Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease



Status:Recruiting
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:4 - 17
Updated:3/30/2019
Start Date:May 2016
End Date:May 2022
Contact:Dan Rudin, MD
Email:drudin@gbt.com
Phone:(650) 534-2574

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A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Exploratory Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

This study consists of three parts, Parts A, B, and C. Part A is a single dose PK study in
pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety,
exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were
12-17 years of age. 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
GBT440 is pediatric participants with Sickle Cell Disease who are 4 to 17 years of age.


Inclusion Criteria:

1. Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0
thalassemia (HbS β0thal)

2. 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.

3. Hemoglobin (HB):

1. Part A - No restriction

2. Part B - Hb ≤ 10.5 g/dL

3. Part C - Hb ≤ 10.5 g/dL

4. Written informed parental/guardian consent and participant assent has been obtained
per institutional review board (IRB)/Ethics Committee (EC) policy and requirements,
consistent with International Council on Harmonisation (ICH) guidelines.

10. 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:

1. 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

- Dactylitis

2. Requires chronic transfusion therapy

3. History of stroke or meeting criteria for primary stroke prophylaxis (history of two
TCD measurements ≥ 200 cm/sec)

4. Transfusion within 30 days prior to signing the ICF
We found this trial at
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New Brunswick, New Jersey 08901
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Atlanta, Georgia
Phone: 732-135-5976
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Pittsburgh, Pennsylvania
Phone: 412-692-6467
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