Recruiting
Phase 3

Pozelimab & Cemdisiran

Sponsor:

Regeneron Pharmaceuticals

Code:

NCT07154745

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Pozelimab

Cemdisiran

Study Details

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 Nocturnal Hemoglobinuria (PNH).

The aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has been not well controlled even after taking other complement component 5 (C5) inhibitors, eculizumab/eculizumab biosimilar, ravulizumab or crovalimab.

The study is looking at several other research questions, including:

  • What side effects may happen from taking the study drugs?
  • How much of the study drugs are in the blood at different times?
  • Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Study ID

NCT07154745

Start date

Jun 11, 2026

Status verified date

Jul, 2026

Completion date

Dec 4, 2031

Anticipated

Primary completion date

Jan 18, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

35 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Pozelimab + Cemdisiran Combo

Interventions

Pozelimab

Administered per the protocol

Cemdisiran

Administered per the protocol

Primary outcome measure

  • Percent change in Lactate Dehydrogenase (LDH) during TP [ Time Frame: From baseline to week 28 ]

Central Contacts and Locations

Central contacts

Clinical Trials Administrator

844-734-6643clinicaltrials@regeneron.com

Locations

CISSS-CA

Recruiting

Lévis, Quebec, Canada, G6V 3Z1

More Information

Sponsor

Regeneron Pharmaceuticals

Last update posted

Aug 5, 2026

Last verified

Jul, 2026

Keywords

  • PNH

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Regeneron Pharmaceuticals on 2026-08-05.