At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Pozelimab (drug), Cemdisiran (drug)
- How long the study runs
- Study runs about 67 months (dates as stated)
- About the drug or intervention
- Pozelimab — drug: Administered per the protocol · Cemdisiran — drug: Administered per the protocol
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 18 Years and over
- Who
- All
- Number of participants
- 202
- Started
- 2023-03-07
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for paroxysmal nocturnal hemoglobinuria
- • Phase3 - 202 participants
- • This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran
Who can take part?
- • Ages 18 Years and over
- • Diagnosed with paroxysmal nocturnal hemoglobinuria
Where?
- • Leeds - Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years and over
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ per protocol
What the study is looking for
- ✓Patients Entering from the Parent Study
- ✓Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations...
- ✓Patients Entering with C5 polymorphism
- ✓Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg,...
- ✓Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
Who cannot take part
- ✗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...
- ✗Patients Entering with C5 polymorphism
- ✗Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except...
- ✗Receipt of an organ transplant, history of bone marrow transplantation or other blood transplant
See the full criteria
Where Is This Study? (1 UK site)
Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology
Leeds LS97TF, United Kingdom
How to Get in Touch
Clinical Trials Administrator
Sponsor contactCONTACT
