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An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03041909
Recruitment Status : Completed
First Posted : February 3, 2017
Results First Posted : January 2, 2019
Last Update Posted : January 2, 2019
Sponsor:
Information provided by (Responsible Party):
Global Blood Therapeutics

Tracking Information
First Submitted Date  ICMJE January 19, 2017
First Posted Date  ICMJE February 3, 2017
Results First Submitted Date  ICMJE October 2, 2018
Results First Posted Date  ICMJE January 2, 2019
Last Update Posted Date January 2, 2019
Study Start Date  ICMJE August 2016
Actual Primary Completion Date June 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 18, 2018)
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months. [ Time Frame: 2 - 6 months ]
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Original Primary Outcome Measures  ICMJE
 (submitted: February 1, 2017)
To evaluate the safety and tolerability of GBT440 for up to 6 months of dosing. [ Time Frame: 2 - 6 months ]
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 18, 2018)
  • To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia [ Time Frame: 2 - 6 months ]
    Data presented are hemoglobin value collected at specific time points.
  • To Observed Pharmacokinetics in Plasma and Whole Blood. [ Time Frame: 2 - 6 months ]
    Measure maximum plasma concentration (Cmax)
  • To Characterize the Effect of GBT440 on Hemolysis. [ Time Frame: 2 - 6 months ]
    Data presented for unconjugated bilirubin at specific time point.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 1, 2017)
  • To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia [ Time Frame: 2 - 6 months ]
    Measurement of haemoglobin
  • To Observed Pharmacokinetics in Plasma and Whole Blood. [ Time Frame: 2 - 6 months ]
    Measure maximum plasma concentration (Cmax)
  • To Characterize the Effect of GBT440 on Hemolysis. [ Time Frame: 2 - 6 months ]
    Measurement of hemolysis include unconjugated bilirubin.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001
Official Title  ICMJE An Open Label Single Arm Extension Study to Further Evaluate the Safety, Tolerability and Treatment Response of GBT440 in Patients With Sickle Cell Disease Who Participated in the Phase 1 Study GBT440-001
Brief Summary This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
Detailed Description

This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).

Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001 (NCT02285088).

The primary objective of the study was to evaluate the safety and tolerability of up to a total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study (NCT02285088).

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Sickle Cell Disease
Intervention  ICMJE Drug: GBT440
Oral drug
Study Arms  ICMJE Experimental: Single Arm
Single Arm / open label
Intervention: Drug: GBT440
Publications * Howard J, Hemmaway CJ, Telfer P, Layton DM, Porter J, Awogbade M, Mant T, Gretler DD, Dufu K, Hutchaleelaha A, Patel M, Siu V, Dixon S, Landsman N, Tonda M, Lehrer-Graiwer J. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease. Blood. 2019 Apr 25;133(17):1865-1875. doi: 10.1182/blood-2018-08-868893. Epub 2019 Jan 17.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 13, 2018)
5
Original Estimated Enrollment  ICMJE
 (submitted: February 1, 2017)
16
Actual Study Completion Date  ICMJE August 2017
Actual Primary Completion Date June 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female subjects with SCD aged 18 to 60 years inclusive and >50 kg who have participated in the GBT440-001 study.
  2. Subjects, who if female and of child bearing potential, agree to continue to use highly effective methods of contraception prior to enrollment in this study and for 3 months after the last dose of study drug.
  3. Subjects, who if male are willing to continue to use barrier methods of contraception, prior to enrollment in this study to 3 months after the last dose of study drug.

Exclusion Criteria:

  1. Subjects requiring chronic transfusion therapy.
  2. Subjects receiving a blood transfusion within 30 days of enrollment in this study.
  3. Female subjects who are pregnant, trying to become pregnant or lactating.
  4. Subjects who have a clinically relevant history or presence of respiratory, gastrointestinal, renal, hepatic, haematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological, dermatological, connective tissue diseases or disorders, or additional risk factors for torsades de pointe (e.g., heart failure, hypokalemia, personal or family history of long QTc interval).
  5. Subjects who have a significant infection or known inflammatory process on admission to this study.
  6. Subjects who have acute gastrointestinal symptoms at the time of admission (e.g. nausea, vomiting, diarrhoea, heartburn).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03041909
Other Study ID Numbers  ICMJE GBT440-024
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Supporting Materials: Study Protocol
Responsible Party Global Blood Therapeutics
Study Sponsor  ICMJE Global Blood Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Josh Lehrer-Graiwer, MD Global Blood Therapeutics, Inc.
Principal Investigator: Timothy Mant, FRCP, FFPM Quintiles, Inc.
PRS Account Global Blood Therapeutics
Verification Date December 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP