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siRNA

Pozelimab for Paroxysmal Nocturnal Hemoglobinuria (ACCESS 2 Trial)

Phase 3
Waitlist Available
Research Sponsored by Regeneron Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 8 (day 57) through week 36
Awards & highlights

ACCESS 2 Trial Summary

This trial is testing a combination therapy of pozelimab and cemdisiran to see if it is more effective than the standard-of-care treatment for PNH (eculizumab or ravulizumab) in reducing transfusion requirements and hemolysis.

Eligible Conditions
  • Paroxysmal Nocturnal Hemoglobinuria

ACCESS 2 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 8 (day 57) through week 36
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 8 (day 57) through week 36 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
Change in Physical Function (PF) score on the European organization for research and treatment of cancer quality-of-Life questionnaire Core 30 Items (EORTC-QLQ-C30)
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale
Change in global health status (GHS)/QoL scale score on the EORTC-QLQ-C30
+20 more

ACCESS 2 Trial Design

2Treatment groups
Experimental Treatment
Group I: Pozelimab and CemdisiranExperimental Treatment4 Interventions
Randomized 1:1
Group II: Anti-C5 standard-of-careExperimental Treatment2 Interventions
Randomized 1:1
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
2021
Completed Phase 4
~1080
Eculizumab
2021
Completed Phase 4
~1190
Pozelimab
2020
Completed Phase 2
~120
Cemdisiran
2020
Completed Phase 2
~80

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
615 Previous Clinical Trials
379,845 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
259 Previous Clinical Trials
251,042 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 available positions for patients in this clinical trial?

"The answer is affirmative, according to information on clinicaltrials.gov. This trial was originally posted on March 31st, 2020 and updated most recently on January 8th, 2020. They are looking for a total of 140 patients from 1 site."

Answered by AI

Does this trial break new ground in the medical field?

"Alexion Pharmaceuticals sponsored the first Pozelimab trial in 2016. That initial study had 270 participants and completed Phase 3 drug approval. To date, there have been 53 trials completed with 30 more currently underway across 265 cities and 45 countries."

Answered by AI

Does the medical literature on Pozelimab date back far?

"30 trials for Pozelimab are ongoing, with 20 in Phase 3. Trials for this medication are being conducted all over the world, though many of them are based in Bethesda, Maryland. There are a total of 1339 medical sites running these clinical studies."

Answered by AI

What are the situations in which Pozelimab is indicated?

"Neuromyelitis optica is most commonly treated with Pozelimab, which can also help patients manage hemolysis, thrombotic microangiopathies, and disease activity."

Answered by AI

What is the Pozelimab drug's official standing with the FDA?

"Pozelimab has undergone multiple rounds of testing and there is some evidence supporting its efficacy, so it received a score of 3."

Answered by AI

How many test subjects are involved in this clinical research?

"Yes, this is an ongoing study that has updated its information as recently as January 28th, 2022. It was first posted on March 31st of the same year. The researchers conducting this trial are looking for a total of 140 patients at 1 location."

Answered by AI
~1 spots leftby Apr 2025