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)

NCT ID: NCT06439082

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

315 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-24

Study Completion Date

2030-04-19

Brief Summary

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A phase III, multi-center, 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.

Detailed Description

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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.

Conditions

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Sickle Cell Disease

Keywords

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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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Double-blind Study

Study Groups

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Crizanlizumab (SEG101) at 5.0 mg/kg

Participants receive Crizanlizumab (SEG101) at 5.0 mg/kg and standard of care.

Group Type EXPERIMENTAL

Crizanlizumab

Intervention Type BIOLOGICAL

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.

Placebo

Participants receive the placebo drug and standard of care.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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.

Interventions

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Crizanlizumab

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.

Intervention Type BIOLOGICAL

Placebo

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.

Intervention Type DRUG

Other Intervention Names

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SEG101, Adakveo®, Ryverna®

Eligibility Criteria

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Inclusion Criteria

1. 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.
2. 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.
3. Experienced 4 to 12 VOCs (refer to Section 8.3.1 for study definition of VOC) that are HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) within the 12 months prior to the screening visit. Baseline VOCs are determined by medical history and are required to be documented at source.
4. 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 and plan to continue taking it 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 and plan to continue taking the drug at the same dose and schedule until the participant has reached 52 weeks of the planned study treatment.

Participants who have not been receiving HU/HC, and/or erythropoietin stimulating agent must not have received it for at least 6 months prior to screening visit.

Exclusion Criteria

1. Fewer than 4 or more than 12 VOCs that are HCP-managed (including VOCs leading to management at a health care facility or those managed via remote consultation) within the 12 months prior to screening visit as determined by medical history and documented at source.
2. History of stem cell transplant and/or gene therapy.
3. Received blood products within 30 days prior to Week 1 Day 1 dosing.
4. 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.
5. Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) 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.
6. 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.
Minimum Eligible Age

12 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Childrens National Hospital

Washington D.C., District of Columbia, United States

Site Status RECRUITING

University of Florida

Jacksonville, Florida, United States

Site Status RECRUITING

Augusta University Georgia

Augusta, Georgia, United States

Site Status RECRUITING

WCG Sonar Clinical Research

Riverdale, Georgia, United States

Site Status RECRUITING

Norton Children s Hospital

Louisville, Kentucky, United States

Site Status RECRUITING

Childrens Hospital at Montefiore

The Bronx, New York, United States

Site Status RECRUITING

East Carolina University

Greenville, North Carolina, United States

Site Status RECRUITING

Spoknwrdclinicaltrials

Easton, Pennsylvania, United States

Site Status RECRUITING

U of TX Health Science Ct

Houston, Texas, United States

Site Status RECRUITING

Novartis Investigative Site

Medellín, Antioquia, Colombia

Site Status RECRUITING

Novartis Investigative Site

Cali, Valle del Cauca Department, Colombia

Site Status RECRUITING

Novartis Investigative Site

Cali, Valle del Cauca Department, Colombia

Site Status RECRUITING

Novartis Investigative Site

Montería, , Colombia

Site Status RECRUITING

Countries

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United States Colombia

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Facility Contacts

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Regine Hyppolite

Role: primary

Victoria Ditty

Role: primary

Tayo Togba

Role: primary

Christina Haig

Role: primary

Rosa Nieves

Role: primary

Sean Segre

Role: primary

Vincent Varra

Role: primary

Arlene White-Brisco

Role: primary

Other Identifiers

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CSEG101A2303

Identifier Type: -

Identifier Source: org_study_id