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)
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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 12 Years to 100 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study tests how well crizanlizumab (5 mg/kg) works and how safe it is compared with a placebo, a treatment with no active drug. It is for people age 12 and older with sickle cell disease who have frequent pain crises caused by blocked blood flow. People may take part with or without hydroxyurea treatment.
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Be age 12 or older when signing the consent form.
- Have sickle cell disease confirmed by a blood test. All types of sickle cell disease are allowed.
- Have 4 to 12 pain crises managed by a health care provider in the past 12 months, with records to confirm them. Care may have been in person or through a remote visit.
- If taking hydroxyurea, also called hydroxycarbamide, have taken it for at least 6 months and at the same dose for at least 3 months. Plan to keep the same dose and schedule through week 52 of study treatment.
- If hydroxyurea was started 6 to 12 months before screening, still have poorly controlled pain. Have 4 to 12 crises in the past 12 months, including at least 2 in the past 6 months while taking it.
- If taking a drug that helps the body make red blood cells, have taken it for at least 6 months. Plan to keep the same dose and schedule through week 52 of study treatment.
- If not currently taking hydroxyurea or a drug that helps make red blood cells, have been off that drug for at least 6 months before screening.
Who can't join
- Have fewer than 4 or more than 12 provider-managed pain crises in the 12 months before screening.
- Have ever had a stem cell transplant or gene therapy.
- Received blood products within 30 days before the first study dose.
- Have a history of a stroke with symptoms or bleeding inside the skull, or an unexplained nerve or brain finding in the past 12 months. A silent stroke found only on a scan does not rule you out.
- Receive regular planned blood transfusions to prevent problems, or plan to have an exchange transfusion during the study. An exchange transfusion replaces some of your blood with donor blood. Occasional transfusions for worsening anemia or a pain crisis are allowed.
- Cannot safely receive, or are allergic to, drugs like the study drug, their breakdown products, or the study drug's ingredients.
- Have had a severe allergic reaction to other antibody medicines that the study doctor believes raises the risk of a serious reaction during treatment.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
Key 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. Key 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.
Study sites (17)
AL
- University Of Alabama · Birmingham, AL 35233Jeanine Dumas · Study contact · +1 205 638 9285 · jdumas@uabmc.eduChibuzo Churchill Ilonze · Principal investigatorMore trials in Birmingham
CA
- Ctr for Inherited Blood Disorders · Orange, CA 92868Emily Lopez · Study contact · elopez@cibd-ca.orgVanessa Salinas · Principal investigatorMore trials in Orange
DC
- Childrens National Hospital · Washington D.C., DC 20010Taylor Quinn · Study contact · +1 202 476 5000 · tquinn@childrensnational.orgAndrew Campbell · Principal investigatorMore trials in Washington D.C.
FL
- University of Florida · Jacksonville, FL 32209Tatiana Elias-Grajeda · Study contact · tatiana.elias-grajeda@jax.ufl.eduGabriela Bastidas · Principal investigatorMore trials in Jacksonville
GA
- Augusta University Georgia · Augusta, GA 30912Latanya Bowman · Study contact · +1 706 721 2941 · lbowman@augusta.eduGirindra Raval · Principal investigatorMore trials in Augusta
- WCG Sonar Clinical Research · Riverdale, GA 30274Ify Anierobi · Study contact · ianierobi@sonarcr.comAnthony Onyegbula · Principal investigatorMore trials in Riverdale
IL
- Uni of Illinois Hospital and HSC · Chicago, IL 60612Taif Hassan · Study contact · tohassan@uic.eduSantosh Saraf · Principal investigatorMore trials in Chicago
KY
- Norton Children s Hospital · Louisville, KY 40202Meagan Despain · Study contact · meagan.despain@louisville.eduAshok Raj · Principal investigatorMore trials in Louisville
MD
- The Johns Hopkins University School of Medicine · Baltimore, MD 21205Goodness Che · Study contact · +1 410 955 2812 · anchang1@jhmi.eduLydia Pecker · Principal investigatorMore trials in Baltimore
MS
- Southern Specialty Research · Flowood, MS 39232Wendy Thomson · Study contact · wendy.thomson@southernspecialty.orgSharon Pennington · Principal investigatorMore trials in Flowood
NC
- East Carolina University · Greenville, NC 27834Tori Donadio · Study contact · donadiov21@ecu.eduBeng Fuh · Principal investigatorMore trials in Greenville
- Wake Forest University Baptist Medical Center · Winston-Salem, NC 27157Julie Fountain · Study contact · julie.fountain@advocatehealth.orgAlex George · Principal investigatorMore trials in Winston-Salem
NY
- Childrens Hospital at Montefiore · The Bronx, NY 10467Karen Ireland · Study contact · +1 718 741 2384 · kireland@montefiore.orgKaitlin Strumph · Principal investigatorMore trials in The Bronx
PA
- Spoknwrdclinicaltrials · Easton, PA 18045Anne Murunga · Study contact · amurunga@spoknwrdclinicaltrials.comHayman Salib · Principal investigatorMore trials in Easton
- Thomas Jefferson University Me · Philadelphia, PA 19107Katie McDonnell · Study contact · Katie.McDonnell@jefferson.eduSanaa Rizk · Principal investigatorMore trials in Philadelphia
TX
- Texas Childrens Cancer and Hematology Center · Houston, TX 77030Bogdan Dinu · Study contact · +1 832 824 1000 · brdinu@texaschildrens.orgVenee Tubman · Principal investigatorMore trials in Houston
- U of TX Health Science Ct · Houston, TX 77030Eyuel Baherey · Study contact · Eyuel.L.Baherey@uth.tmc.eduModupe Idowu · Principal investigatorMore trials in Houston