A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in P… (NCT04654468) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition
China51 participantsStarted 2021-03-17
Plain-language summary
This study will enroll participants aged 12 years or older with a body weight ≥ 40 kilograms (kg) diagnosed with PNH who have not been previously treated with complement inhibitor therapy. Approximately 50 participants will be treated with Crovalimab for at least 24 weeks.
Who can participate
Age range12 Years
SexALL
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Inclusion Criteria:
* Body weight ≥ 40 kg at screening
* Willingness and ability to comply with all study visits and procedures
* Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry
* LDH Levels ≥ 2x the ULN at screening
* Participants who have at least four transfusions during 12 months prior to screening (documented in the medical record)
* Presence of one or more of the PNH-related signs or symptoms within 3 months of screening
* Vaccination against Neisseria meningitidis serotypes A, C, W, and Y \< 3 years prior to initiation of study treatment (Day 1)
* Vaccination against Haemophilius influenzae type B and Streptococcus pneumonia according to national vaccination recommendations
* For participants receiving other therapies (e.g., immunosuppressants, corticosteroids): stable dose for ≥ 28 days prior to screening and up to the first drug administration
* Adequate hepatic and renal function
* Women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception during the treatment period and for 46 weeks (approximately 10.5 months) after the final dose of crovalimab
* Platelet count ≥30,000 per cubic millimeter (mm\^3) at screening
* ANC \> 500/microlitres (μl) at screening
Exclusion Criteria:
* Current or previous treatment with a complement inhibitor
* History of allogeneic bone marrow transplantation
* History of Neisseria meningitidis infection within 6 months prior to screening and up to f…
What they're measuring
1
Mean Percentage of Participants With Hemolysis Control
Timeframe: From Week 5 up to Week 25
2
Difference in Percentage of Participants With Transfusion Avoidance (TA) From Baseline Through Week 25 and Within 24 Weeks Prior to Screening
Timeframe: 24 Weeks Prior to Screening, Baseline to Week 25