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Trial details
Trial details
Open-Label Extension of Voxelotor
Current status:
Approved
|
Date registered:
26/10/2022
Trial version(s)
History: 12/06/2020
Current: 12/06/2020
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Main Information
Primary registry identifying number
LBCTR2020063513
Protocol number
GBT440-038
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
25/06/2020
Primary sponsor
Global Blood Therapeutics Inc.
Primary sponsor: Country of origin
United States of America
Public title
Open-Label Extension of Voxelotor
Acronym
Scientific title
An Open-Label Extension Study of Voxelotor Administered Orally to Participants with Sickle Cell Disease Who Have Participated in Voxelotor Clinical Trials
Acronym
Brief summary of the study: English
Open-label extension (OLE) study of Voxelotor for participants with Sickle Cell Disease who have participated in Voxelotor clinical trials. Approximately 600 participants with sickle cell disease (SCD), aged ≥ 4 to >18year years will be enrolled at approximately 70 global clinical sites. Participants aged ≥ 12 years will receive a voxelotor dose of 1500 mg QD, regardless of their body weight. Participants aged < 12 years will receive a voxelotor dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg QD. The participant’s weight at study entry will be used to determine the starting voxelotor dose in this study. The dose should be adjusted if the participant’s weight increases or decreases at a scheduled clinic visit. The objective of this OLE is to assess the safety of, and SCD-related complications of, long-term treatment with Voxelotor, in participants who have completed treatment in a Global Blood Therapeutics (GBT)-sponsored Voxelotor clinical study.
Brief summary of the study: Arabic
دراسة تكميلية على voxelotor للمشاركين ابتداءاً من عمر 4 أعوام و المصابين بمرض الخلايا المنجلية و الذين شاركوا في التجارب السريرية السابقة على مستحضر voxelotor
Health conditions/problem studied: Specify
Sickle Cell Disease
Interventions: Specify
Drug: Voxelotor (GBT440) All participants will receive voxelotor once daily (QD), administered orally as tablets, dispersible tablets, or powder for oral suspension formulation
Key inclusion and exclusion criteria: Inclusion criteria
1. Male or female participant with SCD, aged ≥ 4 years, who participated and received study drug in a GBT-sponsored voxelotor pediatric clinical study Note: Participants 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 voxelotor. 2. Female participants of childbearing potential are required to have a negative urine pregnancy test before dosing on Day 1. Note: Female participants who become childbearing during the study must be willing to have a negative urine pregnancy test to remain in the study. 3. If sexually active, female participants of childbearing potential must use highly effective methods of contraception until 30 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 30 days after the last dose of study drug. 4. Participant has provided written assent (both the consent of the participant’s legal representative or legal guardian and the participant’s assent [where applicable] must be obtained) 5. Subjects with abnormal TCD who have not completed Study GBT440-032 can participate in OLE study
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
4
Key inclusion and exclusion criteria: Age maximum
99
Key inclusion and exclusion criteria: Exclusion criteria
1. Female participant who is breastfeeding or pregnant 2. Participant withdrew consent from a GBT-sponsored voxelotor pediatric clinical study 3. Participant was lost to follow-up from a GBT-sponsored voxelotor pediatric clinical study 4. Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent 5. Based on the most recent Oxbryta® US label (December 2021), co-administration with both moderate and strong CYP3A4 inducers should be avoided
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
N/A
Study design: Masking
Open (masking not used)
Study design: Control
Active
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
Yes, Worldwide
IMP has market authorization: Specify the countries
United States of America
Name of IMP
Voxelotor (Oxbryta)
Year of authorization
2019
Month of authorization
11
Type of IMP
Others
Type of IMP: Specify
Chemical
Pharmaceutical class
Allosteric modulator of hemoglobin oxygen affinity
Therapeutic indication
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
Target sample size
24
Actual enrollment target size
21
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
11/08/2020
Date of study closure: Type
Anticipated
Date of study closure: Date
30/06/2026
Recruitment status
Pending
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
N/A
Additional data URL
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Clinicaltrials.gov
NCT04188509
WHO International Clinical Trials Registry Platform
EUCTR2019-003144-76-GB
Sources of Monetary or Material Support
Name
Global Blood Therapeutics Inc. USA
Secondary Sponsors
Name
N/A
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Dr. Miguel
Abboud
Beirut
Lebanon
9611350000
ma56@aub.edu.lb
American University of Beirut Medical Center
Scientific
Margaret
Tonda
181 Oyster Point Blvd. South San Francisco, CA 94080
United States of America
650 741 7761
mtonda@gbt.com
Global Blood Therapeutics Inc.
Public
Dr. Adlette
Inati
Tripoli
Lebanon
9613228033
adlette.inati@lau. edu.lb
Nini Hospital
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
Hematology
Not approved
Nini Hospital
Dr. Adlette Inati
Hematology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Nini Hospital
15/06/2020
Dr. Nabil Kabbara
N/A
9616431400
Countries of Recruitment
Name
United States of America
United Kingdom
Lebanon
Brazil
Egypt
Oman
Kenya
Nigeria
Ghana
Health Conditions or Problems Studied
Condition
Code
Keyword
Sickle-Cell Disorder
Sickle-cell disorders (D57)
Hematology, Sickle Cell, Disorder
Interventions
Intervention
Description
Keyword
Drug
Voxelotor
GBT440
Primary Outcomes
Name
Time points
Measure
Treatment Emergent Adverse Events and Serious Adverse Events
Throughout entire study
N/A
Sickle Cell Disease-Related Complications
Throughout entire study
Frequency of SCD-related complications
Key Secondary Outcomes
Name
Time points
Measure
N/A
N/A
N/A
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