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

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2016 by Global Blood Therapeutics
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics
ClinicalTrials.gov Identifier:
NCT02850406
First received: July 1, 2016
Last updated: April 20, 2017
Last verified: July 2016
  Purpose
This study consists of two parts, Part A and Part B. 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.

Condition Intervention Phase
Sickle Cell Disease Drug: GBT440 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
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

Resource links provided by NLM:


Further study details as provided by Global Blood Therapeutics:

Primary Outcome Measures:
  • Part A: Pharmacokinetic profile of GBT440 including maximum concentration [ Time Frame: Pre-dose to Day 15 ]
  • Part A: Pharmacokinetic profile of GBT440 including the time taken to reach the maximum concentration [ Time Frame: Pre-dose to Day 15 ]
  • Part A: Pharmacokinetic profile of GBT440 including the total drug concentration over time [ Time Frame: Pre-dose to Day 15 ]
  • Part B: Change in hemoglobin [ Time Frame: Basline to Week 24 ]

Secondary Outcome Measures:
  • Part A: Number of participants with treatment-related adverse events as assessed by CTCAE v4.03 [ Time Frame: Days 1 - 15 ]
  • Part B Multiple Dose effect on Clinical Measures of Hemolysis [ Time Frame: Day 1 - Week 24 ]
    To evaluate the effect of GBT440 on clinical measures of hemolysis

  • Part B: Pharmacokinetic profile of GBT440 including maximum concentration [ Time Frame: Pre-dose to Week 24 ]
  • Part B: Pharmacokinetic profile of GBT440 including the time taken to reach the maximum concentration [ Time Frame: Pre-dose to Week 24 ]
  • Part B: Pharmacokinetic profile of GBT440 including the total drug concentration over time [ Time Frame: Pre-dose to Week 24 ]

Estimated Enrollment: 41
Study Start Date: May 2016
Estimated Study Completion Date: May 2018
Estimated Primary Completion Date: May 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: GBT440
Subjects to receive daily oral dosing of GBT440 for 1 day (single dose) or up to 24 weeks (multiple dose)
Drug: GBT440
GBT440 will be administered as oral capsules

  Eligibility

Ages Eligible for Study:   12 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female age 12 to 17 years of age (inclusive) with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0 thal).
  2. A subject taking hydroxyurea may be enrolled if the dose has been stable for at least 3 months with no anticipated need for dose adjustments during the study and no sign of hematological toxicity.
  3. Written informed parental/ guardian consent and subject assent has been obtained per institutional review board (IRB)/Ethics Committee (EC) policy and requirements, consistent with International Council on Harmonisation (ICH) guidelines.

Exclusion Criteria:

  1. Any one of the following requiring medical attention within 14 days prior to signing the informed consent form (ICF):

    • Vaso-occlusive crisis (VOC)
    • Acute chest syndrome (ACS)
    • Stroke
    • Splenic sequestration crisis
    • Dactylitis
  2. Requires chronic transfusion therapy
  3. History of stroke or meeting criteria for primary stroke prophylaxis (history of two Transcranial Doppler (TCD) measurements ≥ 200 cm/sec)
  4. Transfusion within 30 days prior to signing the Informed Consent Form.
  5. Renal dysfunction requiring chronic dialysis or creatinine ≥ 1.5 milligrams per deciliter (mg/dL)
  6. History of hepatic compromise or severe liver disease
  7. Clinically relevant cardiac abnormality
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02850406

Contacts
Contact: Ganesh Balaratnam, MBChB BAO (NUI) 650-741-7700 GBalaratnam@globalbloodtx.com

Locations
United States, California
Recruiting
Oakland, California, United States, 94609
Contact: Kacie Smith    510-428-3885 ext 2752    kasmith@mail.cho.org   
United States, Georgia
Recruiting
Atlanta, Georgia, United States, 30342
Contact: Leann Schilling    404-785-2025    leann.hassen@choa.org   
United States, Illinois
Recruiting
Chicago, Illinois, United States, 60611
Contact: Trecia Malcolm    312-227-4825    tmalcolm@luriechildrens.org   
Recruiting
Chicago, Illinois, United States, 60612
Contact: Lani Krauz    312-413-0242    lignacio@uic.edu   
United States, Missouri
Recruiting
Kansas City, Missouri, United States, 64108
Contact: Ann Mehrhof    816-302-6808    amehrhof@cmh.edu   
United States, New Jersey
Recruiting
New Brunswick, New Jersey, United States, 08901
Contact: Lisa Cerracchio    732-235-5976    lisa.cerracchio@rutgers.edu   
United States, Ohio
Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Adriane Hausfeld    513-803-1917    Adriane.Hausfeld@cchmc.org   
Recruiting
Cleveland, Ohio, United States, 38105
Contact: Arika Maxwell    216-844-3681    Arika.Maxwell@uhhospitals@.org   
United States, Tennessee
Recruiting
Memphis, Tennessee, United States, 38105
Contact: Bettina Utzelmann    901-595-6216    Bettina.Utzelmann@stjude.org   
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
Study Director: Ganesh Balaratnam, MBChB BAO (NUI) Global Blood Therapeutics
  More Information

Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT02850406     History of Changes
Other Study ID Numbers: GBT440-007
Study First Received: July 1, 2016
Last Updated: April 20, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 24, 2017