Clinical trial NCT03041909: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Lead sponsor: Global Blood Therapeutics
Sponsor class: INDUSTRY
First posted: 2017-02-03
Enrollment: 5
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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).

Eligibility 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).

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
timeFrame: 2 - 6 months
Secondary outcomes:
measure: To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
timeFrame: 2 - 6 months
measure: To Observed Pharmacokinetics in Plasma and Whole Blood.
timeFrame: 2 - 6 months
measure: To Characterize the Effect of GBT440 on Hemolysis.
timeFrame: 2 - 6 months
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: The BRC Research Facility, Floor 15 The Tower Wing
Study officials:
affiliation: Global Blood Therapeutics, Inc.
name: Josh Lehrer-Graiwer, MD
role: STUDY_DIRECTOR
affiliation: Quintiles, Inc.
name: Timothy Mant, FRCP, FFPM
role: PRINCIPAL_INVESTIGATOR
References:
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.