A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors (NCT04434092) — Japan Trial Details

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Investigational drug(s) / intervention(s)

Crovalimab: Dosing depends on body weight. Participants will be dosed as follows: * 5 kg to \< 12 kg: 100 mg IV on Week 1 Day 1 (W1D1); 85 mg SC on Week 1 Day 2 (W1D2) and Q2W from Week 3 until end of study * 12 kg to \< 20 kg: 200 mg IV on W1D1; 85 mg SC on W1D2, Weeks 2, 3 and 4; 170 mg SC, Q2W from Week 5 until end of study * 20 kg to \< 30 kg: 300 mg IV on W1D1; 85 mg SC on W1D2, Weeks 2, 3 and 4; 340 mg SC, Q4W from Week 5 until end of study * 30 kg to \< 40 kg: 400 mg IV on W1D1; 170 mg SC on W1D2, Weeks 2, 3 and 4; 510 mg SC, Q4W from Week 5 until end of study * 40 kg to \< 100 kg: 1000 mg IV on W1D1; 340 mg SC W1D2, Weeks 2, 3 and 4; 680 mg SC, Q4W from Week 5 until end of study * 100 kg: 1500 mg IV on W1D1; 340 mg SC W1D2, Weeks 2, 3 and 4; 1020 mg SC, Q4W from Week 5 until end of study.

Eculizumab: Eculizumab will be administered as specified in the respective arm.

Study 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

Sex

ALL

Min age

2 Years

Max age

—

Healthy volunteers

No

Inclusion Criteria: * 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 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 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

Primary outcome measure(s)

Percentage of Participants With Transfusion Avoidance (TA) — Baseline to Week 25
TA is defined as participants who are packed red blood cell (pRBC) transfusion-free and do not require transfusion per protocol-specified guidelines. 95% Confidence Interval (CI) for percentage of participants with TA was calculated using the Wilson's method with continuity correction. Participants who withdrew before Week 25 were deemed to have had a transfusion. Percentages are rounded off to the nearest single decimal.
Percentage of Participants With Hemolysis Control Measured by LDH — Week 5 to Week 25
A Generalized Estimating Equation (GEE) was used to estimate the population-average percentage of the participants with hemolysis control (measured by LDH ≤1.5 x upper limit of normal (ULN)) from Week 5 to Week 25 taking account of the intra-patient and inter-patient correlation between LDH control statuses across visits. Percentages are rounded off to the nearest single decimal.

Trial sites (63)

Facility	City	Region
Organizacion Medica de Investigacion (OMI)	Ciudad Autonoma Buenos Aires	Argentina
XCEPHO - Centro de Estudos e Pesquisas em Hematologia e Oncologia	Santo André	São Paulo
Beneficencia Portuguesa de Sao Paulo	São Paulo	Brazil
Peking Union Medical College Hospital	Beijing	China
Nanfang Hospital, Southern Medical University	Guangzhou	China
The First Affiliated Hospital, Zhejiang University	Hangzhou	China
Jiangsu Province Hospital	Nanjing	China
Affiliated Hospital of Nantong University	Nantong	China
Huashan Hospital, Fudan University	Shanghai	China
Union Hospital Tongji Medical College Huazhong University of Science and Technology	Wuhan	China
Centre Hospitalier Lyon Sud	Pierre-Bnite	Rhone
Hopital Claude Huriez - CHU Lille	Lille	France
Universitaetsklinkm	Essen	Germany
Universitaetsklinikum Ulm	Ulm	Germany
General Hospital of Thessaloniki G. Papanikolaou	Thessaloniki	Greece
Sapporo Medical University Hospital	Hokkaido	Japan
University of Tsukuba Hospital	Ibaraki	Japan
NTT Medical Center Tokyo	Tokyo	Japan
Tokyo Medical University Hospital	Tokyo	Japan
Vilnius University Hospital Santariskiu Clinics, Public Institution	Vilnius	Lithuania
Hospital Raja Permaisuri Bainun	Ipoh	Perak
Hospital Pulau Pinang	George Town	Pulau Pinang
Hospital Ampang	Ampang	Selangor
Hospital Tengku Ampuan Afzan	Kuantan	Malaysia
Instituto Nacional de Ciencias Medicas y Nutricion Salvador Zubiran	Mexico City	Mexico CITY (federal District)
Global Trial Research Center S.A. de C.V.	Monterrey	Nuevo León
Amsterdam UMC, Locatie AMC	Amsterdam	Netherlands
Mary Mediatrix Medical Center	Lipa City	Philippines
Philippine General Hospital	Manila	Philippines
St Lukes Medical Center	Quezon	Philippines
Szpital Uniwersytecki nr2 im. dr J. Biziela	Bydgoszcz	Poland
Uniwersyteckie Centrum Kliniczne	Gdansk	Poland
Samodzielny Publiczny Szpital Kliniczny nr 1	Lublin	Poland
Pratia Poznan	Skórzewo	Poland
MTZ Clinical Research Powered by Pratia	Warsaw	Poland
Centro Hospitalar do Baixo Vouga E.P.E. - Hospital de Aveiro	Aveiro	Portugal
Instituto Portugues Oncologia de Lisboa Francisco Gentil EPE	Lisbon	Portugal
Centro Hospitalar do Porto - Hospital de Santo António	Porto	Portugal
Spitalul Universitar de Urgenta Bucuresti	Bucharest	Romania
Spitalul Clinic Municipal Filantropia Craiova	Craiova	Romania

+ 23 more sites — see the full list on the official registry below.

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