Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2020-002761-33 | EudraCT Number |
Not provided
Not provided
Sponsor decision not related to efficacy or safety.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The primary objective of the study is:
To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy
The secondary objectives of the study are to:
Evaluate the effect of pozelimab and cemdisiran combination treatment versus anti-C5 standard-of-care treatment (eculizumab or ravulizumab) on the following:
To assess the concentrations of total pozelimab and either total eculizumab or total ravulizumab in serum and total cemdisiran and total C5 protein in plasma
To assess the immunogenicity of pozelimab and cemdisiran
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Pozelimab and Cemdisiran | Experimental | Randomized 1:1 |
|
| Anti-C5 standard-of-care | Experimental | Randomized 1:1 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cemdisiran | Drug | Administered per protocol |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change in Lactate Dehydrogenase (LDH) From Baseline to Week 36 | From baseline to week 36 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Transfusion Avoidance After Day 1 Through Week 36 | Participants who do not receive a red blood cell (RBC) transfusion as per protocol algorithm based on post baseline hemoglobin values | Day 1 through week 36 |
| Percentage of Participants With Transfusion Avoidance From Week 4 Through Week 36 |
Not provided
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Trial Management | Regeneron Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Regeneron Research Facility | Whittier | California | 90603 | United States |
Not provided
| Label | URL |
|---|---|
| A Plain Language Summary is available on TrialSummaries.com | View source |
Not provided
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
A total of 140 participants were expected to be enrolled, however, due to feasibility: five participants were screened and 3 were randomized and treated. There were 2 screen failures.
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Pozelimab and Cemdisiran | Randomized 1:1 |
| FG001 | Anti-C5 Standard-of-care | Randomized 1:1 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 21, 2022 | Jul 9, 2024 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Eculizumab | Drug | Administered per protocol |
|
|
| Pozelimab | Drug | Administered per protocol |
|
|
| Ravulizumab | Drug | Administered per protocol |
|
|
Participants who do not receive an RBC transfusion as per protocol algorithm based on post baseline hemoglobin values |
| Week 4 through week 36 |
| Percentage of Participants With Breakthrough Hemolysis After Day 1 Through Week 36 | Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol | Day 1 through week 36 |
| Percentage of Participants With Breakthrough Hemolysis From Week 4 Through Week 36 | Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol | Week 4 (day 29) through week 36 |
| Percentage of Participants With Hemoglobin Stabilization After Day 1 Through Week 36 | Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol | Day 1 through week 36 |
| Percentage of Participants With Hemoglobin Stabilization From Week 4 Through Week 36 | Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol | Week 4 (day 29) through week 36 |
| Percentage of Participants With Adequate Control of LDH From Week 8 Through Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Week 8 (day 57) through week 36 |
| Percentage of Participants With Adequate Control of LDH After Day 1 Though Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Day 1 through week 36 |
| Percentage of Participants Who Maintained Adequate Control of Hemolysis From Week 8 Through Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Week 8 through week 36 |
| Percentage of Participants Who Maintained Adequate Control of Hemolysis After Day 1 Through Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Day 1 through week 36 |
| Percentage of Participants With Normalization of LDH From Week 8 Through Week 36 | Percentage of participants with normalization of LDH as defined in the protocol | Week 8 (day 57) through week 36 |
| Percentage of Participants With Normalization of LDH After Day 1 Through Week 36 | Percentage of participants with normalization of LDH as defined in the protocol | Day 1 through week 36 |
| Change in Fatigue as Measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline to Week 36 | The FACIT-Fatigue is a 13 item, self-administered clinical outcome assessment (COA) assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-Fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue. | From baseline to week 36 |
| Change in Physical Function (PF) Score on the European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire Core 30 Items (EORTC-QLQ-C30) From Baseline to Week 36 | EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much." | From baseline to week 36 |
| Change in Global Health Status (GHS)/QoL Scale Score on the EORTC-QLQ-C30 From Baseline to Week 36 | EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much." | From baseline to week 36 |
| Rate of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36 | Per protocol algorithm | Day 1 through week 36 |
| Rate of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36 | Per protocol algorithm | Week 4 through week 36 |
