← Back to Search

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

Side effects data

From 2024 Phase 2 & 3 trial • 10 Patients • NCT04209634
20%
Pyrexia
20%
Rhinitis
20%
Iron deficiency
20%
Urticaria
10%
Proteinuria
10%
Headache
10%
Abdominal pain
10%
Alopecia
10%
Hepatic enzyme increased
10%
Dehydration
10%
Tonsillitis
10%
Immunisation reaction
10%
Hypokalaemia
10%
Dermatitis contact
10%
Vomiting
10%
Blood glucose increased
10%
Gingival bleeding
10%
Haematuria
10%
Acarodermatitis
10%
Metabolic acidosis
10%
Alopecia areata
10%
Constipation
10%
Nasopharyngitis
10%
Blood uric acid increased
10%
Anaemia folate deficiency
100%
80%
60%
40%
20%
0%
Study treatment Arm
Pozelimab

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 3
~130
Cemdisiran
2020
Completed Phase 2
~80

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
628 Previous Clinical Trials
382,240 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
266 Previous Clinical Trials
252,310 Total Patients Enrolled
~1 spots leftby Jun 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top