg/dL)
Standard Deviation 318.818
|
|
Change From Baseline in Alpha-1 Antitrypsin Levels in Stool at Week 12 and Week 24
Change from Baseline to Week 24
|
-463.71 milligrams per deciliter (mg/dL)
Standard Deviation 204.250
|
SECONDARY outcome
Timeframe: Week 12, Week 24Population: Number of participants based on the total in the safety analysis set (SAF)
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Alpha-1 Antitrypsin Levels in Blood at Week 12 and Week 24
Change from Baseline to Week 12
|
-57.8 mg/dL
Standard Deviation 33.15
|
|
Change From Baseline in Alpha-1 Antitrypsin Levels in Blood at Week 12 and Week 24
Change from Baseline to Week 24
|
-43.4 mg/dL
Standard Deviation 35.89
|
SECONDARY outcome
Timeframe: Weeks 12 to 48; Weeks 12 to 144; Weeks 24 to 48; Weeks 48 to 96; Weeks 96 to 144Population: All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5, and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144.
Measured by normalization of serum albumin, no worsening of facial or peripheral edema, increase in bowel movement, or increase in abdominal pain frequency, no increase in dose of permitted concomitant medication for the treatment of PLE at any time as described in the protocol
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control
Weeks 12 to 48
|
100 percentage of participants
|
|
Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control
Weeks 12 to 144
|
100 percentage of participants
|
|
Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control
Weeks 24 to 48
|
100 percentage of participants
|
|
Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control
Weeks 48 to 96
|
100 percentage of participants
|
|
Percentage of Participants With Active Disease at Baseline Who Maintained Disease Control
Weeks 96 to 144
|
100 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
The physician assessment of facial edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5).
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Physician Assessment of Facial Edema Based on a 5-point Likert Rating Scale
|
-1.7 score on a scale
Standard Deviation 0.87
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
The physician assessment of peripheral edema is based on a 5-point Likert scale ranging from no edema (1) to very severe edema (5).
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Physician Assessment of Peripheral Edema Based on a 5-point Likert Rating Scale
|
-1.7 score on a scale
Standard Deviation 0.71
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure.
Frequency of limitations is assessed on a 5-point Likert response scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse scored and linearly transformed to a 0 to 100 scale, where lower scores indicate more frequent problems with food and drink limitations.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Food and Drink Limitations as Assessed by the PedsQL™ GI Symptom Scales' Food and Drink Limits Sub-scale
|
47.40 score on a scale
Standard Deviation 36.653
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure.
Physical functioning, emotional functioning, social functioning, and school/work/studies functioning is assessed using a 5-point Likert scale, ranging from never a problem (0) to almost always a problem (4). Items were reverse-scored and linearly transformed to a 0 to 100 scale, with higher scores indicating better HRQoL. The total scale score is computed as the sum of all the items over the number of items answered on individual scales. Subscale score is calculated as the sum of the items in the scale divided by the number of items answered in the scale.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales
Week 144 Change from Baseline Total score of PedsQL Core Score
|
26.55 score on a scale
Standard Deviation 17.023
|
|
Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales
Week 144 Change from Baseline Subscale score: Physical functioning
|
14.45 score on a scale
Standard Deviation 39.900
|
|
Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales
Week 144 Change from Baseline Subscale score: Emotional functioning
|
26.3 score on a scale
Standard Deviation 28.88
|
|
Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales
Week 144 Change from Baseline Subscale score: Social functioning
|
30.6 score on a scale
Standard Deviation 20.43
|
|
Change From Baseline in Health-Related Quality of Life (HRQoL) as Assessed by the PedsQL™ Generic Core Scales
Week 144 Change from Baseline Subscale score: School functioning
|
41.43 score on a scale
Standard Deviation 19.518
|
SECONDARY outcome
Timeframe: Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144Population: All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5, and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Number of Participants With Albumin Infusion by 24 Week Periods
Weeks 0 to 24
|
1 Participants
|
|
Number of Participants With Albumin Infusion by 24 Week Periods
Weeks 24 to 48
|
0 Participants
|
|
Number of Participants With Albumin Infusion by 24 Week Periods
Weeks 48 to 72
|
0 Participants
|
|
Number of Participants With Albumin Infusion by 24 Week Periods
Weeks 72 to 96
|
0 Participants
|
|
Number of Participants With Albumin Infusion by 24 Week Periods
Weeks 96 to 120
|
0 Participants
|
|
Number of Participants With Albumin Infusion by 24 Week Periods
Weeks 120 to 144
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline and Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Albumin Values
|
2.400 grams per deciliter (g/dL)
Standard Deviation 0.6103
|
SECONDARY outcome
Timeframe: Baseline and Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Percentage Change From Baseline in Albumin Values
|
127.489 percentage of change
Standard Deviation 81.0666
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Albumin Values
|
15.5 days
Interval 8.0 to 28.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at this time point for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Protein Values
|
37.13 grams per liter (g/L)
Standard Deviation 10.789
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Total Protein
|
22.0 days
Interval 9.0 to 35.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Immunoglobulin (Ig) Values
|
1271.2 mg/dL
Standard Deviation 261.91
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Population: All enrolled participants with available Ig lab reference ranges.
