Status:

RECRUITING

A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently

Lead Sponsor:

Regeneron Pharmaceuticals

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

Eligibility:

All Genders

18+ years

Phase:

PHASE3

Brief Summary

This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are looking for a better way to treat Paroxysmal...

Detailed Description

The treatment period has two parts, a Treatment Period (TP, 28 weeks) and an Extension treatment Period (EP, 52 weeks).

Eligibility Criteria

Key Inclusion Criteria:

  1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
  2. Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months
  3. LDH persistently > 1.5 × Upper Limit of Normal (ULN) in the previous 6 months that the Principal Investigator (PI) attributes is due to intravascular hemolysis
  4. At least 2 screening LDH values from different visits as described in the protocol
  5. Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol and agreement to continue to remain up to date with these vaccinations during the study

Key Exclusion Criteria:

  1. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
  2. Body weight <40 kilograms at screening visit
  3. Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol
  4. Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol
  5. Known hereditary complement deficiency

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Key Trial Info

Start Date :

March 31 2026

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

September 23 2031

Estimated Enrollment :

35 Patients enrolled

Trial Details

Trial ID

NCT07154745

Start Date

March 31 2026

End Date

September 23 2031

Last Update

April 14 2026

Active Locations (7)

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Page 1 of 2 (7 locations)

1

AOU Careggi

Florence, Tuscany, Italy, 50134

2

SC Hematology, AOU Città della Salute e della Scienza di Torino

Torino, Italy, 10126

3

In-Vivo Sp. z o.o.

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-048

4

Seoul National University Hospital

Seoul, South Korea, 03080