A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works (ACCESS-EXT)

July 29, 2026 updated by: Regeneron Pharmaceuticals

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section.

This study is looking at several other research questions, including:

  • How effective is the pozelimab + cemdisiran combination?
  • What side effects may happen from taking the study drugs?
  • How much of each study drug is in the blood at different times?
  • Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

202

Phase

  • Phase 3

Contacts and Locations

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

Study Contact

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 2C4
        • Recruiting
        • Toronto General Hospital
  • Colombia
    • Antioquia
      • Medellín, Antioquia, Colombia, 050034
        • Recruiting
        • Hospital Pablo Tobon Uribe
  • Hungary
      • Budapest, Hungary, 1083
        • Recruiting
        • Semmelweis University/Semmelweis Egyetem
  • India
      • Chandigarh, India, 160012
        • Recruiting
        • Postgraduate Institute of Medical Education & Research
      • Jaipur, India, 302017
        • Recruiting
        • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
    • Kerala
      • Kochi, Kerala, India, 682041
        • Recruiting
        • Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
    • Maharashtra
      • Mumbai, Maharashtra, India, 400022
        • Recruiting
        • K J Somaiya Super Specialty Hospital & Research Centre
    • National Capital Territory of Delhi
      • New Delhi, National Capital Territory of Delhi, India, 110085
        • Recruiting
        • Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
  • Italy
      • Torino, Italy, 10126
        • Recruiting
        • SC Hematology, AOU Città della Salute e della Scienza di Torino
    • Firenze
      • Florence, Firenze, Italy, 50139
        • Recruiting
        • AOU Careggi
  • Japan
    • Aichi-ken
      • Nagoya, Aichi-ken, Japan, 466-8650
        • Withdrawn
        • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
    • Ibaraki
      • Tsukuba, Ibaraki, Japan, 305-8576
        • Recruiting
        • University of Tsukuba Hospital
  • Jordan
      • Amman, Jordan, 11942
        • Recruiting
        • Jordan University Hospital (JUH)
  • Malaysia
    • Pahang
      • Kuantan, Pahang, Malaysia, 25200
        • Recruiting
        • Hospital Tg Ampuan Afzan
    • Sabah
      • Kota Kinabalu, Sabah, Malaysia, 88200
        • Recruiting
        • Hospital Queen Elizabeth
    • Selangor
      • Ampang, Selangor, Malaysia, 68000
        • Recruiting
        • Hospital Ampang
  • Peru
      • Lima, Peru, 15072
        • Recruiting
        • Clinica San Felipe
  • Philippines
    • National Capital Region
      • Quezon City, National Capital Region, Philippines, 1634
        • Recruiting
        • St Lukes Medical Center
  • Poland
      • Bydgoszcz, Poland, 85-168
        • Completed
        • Szpital Uniwersytecki Nr2 Bydgoszcz
    • Kuyavian-Pomeranian Voivodeship
      • Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-048
        • Recruiting
        • In-Vivo Sp. z o.o.
    • Pomeranian Voivodeship
      • Gdansk, Pomeranian Voivodeship, Poland, 80-214
        • Recruiting
        • University Clinical Center Medical University of Gdansk
  • Romania
    • Cluj
      • Cluj-Napoca, Cluj, Romania, 400015
        • Recruiting
        • Prof Dr Ion Chiricuta Cancer Institute
  • Singapore
      • Singapore, Singapore, 119074
        • Recruiting
        • National University Hospital
  • South Korea
      • Busan, South Korea, 49241
        • Recruiting
        • Pusan National University Hospital
      • Seoul, South Korea, 06351
        • Recruiting
        • Samsung Medical Center
      • Seoul, South Korea, 03722
        • Recruiting
        • Severance Hospital Yonsei University Health System
      • Seoul, South Korea, 06591
        • Recruiting
        • The Catholic University of Korea, Seoul St. Mary's Hospital
      • Seoul, South Korea, 07985
        • Recruiting
        • Ewha Womans University MokDong Hospital
    • Gyeonggi-do
      • Suwon, Gyeonggi-do, South Korea, 16247
        • Recruiting
        • St. Vincent Hospital
      • Suwon, Gyeonggi-do, South Korea, 16499
        • Recruiting
        • Ajou University Medical Center
    • Namdong-Gu
      • Incheon, Namdong-Gu, South Korea, 21565
        • Recruiting
        • Gachon University Gil Medical Center
  • Spain
      • Barcelona, Spain, 08036
        • Recruiting
        • Hospital Clinic de Barcelona
    • Vizcaya
      • Bilbao, Vizcaya, Spain, 48013
        • Recruiting
        • Hospital Universitario Basurto
  • Taiwan
      • Changhua, Taiwan, 500
        • Recruiting
        • Changhua Christian Hospital
      • Hualien City, Taiwan, 97002
        • Recruiting
        • Hualien Tzu Chi Hospital
      • Taipei, Taiwan, 10002
        • Recruiting
        • National Taiwan University Hospital
      • Taipei, Taiwan, 11490
        • Recruiting
        • Tri-Service General Hospital
    • Central Taiwan
      • Taichung, Central Taiwan, Taiwan, 40447
        • Recruiting
        • China Medical University Hospital
    • Hunan Province
      • Taoyuan, Hunan Province, Taiwan, 33305
        • Recruiting
        • Chang Gung Memorial Hospital - Linkou Branch
  • Thailand
      • Bangkok, Thailand, 10330
        • Recruiting
        • King Chulalongkorn Memorial Hospital
      • Chiang Mai, Thailand, 50200
        • Recruiting
        • Chiang Mai University
      • Khon Kaen, Thailand, 40002
        • Recruiting
        • Faculty of Medicine Khon Kaen University
    • Changwat Songkhla
      • Hat Yai, Changwat Songkhla, Thailand, 90110
        • Recruiting
        • Prince Of Songkla Hospital, Prince Of Songkhla University
  • Turkey (Türkiye)
      • Istanbul, Turkey (Türkiye), 34418
        • Recruiting
        • Istanbul University
    • İzmir
      • Bornova, İzmir, Turkey (Türkiye), 35100
        • Recruiting
        • Ege University Faculty of Medicine
  • United Kingdom
      • Leeds, United Kingdom, LS97TF
        • Recruiting
        • Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology

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

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

Patients Entering from the Parent Study

  1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021[NCT05133531]), including the post-Open-label treatment period (OLTP) transition period, if applicable.
  2. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol.

Patients Entering with C5 polymorphism

  1. Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
  2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
  3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
  4. LDH level ≥2 × upper limit of normal (ULN) at the screening visit
  5. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol

Key Exclusion Criteria:

Patients Entering from the Parent Study

  1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
  2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study

Patients Entering with C5 polymorphism

  1. Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
  2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
  3. Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol
  4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening
  5. Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol
  6. Known hereditary complement deficiency
  7. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
  8. Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol

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: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: C5 Polymorphism Patients
Patients who have not been treated in either parent study but who have a documented complement component 5 (C5) variation rendering them refractory to eculizumab/ravulizumab. Note: Loading dose of pozelimab administered IV on Day 1.
Drug: Pozelimab
Administered per the protocol
Other Names:
  • REGN3918
Drug: Cemdisiran
Administered per the protocol
Other Names:
  • ALN-CC5
Experimental: PNH Transition Patients
Patients with PNH who completed treatment/ protocol requirements (as applicable) in the parent study (R3918-PNH-2021 [NCT05133531])
Drug: Pozelimab
Administered per the protocol
Other Names:
  • REGN3918
Drug: Cemdisiran
Administered per the protocol
Other Names:
  • ALN-CC5

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of treatment-emergent serious adverse events (SAEs)
Time Frame: Up to week 108

An SAE is any untoward medical occurrence that at any dose:

  • Results in death
  • Is life-threatening
  • Requires in-patient hospitalization or prolongation of existing hospitalization.
  • Results in persistent or significant disability/incapacity
  • Is a congenital anomaly/birth defect.
  • Is an important medical event
Up to week 108
Severity of treatment-emergent SAEs
Time Frame: Up to week 108
Up to week 108
Incidence of treatment emergent adverse events of special interest (AESIs)
Time Frame: Up to week 108
An AESI (serious or non-serious) is one of scientific and medical concern specific to the sponsor's product or program, for which ongoing monitoring and rapid communication by the Investigator to the sponsor can be appropriate. Such an event might warrant further investigation in order to characterize and understand it
Up to week 108
Severity of treatment emergent AESIs
Time Frame: Up to week 108
Up to week 108
Percent change from baseline in lactate dehydrogenase (LDH)
Time Frame: Baseline to week 36
Baseline to week 36
Incidence of adverse events (AEs) leading to permanent treatment discontinuation
Time Frame: Up to week 108
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug.
Up to week 108
Severity of adverse events (AEs) leading to permanent treatment discontinuation
Time Frame: Up to week 108
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug.
Up to week 108

