Sickle Cell Disease (SCD)
Conditions
Sponsors
Roche Farma S. A. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Signed ICF or Assent Form (as determined by patient’s age and individual site and country standards) - Age >=12 to =40 kg - Male or female with confirmed diagnosis of HbSS (SCD genotype of sickle cell anemia) or HbSß0 (SCD genotype of sickle cell beta zero thalassemia) - Two or more (>=2) to =65 years) no F.1.3.1 Number of subjects for this age range
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 6 months after the final dose of study treatment - Known HIV infection with documented CD4 count <200 cells/microliter within 24 weeks prior to screening - History of N. meningitidis infection within the prior 6 months
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To evaluate the efficacy of crovalimab compared with placebo;Secondary Objective: - To evaluate the efficacy of crovalimab compared with placebo - To evaluate the safety and tolerability of crovalimab compared with placebo - To evaluate the pharmacokinetics of crovalimab - To evaluate the immune response to crovalimab;Primary end point(s): 1. Annualized rate of medical facility VOEs (AVR);Timepoint(s) of evaluation of this end point: 1. Up to 48 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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;Timepoint(s) of evaluation of this end point: 1-5. Baseline to Week 49 6. Baseline to Week 49 7. Up to Week 49 8-9. Baseline to Week 49 10. At Week 49 11. Baseline to Week 49 | — |
Countries
Brazil, France, Italy, Spain, Turkey
Contacts
Public ContactTrial Information Support Line-TISL
Roche Farma S.A
Outcome results
None listed