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The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Zilucoplan (RA101495) | Experimental | Subjects will receive 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 |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Zilucoplan (RA101495) | Drug | 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC |
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| Measure | Description | Time Frame |
|---|---|---|
| Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. | Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels | Through Week 12 of the study |
| Measure | Description | Time Frame |
|---|---|---|
| Change-from-baseline Bilirubin Values | Changes from baseline at each of the scheduled postbaseline time-points | Through week 12 |
| Change-from-baseline Total Hemoglobin Values | Changes from baseline at each of the scheduled postbaseline time-points |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Dr. Anita Hill | St James' Institute of Oncology | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site | Duarte | California | 91010 | United States | ||
| Investigative Site |
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| Label | URL |
|---|---|
| Related Info | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | 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 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 17, 2017 | Jul 1, 2019 |
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| Through week 12 |
| Change-from-baseline Free Hemoglobin Values | Changes from baseline at each of the scheduled postbaseline time-points | Through week 12 |
| Change-from-baseline Haptoglobin Values | Changes from baseline at each of the scheduled postbaseline time-points | Through week 12 |
| Change-from-baseline Reticulocyte Values | Changes from baseline at each of the scheduled postbaseline time-points | Through week 12 |
| Change-from-baseline Hemoglobinuria Values | 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. | Through week 12 |
| Los Angeles |
| California |
| 90033 |
| United States |
| Investigative Site | Chicago | Illinois | 60612 | United States |
| Investigative Site | Manhasset | New York | 11030 | United States |
| Investigative Site | Durham | North Carolina | 27710 | United States |
| Investigative Site | Dallas | Texas | 75390 | United States |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 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): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Region of Enrollment | Number | participants |
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| Weight | Mean | Standard Deviation | kg |
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| Height | Mean | Standard Deviation | cm |
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| BMI | Mean | Standard Deviation | kg/m2 |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change-from-baseline in Serum Lactate Dehydrogenase (LDH) Levels. | Change-from-baseline through Week 12 in serum lactate dehydrogenase (LDH) levels | Efficacy Evaluable | Posted | Mean | Standard Deviation | U/L | Through Week 12 of the study |
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| Secondary | Change-from-baseline Bilirubin Values | Changes from baseline at each of the scheduled postbaseline time-points | Efficacy Evaluable | Posted | Mean | Standard Deviation | umol/L | Through week 12 |
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| Secondary | Change-from-baseline Total Hemoglobin Values | Changes from baseline at each of the scheduled postbaseline time-points | Efficacy Evaluable | Posted | Mean | Standard Deviation | g/L | Through week 12 |
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| Secondary | Change-from-baseline Free Hemoglobin Values | Changes from baseline at each of the scheduled postbaseline time-points | Efficacy Evaluable | Posted | Number | mg/dL | Through week 12 |
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| Secondary | Change-from-baseline Haptoglobin Values | Changes from baseline at each of the scheduled postbaseline time-points | Efficacy Evaluable | Posted | Mean | Standard Deviation | g/L | Through week 12 |
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| Secondary | Change-from-baseline Reticulocyte Values | Changes from baseline at each of the scheduled postbaseline time-points | Efficacy Evaluable | Posted | Mean | Standard Deviation | 10^12 cells/L | Through week 12 |
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| Secondary | Change-from-baseline Hemoglobinuria Values | 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. | Efficacy Evaluable | Posted | Mean | Standard Deviation | score on a scale | Through week 12 |
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Through week 12 of the Study
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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): 0.3 mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC | 0 | 3 | 0 | 3 | 3 | 3 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
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| Influenza like illness | General disorders | Systematic Assessment |
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| Lipase increased | Investigations | Systematic Assessment |
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| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Haemoglobinuria | Renal and urinary disorders | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | Systematic Assessment |
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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
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Sponsor Ra Pharmaceutical, Inc | Ra Pharmaceutical, Inc | 1 617 401 4060 | trials@rapharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 12, 2018 | Jul 1, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
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| ID | Term |
|---|---|
| C000719268 | zilucoplan |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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