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adequate control of hemolysis (LDH ≤1.5 × ULN)
Time Frame: Post-baseline through week 108
Post-baseline through week 108
Transfusion avoidance
Time Frame: Post-baseline through week 36
Not requiring red blood cell (RBC) transfusion as per protocol algorithm based on hemoglobin values
Post-baseline through week 36
Transfusion avoidance
Time Frame: Post-baseline through week 48
Not requiring red blood cell (RBC) transfusion as per protocol algorithm based on hemoglobin values
Post-baseline through week 48
Transfusion avoidance
Time Frame: Post-baseline through week 76
Not requiring red blood cell (RBC) transfusion as per protocol algorithm based on hemoglobin values
Post-baseline through week 76
Transfusion avoidance
Time Frame: Post-baseline through week 108
Not requiring red blood cell (RBC) transfusion as per protocol algorithm based on hemoglobin values
Post-baseline through week 108
Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)
Time Frame: Post-baseline through week 36
Post-baseline through week 36
Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)
Time Frame: Post-baseline through week 48
Post-baseline through week 48
Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)
Time Frame: Post-baseline through week 76
Post-baseline through week 76
Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)
Time Frame: Post-baseline through week 108
Post-baseline through week 108
Hemoglobin stabilization
Time Frame: Post-baseline through week 36
Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level of ≥2 g/dL
Post-baseline through week 36
Hemoglobin stabilization
Time Frame: Post-baseline through week 48
Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level of ≥2 g/dL
Post-baseline through week 48
Hemoglobin stabilization
Time Frame: Post-baseline through week 76
Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level of ≥2 g/dL
Post-baseline through week 76
Hemoglobin stabilization
Time Frame: Post-baseline through week 108
Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level of ≥2 g/dL
Post-baseline through week 108
Percent change in LDH
Time Frame: From baseline to week 48
From baseline to week 48
Percent change in LDH
Time Frame: From baseline to week 76
From baseline to week 76
Percent change in LDH
Time Frame: From baseline to week 108
From baseline to week 108
Change in fatigue
Time Frame: From baseline to week 36
Measured by the FACIT-Fatigue scale The FACIT-F is a 13-item, self-reported PRO 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 QoL in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a 5-point 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 fatigue
Time Frame: From baseline to week 48
Measured by the FACIT-Fatigue scale The FACIT-F is a 13-item, self-reported PRO 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 QoL in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a 4-point 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 48
Change in fatigue
Time Frame: From baseline to week 76
Measured by the FACIT-Fatigue scale The FACIT-F is a 13-item, self-reported PRO 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 QoL in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a 4-point 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 76
Change in fatigue
Time Frame: From baseline to weeks 108
Measured by the FACIT-Fatigue scale The FACIT-F is a 13-item, self-reported PRO 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 QoL in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a 4-point 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 weeks 108
Change in physical function (PF) scores on the EORTC QLQ-C30
Time Frame: From baseline to week 36

EORTC QLQ-C30 (European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire)

EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 36
Change in PF scores on the EORTC QLQ-C30
Time Frame: From baseline to week 48

EORTC QLQ-C30 (European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire)

EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 48
Change in PF scores on the EORTC QLQ-C30
Time Frame: From baseline to week 76

EORTC QLQ-C30 (European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire)

EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 76
Change in PF scores on the EORTC QLQ-C30
Time Frame: From baseline to week 108

EORTC QLQ-C30 (European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire)

EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including a global health status/quality of life (GHS/QoL) scale. Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 108
Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30
Time Frame: From baseline to week 36

GHS/QoL (Global Health Status/ Quality of Life)

Global Health Status/Quality of Life (GHS/Qol) Score (Items 29 and 30) Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 36
Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30
Time Frame: From baseline to week 48

GHS/QoL (Global Health Status/ Quality of Life)

