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

• 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: Global Blood Therapeutics
• Information provided by (Responsible Party): Global Blood Therapeutics

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).

Condition or disease	Intervention/treatment	Phase
Sickle Cell Disease	Drug: GBT440	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 5 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• 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 Start Date: August 2016
• Actual Primary Completion Date: June 2017
• Actual Study Completion Date: August 2017

Arms and Interventions

Arm	Intervention/treatment
Experimental: Single Arm; Single Arm / open label	Drug: GBT440; Oral drug

Outcome Measures

Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. 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.
2. To Observed Pharmacokinetics in Plasma and Whole Blood. [ Time Frame: 2 - 6 months ]
   Measure maximum plasma concentration (Cmax)
3. To Characterize the Effect of GBT440 on Hemolysis. [ Time Frame: 2 - 6 months ]
   Data presented for unconjugated bilirubin at specific time point.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 60 Years (Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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).

Contacts and Locations

Locations

United Kingdom

The BRC Research Facility, Floor 15 The Tower Wing

London, United Kingdom, SE1 9RT

Sponsors and Collaborators

Global Blood Therapeutics

Investigators

• Study Director: Josh Lehrer-Graiwer, MD; Global Blood Therapeutics, Inc.
• Principal Investigator: Timothy Mant, FRCP, FFPM; Quintiles, Inc.

  Study Documents (Full-Text)

Documents provided by Global Blood Therapeutics:

Study Protocol and Statistical Analysis Plan  [PDF] September 28, 2016

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: Global Blood Therapeutics
• ClinicalTrials.gov Identifier: NCT03041909
• Other Study ID Numbers: GBT440-024
• First Posted: February 3, 2017
• Results First Posted: January 2, 2019
• Last Update Posted: January 2, 2019
• Last Verified: December 2018

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Supporting Materials: Study Protocol

Additional relevant MeSH terms:

Anemia, Sickle Cell; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn