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

This study is ongoing, but not recruiting participants.

Sponsor:

Information provided by (Responsible Party):

Global Blood Therapeutics

ClinicalTrials.gov Identifier:

NCT03041909

First received: January 19, 2017

Last updated: February 3, 2017

Last verified: January 2017

History of Changes

Purpose

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	Intervention	Phase
Sickle Cell Disease	Drug: GBT440	Phase 2


Study Type:	Interventional
Study Design:	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

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Sickle Cell Anemia

Genetic and Rare Diseases Information Center resources: Sickle Cell Anemia

U.S. FDA Resources

Further study details as provided by Global Blood Therapeutics:

Primary Outcome Measures:

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:

To assess the efficacy of GBT440 as measured by improvements in anemia [ Time Frame: 2 - 6 months ]
Measurement of haemoglobin
To observed pharmacokinetics in plasma and whole blood. [ Time Frame: 2 - 6 months ]
Measure maximum plasma concentration (Cmax)
To characterize the effect of GBT440 on hemolysis. [ Time Frame: 2 - 6 months ]
Measurement of hemolysis include unconjugated bilirubin.


Estimated Enrollment:	16
Study Start Date:	August 2016
Estimated Study Completion Date:	August 2017
Estimated Primary Completion Date:	May 2017 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Single Arm Single Arm / open label	Drug: GBT440 Oral drug

Eligibility


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

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).

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03041909

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


Responsible Party:	Global Blood Therapeutics
ClinicalTrials.gov Identifier:	NCT03041909 History of Changes
Other Study ID Numbers:	GBT440-024
Study First Received:	January 19, 2017
Last Updated:	February 3, 2017
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

ClinicalTrials.gov processed this record on September 25, 2017

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