Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

August 27, 2025 updated by: Regeneron Pharmaceuticals

A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

The primary objective of the study is to evaluate the safety and tolerability of pozelimab and cemdisiran combination therapy in participants with PNH who switch from eculizumab therapy

The secondary objectives of the study are:

  • To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of CH50
  • To evaluate the effect of the combination treatment on the stability of LDH during the transition period from eculizumab monotherapy to combination with pozelimab and cemdisiran
  • To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements
  • To evaluate the effect of the combination treatment on hemoglobin levels
  • To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life (HRQoL)
  • To assess the concentrations of total pozelimab and eculizumab in serum; and total cemdisiran and C5 protein in plasma
  • To assess the immunogenicity of pozelimab and cemdisiran
  • To assess safety after dose intensification
  • To evaluate the long-term safety and efficacy of the combination treatment in an optional open-label extension period (OLEP)

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
6

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  1. Diagnosis of paroxysmal nocturnal hemoglobinuria confirmed by a history of high-sensitivity flow cytometry from prior testing
  2. Treated with stable (ie, no change in dose or frequency) eculizumab therapy at the labeled dosing regimen or a higher dose and/or more frequently administered than labeled for at least 12 weeks prior to screening visit

Key Exclusion Criteria:

  1. History of bone marrow transplantation or receipt of an organ transplant
  2. Body weight <40 kg at screening
  3. Current plans for modification of the following background concomitant medications, as applicable, during screening and treatment period: erythropoietin, immunosuppressive drugs, corticosteroids, anti-thrombotic agents, anticoagulants, iron supplements, and folic acid as described in the protocol
  4. Any use of complement inhibitor therapy other than eculizumab in the 12 weeks prior to the screening visit or planned use during the study
  5. Known hypocellular bone marrow based on a history of reduced age-adjusted bone marrow cellularity and/or bone marrow cellularity ≤25%
  6. No documented meningococcal vaccination within 5 years prior to screening visit unless it is documented that vaccination has been administered during the screening period and prior to initiation of study treatment
  7. Unable to take antibiotics for meningococcal prophylaxis, if required by local standard of care
  8. Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
  9. Documented positive polymerase chain reaction (PCR) or equivalent test based on regional recommendations for COVID-19 or suspected SARS-CoV-2 infection as described in the protocol
  10. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
  11. Recent, unstable medical conditions, excluding PNH and PNH-related complications, within the past 3 months prior to screening visit as described in the protocol
  12. Anticipated need for major surgery during the study

NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Pozelimab+Cemdisiran
Drug: Pozelimab
Intravenous (IV) loading dose (once) followed after 30 minutes by sub-cutaneous (SC) administration
Other Names:
  • REGN3918
Drug: Cemdisiran
SC administration
Other Names:
  • ALN-CC5

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
OLTP: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time Frame: Up to Day 225
Up to Day 225

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
OLTP: Percent Change in Lactate Dehydrogenase (LDH) From Pre-treatment to End-of-treatment Period
Time Frame: Screening (Day 1) through Day 225
Screening (Day 1) through Day 225
OLTP: Percent Change in LDH From Pre-treatment Through Day 29
Time Frame: Screening (Day 1) through Day 29
Screening (Day 1) through Day 29
OLTP: Percentage of Participants Who Were Transfusion-free From Baseline Through Week 32
Time Frame: Baseline through Week 32
Baseline through Week 32
OLTP: Percentage of Participants Who Were Transfusion-free From Week 4 Through Week 32
Time Frame: Week 4 through Week 32
Week 4 through Week 32
OLTP: Rate of Red Blood Cell (RBC) Transfusions From Baseline Through Week 32
Time Frame: Baseline through Week 32
The rate of units of transfusion for a participant was calculated based on the duration of treatment exposure of the participant.
Baseline through Week 32
OLTP: Rate of RBC Transfusions From Week 4 Through Week 32
Time Frame: Week 4 through Week 32
The rate of units of transfusion for a participant was calculated based on the duration of treatment exposure of the participant.
Week 4 through Week 32
OLTP: Number of RBC Units Transfused From Baseline Through Week 32
Time Frame: Baseline through Week 32
Baseline through Week 32
OLTP: Number of RBC Units Transfused From Week 4 Through Week 32
Time Frame: Week 4 through Week 32
Week 4 through Week 32
OLTP: Percentage of Participants With Breakthrough Hemolysis From Baseline Through Week 32
Time Frame: Baseline through Week 32
Breakthrough hemolysis was defined as having an LDH ≥ 2 x upper limit of normal (ULN) and having signs or symptoms.
Baseline through Week 32
OLTP: Percentage of Participants With Breakthrough Hemolysis From Week 4 Through Week 32
Time Frame: Week 4 through Week 32
Breakthrough hemolysis was defined as having an LDH ≥ 2 x ULN and having signs or symptoms.
Week 4 through Week 32
OLTP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Day 1 Through Week 32
Time Frame: Day 1 through Week 32
Adequate control was defined as LDH ≤ 1.5 x ULN from Day 1 through Week 32.
Day 1 through Week 32
OLTP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Week 8 Through Week 32
Time Frame: Week 8 through Week 32
Adequate control was defined as LDH ≤ 1.5 x ULN from Week 8 through Week 32.
Week 8 through Week 32
OLTP: Number of Participants With Adequate Control of Hemolysis at Each Visit
Time Frame: Days 1, 8, 15, 29, 43, 57, 71, 85, 113, 141, 169, 197, 225
Adequate control at a visit was defined as having LDH ≤1.5 x ULN at that visit.
Days 1, 8, 15, 29, 43, 57, 71, 85, 113, 141, 169, 197, 225
OLTP: Number of Participants With Normalization of Their LDH at Each Visit
Time Frame: Days 1, 8, 15, 29, 43, 57, 71, 85, 113, 141, 169, 197, 225
Normalization was defined as LDH ≤ 1.0 x ULN at that visit.
Days 1, 8, 15, 29, 43, 57, 71, 85, 113, 141, 169, 197, 225
OLTP: Average LDH From Baseline Through Week 32
Time Frame: Baseline through Week 32
The trapezoidal rule was used to calculate area under the curve (AUC). Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period.
Baseline through Week 32
OLTP: Average LDH From Week 8 Through Week 32
Time Frame: Week 8 through Week 32
The trapezoidal rule was used to calculate AUC. Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period.
Week 8 through Week 32
OLTP: Percentage of Participants With Hemoglobin Stabilization From Baseline Through Week 32
Time Frame: Baseline through Week 32
Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 grams per deciliter (g/dL).
Baseline through Week 32
OLTP: Percentage of Participants With Hemoglobin Stabilization From Week 4 Through Week 32
Time Frame: Week 4 through Week 32
Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 g/dL.
Week 4 through Week 32
OLTP: Change From Baseline in Hemoglobin Levels
Time Frame: Baseline, Week 32
Baseline, Week 32
OLTP: Change From Baseline in Fatigue as Measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score
Time Frame: Baseline, Week 32
The FACIT-Fatigue is a 13-item, self-reported measure 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 participants 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). Total scores range from 0 to 52, with higher scores indicating a higher quality of life.
Baseline, Week 32