Outcome measures
| Measure |
Pozelimab Injection
n=1 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Ig Values
|
341 days
Interval 341.0 to 341.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in IgG Values
|
10.626 g/L
Standard Deviation 2.2486
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for IgG Values
|
29.0 days
Interval 8.0 to 29.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in IgM Values
|
112.0 mg/dL
Standard Deviation 51.21
|
SECONDARY outcome
Timeframe: Baseline up to Week 44Population: All enrolled participants who received the study drug who were evaluable for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for IgM Values
|
1 days
Interval 1.0 to 9.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in IgA Values
|
96.7 mg/dL
Standard Deviation 28.78
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for IgA Values
|
1 days
Interval 1.0 to 9.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=6 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Vitamin B12 Values
|
138.8 picomoles per liter (pmol/L)
Standard Deviation 56.07
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Vitamin B12 Values
|
8 days
Interval 1.0 to 8.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=6 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Vitamin B9 (Folate) Values
|
2.92 nanomole per liter (nmol/L)
Standard Deviation 10.241
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Vitamin B9 (Folate) Values
|
1 days
Interval 1.0 to 8.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Iron Values
|
7.82 micromole/liter (umol/L)
Standard Deviation 4.091
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Iron Values
|
97.5 days
Interval 1.0 to 337.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Unsaturated Iron Binding Capacity
|
16.20 umol/L
Standard Deviation 13.811
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Unsaturated Iron Binding Capacity
|
1 days
Interval 1.0 to 56.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=6 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Ferritin Values
|
32.0 microgram per liter (ug/L)
Standard Deviation 23.67
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Ferritin Values
|
28 days
Interval 1.0 to 505.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=6 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Magnesium Values
|
0.083 millimoles per liter (mmol/L)
Standard Deviation 0.0606
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Magnesium Values
|
1 days
Interval 1.0 to 2.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=6 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Fasting Cholesterol Values
|
-0.072 mmol/L
Standard Deviation 0.7299
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Fasting Cholesterol Values
|
1 days
Interval 1.0 to 29.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=6 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Fasting Triglycerides Values
|
-0.710 mmol/L
Standard Deviation 0.9086
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Time to First Normalization for Fasting Triglycerides Values
|
4 days
Interval 1.0 to 57.0
|
SECONDARY outcome
Timeframe: Baseline up to Week 144Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Number of Participants Who Used Concomitant Medication
|
10 Participants
|
SECONDARY outcome
Timeframe: Weeks 0 to 24; Weeks 24 to 48; Weeks 48 to 72; Weeks 72 to 96; Weeks 96 to 120; Weeks 120 to 144Population: All enrolled participants who received the study drug. 1 participant was consented to Protocol Amendment 5 and completed the study at week 104. The remaining 9 participants were consented to Protocol Amendment 6 and completed the study at week 144.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Number of Hospitalization Days by 24 Week Period
Weeks 0 to 24
|
0.0 days
Interval 0.0 to 4.0
|
|
Number of Hospitalization Days by 24 Week Period
Weeks 24 to 48
|
0.0 days
Interval 0.0 to 0.0
|
|
Number of Hospitalization Days by 24 Week Period
Weeks 48 to 72
|
0.0 days
Interval 0.0 to 0.0
|
|
Number of Hospitalization Days by 24 Week Period
Weeks 72 to 96
|
0.0 days
Interval 0.0 to 0.0
|
|
Number of Hospitalization Days by 24 Week Period
Weeks 96 to 120
|
0.0 days
Interval 0.0 to 2.0
|
|
Number of Hospitalization Days by 24 Week Period
Weeks 120 to 144
|
0.0 days
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Weight-for-age z-score compares a participant's weight to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Body weight z-score, regardless of magnitude, represents catch-up growth.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Body Weight Z-Score
|
0.924 z-score
Standard Deviation 0.7529
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Height-for-age z-score compares a participant's height to children of the same age and sex from a healthy reference population. A z-score reflects an individual score as compared to a population mean and is expressed in units of standard deviation above (positive values) and below (negative values). Any increase in height z-score, regardless of magnitude, represents catch-up growth.
Outcome measures
| Measure |
Pozelimab Injection
n=8 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Height Z-Score
|
1.079 z-score
Standard Deviation 0.6469
|
SECONDARY outcome
Timeframe: Baseline and Week 144Population: All enrolled participants who received the study drug and consented to Protocol Amendment 6 and who were evaluable at both time points for this outcome measure.
Outcome measures
| Measure |
Pozelimab Injection
n=9 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Change From Baseline in Total Complement Activity Complement Hemolytic Assay (CH50)
|
-241.6 international units/milliliter (IU/mL)
Standard Deviation 39.79
|
SECONDARY outcome
Timeframe: Baseline up to Week 164Population: The Pharmacokinetic Analysis Set (PKAS) includes all treated participants who received any amount of study drug and had at least 1 non-missing total pozelimab measurement following the first dose of study drug. The PKAS is based on the actual treatment received (as treated) rather than as randomized.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Concentrations of Total Pozelimab in Serum
Week 24
|
421 mg/L
Standard Deviation 129
|
|
Concentrations of Total Pozelimab in Serum
Week 36
|
467 mg/L
Standard Deviation 127
|
|
Concentrations of Total Pozelimab in Serum
Week 48
|
517 mg/L
Standard Deviation 142
|
|
Concentrations of Total Pozelimab in Serum
Week 72
|
492 mg/L
Standard Deviation 131
|
|
Concentrations of Total Pozelimab in Serum
Week 96
|
461 mg/L
Standard Deviation 115
|
|
Concentrations of Total Pozelimab in Serum
Week 144
|
430 mg/L
Standard Deviation 134
|
|
Concentrations of Total Pozelimab in Serum
Week 145
|
NA mg/L
Standard Deviation NA
Mean and Standard Deviation (SD) not calculable with n=1
|
|
Concentrations of Total Pozelimab in Serum
Week 146
|
NA mg/L
Standard Deviation NA
Mean and Standard Deviation (SD) not calculable with n=1
|
|
Concentrations of Total Pozelimab in Serum
Week 1
|
182 mg/L
Standard Deviation 68.9
|
|
Concentrations of Total Pozelimab in Serum
Week 2
|
185 mg/L
Standard Deviation 103
|
|
Concentrations of Total Pozelimab in Serum
Week 4
|
227 mg/L
Standard Deviation 110
|
|
Concentrations of Total Pozelimab in Serum
Week 6
|
268 mg/L
Standard Deviation 106
|
|
Concentrations of Total Pozelimab in Serum
Week 8
|
306 mg/L
Standard Deviation 111
|
|
Concentrations of Total Pozelimab in Serum
Week 12
|
341 mg/L
Standard Deviation 115
|
|
Concentrations of Total Pozelimab in Serum
Week 16
|
402 mg/L
Standard Deviation 141
|
|
Concentrations of Total Pozelimab in Serum
Week 20
|
418 mg/L
Standard Deviation 136
|
|
Concentrations of Total Pozelimab in Serum
Week 164
|
410 mg/L
Standard Deviation 104
|
SECONDARY outcome
Timeframe: Baseline up to Week 164Population: The Anti-Drug Antibody Analysis Set (AAS) includes all treated participants who received any amount of study drug and had at least 1 non-missing ADA result following the first dose of study drug. The AAS is based on the actual treatment received (as treated) rather than as randomized.
Outcome measures
| Measure |
Pozelimab Injection
n=10 Participants
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Number of Participants With Treatment-emergent Anti-drug Antibodies (ADA) to Pozelimab
|
0 Participants
|
Adverse Events
Pozelimab Injection
Serious events: 5 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pozelimab Injection
n=10 participants at risk
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Gastrointestinal disorders
Vomiting
|
20.0%
2/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Dehydration
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Abdominal pain
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Head injury
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Urinary tract infection
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
Other adverse events
| Measure |
Pozelimab Injection
n=10 participants at risk
Participants received a single loading dose of pozelimab 30 mg/kg IV injection on day 1, followed by weekly SC injection as maintenance dosing of 125 mg for body weight \<10 kg, 200 mg for \>=10 kg and \<20 kg, 350 mg for \>=20 kg and \<40 kg, 500 mg for \>=40 kg and \<60 kg, and 800 mg for \>=60 kg over the treatment period.
|
|---|---|
|
Infections and infestations
Rhinitis
|
20.0%
2/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Acarodermatitis
|
20.0%
2/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Nasopharyngitis
|
30.0%
3/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Tonsillitis
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Iron deficiency
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
10.0%
1/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Skin and subcutaneous tissue disorders
Alopecia areata
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Abdominal pain
|
40.0%
4/10 • Number of events 5 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Constipation
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Gingival bleeding
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
4/10 • Number of events 4 • From signing of informed consent up to 164 weeks (end of study)
|
|
General disorders
Pyrexia
|
20.0%
2/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Investigations
Hepatic enzyme increased
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Blood and lymphatic system disorders
Anaemia folate deficiency
|
10.0%
1/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Immune system disorders
Immunisation reaction
|
10.0%
1/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Nervous system disorders
Headache
|
20.0%
2/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Renal and urinary disorders
Haematuria
|
10.0%
1/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
Renal and urinary disorders
Proteinuria
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Upper respiratory tract infection
|
30.0%
3/10 • Number of events 6 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
COVID-19
|
20.0%
2/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Coronavirus infection
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Gastroenteritis
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Influenza
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Pneumonia
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Infections and infestations
Viral infection
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Diarrhoea
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Gastrointestinal disorders
Nausea
|
10.0%
1/10 • Number of events 5 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Contusion
|
20.0%
2/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
20.0%
2/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Fall
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Folate deficiency
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Hypoalbuminaemia
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Vitamin B12 deficiency
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Skin and subcutaneous tissue disorders
Chronic spontaneous urticaria
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Skin and subcutaneous tissue disorders
Skin mass
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
General disorders
Injection site erythema
|
10.0%
1/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
|
General disorders
Oedema peripheral
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
General disorders
Xerosis
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Investigations
Activated partial thromboplastin time prolonged
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Investigations
"Blood 1,25-dihydroxycholecalciferol increased"
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Investigations
Blood bilirubin increased
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Investigations
Blood immunoglobulin M decreased
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Investigations
Blood zinc decreased
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
10.0%
1/10 • Number of events 2 • From signing of informed consent up to 164 weeks (end of study)
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Cardiac disorders
Tachycardia
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Eye disorders
Eyelid oedema
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Psychiatric disorders
Irritability
|
10.0%
1/10 • Number of events 1 • From signing of informed consent up to 164 weeks (end of study)
|
|
Renal and urinary disorders
Dysuria
|
10.0%
1/10 • Number of events 3 • From signing of informed consent up to 164 weeks (end of study)
|
Additional Information
Clinical Trials Administrator
Regeneron Pharmaceuticals, Inc.
Phone: 844-734-6643
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.
- Publication restrictions are in place
Restriction type: OTHER