Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of Imsidolimab (ANB019) in the Treatment of Participants With Ichthyosis (NCT NCT04697056)
NCT ID: NCT04697056
Last Updated: 2025-09-16
Results Overview
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling \& percentage of body surface area (BSA) affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 body regions (BR) \[A1: head \& neck (H\&N), A2: upper limbs (UL), A3: trunk (T), A4: lower limbs (LL)\]. Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value(0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6).Total extent was determined using multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-Erythema (E)= A1E x B1 x C1 + A2E x B2 x C2 + A3E x B3 x C3 + A4E x B4 x C4 (score 0 to 24) IASI-Scaling (S)= A1S x B1 X C1 + A2S x B2 x C2 + A3S x B3 x C3 + A4S x B4 x C4 (score 0 to 24) IASI total score= IASI-E + IASI-S score ranged from 0 - 48, higher score indicated worse disease state.
TERMINATED
PHASE2
5 participants
Baseline and Week 16
2025-09-16
Participant Flow
Participants with confirmed diagnosis of ichthyosis by genetic testing of ichthyosis were enrolled into the study.
7 participants were screened for eligibility and 5 participants were randomized into the study.
Participant milestones
| Measure |
Imsidolimab
Participants received a starting dose of 400 milligrams (mg) of imsidolimab on Day 1 followed by 200 mg imsidolimab every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
Placebo
Participants received imsidolimab matching placebo on Day 1 and thereafter every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
|---|---|---|
|
Overall Study
STARTED
|
4
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
4
|
1
|
Reasons for withdrawal
| Measure |
Imsidolimab
Participants received a starting dose of 400 milligrams (mg) of imsidolimab on Day 1 followed by 200 mg imsidolimab every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
Placebo
Participants received imsidolimab matching placebo on Day 1 and thereafter every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
|---|---|---|
|
Overall Study
Termination of the participant participation by the Investigator or Sponsor
|
4
|
1
|
Baseline Characteristics
A Study to Evaluate the Efficacy and Safety of Imsidolimab (ANB019) in the Treatment of Participants With Ichthyosis
Baseline characteristics by cohort
| Measure |
Imsidolimab
n=4 Participants
Participants received a starting dose of 400 mg of imsidolimab on Day 1 followed by 200 mg imsidolimab every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
Placebo
n=1 Participants
Participants received imsidolimab matching placebo on Day 1 and thereafter, every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
39.0 years
STANDARD_DEVIATION 20.15 • n=99 Participants
|
66.0 years
n=107 Participants
|
44.4 years
STANDARD_DEVIATION 21.22 • n=206 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 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
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline and Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling \& percentage of body surface area (BSA) affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 body regions (BR) \[A1: head \& neck (H\&N), A2: upper limbs (UL), A3: trunk (T), A4: lower limbs (LL)\]. Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value(0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6).Total extent was determined using multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-Erythema (E)= A1E x B1 x C1 + A2E x B2 x C2 + A3E x B3 x C3 + A4E x B4 x C4 (score 0 to 24) IASI-Scaling (S)= A1S x B1 X C1 + A2S x B2 x C2 + A3S x B3 x C3 + A4S x B4 x C4 (score 0 to 24) IASI total score= IASI-E + IASI-S score ranged from 0 - 48, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling, \& percentage of BSA affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 BR (A1: H\&N, A2: UL, A3: T, A4: LL). Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value (0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6). Total extent was determined using a multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-E= A1E x B1 x C1 + A2E x B2 x C2 + A3E x B3 x C3 + A4E x B4 x C4 (score 0 to 24) IASI-S= A1S x B1 X C1 + A2S x B2 x C2 + A3S x B3 x C3 + A4S x B4 x C4 (score 0 to 24) IASI total score= IASI-E + IASI-S score ranged from 0 - 48, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling, \& percentage of BSA affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 BR (A1: H\&N, A2: UL, A3: T, A4: LL). Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value (0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6). Total extent was determined using a multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-E= A1E x B1 x C1 + A2E x B2 x C2 + A3E x B3 x C3 + A4E x B4 x C4 (score 0 to 24) IASI-S= A1S x B1 X C1 + A2S x B2 x C2 + A3S x B3 x C3 + A4S x B4 x C4 (score 0 to 24) IASI total score= IASI-E + IASI-S score ranged from 0 - 48, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling, \& percentage of BSA affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 BR (A1: H\&N, A2: UL, A3: T, A4: LL). Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value (0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6). Total extent was determined using a multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-E= A1E x B1 x C1 + A2E x B2 x C2 + A3E x B3 x C3 + A4E x B4 x C4 IASI-E score ranged from 0 - 24, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling, \& percentage of BSA affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 BR (A1: H\&N, A2: UL, A3: T, A4: LL). Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value (0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6). Total extent was determined using a multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-S= A1S x B1 X C1 + A2S x B2 x C2 + A3S x B3 x C3 + A4S x B4 x C4 IASI-S score ranged from 0 - 24, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling, \& percentage of BSA affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 BR (A1: H\&N, A2: UL, A3: T, A4: LL). Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value (0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6). Total extent was determined using a multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-E= A1E x B1 x C1 + A2E x B2 x C2 + A3E x B3 x C3 + A4E x B4 x C4 IASI-E score ranged from 0 - 24, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline and Week 16Population: Due to early study termination before the primary time point, efficacy data was not collected, therefore, not analyzed.
