Trial · NCT03078582
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
NCT03078582, Phase 2, Completed, Ra Pharmaceuticals; results first posted 2020-03-10
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients. Trials, record NCT03078582. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03078582
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients
- NCT number
- NCT03078582
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 26 participants, actual
- Lead sponsor
- Ra Pharmaceuticals
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Interventions
- Zilucoplan (RA101495)
- First posted
- 2017-03-13
- Last updated
- 2022-07-27
- Other registries' IDs
- eudract:2016-003522-16
- Results first posted
- 2020-03-10
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. | Through Week 12 of the study | The primary efficacy endpoint is the change-from-baseline in serum LDH levels during this period, defined as the mean LDH values of Weeks 6, 8, 10, and 12 minus the baseline value of LDH. |
| Secondary | Changes From Baseline in Bilirubin Values | Through Week 12 of the study | Changes from baseline at each of the scheduled post-baseline time-points |
| Secondary | Total Hemoglobin | Through Week 12 of the Study | Changes from baseline at each of the scheduled post-baseline time-points |
| Secondary | Changes From Baseline in Free Hemoglobin Values | Through Week 12 of the study | Changes from baseline at each of the scheduled post-baseline time-points |
| Secondary | Haptoglobin Values | Through Week 12 of the Study | Changes from baseline at each of the scheduled post-baseline time-points |
| Secondary | Reticulocyte Values | Through Week 12 of the Study | Changes from baseline at each of the scheduled post-baseline time-points |
| Secondary | Hemoglobinuria Values | Through Week 12 of the Study | Changes from baseline at each of the scheduled post-baseline 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. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. | Through Week 12 of the study | Efficacy Evaluable | U/L | MEAN |
| SECONDARY | Changes From Baseline in Bilirubin Values | Through Week 12 of the study | Efficacy Evaluable | umol/L | MEAN |
| SECONDARY | Total Hemoglobin | Through Week 12 of the Study | Efficacy Evaluable | g/L | MEAN |
| SECONDARY | Changes From Baseline in Free Hemoglobin Values | Through Week 12 of the study | Efficacy Evaluable | mg/dL | MEAN |
| SECONDARY | Haptoglobin Values | Through Week 12 of the Study | Efficacy Evaluable | g/L | MEAN |
| SECONDARY | Reticulocyte Values | Through Week 12 of the Study | Efficacy Evaluable | 10^12 cells/L (SI Units) | MEAN |
| SECONDARY | Hemoglobinuria Values | Through Week 12 of the Study | Efficacy Evaluable | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group98
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. | Cohort A (Treatment Naive) | -695.2 | Standard Deviation 589.2 | U/L | |
| Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Level. | Cohort B (Previously on Eculizumab) | 216.7 | Standard Deviation 209.2 | U/L | |
| Changes From Baseline in Bilirubin Values | Cohort A (Treatment Naive) | Week 1 | 1.3 | Standard Deviation 7.2 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort B (Previously on Eculizumab) | Week 1 | 1.9 | Standard Deviation 4.5 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort A (Treatment Naive) | Week 2 | 3.1 | Standard Deviation 7.7 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort B (Previously on Eculizumab) | Week 2 | 4.4 | Standard Deviation 6.5 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort A (Treatment Naive) | Week 3 | 4.4 | Standard Deviation 9.0 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort B (Previously on Eculizumab) | Week 3 | 6.8 | Standard Deviation 7.5 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort A (Treatment Naive) | Week 4 | 4.0 | Standard Deviation 6.7 | umol/L |
| Changes From Baseline in Bilirubin Values | Cohort B (Previously on Eculizumab) | Week 4 | 4.3 | Standard Deviation 6.5 | umol/L |
1 to 10 of 98Source ClinicalTrials.gov
See all 98 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort A (Treatment Naive) | 10 | |
| Overall Study | STARTED | Cohort B (Previously on Eculizumab) | 16 | |
| Overall Study | COMPLETED | Cohort A (Treatment Naive) | 10 | |
| Overall Study | COMPLETED | Cohort B (Previously on Eculizumab) | 8 | |
| Overall Study | NOT COMPLETED | Cohort A (Treatment Naive) | 0 | |
| Overall Study | NOT COMPLETED | Cohort B (Previously on Eculizumab) | 8 |
Source ClinicalTrials.gov
Adverse events116
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal Pain | Gastrointestinal disorders | Cohort A (Treatment Naive) | 2 | 10 | 2 |
| other | Abdominal Pain | Gastrointestinal disorders | Cohort B (Previously on Eculizumab) | 1 | 16 | 3 |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Cohort A (Treatment Naive) | 0 | 10 | 0 |
| other | Abdominal Pain Upper | Gastrointestinal disorders | Cohort B (Previously on Eculizumab) | 3 | 16 | 3 |
| other | Diarrhoea | Gastrointestinal disorders | Cohort A (Treatment Naive) | 1 | 10 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Cohort B (Previously on Eculizumab) | 1 | 16 | 1 |
| other | Constipation | Gastrointestinal disorders | Cohort A (Treatment Naive) | 0 | 10 | 0 |
| other | Constipation | Gastrointestinal disorders | Cohort B (Previously on Eculizumab) | 1 | 16 | 1 |
| other | Enteritis | Gastrointestinal disorders | Cohort A (Treatment Naive) | 1 | 10 | 1 |
| other | Enteritis | Gastrointestinal disorders | Cohort B (Previously on Eculizumab) | 0 | 16 | 0 |
1 to 10 of 116Source ClinicalTrials.gov
See all 116 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort A (Treatment Naive) | 0 | 10 |
| Total, all-cause mortality | Cohort B (Previously on Eculizumab) | 0 | 16 |
| Total, other adverse events | Cohort A (Treatment Naive) | 10 | 10 |
| Total, other adverse events | Cohort B (Previously on Eculizumab) | 15 | 16 |
| Total, serious adverse events | Cohort A (Treatment Naive) | 2 | 10 |
| Total, serious adverse events | Cohort B (Previously on Eculizumab) | 1 | 16 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Cohort A (Treatment Naive) vs Cohort B (Previously on Eculizumab): deaths from any cause | Trial comparisons |
| Cohort A (Treatment Naive) vs Cohort B (Previously on Eculizumab): serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03078582
GET https://conductscience.org/api/trials/v1/NCT03078582/results?page=2