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)

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

• Ages 12 to 100 years
• All sexes
• No healthy volunteers
• Child, Adult, Older adult

Trial locations and contacts

36 sites in 5 countries. Contact the study team or a site near you to ask about taking part.

About this study

Official title: A Phase III, Multicenter, Randomized, Placebo Controlled, Double-blind Study to Assess Efficacy and Safety of Crizanlizumab (5 mg/kg) Versus Placebo, With or Without Hydroxyurea/Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Frequent Vaso-Occlusive Crises

Conditions

Sickle Cell Disease

Being tested

Crizanlizumab, Placebo

Keywords

Sickle Cell Disease, SCD, SEG101, Crizanlizumab, Hydroxyurea/ Hydroxycarbamide Therapy, Vaso-Occlusive Crises, Sickle Cell Anemia, blood disorders, hemoglobin, red blood cells, sickle-like shape, mutation in hemoglobin gene, sickle-cell trait, sickle-cell crisis

Medical subject headings

Anemia, Sickle Cell, Vaso-Occlusive Crises, Hematologic Diseases, Sickle Cell Trait

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• CrizanlizumabBiological

  Also called SEG101, Adakveo®, Ryverna®

  Crizanlizumab is supplied in single use 10 mL glass vials at a concentration of 10 mg/mL. One vial contains 100 mg of crizanlizumab. This is a concentrate for solution for IV infusion.

• PlaceboDrug

  Placebo is supplied in single use 10 mL glass vials at a concentration of 0 mg/mL. This is a concentrate for solution for IV infusion.

What is being measured

The primary outcome is the main question the study is designed to answer.

1. Annualized rate of VOCs that are healthcare professional (HCP)-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) in each treatment arm

   Vaso oclusive crisis (VOC) is defined as a pain crisis (acute onset of pain for which there is no other medically determined explanation other than vaso-occlusion) lasting for at least 4 hours which is treated as per local guidelines with standard of care therapy used to treat VOC. Acute chest syndrome (ACS), priapism and hepatic or splenic sequestration will be considered VOC in this study.

   VOCs included are those HCP-managed in a healthcare facility and HCP-managed via remote consultation.

   Annualized rate of VOC events = (Number of VOC events * 365)/(number of days in the observation period).

   Observation period = time from date of randomization to minimum of (last dose date until treatment discontinuation + 27 days, date of initiation or discontinuation of HU/HC or L-Glutamine (or other therapies such as Voxelotor and erythropoietin therapies to treat SCD and/or to prevent/reduce VOCs), date of randomization + 365 days).

   Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted3 Jun 2024
2. Study start24 Oct 2024
3. Primary completion2 Jul 2029 (estimated)
4. Study completion29 Jul 2030 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
• More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
• What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.