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

Study Description

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Brief Summary:

This is an open label, single arm study which will enroll up to 16 subjects with SCD who previously participated in the GBT440-001 study.

Dosing of study drug will be for 2 to 6 months, depending on subject's dose assignment in the last administration of study drug in GBT440-001.

The primary objective of the study is 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.

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

Study Design

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Study Type :	Interventional (Clinical Trial)
Actual Enrollment :	5 participants
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

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

Outcome Measures

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Primary Outcome Measures :

1. The Incidence of 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 ]
   Measurement of haemoglobin
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 ]
   Measurement of hemolysis include unconjugated bilirubin.

Eligibility Criteria

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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

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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.

More Information

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Responsible Party:	Global Blood Therapeutics
ClinicalTrials.gov Identifier:	NCT03041909 History of Changes
Other Study ID Numbers:	GBT440-024
First Posted:	February 3, 2017
Last Update Posted:	February 15, 2018
Last Verified:	February 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:	Yes

Additional relevant MeSH terms:

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