Last updated 17 days ago
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
315 patients around the world
Available in Colombia, United States
Study CSEG101A2303 (SPARKLE) is a Phase III, multicenter, randomized, double-blind study
to assess efficacy and safety of crizanlizumab 5 mg/kg versus placebo, with or without
hydroxyurea/ hydroxycarbamide therapy (HU/HC), in Sickle Cell Disease patients aged 12
years and older with frequent vaso-occlusive crises (4-12 events in 12 months prior to
the screening visit).
Participants will be randomized in a 2:1 ratio to the crizanlizumab 5 mg/kg or placebo
treatment arm. Central randomization will be stratified by concomitant HU/HC usage
(yes/no) and region (South America, North America, and sub-Saharan Africa) at baseline.
Novartis Pharmaceuticals
315Patients around the world
This study is for people with
Rare diseases
Sickle cell disease
Requirements for the patient
To 100 Years
All Gender
Medical requirements
- Participants must be aged 12 years and older on the day of signing informed consent. Adolescents include participants aged 12 to 18 years old and adults include participants aged 18 years and older.Confirmed diagnosis of SCD by Hb electrophoresis or high-performance liquid chromatography (HPLC) performed locally or by central laboratory if not available locally.All SCD genotypes are eligible.Experienced 4 to 12 VOCs that are HCP-managed within the 12 months prior to the screening visit. Baseline VOCs are determined by medical history and are required to be documented at source.If the participant is on HU/HC, they must be taking it for at least 6 months and at stable dose for at least 3 months prior to the Screening visit. Participants must plan to continue taking HU/HC at the same dose and schedule until at least the participant has reached 52 weeks of the planned study treatment. Participants who have initiated HU/HC 6-12 months prior to the screening visit must have evidence of insufficient control of acute pain despite initiation. These participants must have a cumulative of 4-12 VOCs in the 12 months prior to the screening period, with at least 2 during the last 6 months while on HU/HC.If receiving erythropoietin stimulating agent, the participant must have been receiving the drug for at least 6 months prior to screening visit. Participants must plan to continue taking the drug at the same dose and schedule until the participant has reached 52 weeks of the planned study treatment.
- Fewer than 4 or more than 12 VOCs that are HCP-managed within the 12 months prior to screening visit as determined by medical history and documented at source.History of stem cell transplant and/or gene therapy.Received blood products within 30 days prior to Week 1 Day 1 dosing.Any documented history of a clinical stroke or intracranial hemorrhage, or an uninvestigated neurologic finding within the past 12 months before screening visit. Silent infarct only present on imaging is not excluded.Participating in a chronic transfusion program and/or planning to undergo an exchange transfusion during the duration of the study. Episodic transfusion in response to worsened anemia or VOC is permitted.Contraindication or hypersensitivity to any drug or metabolites from similar class as study drug or to any excipients of the study drug formulation.History of severe hypersensitivity reaction to other monoclonal antibodies, which in the opinion of the investigator may pose an increased risk of serious infusion reaction.
StudySPARKLE
SponsorNovartis Pharmaceuticals
Study typeInterventional
Conditions
Sickle cell disease
Requirements
To 100 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06439082