| Number of Units of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36 | Per protocol algorithm | Day 1 through week 36 |
| Number of Units of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36 | Per protocol algorithm | Week 4 through week 36 |
| Change in Hemoglobin Levels From Baseline to Week 36 | Per protocol algorithm | From baseline to week 36 |
| Incidence of Treatment Emergent Serious Adverse Events (SAEs) | Treatment period and safety follow up period | Up to week 29 |
| Incidence of Treatment-emergent Adverse Events (TEAEs) of Special Interest | Treatment period and safety follow up period | Up to week 29 |
| Incidence of TEAEs Leading to Treatment Discontinuation | Treatment period and safety follow up period | Up to week 29 |
| Change in Total CH50 From Baseline to Week 36 | From baseline to week 36 |
| Percent Change in Total CH50 From Baseline to Week 36 | From baseline to week 36 |
| Concentration of Total C5 in Plasma Through Week 62 | Treatment period and safety follow up period | Through week 62 |
| Concentrations of Total Pozelimab in Serum Through Week 62 | Treatment period and safety follow up period | Through week 62 |
| Concentrations of Total Cemdisiran in Plasma Through Week 32 | Treatment period | Through week 32 |
| Concentrations of Total Eculizumab in Serum Through Week 40 | Treatment period | Through week 40 |
| Concentrations of Total Ravulizumab in Plasma Through Week 44 | Treatment period | Through week 44 |
| Incidence of Treatment Emergent Anti-drug Antibodies (ADAs) to Pozelimab Through Week 62 | Treatment period and safety follow up period | Through week 62 |
| Incidence of Treatment Emergent ADAs to Cemdisiran Through Week 62 | Treatment period and safety follow up period | Through week 62 |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Pozelimab and Cemdisiran | Randomized 1:1 |
| BG001 | Anti-C5 Standard-of-care | Randomized 1:1 |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Number | Participants |
|
| 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 | Percent Change in Lactate Dehydrogenase (LDH) From Baseline to Week 36 | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | From baseline to week 36 |
|
| |||||||||||||||||||||||
| Secondary | Percentage of Participants With Transfusion Avoidance After Day 1 Through Week 36 | Participants who do not receive a red blood cell (RBC) transfusion as per protocol algorithm based on post baseline hemoglobin values | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Transfusion Avoidance From Week 4 Through Week 36 | Participants who do not receive an RBC transfusion as per protocol algorithm based on post baseline hemoglobin values | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Week 4 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Breakthrough Hemolysis After Day 1 Through Week 36 | Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Breakthrough Hemolysis From Week 4 Through Week 36 | Participants with an increase in LDH with concomitant signs or symptoms associated with hemolysis as described in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Week 4 (day 29) through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Hemoglobin Stabilization After Day 1 Through Week 36 | Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Hemoglobin Stabilization From Week 4 Through Week 36 | Participants who do not receive an RBC transfusion and have no decrease in hemoglobin level as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Week 4 (day 29) through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Adequate Control of LDH From Week 8 Through Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Week 8 (day 57) through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Adequate Control of LDH After Day 1 Though Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants Who Maintained Adequate Control of Hemolysis From Week 8 Through Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Week 8 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants Who Maintained Adequate Control of Hemolysis After Day 1 Through Week 36 | Percentage of participants with adequate control of LDH as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Normalization of LDH From Week 8 Through Week 36 | Percentage of participants with normalization of LDH as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Week 8 (day 57) through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Percentage of Participants With Normalization of LDH After Day 1 Through Week 36 | Percentage of participants with normalization of LDH as defined in the protocol | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Change in Fatigue as Measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline to Week 36 | The FACIT-Fatigue is a 13 item, self-administered clinical outcome assessment (COA) assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-Fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue. | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | From baseline to week 36 |
|
| ||||||||||||||||||||||
| Secondary | Change in Physical Function (PF) Score on the European Organization for Research and Treatment of Cancer Quality-of-Life Questionnaire Core 30 Items (EORTC-QLQ-C30) From Baseline to Week 36 | EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 6 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much." | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | From baseline to week 36 |
|
| ||||||||||||||||||||||
| Secondary | Change in Global Health Status (GHS)/QoL Scale Score on the EORTC-QLQ-C30 From Baseline to Week 36 | EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much." | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints.. | Posted | From baseline to week 36 |
|
| ||||||||||||||||||||||
| Secondary | Rate of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36 | Per protocol algorithm | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Rate of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36 | Per protocol algorithm | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Week 4 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Number of Units of RBCs Transfused Per Protocol Algorithm After Day 1 Through Week 36 | Per protocol algorithm | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Day 1 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Number of Units of RBCs Transfused Per Protocol Algorithm From Week 4 Through Week 36 | Per protocol algorithm | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Week 4 through week 36 |
|
| ||||||||||||||||||||||
| Secondary | Change in Hemoglobin Levels From Baseline to Week 36 | Per protocol algorithm | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | From baseline to week 36 |
|
| ||||||||||||||||||||||
| Secondary | Incidence of Treatment Emergent Serious Adverse Events (SAEs) | Treatment period and safety follow up period | Posted | Number | Events | Up to week 29 |
|
| |||||||||||||||||||||
| Secondary | Incidence of Treatment-emergent Adverse Events (TEAEs) of Special Interest | Treatment period and safety follow up period | Posted | Number | Events | Up to week 29 |
|
| |||||||||||||||||||||
| Secondary | Incidence of TEAEs Leading to Treatment Discontinuation | Treatment period and safety follow up period | Posted | Number | Events | Up to week 29 |
|
| |||||||||||||||||||||
| Secondary | Change in Total CH50 From Baseline to Week 36 | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | From baseline to week 36 |
|
| |||||||||||||||||||||||
| Secondary | Percent Change in Total CH50 From Baseline to Week 36 | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | From baseline to week 36 |
|
| |||||||||||||||||||||||
| Secondary | Concentration of Total C5 in Plasma Through Week 62 | Treatment period and safety follow up period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 62 |
|
| ||||||||||||||||||||||
| Secondary | Concentrations of Total Pozelimab in Serum Through Week 62 | Treatment period and safety follow up period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 62 |
|
| ||||||||||||||||||||||
| Secondary | Concentrations of Total Cemdisiran in Plasma Through Week 32 | Treatment period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 32 |
|
| ||||||||||||||||||||||
| Secondary | Concentrations of Total Eculizumab in Serum Through Week 40 | Treatment period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 40 |
|
| ||||||||||||||||||||||
| Secondary | Concentrations of Total Ravulizumab in Plasma Through Week 44 | Treatment period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 44 |
|
| ||||||||||||||||||||||
| Secondary | Incidence of Treatment Emergent Anti-drug Antibodies (ADAs) to Pozelimab Through Week 62 | Treatment period and safety follow up period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 62 |
|
| ||||||||||||||||||||||
| Secondary | Incidence of Treatment Emergent ADAs to Cemdisiran Through Week 62 | Treatment period and safety follow up period | Due to Early Termination, this endpoint was removed from the Statistical Analysis Plan and data was not collected for primary and secondary endpoints. | Posted | Through week 62 |
|
|
From first dose to week 29
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Pozelimab and Cemdisiran | Randomized 1:1 | 0 | 2 | 0 | 2 | 2 | 2 |
| EG001 | Anti-C5 Standard-of-care | Randomized 1:1 | 0 | 1 | 0 | 1 | 1 | 1 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Injection site rash | General disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (26.0) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
|
20Dec2022 - Due to feasibility challenges related to enrollment and not due to efficacy or safety concerns, the sponsor terminated the study early and discontinued participant enrollment.
The investigator has the right to independently publish study results from the investigator's site after a multi-center publication, or a defined period after the completion of the study by all sites. The investigator must provide the sponsor a copy of any such publication derived from the study for review and comment in advance of any submission, and delay publication, if requested, to allow the Sponsor to preserve its proprietary rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trials Administrator | Regeneron Pharmaceuticals, Inc. | 844-734-6643 | clinicaltrials@regeneron.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jul 22, 2023 | Jul 9, 2024 | SAP_001.pdf |
Not provided
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C481642 | eculizumab |
| C000629409 | ravulizumab |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
| Asian |
|
| American Indian or Alaska Native |
|
| Other |
|
| Unknown |
|
| Not Reported |
|
|
|
|