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siRNA

Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With 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
No Placebo-Only Group
Pivotal Trial

Summary

This trial tests a new combination of drugs, pozelimab and cemdisiran, for patients with PNH who are not fully benefiting from their current treatments. The goal is to see if this new combination can better control the breakdown of red blood cells and improve patients' quality of life.

Eligible Conditions
  • Paroxysmal Nocturnal Hemoglobinuria

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Change in Fatigue as Measured by the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale From Baseline to Week 36
Change in Global Health Status (GHS)/QoL Scale Score on the EORTC-QLQ-C30 From Baseline to Week 36
Change in Hemoglobin Levels From Baseline to Week 36
+27 more

Side effects data

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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
2016
Completed Phase 4
~1090
Eculizumab
2009
Completed Phase 4
~1200
Pozelimab
2020
Completed Phase 3
~130
Cemdisiran
2020
Completed Phase 2
~80

Find a Location

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
671 Previous Clinical Trials
385,840 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
284 Previous Clinical Trials
254,914 Total Patients Enrolled
~1 spots leftby Dec 2025
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