Pozelimab for Paroxysmal Nocturnal Hemoglobinuria

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Paroxysmal Nocturnal HemoglobinuriaPozelimab - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study 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.

Eligible Conditions
  • Paroxysmal Nocturnal Hemoglobinuria

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

4 Primary · 33 Secondary · Reporting Duration: Between week 8 and week 26, inclusive

Week 24
Proportion of patients with adequate control of hemolysis
Week 24
Proportion of patients with normalization of LDH
Week 26
Percentage of days with LDH ≤1.5 × ULN
Week 26
Normalization of LDH
Week 26
Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30)
Week 24
Proportion of patients with hemoglobin stabilization
Week 24
Number of units of RBC transfusion per protocol algorithm from day 1 through week 24
Proportion of patients with transfusion avoidance
Rate of RBC transfusion per protocol algorithm from day 1 through week 24
Week 26
Transfusion avoidance
Week 24
Lactate Dehydrogenase
Week 26
Change in fatigue as measured by the FACIT-Fatigue Scale
Change in global health status (GHS)/QoL scale score on the EORTC-QLC-C30
Change in hemoglobin levels
Change in total CH50
Percent change in lactate dehydrogenase (LDH)
Percent change in total CH50
Week 26
Hemoglobin stabilization
Week 26
Breakthrough hemolysis
Week 26
Adequate control of hemolysis
Maintenance of adequate control of hemolysis
Week 26
Number of units of RBC transfused per protocol algorithm from day 1 through week 26
Rate of RBC transfused per protocol algorithm from day 1 through week 26
Week 24
Proportion of patients with breakthrough hemolysis
Up to 20 weeks
Concentrations of cemdisiran in plasma
Concentrations of total cemdisiran in plasma
Up to 24 weeks
Incidence and severity of TEAE leading to treatment discontinuation
Up to 26 weeks
Incidence and severity of TEAEs leading to treatment discontinuation
Incidence and severity of treatment emergent serious adverse events (SAEs)
Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
Up to 34 weeks
Concentrations of total ravulizumab in serum
Up to 52 weeks
Concentration of total C5 in plasma
Concentrations of total pozelimab in serum
Therapeutic procedure
Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
Up to Week 26
Time to first LDH ≤1.0 × ULN
Time to first LDH ≤1.5 × ULN

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

Eculizumab
20%Nasopharyngitis
17%Headache
10%Cough
10%Upper respiratory tract infection
9%Abdominal pain
9%Oropharyngeal pain
9%Nausea
9%Chest pain
8%Influenza like illness
7%Dizziness
7%Diarrhoea
6%Fatigue
6%Dyspnoea
5%Constipation
5%Musculoskeletal pain
4%Vomiting
4%Pain in extremity
4%Rhinitis
3%Pyrexia
3%Anaemia
2%Haemolysis
1%Palpitations
1%Cholelithiasis
1%Pyelonephritis acute
This histogram enumerates side effects from a completed 2022 Phase 3 trial (NCT03056040) in the Eculizumab ARM group. Side effects include: Nasopharyngitis with 20%, Headache with 17%, Cough with 10%, Upper respiratory tract infection with 10%, Abdominal pain with 9%.

Trial Design

2 Treatment Groups

Ravulizumab
1 of 2
Pozelimab and Cemdisiran
1 of 2

Experimental Treatment

148 Total Participants · 2 Treatment Groups

Primary Treatment: Pozelimab · No Placebo Group · Phase 3

Ravulizumab
Drug
Experimental Group · 1 Intervention: Ravulizumab · Intervention Types: Drug
Pozelimab and CemdisiranExperimental Group · 2 Interventions: Pozelimab, Cemdisiran · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
FDA approved
Pozelimab
Not yet FDA approved
Cemdisiran
Not yet FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: between week 8 and week 26, inclusive

Who is running the clinical trial?

Regeneron PharmaceuticalsLead Sponsor
546 Previous Clinical Trials
199,667 Total Patients Enrolled
Clinical Trial ManagementStudy DirectorRegeneron Pharmaceuticals
229 Previous Clinical Trials
88,710 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 4 Total Inclusion Criteria

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