IASI quantified the severity of participants ichthyosis based on severity of erythema/scaling, \& percentage of BSA affected. Degree of erythema \& scaling scored from 0 (none) to 4 (very severe) for each of 4 BR (A1: H\&N, A2: UL, A3: T, A4: LL). Percentage of BSA involved for each BR (B1: % in H\&N, B2: % in UL, B3: % in T, B4: % in LL). Percentage involvement was assigned numerical value (0= 0, 1%-9%= 1, 10%-29%= 2, 30%-49%= 3, 50%-69%= 4, 70%-89%= 5, 90%-100%= 6). Total extent was determined using a multiplier considering % of total BSA by each BR (C1= 0.1 for H\&N; C2= 0.2 for UL; C3= 0.3 for T; C4= 0.4 for LL). IASI-S= A1S x B1 X C1 + A2S x B2 x C2 + A3S x B3 x C3 + A4S x B4 x C4 IASI-S score ranged from 0 - 24, higher score indicated worse disease state.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From first dose up to study termination (maximum up to 9.4 weeks)Population: Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
An AE was any untoward medical occurrence in a participant temporally associated with the use of a study treatment, whether or not considered related to the study treatment. An AE could therefore be any unfavorable and unintended sign (including abnormal laboratory finding), symptom, or disease temporally associated with use of study treatment that did not necessarily have a causal relationship with this treatment. An AE was considered "serious" if there was any of the following outcomes: death, life-threatening adverse event, Inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, other important medical events. An adverse event was considered TE if the date of onset was during or after first dose of study treatment, or if the AE present at baseline worsened in either intensity or frequency after first dose of study treatment.
Outcome measures
| Measure |
Imsidolimab
n=4 Participants
Participants received a starting dose of 400 mg of imsidolimab on Day 1 followed by 200 mg imsidolimab every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
Placebo
n=1 Participants
Participants received imsidolimab matching placebo on Day 1 and thereafter every 4 weeks (Days 29, 57 and 85) by subcutaneous injection.
|
|---|---|---|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Any TEAEs
|
1 participants
|
1 participants
|
|
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Serious TEAEs
|
0 participants
|
0 participants
|
Adverse Events
Imsidolimab
Placebo
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Imsidolimab
n=4 participants at risk
Participants received a starting dose of 400 mg of imsidolimab on Day 1 followed by 200 mg imsidolimab every four 4 (Days 29, 57 and 85) by subcutaneous injection.
|
Placebo
n=1 participants at risk
Participants received imsidolimab matching placebo on Day 1 and thereafter every four 4 (Days 29, 57 and 85) by subcutaneous injection.
|
|---|---|---|
|
General disorders
Fatigue
|
25.0%
1/4 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
0.00%
0/1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
General disorders
Peripheral swelling
|
0.00%
0/4 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
100.0%
1/1 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
Infections and infestations
Herpes simplex reactivation
|
25.0%
1/4 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
0.00%
0/1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/4 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
100.0%
1/1 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
Musculoskeletal and connective tissue disorders
Inguinal mass
|
0.00%
0/4 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
100.0%
1/1 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
0.00%
0/1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
0.00%
0/4 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
100.0%
1/1 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
0.00%
0/4 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
100.0%
1/1 • Number of events 1 • From first dose up to study termination (maximum up to 9.4 weeks)
Safety analysis set included all randomized participants who received at least 1 dose of imsidolimab or placebo.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place