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siRNA

Pozelimab + Cemdisiran for Paroxysmal Nocturnal Hemoglobinuria (ACCESS-1 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
Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
Timeline
Screening 3 weeks
Treatment Varies
Follow Up between week 8 and week 26, inclusive
Awards & highlights

ACCESS-1 Trial Summary

This trial will compare the effects of two different treatments for hemolysis (red blood cell breakdown) in patients with PNH. One treatment is a combination of two drugs, pozelimab and cemdisiran, and the other is ravulizumab. The trial will last 24 weeks, and the primary outcome measure is the effect on transfusions of RBCs. Secondary outcome measures include fatigue, quality of life, safety and tolerability, and complement activation.

Who is the study for?
Adults with confirmed PNH and active disease symptoms, including LDH levels at least twice the upper normal limit. Participants must be new to complement inhibitor treatments or not have received them recently. They should meet vaccination requirements for meningococcal disease, be able to take prophylactic antibiotics if needed, weigh over 40 kg, and have no recent serious infections or history of organ/bone marrow transplants.Check my eligibility
What is being tested?
The trial is testing a combination therapy of two experimental drugs (pozelimab + cemdisiran) against existing treatments ravulizumab and eculizumab in patients with PNH. It aims to compare their effectiveness and safety while monitoring drug levels in blood, potential antibody development against the study drugs, and side effects experienced by participants.See study design
What are the potential side effects?
Potential side effects from pozelimab + cemdisiran may include reactions where the medication is given, changes in liver function tests, allergic responses that could affect various organs like kidneys or lungs, fatigue, headache or nausea. The exact profile will be compared to those of ravulizumab and eculizumab.

ACCESS-1 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have symptoms related to PNH as described.
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My PNH diagnosis was confirmed by a specific blood test.

ACCESS-1 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~between week 8 and week 26, inclusive
This trial's timeline: 3 weeks for screening, Varies for treatment, and between week 8 and week 26, inclusive for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percent change in lactate dehydrogenase (LDH)
Secondary outcome measures
Adequate control of hemolysis
Breakthrough hemolysis
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
+25 more

ACCESS-1 Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort BExperimental Treatment3 Interventions
Randomized 1:1
Group II: Cohort AExperimental Treatment3 Interventions
Randomized 1:1
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
2021
Completed Phase 4
~1080
Pozelimab
2020
Completed Phase 2
~120
Cemdisiran
2020
Completed Phase 2
~80
Eculizumab
2021
Completed Phase 4
~1190

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
613 Previous Clinical Trials
379,568 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
258 Previous Clinical Trials
250,808 Total Patients Enrolled

Media Library

Cemdisiran (siRNA) Clinical Trial Eligibility Overview. Trial Name: NCT05133531 — Phase 3
Paroxysmal Nocturnal Hemoglobinuria Research Study Groups: Cohort A, Cohort B
Paroxysmal Nocturnal Hemoglobinuria Clinical Trial 2023: Cemdisiran Highlights & Side Effects. Trial Name: NCT05133531 — Phase 3
Cemdisiran (siRNA) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05133531 — Phase 3

Frequently Asked Questions

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

Does Pozelimab have a positive long-term effect on patients?

"Pozelimab has undergone multiple rounds of testing which have provided evidence for both its efficacy and safety, resulting in a score of 3."

Answered by AI

What are the most recent findings on Pozelimab's efficacy?

"There are currently 21 clinical trials underway testing the efficacy of Pozelimab. Of those, 15 have reached Phase 3. The largest concentration of these studies is located in Bethesda, Maryland; however, there are 926 total research sites for this medication."

Answered by AI

Are there currently any available positions for participants in this experiment?

"The information on clinicaltrials.gov confirms that this study is looking for patients at the moment. This trial was posted on 7/31/2022 and last updated on 5/3/2022. They are hoping to find 124 people total from 2 different locations."

Answered by AI

What are the most frequent treatments that Pozelimab is used for?

"Pozelimab is most commonly used for those who have not previously tried a complement inhibitor. It can also be used to fight conditions such as hemolysis, thrombotic microangiopathies, and paroxysmal nocturnal haemoglobinuria (pnh)."

Answered by AI
~121 spots leftby Mar 2027