sickle cell anaemia Sickle Cell Disease (SCD)
Conditions
Interventions
The investigational medicinal products (IMPs) for this study are crovalimab and
placebo.
The first dose of the study drug will be given as an IV infusion (into the
vein) and will last approximatel
Sponsors
Roche Nederland B.V.
Eligibility
Age
18 Years to 64 Years
Inclusion criteria
Inclusion criteria: - Signed ICF - Age >= 12 to = 40 kg - Male or female with confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSβ0 (SCD genotype of sickle cell beta zero thalassemia) - Two or more (>= 2) to
Exclusion criteria
Exclusion criteria: - History of hematopoietic stem cell transplant - Participating in a chronic transfusion program and/or planning on undergoing an exchange transfusion during the duration of the study - History of hypersensitivity, allergic, or anaphylactic reactions to any ingredient contained in the study treatment - Received active treatment on another investigational trial within 28 days (or within five half-lives of that agent, whichever is greater) prior to screening visit, or plans to participate in another investigational drug trial - Hemoglobin = 38 degrees Celsius) within 7 days before the first drug administration - Immunized with a live attenuated vaccine within 1 month before first drug administration - Pregnant or breastfeeding, or intending to become pregnant during the study or within 46 weeks (approximately 10.5 months) after the final dose of study treatment - Known HIV infection with documented CD4 count
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary efficacy objective for this study is to evaluate the efficacy of crovalimab compared with placebo on the basis of the following endpoint: Annualized rate of medical facility VOEs (AVR). | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Annualized rate of home VOE captured by patient report 2. Annualized rate of uncomplicated medical facility VOE 3. Annualized rate of acute chest syndrome (ACS) 4. Annualized rate of days hospitalized for medical facility VOE 5. Annualized rate of days hospitalized for treatment of non-VOE complications of SCD 6. Change in hematologic measures from baseline to Week 49 7. Time to first medical facility VOE from randomization 8. Change in urinary albumin-creatinine ratio from baseline to Week 49 9. Change from baseline to Week 49 in tricuspid regurgitant jet velocity (TRV) 10. Proportion of patients with TRV >2.5 m/s at Week 49 11. Change from baseline to Week 49 in Patient-Reported Outcomes Measurement Information System (PROMIS)-Fatigue score in adults Please refer to Protocol Section 2. Objectives and Endpoints for Exploratory efficacy, Safety, Phamacokinetic, Immunogenicity and Biomarker objectives. | — |
Countries
Netherlands
Outcome results
None listed