A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (ACCESS-2) | Aplastic Anemia & MDS International Foundation Return to top.

ClinicalTrials.gov Identifier:

NCT05131204

Company

Regeneron Logo

Dates

Start: March 2022
End: September 2026

Official Title

A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (ACCESS-2)

Purpose

The primary objective of the study is to evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy.

A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (ACCESS-2)

Clinical Trial: NCT05131204

Instructions

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.

Status: 
Recruiting
Phase: 
Phase 3
Gender: 
Female
Male
Age Group: 
18 years and older
Accepts Healthy Volunteers: 
No
Inclusion Criteria: 
  1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
  2. Treated with eculizumab or ravulizumab prior to screening visit as described in the protocol Note: Biosimilars are not permitted, unless approved by the Sponsor
Exclusion Criteria: 
  1. Patients with a screening LDH >1.5 × ULN who have not taken their C5 inhibitor within the labeled dose interval at the dose prior to the screening LDH assessment
  2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
  3. Body weight < 40 kilograms at screening visit
  4. Any use of complement inhibitor therapy other than eculizumab or ravulizumab in the 26 weeks prior to the screening visit or planned use during the study with the exception of study treatments
  5. Not meeting meningococcal vaccination requirements for eculizumab or ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit.
  6. Any contraindication for receiving Neisseria meningitidis vaccination.
  7. Positive for hepatitis B, and/ or hepatitis C as described in the protocol
  8. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
  9. Participation in another interventional clinical study (except R3918-PNH-2021) or use of any experimental therapy within 30 days before screening visit or within 5 half-lives of that investigational product, whichever is greater, with the exception of eculizumab or ravulizumab.
  10. Patients with functional or anatomic asplenia

Note: Other protocol-defined inclusion/ exclusion criteria apply

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