Status:

ACTIVE_NOT_RECRUITING

A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors

Lead Sponsor:

Hoffmann-La Roche

Collaborating Sponsors:

Chugai Pharmaceutical

Conditions:

Paroxysmal Nocturnal Hemoglobinuria

Eligibility:

All Genders

2+ years

Phase:

PHASE3

Brief Summary

A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH who have not been previously treated with complement inhibitor therapy.

Eligibility Criteria

Inclusion

  • Body weight ≥ 40 kg at screening (pediatric participants with body weight \< 40 kg)
  • Willingness and ability to comply with all study visits and procedures
  • Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry
  • LDH level ≥ 2x ULN at screening (as per local assessment)
  • Vaccination against Neisseria meningitidis serotypes A, C, W, and Y\< 3 years prior to initiation of study treatment; or, if not previously done, vaccination administered no later than one week after the first drug administration
  • Women of childbearing potential: agreement to remain abstinent or use contraception during the treatment period and for 10.5 months after the final dose of crovalimab or for 3 months after the final dose of eculizumab (or longer if required by the local product label)

Exclusion

  • 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 first study drug administration
  • History of myelodysplastic syndrome with Revised International Prognostic Scoring System (IPSS-R) prognostic risk categories of intermediate, high and very high
  • Pregnant or breastfeeding, or intending to become pregnant during the study, within 10.5 months after the final dose of crovalimab, or 3 months after the final dose of eculizumab (or longer if required by the local product label)
  • 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
  • Concurrent disease, treatment, procedure or surgery, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose any additional risk for the participant, or would, in the opinion of the Investigator, preclude the participant's safe participation in and completion of the study
  • Splenectomy \< 6 months before screening
  • Positive for Active Hepatitis B and C infection (HBV/HCV)
  • History of or ongoing cryoglobulinemia at screening

Key Trial Info

Start Date :

October 8 2020

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

September 30 2027

Estimated Enrollment :

210 Patients enrolled

Trial Details

Trial ID

NCT04434092

Start Date

October 8 2020

End Date

September 30 2027

Last Update

November 10 2025

Active Locations (63)

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Page 1 of 16 (63 locations)

1

Organizacion Medica de Investigacion (OMI)

Ciudad Autonoma Buenos Aires, Argentina, C1015ABO

2

Centro Integrado de Oncologia de Curitiba

Curitiba, Paraná, Brazil, 80810-050

3

*X*CEPHO - Centro de Estudos e Pesquisas em Hematologia e Oncologia

Santo André, São Paulo, Brazil, 09060-870

4

Beneficencia Portuguesa de Sao Paulo

São Paulo, Brazil, 01321-00