Back to results

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

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:

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).

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

NCT ID

NCT03041909

First Posted

January 19, 2017

Last Updated

December 18, 2018

Sponsor

Global Blood Therapeutics

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

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

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

We'll reach out to this number within 24 hrs