A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition

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About the study

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.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Inclusion Criteria:


  1. Body weight ≥ 40 kg at screening
  2. Willingness and ability to comply with all study visits and procedures
  3. Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry
  4. LDH Levels ≥ 2x the ULN at screening
  5. Participants who have at least four transfusions during 12 months prior to screening (documented in the medical record)
  6. Presence of one or more of the PNH-related signs or symptoms within 3 months of screening
  7. Vaccination against Neisseria meningitidis serotypes A, C, W, and Y < 3 years prior to initiation of study treatment (Day 1)
  8. Vaccination against Haemophilius influenzae type B and Streptococcus pneumonia according to national vaccination recommendations
  9. For participants receiving other therapies (e.g., immunosuppressants, corticosteroids): stable dose for ≥ 28 days prior to screening and up to the first drug administration
  10. Adequate hepatic and renal function
  11. 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
  12. Platelet count ≥30,000 per cubic millimeter (mm^3) at screening
  13. ANC > 500/microlitres (μl) at screening

EXCLUSION CRITERIA

Exclusion Criteria:


  1. Current or previous treatment with a complement inhibitor
  2. History of allogeneic bone marrow transplantation
  3. History of Neisseria meningitidis infection within 6 months prior to screening and up to first drug administration
  4. Known or suspected immune or hereditary complement deficiency
  5. Known HIV infection with cluster of differentiation 4 (CD4) count < 200 cells/µl within 24 weeks prior to screening
  6. Infection requiring hospitalization or treatment with IV antibiotics within 28 days prior to screening and up to the first drug administration, or oral antibiotics within 14 days prior to screening and up to the first drug administration
  7. Active systemic bacterial, viral, or fungal infection within 14 days before first drug administration
  8. Presence of fever (≥ 38˚C) within 7 days before the first drug administration
  9. Splenectomy < 6 months before screening
  10. History of malignancy within 5 years prior to screening and up to the first drug administration
  11. Pregnant or intending to become pregnant during the study or within 46 weeks (10.5 months) after the final dose of study treatment
  12. Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within 28 days of screening or within 5 half-lives of that investigational product, whichever is greater

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Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply


Contact the study center to learn if this study is a good match for you.

Study’s details


Contition
Paroxysmal Nocturnal Hemoglobinuria
Age (in years)
12+
Phase
PHASE3
Participants needed
51
Est. Completion Date
Dec 31, 2026
Treatment type
INTERVENTIONAL

Sponsor
Hoffmann-La Roche
ClinicalTrials.gov identifier
NCT04654468
Study number
YO42311

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