Toggle navigation
Lebanon Clinical Trials Registry
Home
About Us
FAQs
Contact Us
Search Trials
Register
Log in
User Guide
Trial details
You are here
Home
Search Trials
Trial details
Trial details
Study to Assess the Effect of Long-term Treatment With GBT440 in Participants Who Have Completed Treatment in Study GBT440-031
Current status:
Approved
|
Date registered:
13/10/2021
Trial version(s)
Current: 27/03/2019
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2019080216
Protocol number
GBT440-034
MOH registration number
2018/2/30053
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Retrospective
Type of registration: Justify
Sponsor's request and registry was not available when study started
Date of registration in national regulatory agency
08/08/2018
Primary sponsor
Global Blood Therapeutics, Inc
Primary sponsor: Country of origin
United States of America
Public title
Study to Assess the Effect of Long-term Treatment With GBT440 in Participants Who Have Completed Treatment in Study GBT440-031
Acronym
Scientific title
An Open Label Extension Study of GBT440 Administered Orally to Patients With Sickle Cell Disease Who Have Participated in GBT440 Clinical Trials
Acronym
Brief summary of the study: English
Study to Assess the Effect of Long-term Treatment With GBT440 in Participants Who Have Completed Treatment in Study GBT440-031
Brief summary of the study: Arabic
دراسة لتقييم تأثير العلاج طويل الأمد مع GBT440 عند المرضى الذين شاركوا في الدراسة GBT440-031 و أتموا العلاج
Health conditions/problem studied: Specify
Sickle Cell Disease
Interventions: Specify
GBT440 (Voxelotor) tablets orally administered
Key inclusion and exclusion criteria: Inclusion criteria
- Male or female study participants with Sickle Cell Disease who participated and received study treatment in Study GBT440-031. (Note: Participants in GBT440-031 who discontinued study drug due to an AE, but who remained on study may be eligible for treatment in this study provided the AE does not pose a risk for treatment with GBT440.) - Females of child-bearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. - Female participants of child-bearing potential must use highly effective methods of contraception to 30 days after the last dose of study drug. Male participants must continue to use barrier methods of contraception to 30 days after the last dose of study drug. - Participant has provided written informed consent or assent.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
12
Key inclusion and exclusion criteria: Age maximum
99
Key inclusion and exclusion criteria: Exclusion criteria
- Female who is breast-feeding or pregnant. - Participant withdrew consent from Study GBT440-031. - Participant was lost to follow-up from Study GBT440-031. - Participant requiring chronic dialysis. - Any medical, psychological, safety, or behavioral conditions, which, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent.
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
Randomized controlled trial
Study design: Masking
Open (masking not used)
Study design: Control
Active
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Parallel
IMP has market authorization
No
Name of IMP
Voxelotor (previously GBT440)
Type of IMP
Others
Type of IMP: Specify
Chemical
Pharmaceutical class
Allosteric modulator of hemoglobin oxygen affinity
Therapeutic indication
Treatment of Sickle Cell Disease
Therapeutic benefit
Voxelotor is an orally bioavailable HbS polymerization inhibitor that binds specifically to HbS with a 1:1 stoichiometry, and exhibits preferential partitioning to RBCs. By increasing Hb's affinity for oxygen, voxelotor inhibits HbS polymerization in a dose dependent manner that may improve deformability, decrease the viscosity of SCD blood, and ultimately increase blood flow in the microcirculation, thus improving net O2 delivery. Therefore, chronically modifying 20% to 30% of HbS with voxelotor in subjects with SCD is expected to deliver the clinical benefits of reducing HbS polymerization while improving O2 delivery to peripheral tissues.
Biospecimen retention
None retained
Biospecimen description
N/A
Target sample size
6
Actual enrollment target size
6
Date of first enrollment: Type
Actual
Date of first enrollment: Date
13/02/2019
Date of study closure: Type
Actual
Date of study closure: Date
31/12/2024
Recruitment status
Other
Recruitment status: Specify
Enrolling by invitation
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
N/A
Additional data URL
https://clinicaltrials.gov/ct2/show/record/NCT03573882
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
ClinicalTrials.gov
NCT03573882
EU Clinical Trials Register
EudraCT: 2017-004045-25
Sources of Monetary or Material Support
Name
Global Blood Therapeutics, Inc. USA
Secondary Sponsors
Name
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Dr. Adlette
Inati
Tripoli
Lebanon
9613228033
adlette.inati@lau.edu.lb
Nini Hospital
Scientific
Margaret
Tonda
171 Oyster Point Boulevard, Suite 300 South San Francisco, CA 94080
United States of America
650-741-7761
mtonda@gbt.com
Global Blood Therapeutics Inc.
Public
Dr. Miguel
Abboud
Beirut
Lebanon
9611350000
ma56@aub.edu.lb
American University of Beirut Medical Center
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
American University of Beirut Medical Center
Dr. Miguel Abboud
Pediatric Hematology and Oncology
Approved
Nini Hospital
Dr. Adlette Inati
Pediatric Hematology and Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
American University of Beirut Medical Center
07/02/2019
Dr. Fuad Ziyadeh
irb@aub.edu.lb
9611350000 ext 5445
Nini Hospital
29/10/2018
Dr. Nabil Kabbara
n/a
9616431400 ext 1061
Countries of Recruitment
Name
United States of America
Lebanon
United Kingdom
Turkey
Oman
Egypt
Kenya
Italy
France
Jamaica
Netherlands
Canada
Health Conditions or Problems Studied
Condition
Code
Keyword
Sickle Cell Disease
Sickle-cell disorders (D57)
Anemia, Sickle Cell, Hemolytic, Congenital, Hemoglobinopathies, Genetic Diseases, Inborn
Interventions
Intervention
Description
Keyword
Drug
Volexotor (GBT440) 300 mg Oral tablet
Open Label Extension, Anemia, Sickle Cell , Hemolytic, Congenital
Primary Outcomes
Name
Time points
Measure
Hemolysis Markers
5 years
total bilirubin, LDH and reticulocyte lab values
Frequency of sickle cell-related complications
5 years
Frequency of SCD-related complications with long-term dosing with GBT440
Key Secondary Outcomes
Name
Time points
Measure
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
5 years
Safety based on adverse event assessed by CTCAE (Common Terminology Criteria for Adverse Events)
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial