Trial Outcomes & Findings for Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab (NCT NCT03030183)
NCT ID: NCT03030183
Last Updated: 2022-07-27
Results Overview
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
3 participants
Primary outcome timeframe
Through Week 12 of the study
Results posted on
2022-07-27
Participant Flow
Participant milestones
| Measure |
Zilucoplan (RA101495)
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0. 3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
Baseline characteristics by cohort
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
35.3 years
STANDARD_DEVIATION 16.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
|
Weight
|
109.67 kg
STANDARD_DEVIATION 13.97 • n=5 Participants
|
|
Height
|
176.20 cm
STANDARD_DEVIATION 7.60 • n=5 Participants
|
|
BMI
|
35.72 kg/m2
STANDARD_DEVIATION 7.76 • n=5 Participants
|
PRIMARY outcome
Timeframe: Through Week 12 of the studyPopulation: Efficacy Evaluable
Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
|
100.3 U/L
Standard Deviation 128.0
|
SECONDARY outcome
Timeframe: Through week 12Population: Efficacy Evaluable
Changes from baseline at each of the scheduled postbaseline time-points
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline Bilirubin Values
Week 1
|
-3.3 umol/L
Standard Deviation 5.1
|
|
Change-from-baseline Bilirubin Values
Week 2
|
-4.7 umol/L
Standard Deviation 12.0
|
|
Change-from-baseline Bilirubin Values
Week 3
|
-4.3 umol/L
Standard Deviation 7.5
|
|
Change-from-baseline Bilirubin Values
Week 4
|
-0.3 umol/L
Standard Deviation 2.9
|
|
Change-from-baseline Bilirubin Values
Week 6
|
-6.7 umol/L
Standard Deviation 17.5
|
|
Change-from-baseline Bilirubin Values
Week 8
|
-2.3 umol/L
Standard Deviation 12.5
|
|
Change-from-baseline Bilirubin Values
Week 10
|
-7.7 umol/L
Standard Deviation 14.8
|
|
Change-from-baseline Bilirubin Values
Week 12
|
-2.0 umol/L
Standard Deviation 6.2
|
SECONDARY outcome
Timeframe: Through week 12Population: Efficacy Evaluable
Changes from baseline at each of the scheduled postbaseline time-points
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline Total Hemoglobin Values
Week 1
|
5.7 g/L
Standard Deviation 4.2
|
|
Change-from-baseline Total Hemoglobin Values
Week 2
|
3.3 g/L
Standard Deviation 3.8
|
|
Change-from-baseline Total Hemoglobin Values
Week 3
|
0.3 g/L
Standard Deviation 4.0
|
|
Change-from-baseline Total Hemoglobin Values
Week 4
|
-0.3 g/L
Standard Deviation 7.2
|
|
Change-from-baseline Total Hemoglobin Values
Week 6
|
0.7 g/L
Standard Deviation 5.8
|
|
Change-from-baseline Total Hemoglobin Values
Week 8
|
-3.3 g/L
Standard Deviation 4.2
|
|
Change-from-baseline Total Hemoglobin Values
Week 10
|
2.3 g/L
Standard Deviation 3.1
|
|
Change-from-baseline Total Hemoglobin Values
Week 12
|
0.3 g/L
Standard Deviation 2.5
|
SECONDARY outcome
Timeframe: Through week 12Population: Efficacy Evaluable
Changes from baseline at each of the scheduled postbaseline time-points
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline Free Hemoglobin Values
Week 1
|
-145.90 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 2
|
-140.60 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 3
|
-147.70 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 4
|
-138.40 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 6
|
-147.30 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 8
|
-148.10 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 10
|
-143.50 mg/dL
|
|
Change-from-baseline Free Hemoglobin Values
Week 12
|
-144.30 mg/dL
|
SECONDARY outcome
Timeframe: Through week 12Population: Efficacy Evaluable
Changes from baseline at each of the scheduled postbaseline time-points
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline Haptoglobin Values
Week 1
|
0.000 g/L
Standard Deviation 0.000
|
|
Change-from-baseline Haptoglobin Values
Week 2
|
0.000 g/L
Standard Deviation 0.000
|
|
Change-from-baseline Haptoglobin Values