OLTP: Change From Baseline in Health Related Quality of Life (HRQoL) as Measured by the Global Health Status Subscale of the European Organization for Research and Treatment of Cancer (EORTC)- Quality of Life Cancer Patients Questionnaire (QLQ) - 30 Scale
Time Frame: Baseline, Week 32
EORTC-QLQ-C30 is a 30-item self-reported questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. The GHS subscale scores ranged from 0 to 100 with higher scores indicating better quality of life.
Baseline, Week 32
OLTP: Change From Baseline in Physical Function (PF) Scores on the EORTC QLQ-C30
Time Frame: Baseline, Week 32
EORTC-QLQ-C30 is a 30-item participant self-report questionnaire composed of both multi-item and single scales, including a PF scale. PF subscale scores ranged from 0 to 100 with higher scores indicating better quality of life.
Baseline, Week 32
OLTP: Change From Baseline in Total Complement Hemolytic Activity Assay (CH50)
Time Frame: Baseline through Week 32
This assay assessed the activity of the classical pathway of complement to measure C5 activity.
Baseline through Week 32
OLTP and OLEP: Concentration of Total Pozelimab in Serum
Time Frame: Pre-dose Days 28, 56, 84, 112, 140, 168, 196, 224, 392, 588; Post-dose Day 28
Pre-dose Days 28, 56, 84, 112, 140, 168, 196, 224, 392, 588; Post-dose Day 28
OLTP: Concentration of Total Eculizumab in Serum
Time Frame: Pre-dose Days 0, 14, 28, 56, 84, 112, 140, 168, 196, 224
Pre-dose Days 0, 14, 28, 56, 84, 112, 140, 168, 196, 224
OLTP and OLEP: Concentration of Total Cemdisiran in Plasma
Time Frame: Pre-dose and Post-dose Days 0, 84, 196, 224, 588
Pre-dose and Post-dose Days 0, 84, 196, 224, 588
OLTP and OLEP: Concentration of Total C5 in Plasma
Time Frame: Pre-dose Days 0, 7, 14, 28, 56, 84, 112, 140, 168, 196, 224, 392, 588
Pre-dose Days 0, 7, 14, 28, 56, 84, 112, 140, 168, 196, 224, 392, 588
OLTP: Number of Participants With Pozelimab Anti-drug Antibodies (ADA)
Time Frame: Baseline through Week 32
Baseline through Week 32
OLTP: Number of Participants With Cemdisiran ADA
Time Frame: Baseline through Week 32
Baseline through Week 32
OLTP: Number of Participants Who Received Dose Intensification With TEAEs
Time Frame: Baseline through Week 32
Baseline through Week 32
OLEP: Number of Participants With TEAEs
Time Frame: Day 1 through Week 52 of the OLEP
Day 1 through Week 52 of the OLEP
OLEP: Percent Change in LDH From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
Day 1 through Week 24 of the OLEP
OLEP: Percent Change in LDH From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
Day 1 through Week 52 of the OLEP
OLEP: Percentage of Participants Who Were Transfusion-free From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
Day 1 through Week 24 of the OLEP
OLEP: Percentage of Participants Who Were Transfusion-free From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
Day 1 through Week 52 of the OLEP
OLEP: Rate of RBC Transfusions From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
The rate of units of transfusion for a participants was calculated based on the duration of treatment exposure of the participant.
Day 1 through Week 24 of the OLEP
OLEP: Rate of RBC Transfusions From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
The rate of units of transfusion for a participants was calculated based on the duration of treatment exposure of the participant.
Day 1 through Week 52 of the OLEP
OLEP: Number of RBC Units Transfused From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
Day 1 through Week 24 of the OLEP
OLEP: Number of RBC Units Transfused From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
Day 1 through Week 52 of the OLEP
OLEP: Percentage of Participants With Breakthrough Hemolysis From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
Breakthrough hemolysis was defined as having an LDH ≥ 2 x ULN and having signs or symptoms.
Day 1 through Week 24 of the OLEP
OLEP: Percentage of Participants With Breakthrough Hemolysis From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
Breakthrough hemolysis was defined as having an LDH ≥ 2 x ULN and having signs or symptoms.
Day 1 through Week 52 of the OLEP
OLEP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
Adequate control was defined as LDH ≤ 1.5 x ULN.
Day 1 through Week 24 of the OLEP
OLEP: Percentage of Participants Who Maintained Adequate Control of Hemolysis From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
Adequate control was defined as LDH ≤ 1.5 x ULN.
Day 1 through Week 52 of the OLEP
OLEP: Number of Participants Who Maintained Adequate Control of Hemolysis at Each Visit in OLEP
Time Frame: Baseline, Days 57, 113, 169, 225, 281, 365 of the OLEP
Adequate control was defined as LDH ≤ 1.5 x ULN at that visit.
Baseline, Days 57, 113, 169, 225, 281, 365 of the OLEP
OLEP: Number of Participants With Normalization of Their LDH at Each Visit in OLEP
Time Frame: Baseline, Days 57, 113, 169, 225, 281, 365 of the OLEP
Normalization was defined as LDH ≤ 1.0 x ULN at that visit.
Baseline, Days 57, 113, 169, 225, 281, 365 of the OLEP
OLEP: Average LDH From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
The trapezoidal rule was used to calculate AUC. Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period.
Day 1 through Week 24 of the OLEP
OLEP: Average LDH From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
The trapezoidal rule was used to calculate AUC. Individual mean LDH is defined as AUC divided by (last assessment date of LDH - first assessment date of LDH) of specific period.
Day 1 through Week 52 of the OLEP
OLEP: Percentage of Participants With Hemoglobin Stabilization From Day 1 Through Week 24 of the OLEP
Time Frame: Day 1 through Week 24 of the OLEP
Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 g/dL.
Day 1 through Week 24 of the OLEP
OLEP: Percentage of Participants With Hemoglobin Stabilization From Day 1 Through Week 52 of the OLEP
Time Frame: Day 1 through Week 52 of the OLEP
Hemoglobin stabilization was defined as not receiving an RBC transfusion and having no decrease in hemoglobin level of ≥ 2 g/dL.
Day 1 through Week 52 of the OLEP
OLEP: Change From Baseline in Hemoglobin Levels
Time Frame: Day 1 and Week 24 of the OLEP
Day 1 and Week 24 of the OLEP
OLEP: Change From Baseline in Hemoglobin Levels
Time Frame: Day 1 and Week 52 of the OLEP
Day 1 and Week 52 of the OLEP
OLEP: Change From Baseline in FACIT-Fatigue Score
Time Frame: Day 1 and Week 52 of the OLEP
The FACIT-Fatigue is a 13-item, self-reported measure 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). Total scores range from 0 to 52, with higher scores indicating a higher quality of life.
Day 1 and Week 52 of the OLEP
OLEP: Change From Baseline in HRQoL as Measured by the Global Health Status Subscale of the EORTC-QLQ-30 Scale
Time Frame: Day 1 and Week 52 of the OLEP
EORTC-QLQ-C30 is a 30-item self-reported questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. GHS subscale scores ranged from 0 to 100 with higher scores indicating better quality of life.
Day 1 and Week 52 of the OLEP
OLEP: Change From Baseline in PF Scores on the EORTC QLQ-C30
Time Frame: Day 1 and Week 52 of the OLEP
EORTC-QLQ-C30 is a 30-item participant self-report questionnaire composed of both multi-item and single scales, including a PF scale. PF subscale scores ranged from 0 to 100 with higher scores indicating better quality of life.
Day 1 and Week 52 of the OLEP
OLEP: Change From Baseline in CH50
Time Frame: Baseline, Week 16, Week 32, Week 52 of the OLEP
This assay assessed the activity of the classical pathway of complement to measure C5 activity.
Baseline, Week 16, Week 32, Week 52 of the OLEP
OLEP: Number of Participants With Pozelimab ADA
Time Frame: Day 1 through Week 52 of the OLEP
Day 1 through Week 52 of the OLEP
OLEP: Number of Participants With Cemdisiran ADA
Time Frame: Day 1 through Week 52 of the OLEP
Day 1 through Week 52 of the OLEP

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Regeneron Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 8, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 5, 2022

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 4, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 23, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 12, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 17, 2021

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 29, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • R3918-PNH-20105
  • 2020-005006-25 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

IPD Sharing Time Frame

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.

IPD Sharing Access Criteria

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

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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