Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
Summary
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.
Timeline
- Start
- 2019-07-26
- Primary completion
- 2022-08-31
- Completion
- 2026-11-05
Outcome
Missed primary endpoint
paper The primary analysis showed no significant difference in efficacy between crizanlizumab and placebo. PMID 40088922 ↗
registry analysis (superiority test); Crizanlizumab (SEG101) at 5.0 mg/kg vs Placebo; p > 0.999; Rate ratio 1.08 (95% CI 0.76 to 1.55); negative binomial regression model NCT03814746 ↗
registry analysis (superiority test); Crizanlizumab (SEG101) at 7.5 mg/kg vs Placebo; p > 0.999; Rate ratio 0.89 (95% CI 0.62 to 1.27); negative binomial regression model NCT03814746 ↗
Publications
- Abboud MR, Cancado RD, De Montalembert M, Smith WR, Rimawi H, Voskaridou E, Guvenc B, Ataga KI, Keefe D, Grosch K, Watson J, Reshetnyak E, Nassin ML, Dei-Adomakoh Y. Crizanlizumab with or without hydroxyurea in patients with sickle cell disease (STAND): primary analyses from a placebo-controlled, randomised, double-blind, phase 3 trial. Lancet Haematol. 2025 Apr;12(4):e248-e257. doi: 10.1016/S2352-3026(24)00384-3. Epub 2025 Mar 12.
- Obadina M, Wilson S, Derebail VK, Little J. Emerging Therapies and Advances in Sickle Cell Disease with a Focus on Renal Manifestations. Kidney360. 2023 Jul 1;4(7):997-1005. doi: 10.34067/KID.0000000000000162. Epub 2023 May 31.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Crizanlizumab | Monoclonal antibody | 5 mg/kg | Intravenous |
| Subject | Crizanlizumab | Monoclonal antibody | 7.5 mg/kg | Intravenous |