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

  • End date
    Dec 25, 2022
  • participants needed
  • sponsor
    Global Blood Therapeutics
Updated on 25 March 2022
hemoglobin s
sickle cell trait
sickle hemoglobin


This study consists of four parts, Parts A, B, C, and D.

  • Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease ages 6 to 17 years.
  • Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent participants with Sickle Cell Disease ages 12 to 17 years.
  • Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease ages 4 to 17 years.
  • Part D is a multiple dose, safety, tolerability, and PK study, which examines the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease ages 6 months to < 4 years.

Condition Sickle Cell Disease
Treatment GBT440, Voxelotor
Clinical Study IdentifierNCT02850406
SponsorGlobal Blood Therapeutics
Last Modified on25 March 2022


Yes No Not Sure

Inclusion Criteria

Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0thal)
Part A - 6 to 17 years of age
Part B - 12 to 17 years of age
Part C - 4 to 17 years of age
Part D - 6 months to <4 years of age
Hydroxyurea (HU) therapy
Parts A, B, and C: A participant taking hydroxyurea (HU) may be enrolled if the dose has been stable for at least 3 months with no anticipated need for dose adjustment during the study and no sign of hematological toxicity
Part D: A participant taking HU may be enrolled if the dose has been stable for at least 1 month. Titration to the maximum tolerated dose (MTD) is allowed during the study
Hemoglobin (HB)
Part A - No restriction
Parts B, C, & D - Hb ≤ 10.5 g/dL
For Part C only: Participants 12 to 17 years of age must have a TCD velocity of ≥ 140
cm/sec measured anytime during screening

Exclusion Criteria

Any one of the following requiring medical attention within 14 days of signing the Informed Consent Form (ICF)
Vaso-occlusive crisis (VOC)
Acute chest syndrome (ACS)
Splenic sequestration crisis
Requires chronic transfusion therapy
Transfusion within 30 days prior to signing the ICF
History of stroke or meeting criteria for primary stroke prophylaxis (history of two TCD measurements ≥ 200 cm/sec by non-imaging TCD or ≥185 cm/sec by TCDi)
Exclusion Criteria for Part D Only
Body weight <5 kg for 1 month prior to the screening visit and at the screening visit
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How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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