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Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease (HOPE Kids 2)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04218084
Recruitment Status : Recruiting
First Posted : January 6, 2020
Last Update Posted : May 20, 2021
Information provided by (Responsible Party):
Global Blood Therapeutics

Brief Summary:
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Voxelotor Drug: Placebos Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Detailed Description:
This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The study will be conducted at approximately 50 international clinical sites, and will enroll approximately 224 participants. Participants will be randomized in a 1:1 ratio to receive voxelotor or placebo. All participants younger than 12 years of age and randomized to voxelotor will receive a dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg/day.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 224 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double-Blind, Placebo-Controlled
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Voxelotor (GBT440) in Pediatric Participants With Sickle Cell Disease
Actual Study Start Date : April 7, 2021
Estimated Primary Completion Date : March 2026
Estimated Study Completion Date : March 2026

Resource links provided by the National Library of Medicine

Drug Information available for: Voxelotor

Arm Intervention/treatment
Experimental: Voxelotor
Voxelotor 1500mg or equivalent daily as a tablet or as powder for oral suspension.
Drug: Voxelotor
Participants are randomized 1:1 to receive voxelotor or placebo.

Placebo Comparator: Placebo
Matching placebo.
Drug: Placebos
Matching placebo.

Primary Outcome Measures :
  1. TCD Measurement [ Time Frame: 24 weeks ]
    The change in time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow, as measured by TCD.

Secondary Outcome Measures :
  1. Change in TCD Flow Velocity [ Time Frame: 48 and 96 weeks ]
  2. Conversion to Abnormal TCD Flow Velocity [ Time Frame: 24, 48, and 96 weeks ]
    Incidence of conversion to abnormal TCD flow velocity (≥ 200 cm/sec)

  3. Reversion to Normal TCD Flow Velocity [ Time Frame: 24, 48, and 96 weeks ]
    Incidence of reversion to normal TCD flow velocity (<170 cm/sec)

  4. TCD Flow Velocity Reduction [ Time Frame: 24, 48, and 96 weeks ]
    Proportion of participants with TCD flow velocity reduction ≥ 15 cm/sec

  5. Number of Participants with Change in Hemoglobin (Hb) [ Time Frame: 24, 48, and 96 weeks ]
  6. Change in Unconjugated Bilirubin [ Time Frame: 24, 48 and 96 weeks ]
  7. Change in Percent of Reticulocyte [ Time Frame: 24, 48, and 96 weeks ]
  8. Change in Absolute Reticulocyte [ Time Frame: 24, 48, and 96 weeks ]
  9. Change in Lactate Dehydrogenase (LDH) [ Time Frame: 24, 48, and 96 weeks ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female participants with Sickle Cell Anemia (SCA)
  2. TCD time averaged maximum of the mean velocity (TAMMV) arterial cerebral blood flow ≥ 170 to < 200cm/sec during the Screening Period
  3. Hb ≥ 5.5 and ≤ 10.5 g/dL during screening
  4. For participants taking HU, the dose of HU (mg/kg) must be stable for at least 90 days prior to signing the informed consent form (ICF) and/or assent form, and with no anticipated need for dose adjustments (other than weight based) or for initiation of HU for non-chronic use during the study, in the opinion of the Investigator
  5. Written informed parental/guardian consent and participant assent (where applicable) has been obtained per IRB/EC policy and requirements, consistent with ICH guidelines.

Exclusion Criteria:

  1. Body weight < 5kg at the screening visit
  2. Hospitalization for VOC or acute chest syndrome (ACS) within the 14 days prior to execution of informed consent/assent (see Section for the definition of VOC).
  3. More than 10 VOCs within the past 12 months that required hospitalization, emergency room, or clinic visit
  4. Stroke resulting in focal neurological deficit; previous silent infarcts are permitted.
  5. Known history or findings, including screening magnetic resonance imaging (MRI)/magnetic imaging angiography (MRA) findings, suggestive of significant cerebral vasculopathy (eg, moyamoya or significant vasculopathy)
  6. History of seizure disorder (History of febrile seizures is permissible if there have been no seizures within the 12 months prior to randomization).
  7. Has been treated with erythropoietin or other hematopoietic growth factors within 28 days of signing informed consent/assent or if, in the opinion of the Investigator, there is an anticipated need for such agents during the study
  8. RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion) or has received an RBC transfusion or exchange transfusion for any reason within 90 days of signing the informed consent/assent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04218084

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Contact: Margaret Tonda, PharmD (650) 741-7761
Contact: Jenni Herber (650) 826-2422

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Sponsors and Collaborators
Global Blood Therapeutics
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Responsible Party: Global Blood Therapeutics Identifier: NCT04218084    
Other Study ID Numbers: GBT440-032
First Posted: January 6, 2020    Key Record Dates
Last Update Posted: May 20, 2021
Last Verified: May 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Global Blood Therapeutics:
Sickle Cell Disease
Transcranial Doppler Ultrasound (TCD)
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn