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Trial details
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Current status:
Approved
|
Date registered:
29/06/2020
Trial version(s)
History: 27/03/2019
History: 27/03/2019
History: 27/03/2019
Current: 27/03/2019
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Main Information
Primary registry identifying number
LBCTR2019080217
Protocol number
GBT440-031
MOH registration number
2017/2/19436
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
24/05/2017
Primary sponsor
Global Blood Therapeutics,Inc.
Primary sponsor: Country of origin
United States of America
Public title
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
Acronym
Scientific title
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Acronym
Brief summary of the study: English
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease
Brief summary of the study: Arabic
دراسة لتقييم تأثير ال-Voxelotor المأخوذ عن طريق الفم عند المرضى الذين يعانون من مرض الخلايا المنجلية
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 - Participant has had at least 1 episode of vaso-occlusive crisis (VOC) in the past 12 months. - Age 12 to 65 years - Hemoglobin (Hb) ≥5.5 and ≤10.5 g/dL during screening - For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable for at least 3 months prior to signing the ICF
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
12
Key inclusion and exclusion criteria: Age maximum
65
Key inclusion and exclusion criteria: Exclusion criteria
- More than 10 VOCs within the past 12 months that required a hospital, emergency room or clinic visit - Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or have received a RBC transfusion for any reason within 60 days of signing the ICF - Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of signing the ICF (i.e., a vaso-occlusive event cannot be within 14 days prior to signing the ICF) - Hepatic dysfunction characterized by alanine aminotransferase (ALT)>4 x ULN - Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory) <30mL/min/1.73m2 or on chronic dialysis
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
Blinded (masking used)
Study design: Control
Placebo
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
Samples without DNA
Biospecimen description
Samples not including DNA
Target sample size
13
Actual enrollment target size
7
Date of first enrollment: Type
Actual
Date of first enrollment: Date
14/09/2017
Date of study closure: Type
Actual
Date of study closure: Date
16/06/2020
Recruitment status
Complete
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
No Plan of data sharing
Additional data URL
https://clinicaltrials.gov/ct2/show/record/NCT03036813
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
International Clinical Trials Registry Platform WHO (EUCTR)
EUCTR2016-003370-40
Clinicaltrials.gov
NCT03036813
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
Dan
Rudin
171 Oyster Point Boulevard Suite 300 South San Francisco, CA 94080
United States of America
+1 650 534 2574
drudin@gbt.com
Global Blood Therapeutics Inc.
Scientific
Dan
Rudin
171 Oyster Point Boulevard Suite 300 South San Francisco, CA 94080
United States of America
+1 650 534 2574
drudin@gbt.com
Global Blood Therapeutics Inc.
Public
Dr. Adlette
Inati
Rafik Hariri University Hospital
Lebanon
+ 961 1 830000
adlette.inati@lau.edu.lb
Rafik Hariri University Hospital
Public
Dr. Miguel
Abboud
American University of Beirut Medical Center
Lebanon
+961 1 350 000
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
Rafik Hariri University 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
21/02/2019
Dr. Fuad Ziyadeh
irb@aub.edu.lb
+961 1 350000 ext 5445
Rafic Hariri University Hospital
31/01/2018
Dr.Iyad Issa
NA
+961 1 830000
Countries of Recruitment
Name
Lebanon
United States of America
United Kingdom
Netherlands
Canada
France
Egypt
Italy
Turkey
Jamaica
Oman
Kenya
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
Voxelotor
Oral tablet
Primary Outcomes
Name
Time points
Measure
Change in hemoglobin (Hb)>1g/dl
Baseline to Week 24
Proportion of participants with increase in Hb >1 g/dL
Key Secondary Outcomes
Name
Time points
Measure
Change from baseline in hemolysis measures
Baseline to Week 24
Analyze hemoglobin, unconjugated bilirubin, absolute reticulocyte, reticulocytes %, and LDH
Annualized VOC incidence rate
Baseline to Week 72
Number of VOC 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
Recruitment completion and study closure
16/06/2020
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