Try the modernized ClinicalTrials.gov beta website. Learn more about the modernization effort.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 2 for:    Phase 1/2 GBT440-001
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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:
Information provided by (Responsible Party):
Global Blood Therapeutics

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
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

Resource links provided by the National Library of Medicine


Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Layout table for eligibility information
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03041909


Locations
Layout table for location information
United Kingdom
The BRC Research Facility, Floor 15 The Tower Wing
London, United Kingdom, SE1 9RT
Sponsors and Collaborators
Global Blood Therapeutics
Investigators
Layout table for investigator information
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Layout table for additonal information
Responsible Party: Global Blood Therapeutics
ClinicalTrials.gov Identifier: NCT03041909    
Other Study ID Numbers: GBT440-024
First Posted: February 3, 2017    Key Record Dates
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:
Layout table for MeSH terms
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn