Trial Outcomes & Findings for Registry Study of Revcovi Treatment in Patients With ADA-SCID (NCT NCT03878069)
NCT ID: NCT03878069
Last Updated: 2024-11-15
Results Overview
A deficiency in the ADA enzyme results in a build-up of dAXP nucleotides, which are toxic to lymphocytes and lead to impairment of immune function. A detoxified level of dAXP concentration is defined as a trough value of 0.02 millimoles per liter (mmol/L) or lower. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Recruitment status
COMPLETED
Target enrollment
32 participants
Primary outcome timeframe
From enrollment to end of treatment up to Month 24
Results posted on
2024-11-15
Participant Flow
Four of the participants had taken part in an earlier trial of Revcovi, study STP-2279-002, and were invited to continue in the registry study. The remaining participants were invited to enroll by their treating physicians.
Participant milestones
| Measure |
Adagen-naive
Subjects starting on ERT for the first time
|
Adagen-transitioning
Subjects switching from Adagen to Revcovi
|
STP-2279-002 Participant
Subjects who had taken part in an earlier Phase III trial of Revcovi
|
|---|---|---|---|
|
Overall Study
STARTED
|
7
|
21
|
4
|
|
Overall Study
COMPLETED
|
7
|
20
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
0
|
Reasons for withdrawal
| Measure |
Adagen-naive
Subjects starting on ERT for the first time
|
Adagen-transitioning
Subjects switching from Adagen to Revcovi
|
STP-2279-002 Participant
Subjects who had taken part in an earlier Phase III trial of Revcovi
|
|---|---|---|---|
|
Overall Study
Death
|
0
|
1
|
0
|
Baseline Characteristics
Registry Study of Revcovi Treatment in Patients With ADA-SCID
Baseline characteristics by cohort
| Measure |
Adagen-naive
n=7 Participants
Subjects starting on ERT for the first time
|
Adagen-transitioning
n=21 Participants
Subjects switching from Adagen to Revcovi
|
STP-2279-002 Participant
n=4 Participants
Subjects who had taken part in an earlier Phase III trial of Revcovi
|
Total
n=32 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
18 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
14 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
20 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
12 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
7 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
25 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
9 Participants
n=7 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
18 Participants
n=7 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Number of subjects at or below the toxicity threshold for dAXP concentration
|
0 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
17 Participants
n=7 Participants
|
|
Number of subjects meeting the optimal threshold for ADA activity
|
6 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
15 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: From enrollment to end of treatment up to Month 24Population: The As-Treated (AT) population, which included all subjects who received at least one dose of Revcovi.
A deficiency in the ADA enzyme results in a build-up of dAXP nucleotides, which are toxic to lymphocytes and lead to impairment of immune function. A detoxified level of dAXP concentration is defined as a trough value of 0.02 millimoles per liter (mmol/L) or lower. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Outcome measures
| Measure |
Adagen-naive
n=7 Participants
Subjects starting on ERT for the first time
|
Adagen-transitioning
n=21 Participants
Subjects switching from Adagen to Revcovi
|
STP-2279-002 Participant
n=4 Participants
Subjects who had taken part in an earlier Phase III trial of Revcovi
|
|---|---|---|---|
|
Number of Subjects Meeting the Toxicity Threshold for Deoxyadenosine Nucleotide (dAXP) Concentration at the Last Measurement
|
6 Participants
|
17 Participants
|
4 Participants
|
PRIMARY outcome
Timeframe: From enrollment to end of treatment up to Month 24Population: The As-Treated (AT) population, which included all subjects who received at least one dose of Revcovi.
An optimal level of ADA plasma activity is defined as a trough value of 30 millimoles per hour per liter (mmol/h/L) or greater. Data are presented as the number of subjects who met the targeted threshold at the final measurement, whether that took place at Month 24 or earlier.
Outcome measures
| Measure |
Adagen-naive
n=7 Participants
Subjects starting on ERT for the first time
|
Adagen-transitioning
n=21 Participants
Subjects switching from Adagen to Revcovi
|
STP-2279-002 Participant
n=4 Participants
Subjects who had taken part in an earlier Phase III trial of Revcovi
|
|---|---|---|---|
|
Number of Subjects Meeting the Optimal Threshold for ADA Activity at the Last Measurement
|
7 Participants
|
14 Participants
|
4 Participants
|
PRIMARY outcome
Timeframe: From enrollment to end of treatment up to Month 24Population: The As-Treated (AT) population, which included all subjects who received at least one dose of Revcovi.
The number of subjects reporting adverse events (AEs). Due to the nature of ADA-SCID, only AEs deemed to be at least possibly related to Revcovi treatment were documented.
Outcome measures
| Measure |
Adagen-naive
n=7 Participants
Subjects starting on ERT for the first time
|
Adagen-transitioning
n=21 Participants
Subjects switching from Adagen to Revcovi
|
STP-2279-002 Participant
n=4 Participants
Subjects who had taken part in an earlier Phase III trial of Revcovi
|
|---|---|---|---|
|
Safety of Revcovi
|
3 Participants
|
1 Participants
|
0 Participants
|
Adverse Events
Patients With ADA-SCID in Need of ERT Treatment
Serious events: 0 serious events
Other events: 4 other events
Deaths: 1 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Patients With ADA-SCID in Need of ERT Treatment
n=32 participants at risk
All participants will receive Revcovi and will be monitored for adverse events (AEs) throughout their participation in the study. Only AEs deemed at least possibly related to Revcovi treatment will be documented.
|
|---|---|
|
Blood and lymphatic system disorders
Thrombocytosis
|
6.2%
2/32 • Number of events 2 • At any time during the study. Duration of participation was a minimum of 24 months, or until undergoing successful treatment with stem cell transplant or hematopoietic stem cell gene therapy.
For mortality, all incidences are reported. For non-fatal serious adverse events (SAEs) and non-serious adverse events, due to the nature of the disease, sites were asked to report only those events deemed to be at least possibly related to Revcovi treatment, along with clinically meaningful laboratory abnormalities.
|
|
Blood and lymphatic system disorders
Neutropenia
|
3.1%
1/32 • Number of events 1 • At any time during the study. Duration of participation was a minimum of 24 months, or until undergoing successful treatment with stem cell transplant or hematopoietic stem cell gene therapy.
For mortality, all incidences are reported. For non-fatal serious adverse events (SAEs) and non-serious adverse events, due to the nature of the disease, sites were asked to report only those events deemed to be at least possibly related to Revcovi treatment, along with clinically meaningful laboratory abnormalities.
|
|
General disorders
Injection site erythema
|
3.1%
1/32 • Number of events 1 • At any time during the study. Duration of participation was a minimum of 24 months, or until undergoing successful treatment with stem cell transplant or hematopoietic stem cell gene therapy.
For mortality, all incidences are reported. For non-fatal serious adverse events (SAEs) and non-serious adverse events, due to the nature of the disease, sites were asked to report only those events deemed to be at least possibly related to Revcovi treatment, along with clinically meaningful laboratory abnormalities.
|
|
Injury, poisoning and procedural complications
Exposure via skin contact
|
3.1%
1/32 • Number of events 1 • At any time during the study. Duration of participation was a minimum of 24 months, or until undergoing successful treatment with stem cell transplant or hematopoietic stem cell gene therapy.
For mortality, all incidences are reported. For non-fatal serious adverse events (SAEs) and non-serious adverse events, due to the nature of the disease, sites were asked to report only those events deemed to be at least possibly related to Revcovi treatment, along with clinically meaningful laboratory abnormalities.
|
|
Investigations
Weight increased
|
3.1%
1/32 • Number of events 1 • At any time during the study. Duration of participation was a minimum of 24 months, or until undergoing successful treatment with stem cell transplant or hematopoietic stem cell gene therapy.
For mortality, all incidences are reported. For non-fatal serious adverse events (SAEs) and non-serious adverse events, due to the nature of the disease, sites were asked to report only those events deemed to be at least possibly related to Revcovi treatment, along with clinically meaningful laboratory abnormalities.
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
3.1%
1/32 • Number of events 1 • At any time during the study. Duration of participation was a minimum of 24 months, or until undergoing successful treatment with stem cell transplant or hematopoietic stem cell gene therapy.
For mortality, all incidences are reported. For non-fatal serious adverse events (SAEs) and non-serious adverse events, due to the nature of the disease, sites were asked to report only those events deemed to be at least possibly related to Revcovi treatment, along with clinically meaningful laboratory abnormalities.
|
Additional Information
Anna Rozova, MD, Clinical Program Leader
Chiesi Canada Corp.
Phone: 1-800-854-3534
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60