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Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab

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ClinicalTrials.gov Identifier: NCT03030183
Recruitment Status : Completed
First Posted : January 24, 2017
Results First Posted : February 26, 2020
Last Update Posted : February 26, 2020
Sponsor:
Information provided by (Responsible Party):
Ra Pharmaceuticals

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Paroxysmal Nocturnal Hemoglobinuria (PNH)
Intervention Drug: Zilucoplan (RA101495)
Enrollment 3
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description

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

Period Title: Overall Study
Started 3
Completed 3
Not Completed 0
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description

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

Overall Number of Baseline Participants 3
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
<=18 years
0
   0.0%
Between 18 and 65 years
3
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 3 participants
35.3  (16.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Female
1
  33.3%
Male
2
  66.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
3
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
  33.3%
White
1
  33.3%
More than one race
0
   0.0%
Unknown or Not Reported
1
  33.3%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 3 participants
3
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 3 participants
109.67  (13.97)
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 3 participants
176.20  (7.60)
BMI  
Mean (Standard Deviation)
Unit of measure:  Kg/m2
Number Analyzed 3 participants
35.72  (7.76)
1.Primary Outcome
Title Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels.
Hide Description Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels
Time Frame Through Week 12 of the study
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: U/L
100.3  (128.0)
2.Secondary Outcome
Title Change-from-baseline Bilirubin Values
Hide Description Changes from baseline at each of the scheduled postbaseline time-points
Time Frame Through week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: umol/L
Week 1 -3.3  (5.1)
Week 2 -4.7  (12.0)
Week 3 -4.3  (7.5)
Week 4 -0.3  (2.9)
Week 6 -6.7  (17.5)
Week 8 -2.3  (12.5)
Week 10 -7.7  (14.8)
Week 12 -2.0  (6.2)
3.Secondary Outcome
Title Change-from-baseline Total Hemoglobin Values
Hide Description Changes from baseline at each of the scheduled postbaseline time-points
Time Frame Through week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: g/L
Week 1 5.7  (4.2)
Week 2 3.3  (3.8)
Week 3 0.3  (4.0)
Week 4 -0.3  (7.2)
Week 6 0.7  (5.8)
Week 8 -3.3  (4.2)
Week 10 2.3  (3.1)
Week 12 0.3  (2.5)
4.Secondary Outcome
Title Change-from-baseline Free Hemoglobin Values
Hide Description Changes from baseline at each of the scheduled postbaseline time-points
Time Frame Through week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: mg/dL
Week 1 Number Analyzed 1 participants
-145.90
Week 2 Number Analyzed 1 participants
-140.60
Week 3 Number Analyzed 1 participants
-147.70
Week 4 Number Analyzed 1 participants
-138.40
Week 6 Number Analyzed 1 participants
-147.30
Week 8 Number Analyzed 1 participants
-148.10
Week 10 Number Analyzed 1 participants
-143.50
Week 12 Number Analyzed 1 participants
-144.30
5.Secondary Outcome
Title Change-from-baseline Haptoglobin Values
Hide Description Changes from baseline at each of the scheduled postbaseline time-points
Time Frame Through week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: g/L
Week 1 0.000  (0.000)
Week 2 0.000  (0.000)
Week 3 0.000  (0.000)
Week 4 0.133  (0.231)
Week 6 0.000  (0.000)
Week 8 0.000  (0.000)
Week 10 0.000  (0.000)
Week 12 0.000  (0.000)
6.Secondary Outcome
Title Change-from-baseline Reticulocyte Values
Hide Description Changes from baseline at each of the scheduled postbaseline time-points
Time Frame Through week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: 10^12 cells/L
Week 1 -0.0190  (0.0382)
Week 2 0.0090  (0.0052)
Week 3 0.0430  (0.0327)
Week 4 0.0477  (0.0499)
Week 6 0.0310  (0.0157)
Week 8 0.0253  (0.0248)
Week 10 0.0350  (0.0350)
Week 12 0.0190  (0.0092)
7.Secondary Outcome
Title Change-from-baseline Hemoglobinuria Values
Hide Description 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.
Time Frame Through week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Efficacy Evaluable
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description:

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

Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 1 Number Analyzed 1 participants
0.0
Week 2 Number Analyzed 2 participants
0.5  (0.7)
Week 3 Number Analyzed 2 participants
0.5  (0.7)
Week 4 Number Analyzed 2 participants
1.0  (1.4)
Week 6 Number Analyzed 2 participants
0.5  (0.7)
Week 8 Number Analyzed 1 participants
2.0
Week 10 Number Analyzed 3 participants
2.3  (2.5)
Week 12 Number Analyzed 3 participants
-0.3  (2.1)
Time Frame Through week 12 of the Study
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Zilucoplan (RA101495)
Hide Arm/Group Description

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

All-Cause Mortality
Zilucoplan (RA101495)
Affected / at Risk (%)
Total   0/3 (0.00%)    
Hide Serious Adverse Events
Zilucoplan (RA101495)
Affected / at Risk (%) # Events
Total   0/3 (0.00%)    
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Zilucoplan (RA101495)
Affected / at Risk (%) # Events
Total   3/3 (100.00%)    
Gastrointestinal disorders   
Nausea   1/3 (33.33%)  1
General disorders   
Influenza like illness   1/3 (33.33%)  1
Investigations   
Lipase increased   1/3 (33.33%)  1
Musculoskeletal and connective tissue disorders   
Musculoskeletal chest pain   1/3 (33.33%)  1
Pain in extremity   1/3 (33.33%)  1
Nervous system disorders   
Headache   1/3 (33.33%)  1
Renal and urinary disorders   
Haemoglobinuria   1/3 (33.33%)  1
Nephrolithiasis   1/3 (33.33%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Sponsor Ra Pharmaceutical, Inc
Organization: Ra Pharmaceutical, Inc
Phone: 1 617 401 4060
EMail: trials@rapharma.com
Layout table for additonal information
Responsible Party: Ra Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03030183    
Other Study ID Numbers: RA101495-01.203
First Submitted: January 20, 2017
First Posted: January 24, 2017
Results First Submitted: August 13, 2019
Results First Posted: February 26, 2020
Last Update Posted: February 26, 2020