Week 3
|
0.000 g/L
Standard Deviation 0.000
|
|
Change-from-baseline Haptoglobin Values
Week 4
|
0.133 g/L
Standard Deviation 0.231
|
|
Change-from-baseline Haptoglobin Values
Week 6
|
0.000 g/L
Standard Deviation 0.000
|
|
Change-from-baseline Haptoglobin Values
Week 8
|
0.000 g/L
Standard Deviation 0.000
|
|
Change-from-baseline Haptoglobin Values
Week 10
|
0.000 g/L
Standard Deviation 0.000
|
|
Change-from-baseline Haptoglobin Values
Week 12
|
0.000 g/L
Standard Deviation 0.000
|
SECONDARY outcome
Timeframe: Through week 12Population: Efficacy Evaluable
Changes from baseline at each of the scheduled postbaseline time-points
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline Reticulocyte Values
Week 1
|
-0.0190 10^12 cells/L
Standard Deviation 0.0382
|
|
Change-from-baseline Reticulocyte Values
Week 2
|
0.0090 10^12 cells/L
Standard Deviation 0.0052
|
|
Change-from-baseline Reticulocyte Values
Week 3
|
0.0430 10^12 cells/L
Standard Deviation 0.0327
|
|
Change-from-baseline Reticulocyte Values
Week 4
|
0.0477 10^12 cells/L
Standard Deviation 0.0499
|
|
Change-from-baseline Reticulocyte Values
Week 6
|
0.0310 10^12 cells/L
Standard Deviation 0.0157
|
|
Change-from-baseline Reticulocyte Values
Week 8
|
0.0253 10^12 cells/L
Standard Deviation 0.0248
|
|
Change-from-baseline Reticulocyte Values
Week 10
|
0.0350 10^12 cells/L
Standard Deviation 0.0350
|
|
Change-from-baseline Reticulocyte Values
Week 12
|
0.0190 10^12 cells/L
Standard Deviation 0.0092
|
SECONDARY outcome
Timeframe: Through week 12Population: Efficacy Evaluable
Changes from baseline at each of the scheduled postbaseline time-points Hemoglobinuria was assessed using a urine colorimetric scoring system with a score of 1 through 10. Where 1 represents no hemoglobinuria and 10 represents maximum hemoglobinuria.
Outcome measures
| Measure |
Zilucoplan (RA101495)
n=3 Participants
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Change-from-baseline Hemoglobinuria Values
Week 1
|
0.0 score on a scale
|
|
Change-from-baseline Hemoglobinuria Values
Week 2
|
0.5 score on a scale
Standard Deviation 0.7
|
|
Change-from-baseline Hemoglobinuria Values
Week 3
|
0.5 score on a scale
Standard Deviation 0.7
|
|
Change-from-baseline Hemoglobinuria Values
Week 4
|
1.0 score on a scale
Standard Deviation 1.4
|
|
Change-from-baseline Hemoglobinuria Values
Week 6
|
0.5 score on a scale
Standard Deviation 0.7
|
|
Change-from-baseline Hemoglobinuria Values
Week 8
|
2.0 score on a scale
|
|
Change-from-baseline Hemoglobinuria Values
Week 10
|
2.3 score on a scale
Standard Deviation 2.5
|
|
Change-from-baseline Hemoglobinuria Values
Week 12
|
-0.3 score on a scale
Standard Deviation 2.1
|
Adverse Events
Zilucoplan (RA101495)
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Zilucoplan (RA101495)
n=3 participants at risk
Subjects will receive zilucoplan (RA101495) at the dose of 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
Zilucoplan (RA101495): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
General disorders
Influenza like illness
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
Investigations
Lipase increased
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
Nervous system disorders
Headache
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
Renal and urinary disorders
Haemoglobinuria
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
|
Renal and urinary disorders
Nephrolithiasis
|
33.3%
1/3 • Number of events 1 • Through week 12 of the Study
|
Additional Information
Sponsor Ra Pharmaceutical, Inc
Ra Pharmaceutical, Inc
Phone: 1 617 401 4060
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee There is an agreement between the Principal Investigator and the Sponsor (or its agents) that restricts the PI's right to discuss or publish trial results after the trial is completed. Disclosure agreements are negotiated separately with each PI
- Publication restrictions are in place
Restriction type: OTHER