Recruiting

ACCESS-1Pozelimab and Cemdisiran for Paroxysmal Nocturnal Hemoglobinuria

Study Aim

This study aims to evaluate if Pozelimab and Cemdisiran can effectively control hemolysis and reduce the need for blood transfusions in adults with Paroxysmal Nocturnal Hemoglobinuria.

What is being tested

Ravulizumab

+ Pozelimab

+ Cemdisiran

Drug

Who is being recruted

Anemia+4

+ Anemia, Hemolytic

+ Bone Marrow Diseases

Over 18 Years

+13 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 3

Interventional

Study Start: August 2022

See protocol details

Summary

Principal SponsorRegeneron Pharmaceuticals

Study ContactClinical Trials Administrator

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: August 1, 2022

Actual date on which the first participant was enrolled.

This clinical trial is exploring a new combination treatment for people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. The study tests the effectiveness and safety of two experimental drugs, pozelimab and cemdisiran, compared to two existing treatments, ravulizumab and eculizumab. Participants in this study have either never received or have not recently had complement inhibitor treatments. The main goal is to find out if the new drug combination can better manage the disease compared to current options, potentially offering a new choice for patients who need alternative treatments. Participants in the study will receive the treatment through injections, and researchers will observe various outcomes. They will measure how well the new combination controls hemolysis, which is the breakdown of red blood cells, by checking levels of a blood enzyme called lactate dehydrogenase (LDH). Other important measures include whether participants can avoid blood transfusions and if their bodies develop antibodies against the new drugs, which could affect how well the treatment works or cause side effects. By closely monitoring these factors, the study aims to gather comprehensive data on the potential benefits and risks of pozelimab and cemdisiran for treating PNH.

Official TitleA Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

NCT05133531

Principal SponsorRegeneron Pharmaceuticals

Study ContactClinical Trials Administrator

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

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

Design Details

190 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

AnemiaAnemia, HemolyticBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinuria, ParoxysmalMyelodysplastic Syndromes

Criteria

4 inclusion criteria required to participate

Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol

Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol

LDH level ≥2 × ULN at the screening visit

Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol

9 exclusion criteria prevent from participating

Body weight <40 kilograms at screening visit

Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol

Any contraindication for receiving Neisseria meningitidis vaccinations (serotypes ACWY and B)

Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

2 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Randomized 1:1

Group II

Experimental

Randomized 1:1

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 69 locations

Recruiting

Kaohsiung Medical University Hospital

Kaohsiung City, TaiwanOpen Kaohsiung Medical University Hospital in Google Maps

Recruiting

The Oncology Institute of Hope & Innovation

Whittier, United States

Recruiting

Centro de Estudos e Pesquisas em Hematologia e Oncologia

Santo André, Brazil

Recruiting

A Beneficencia Portuguesa de Sao Paulo, BP Mirante

São Paulo, Brazil

Recruiting

69 Study Centers