A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Pozelimab, Cemdisiran.
- Who it may be relevant to
- Registry conditions: Paroxysmal Nocturnal Hemoglobinuria. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Brazil, Canada, Italy, Poland, South Korea +2
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy
Overview
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)
Detailed description
The treatment period has two parts, a Treatment Period (TP, 28 weeks) and an Extension treatment Period (EP, 52 weeks).
Interventions
- Drug Pozelimab
Administered per the protocol - Drug Cemdisiran
Administered per the protocol
Primary outcome measures
- Percent change in Lactate Dehydrogenase (LDH) during TP [Time frame: From baseline to week 28]
Secondary outcome measures (12)
- Normalization of LDH [Time frame: Through week 52]
- Adequate control of hemolysis (LDH ≤1.5 × ULN) [Time frame: Through week 52]
- Transfusion avoidance [Time frame: Through week 52]
- Hemoglobin stabilization [Time frame: Through week 52]
- Change in hemoglobin from baseline [Time frame: Through week 52]
- Change in fatigue [Time frame: Through week 52]
- Occurrence of all Adverse Events (AEs) [Time frame: Through week 52]
- Severity of all AEs [Time frame: Through week 52]
- Occurrence of all Treatment-Emergent Adverse Events (TEAEs) [Time frame: Through week 52]
- Severity of all TEAEs [Time frame: Through week 52]
- Change from baseline in Total Complement Hemolytic Activity Assay (CH50) [Time frame: Through week 52]
- Concentrations of total pozelimab [Time frame: Through week 52]
Eligibility criteria
Inclusion criteria
- Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
- Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months
- 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
- At least 2 screening LDH values from different visits as described in the protocol
- 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
Exclusion criteria
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
- Body weight <40 kilograms at screening visit
- Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol
- Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol
- Known hereditary complement deficiency
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
South Korea · 5 centers
- Seoul National University Hospital — Seoul
- Severance Hospital — Seoul
- Seoul St. Mary's Hospital - The Catholic University of Korea — Seoul
- Ewha Womans University Mokdong Hospital — Seoul
- Samsung Medical Center — Seoul
Spain · 5 centers
- Hospital Universitario Basurto — Bilbao
- Institut Catala d'Oncologia — Barcelona
- Hospital San Pedro de Alcantara — Cáceres
- Hospital General JM Morales Meseguer — Murcia
- Hospital Universitario de Salamanca — Salamanca
Brazil · 3 centers
- Hospital das Clinicas da FMRP USP — Ribeirão Preto
- Faculdade de Medicina do ABC — Santo André
- Hospital Santa Marcelina — São Paulo
Italy · 3 centers
- Universita Cattolica del Sacro Cuore - Policlinico Universitario A. Gemelli — Rome
- AOU Careggi — Florence
- SC Hematology, AOU Città della Salute e della Scienza di Torino — Torino
Turkey (Türkiye) · 2 centers
- Istanbul University Istanbul Faculty of Medicine — Istanbul
- Ege University — Izmir
Canada · 1 center
- CISSS-CA — Lévis
Poland · 1 center
- In-Vivo Sp. z o.o. — Bydgoszcz
Identifiers
NCT: NCT07154745 · R3918-PNH-2483 · 2024-519709-37-00