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

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.
 
ClinicalTrials.gov Identifier: NCT02850406
Recruitment Status : Recruiting
First Posted : August 1, 2016
Last Update Posted : February 18, 2020
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Brief Summary:
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. 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 voxelotor in pediatric participants with Sickle Cell Disease who are 4 to 17 years of age. Part D is a multiple dose, safety, tolerability, and PK study, which will examine the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease who are between 9 months to < 4 years of age.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Voxelotor Phase 2

Expanded Access : An investigational treatment associated with this study has been approved for sale to the public.   More info ...

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 155 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : May 18, 2016
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Voxelotor

Subjects to receive daily oral dosing of voxelotor according to which Part (A, B, C, or D), the subject is participating in:

  • Part A: Subjects to receive daily oral dosing of voxelotor for 1 day (single dose)
  • Part B: Subjects to receive daily oral dosing of voxelotor for up to 24 weeks (multiple dose)
  • Part C: Subjects to receive daily oral dosing of voxelotor for up to 48 weeks (1500mg or 1500mg equivalent dose)
  • Part D: Subjects to receive daily oral dosing of voxelotor for up to 48 weeks (1500mg or 1500mg equivalent dose)
Drug: Voxelotor
Part A: Voxelotor will be administered as oral capsules or tablets Part B: Voxelotor will be administered as oral capsules or tablets Part C: Voxelotor will be administered as oral dispersible tablets or powder for oral suspension Part D: Voxelotor will be administered as powder for oral suspension




Primary Outcome Measures :
  1. Part A: Pharmacokinetic profile of voxelotor including maximum concentration [ Time Frame: Pre-dose to Day 15 ]
  2. Part A: Pharmacokinetic profile of voxelotor including the time taken to reach the maximum concentration [ Time Frame: Pre-dose to Day 15 ]
  3. Part A: Pharmacokinetic profile of voxelotor including the total drug concentration over time [ Time Frame: Pre-dose to Day 15 ]
  4. Part B: Change in hemoglobin [ Time Frame: Baseline to Week 24 ]
  5. Part C: Change in cerebral blood flow as measured by the TAMM TCD velocity [ Time Frame: Baseline to Week 48 ]
  6. Part D: Treatment-Emergent Adverse Events and Serious Adverse Events [ Time Frame: Baseline to Week 48 ]

Secondary Outcome Measures :
  1. Part A: Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Days 1 - 15 ]
  2. Part B: Multiple Dose effect on Clinical Measures of Hemolysis [ Time Frame: Day 1 - Week 24 ]
  3. Part B: Pharmacokinetic profile of voxelotor including maximum concentration [ Time Frame: Pre-dose to Week 24 ]
  4. Part B: Pharmacokinetic profile of voxelotor including the time taken to reach the maximum concentration [ Time Frame: Pre-dose to Week 24 ]
  5. Part B: Pharmacokinetic profile of voxelotor including the total drug concentration over time [ Time Frame: Pre-dose to Week 24 ]
  6. Part C: Multiple dose effect on clinical measures of hemolysis [ Time Frame: Baseline to Week 24 and Week 48 ]
  7. Part C: Change in cerebral blood flow [ Time Frame: Baseline to Week 24 ]
  8. Part C: Pharmacokinetic profile of voxelotor including maximum concentration [ Time Frame: Pre-Dose to Week 48 ]
  9. Part C: Pharmacokinetic profile of voxelotor including the time taken to reach the maximum concentration [ Time Frame: Pre-Dose to Week 48 ]
  10. Part C: Pharmacokinetic profile of voxelotor including the total drug concentration over time [ Time Frame: Pre-Dose to Week 48 ]
  11. Part D: Pharmacokinetic profile of voxelotor including percent Hemoglobin occupancy [ Time Frame: Baseline to Week 48 ]
  12. Part D: Change in Hemoglobin [ Time Frame: Baseline to Week 24 ]
  13. Part D: Change in Lactate Dehydrogenase [ Time Frame: Baseline to Week 24 ]
  14. Part D: Change in Indirect Bilirubin [ Time Frame: Baseline to Week 24 ]
  15. Part D: Change in Reticulocyte Count [ Time Frame: Baseline to Week 24 ]
  16. Part D: Time to initial Hemoglobin response [ Time Frame: Baseline to Week 48 ]
    Change in baseline in Hb > 1g/dL



Information from the National Library of Medicine

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Ages Eligible for Study:   9 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

    1. Part A - 6 to 17 years of age
    2. Part B - 12 to 17 years of age
    3. Part C - 4 to 17 years of age
    4. Part D - 9 months to <4 years of age
  3. 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.
  4. Hemoglobin (HB):

    1. Part A - No restriction
    2. Parts B, C, & D - Hb ≤ 10.5 g/dL

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 by non-imaging TCD or ≥185 cm/sec by TCDi).
  4. Transfusion within 30 days prior to signing the ICF

Exclusion Criteria for Part D Only:

16. Body weight <5 kg for 1 month prior to the screening visit and at the screening visit.


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 ClinicalTrials.gov identifier (NCT number): NCT02850406


Contacts
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Contact: Margaret Tonda, PharmD (650) 741-7761 mtonda@gbt.com
Contact: Aimee Enriquez (650) 741-7777 aenriquez@gbt.com

Locations
Show Show 17 study locations
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
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Study Director: Margaret Tonda, PharmD Global Blood Therapeutics, Inc.
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Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT02850406    
Other Study ID Numbers: GBT440-007
First Posted: August 1, 2016    Key Record Dates
Last Update Posted: February 18, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn