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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

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 2
Locations
United Kingdom
Study Type
Interventional
Intervention
GBT440
Sponsored by
Global Blood Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Sickle Cell Disease

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • The BRC Research Facility, Floor 15 The Tower Wing

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single Arm

Arm Description

Single Arm / open label

Outcomes

Primary Outcome Measures

Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.

Secondary Outcome Measures

To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
Data presented are hemoglobin value collected at specific time points.
To Observed Pharmacokinetics in Plasma and Whole Blood.
Measure maximum plasma concentration (Cmax)
To Characterize the Effect of GBT440 on Hemolysis.
Data presented for unconjugated bilirubin at specific time point.

Full Information

First Posted
January 19, 2017
Last Updated
December 18, 2018
Sponsor
Global Blood Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT03041909
Brief Title
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
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2018
Overall Recruitment Status
Completed
Study Start Date
August 2016 (undefined)
Primary Completion Date
June 2017 (Actual)
Study Completion Date
August 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Global Blood Therapeutics

4. Oversight

Data Monitoring Committee
No

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease

7. Study Design

Primary Purpose
Other
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
5 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Single Arm
Arm Type
Experimental
Arm Description
Single Arm / open label
Intervention Type
Drug
Intervention Name(s)
GBT440
Intervention Description
Oral drug
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
Description
The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
Time Frame
2 - 6 months
Secondary Outcome Measure Information:
Title
To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
Description
Data presented are hemoglobin value collected at specific time points.
Time Frame
2 - 6 months
Title
To Observed Pharmacokinetics in Plasma and Whole Blood.
Description
Measure maximum plasma concentration (Cmax)
Time Frame
2 - 6 months
Title
To Characterize the Effect of GBT440 on Hemolysis.
Description
Data presented for unconjugated bilirubin at specific time point.
Time Frame
2 - 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female subjects with SCD aged 18 to 60 years inclusive and >50 kg who have participated in the GBT440-001 study. 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. 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: Subjects requiring chronic transfusion therapy. Subjects receiving a blood transfusion within 30 days of enrollment in this study. Female subjects who are pregnant, trying to become pregnant or lactating. 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). Subjects who have a significant infection or known inflammatory process on admission to this study. Subjects who have acute gastrointestinal symptoms at the time of admission (e.g. nausea, vomiting, diarrhoea, heartburn).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Josh Lehrer-Graiwer, MD
Organizational Affiliation
Global Blood Therapeutics, Inc.
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Timothy Mant, FRCP, FFPM
Organizational Affiliation
Quintiles, Inc.
Official's Role
Principal Investigator
Facility Information:
Facility Name
The BRC Research Facility, Floor 15 The Tower Wing
City
London
ZIP/Postal Code
SE1 9RT
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
Citations:
PubMed Identifier
30655275
Citation
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.
Results Reference
derived

Learn more about this trial

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

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