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

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03041909
• Other study ID #: GBT440-024
• Status: Completed
• Phase: Phase 2
• Start date: August 2016
• Est. completion date: August 2017

Study information

• Verified date: December 2018
• Source: Global Blood Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 5
• Est. completion date: August 2017
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

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).

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• GBT440
Oral drug

Locations

Country	Name	City	State
United Kingdom	The BRC Research Facility, Floor 15 The Tower Wing	London

Sponsors (1)

Lead Sponsor	Collaborator
Global Blood Therapeutics

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	2 - 6 months
Secondary	To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia	Data presented are hemoglobin value collected at specific time points.	2 - 6 months
Secondary	To Observed Pharmacokinetics in Plasma and Whole Blood.	Measure maximum plasma concentration (Cmax)	2 - 6 months
Secondary	To Characterize the Effect of GBT440 on Hemolysis.	Data presented for unconjugated bilirubin at specific time point.	2 - 6 months