A Study in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment with Pozelimab + Cemdisiran Combination Therapy is and How Well it Works.
EU CTIS ID: 2023-510336-36-00
What this study is testing
The primary objective of the study is to describe the long-term safety, tolerability, and efficacy of pozelimab + cemdisiran combination therapy in patients with PNH.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Patients Entering from the Parent Study: Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021[NCT05133531]), including the post-Open-label treatment period (OLTP) transition period, if applicable.
- Patients Entering from the Parent Study: Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol
- Patients Entering with C5 polymorphism: Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
- Patients Entering with C5 polymorphism: Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
- Patients Entering with C5 polymorphism: Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
- Patients Entering with C5 polymorphism: LDH level ≥2 × upper limit of normal (ULN) at the screening visit
You likely can't join if
- Patients Entering from the Parent Study: Significant protocol deviation(s) in the parent study based on the investigator’s judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
- Patients Entering with C5 polymorphism: Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
- Patients Entering from the Parent Study: Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study
- Patients Entering with C5 polymorphism: Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
- Patients Entering with C5 polymorphism: Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- Patients Entering with C5 polymorphism: Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol
See the full eligibility criteria
- Patients Entering from the Parent Study: Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021[NCT05133531]), including the post-Open-label treatment period (OLTP) transition period, if applicable.
- Patients Entering from the Parent Study: Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol
- Patients Entering with C5 polymorphism: Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
- Patients Entering with C5 polymorphism: Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
- Patients Entering with C5 polymorphism: Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
- Patients Entering with C5 polymorphism: LDH level ≥2 × upper limit of normal (ULN) at the screening visit
- Patients Entering with C5 polymorphism: Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol
- Patients Entering from the Parent Study: Significant protocol deviation(s) in the parent study based on the investigator’s judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
- Patients Entering with C5 polymorphism: Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
- Patients Entering from the Parent Study: Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study
- Patients Entering with C5 polymorphism: Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
- Patients Entering with C5 polymorphism: Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- Patients Entering with C5 polymorphism: Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol
- Patients Entering with C5 polymorphism: Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening
- Patients Entering with C5 polymorphism: Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol
- Patients Entering with C5 polymorphism: Known hereditary complement deficiency
- Patients Entering with C5 polymorphism: Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
The study team makes the final eligibility decision.
Where it's taking place
- Colombia
- Peru
- Singapore
- China
- Korea, Republic of
- United Kingdom
- Brazil
- Thailand
- South Africa
- Taiwan
- India
- United States
- Malaysia
- Japan
- Turkey
- Mexico
- Canada
- Jordan
- Philippines
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Colombia; Peru; Singapore; China; Korea, Republic of; United Kingdom and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.