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New treatment option for Paroxysmal Nocturnal Hemoglobinuria
Official title 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
ClinicalTrials.gov ID: NCT05744921
What this study is testing
What is Pozelimab?
Pozelimab is an investigational medicine, being studied as a potential treatment for paroxysmal nocturnal hemoglobinuria.
Also referred to as REGN3918.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH).
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Key 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...
- Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol. Patients...
- Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as...
- Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
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...
- Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment...
- Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab...
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to...
See the full eligibility criteria
- Key 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...
- 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
- Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
- Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol
- LDH level ≥2 × upper limit of normal (ULN) at the screening visit
- Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol Key
- 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
- 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...
- 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
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- 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...
- Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening
- Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol
- Known hereditary complement deficiency
- Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
- 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)...
The study team makes the final eligibility decision.
Where it's taking place
- Toronto, Ontario, Canada
- Medellín, Antioquia, Colombia
- Budapest, Hungary
- Kochi, Kerala, India
- Mumbai, Maharashtra, India
- New Delhi, National Capital Territory of Delhi, India
- Chandigarh, India
- Jaipur, India
- Florence, Firenze, Italy
- Torino, Italy
- Nagoya, Aichi-ken, Japan
- Tsukuba, Ibaraki, Japan
- Amman, Jordan
- Kuantan, Pahang, Malaysia
- Kota Kinabalu, Sabah, Malaysia
- Ampang, Selangor, Malaysia
- Lima, Peru
- Quezon City, National Capital Region, Philippines
- Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland
- Gdansk, Pomeranian Voivodeship, Poland
+ 20 more site(s).
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 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Toronto, Ontario, Canada; Medellín, Antioquia, Colombia; Budapest, Hungary; Kochi, Kerala, India; Mumbai, Maharashtra, India; New Delhi, National Capital Territory of Delhi, India and 34 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 ClinicalTrials.gov; the study team decides eligibility.