Myelodysplastic Syndrome Clinical Trial

A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

Summary

The primary objective of the study is:

To evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 24-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor therapy

The secondary objectives of the study are to:

Evaluate the effect of pozelimab and cemdisiran combination treatment versus ravulizumab treatment on the following:

Measures of hemolysis
Transfusion parameters
Hemoglobin levels
Fatigue as assessed by Clinical Outcome Assessments (COAs)
Health-related quality of life (HRQoL) as assessed by COAs
Safety and tolerability
Complement activation
To assess the concentrations of total pozelimab and total ravulizumab in serum and total cemdisiran and total complement factor 5 (C5) protein in plasma
To assess the immunogenicity of pozelimab and cemdisiran

View Eligibility Criteria

Eligibility Criteria

Key Inclusion Criteria:

Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
LDH level ≥2 × ULN at the screening visit
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

Key Exclusion Criteria:

Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
Body weight <40 kilograms at screening visit
Planned use of any complement inhibitor therapy other than study drugs during the treatment period
Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine (when available) within 3 years prior to the screening visit as described in the protocol.
Any contraindication for receiving Neisseria meningitidis vaccinations (serotypes ACWY and B).
Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, where available, or national guidelines/local practice, or if necessary when administration of the first dose of the vaccination is less than 2 weeks prior to study treatment initiation)
Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

190

Study ID:

NCT05133531

Recruitment Status:

Recruiting

Sponsor:

Regeneron Pharmaceuticals

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There are 48 Locations for this study

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The Oncology Institute of Hope and Innovation
Whittier California, 90603, United States
Toronto General Hospital
Toronto Ontario, M5G 2, Canada
George Papanikolaou Hospital
Thessaloniki , 570 1, Greece
Semmelweis Egyetem
Budapest , 1089, Hungary
Fondazione Policlinico Universitario A Gemelli
Roma Lazio, 168, Italy
Azienda Ospedaliera Città della Salute e della Scienza di Torino
Torino Piemonte, 10126, Italy
Azienda Ospedaliera Universitaria Careggi
Firenze , 50134, Italy
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya Aichi, 466-8, Japan
Ogaki Municipal Hospital
Ogaki city Gifu, 503-8, Japan
University of Tsukuba Hospital
Tsukuba-shi Ibaraki, 305-8, Japan
Panasonic Health Insurance Organization Matsushita Memorial Hospital
Moriguchi-city Osaka, 570-8, Japan
NTT Medical Center Tokyo
Shinagawa-ku Tokyo, 141-8, Japan
Pusan National University Hospital
Busan , 49241, Korea, Republic of
Ajou University Medical Center
Gyeonggi-do , 16499, Korea, Republic of
Gachon University Gil Medical Center
Incheon , 21565, Korea, Republic of
Korea University Hospital
Seoul , 02841, Korea, Republic of
Severance Hospital
Seoul , 03722, Korea, Republic of
Samsung Medical Center - PPDS
Seoul , 06351, Korea, Republic of
Ewha Womans University Mokdong Hospital
Seoul , 7895, Korea, Republic of
St. Vincent Hospital
Suwon , 16247, Korea, Republic of
Hospital Tengku Ampuan Afzan
Kuantan Pahang, 25200, Malaysia
Hospital Ampang
Ampang Selangor, 68000, Malaysia
Queen Elizabeth Hospital - Kota Kinabalu
Kota Kinabalu , 88586, Malaysia
Hospital Universitario Dr. Jose Eleuterio González
Monterrey Nuevo León, 64460, Mexico
Klinika Hematologii, Szpital Uniwersytecki Nr 2 im. Jana Biziela w Bydgoszczy
Bydgoszcz , 85-16, Poland
Uniwersyteckie Centrum Kliniczne
Gdansk , 80-21, Poland
Instytut Hematologii i Transfuzjologii
Warszawa , 02-77, Poland
Prof Dr I Chiricuta Institute of Oncology
Cluj-Napoca Cluj, 40001, Romania
Filantropia Municipal Clinical Hospital
Craiova Dolj, 20014, Romania
Targu-Mures Emergency Clinical County Hospital
Targu Mures Mures, 54013, Romania
National University Hospital
Singapore , 11907, Singapore
Hospital Universitario Basurto
Bilbao Vizcaya, 48013, Spain
Hospital Clinic de Barcelona
Barcelona , 8036, Spain
Changhua Christian Hospital
Changhua City , 50006, Taiwan
Hualien Tzu Chi Hospital
Hualien City , 97002, Taiwan
Kaohsiung Medical University - Chung-Ho Memorial Hospital
Kaohsiung , 807, Taiwan
China Medical University Hospital - PPDS
Taichung , 40447, Taiwan
Tri-Service General Hospital
Taipei , 11490, Taiwan
Chang Gung Hospital
Taoyuan City , 33305, Taiwan
National Taiwan University Hospital
Tapei City , 10022, Taiwan
Rajavithi Hospital
Ratchathewi Krung Thep Maha Nakhon-Bangkok, 10400, Thailand
Songklanagarind Hospital Prince of Songkla University
Hat Yai Songkhla, 90110, Thailand
King Chulalongkorn Memorial Hospital
Bangkok , 10330, Thailand
Chiang Mai University
Chiang Mai , 50200, Thailand
Srinagarind Hospital
Khon Kaen , 40000, Thailand
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul , 34418, Turkey
Ege Universitesi Tip Fakultesi Hastanesi
Izmir , 35100, Turkey
St James University Hospital
Leeds , LS9 7, United Kingdom

How clear is this clinincal trial information?

Study is for people with:

Myelodysplastic Syndrome

Phase:

Phase 3

Estimated Enrollment:

190

Study ID:

NCT05133531

Recruitment Status:

Recruiting

Sponsor:


Regeneron Pharmaceuticals

How clear is this clinincal trial information?

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