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RNAi Therapeutics

Pozelimab + Cemdisiran for Paroxysmal Nocturnal Hemoglobinuria (ACCESS-EXT Trial)

Phase 3

Recruiting

Research Sponsored by Regeneron Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to week 108

Awards & highlights

ACCESS-EXT Trial Summary

This trial is researching an experimental drug combo to treat paroxysmal nocturnal hemoglobinuria (PNH) to see how safe & effective it is.

Who is the study for?

Adults with a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria (PNH) who have specific genetic variations making them unresponsive to certain other treatments. They should have high levels of an enzyme called LDH, active disease symptoms, and must have completed previous related studies without dropping out.Check my eligibility

What is being tested?

The trial is testing the long-term safety and effectiveness of combining two experimental drugs, Pozelimab and Cemdisiran, for treating PNH. It will also assess how much drug remains in the blood over time and if the body creates antibodies against these drugs.See study design

What are the potential side effects?

Potential side effects from taking Pozelimab and Cemdisiran may include reactions where the drug enters the body, changes in liver function tests, allergic responses or possible development of antibodies that could reduce drug effectiveness or cause additional side effects.

ACCESS-EXT Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have PNH and a specific genetic variation making me unresponsive to eculizumab or ravulizumab.

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My PNH diagnosis was confirmed with a specific blood test.

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I have the C5 polymorphism.

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I have symptoms related to PNH as listed in the study.

ACCESS-EXT Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to week 108

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to week 108

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to week 108 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Incidence of adverse events (AEs) leading to permanent treatment discontinuation

Incidence of treatment emergent adverse events of special interest (AESIs)

Incidence of treatment-emergent serious adverse events (SAEs)

+4 more

Secondary outcome measures

Adequate control of hemolysis (LDH ≤1.5 × ULN)

Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)

Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30

+22 more

ACCESS-EXT Trial Design

2Treatment groups

Experimental Treatment

Group I: PNH Transition PatientsExperimental Treatment2 Interventions

Patients with PNH who completed treatment/ protocol requirements (as applicable) in the parent study (R3918-PNH-2021 [NCT05133531]) Note: Dose of pozelimab may be administered intravenously [IV] as needed or as warranted in some circumstances and with sponsor approval for patients who experience breakthrough hemolysis, an increase in lactate dehydrogenase (LDH), a type III hypersensitivity reaction, or reintroduction of study treatment after a period of treatment interruption.

Group II: C5 Polymorphism PatientsExperimental Treatment2 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Cemdisiran

2020

Completed Phase 2

~80

Pozelimab

2020

Completed Phase 2

~120

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor

622 Previous Clinical Trials

380,529 Total Patients Enrolled

Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals

263 Previous Clinical Trials

251,212 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any openings at the moment for this research?

"According to clinicaltrials.gov, this experimental trial is still recruiting eligible individuals for participation; the study was initially posted on March 7th 2023 and had its latest update on November 28th of that same year."

Answered by AI

How many people are participating in this clinical research?

"Affirmative. Clinicaltrials.gov confirms that the trial, first posted on March 7th 2021, is currently accepting volunteers and has been recently edited on November 28th 2020. 202 participants are sought from 18 distinct medical sites for this research project."

Answered by AI

Has the FDA sanctioned C5 Polymorphism Patients as a viable therapeutic option?

"Our team at Power rated C5 Polymorphism Patients a 3 for safety, which is indicative of the fact that there is clinical evidence both supporting its efficacy and confirming its security."

Answered by AI

How many areas are presently hosting this trial?

"Clinical data for this study is being collected from 18 different sites, such as Hualien Tzu Chi Hospital in Hualien City, Gachon University Gil Medical Center in Incheon, and National Taiwan University Hospital in Taipei."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria

2023. "A Study to Examine the Long-term Safety, Tolerability, and Effectiveness of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05744921.