Global Health Status/Quality of Life (GHS/Qol) Score (Items 29 and 30) Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 48
Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30
Time Frame: From baseline to week 76

GHS/QoL (Global Health Status/ Quality of Life)

Global Health Status/Quality of Life (GHS/Qol) Score (Items 29 and 30) Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 76
Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30
Time Frame: From baseline to week 108

GHS/QoL (Global Health Status/ Quality of Life)

Global Health Status/Quality of Life (GHS/Qol) Score (Items 29 and 30) Using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30). Participants rate items on a four-point scale, with 1 as "not at all" and 4 as "very much." A change of 5 - 10 points is considered a small change. A change of 10 - 20 points is considered a moderate change.
From baseline to week 108
Normalization of LDH
Time Frame: From post-baseline through week 108
From post-baseline through week 108
Rate of red blood cell (RBC) transfusion
Time Frame: Post-baseline through week 36
Per protocol algorithm
Post-baseline through week 36
Rate of RBC transfusion
Time Frame: Post-baseline through week 48
Per protocol algorithm
Post-baseline through week 48
Rate of RBC transfusion
Time Frame: Post-baseline through week 76
Per protocol algorithm
Post-baseline through week 76
Rate of RBC transfusion
Time Frame: Post-baseline through week 108
Per protocol algorithm
Post-baseline through week 108
Number of units of RBC transfusion
Time Frame: Post-baseline through week 36
Per protocol algorithm
Post-baseline through week 36
Number of units of RBC transfusion
Time Frame: Post-baseline through week 48
Per protocol algorithm
Post-baseline through week 48
Number of units of RBC transfusion
Time Frame: Post-baseline through week 76
Per protocol algorithm
Post-baseline through week 76
Number of units of RBC transfusion
Time Frame: Post-baseline through week 108
Per protocol algorithm
Post-baseline through week 108
Percentage of days with LDH ≤1.5x upper limit of normal (ULN)
Time Frame: Post-baseline through week 36
Post-baseline through week 36
Percentage of days with LDH ≤1.5x ULN
Time Frame: Post-baseline through week 48
Post-baseline through week 48
Percentage of days with LDH ≤1.5x ULN
Time Frame: Post-baseline through week 76
Post-baseline through week 76
Percentage of days with LDH ≤1.5x ULN
Time Frame: Post-baseline through week 108
Post-baseline through week 108
Change in hemoglobin levels
Time Frame: From baseline to week 36
From baseline to week 36
Change in hemoglobin levels
Time Frame: From baseline to week 48
From baseline to week 48
Change in hemoglobin levels
Time Frame: From baseline to week 76
From baseline to week 76
Change in hemoglobin levels
Time Frame: From baseline to week 108
From baseline to week 108
Change in total complement hemolytic activity assay (CH50)
Time Frame: Through week 108
Through week 108
Percent change in CH50
Time Frame: Through week 108
Through week 108
Concentrations of total pozelimab in serum
Time Frame: Through week 108
Through week 108
Concentrations of cemdisiran in plasma
Time Frame: Through week 24
Through week 24
Incidence of treatment-emergent anti-drug antibodies to pozelimab
Time Frame: Through week 108
Through week 108
Incidence of treatment-emergent anti-drug antibodies to cemdisiran
Time Frame: Through week 108
Through week 108
Concentration of total complement component 5 (C5) in plasma
Time Frame: Through week 108
Through week 108
Percent change of concentration of total C5 in plasma
Time Frame: Through week 108
Through week 108

Collaborators and Investigators

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

Sponsor

Regeneron Pharmaceuticals

Investigators

  • Study Director: Clinical Trial Management, Regeneron Pharmaceuticals

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)

March 7, 2023

Primary Completion (Estimated)

October 10, 2028

Study Completion (Estimated)

October 10, 2028

Study Registration Dates

First Submitted

February 6, 2023

First Submitted That Met QC Criteria

February 22, 2023

First Posted (Actual)

February 27, 2023

Study Record Updates

Last Update Posted (Actual)

July 30, 2026

Last Update Submitted That Met QC Criteria

July 29, 2026

Last Verified

July 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • R3918-PNH-2050
  • 2021-004931-10 (EudraCT Number)
  • 2023-510336-36-00 (Ctis: EU CT 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 or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
  • made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry),
  • the legal authority to share the data, and
  • 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

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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