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Trial details
Trial details
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
Current status:
Approved
|
Date registered:
30/09/2019
Trial version(s)
History: 19/02/2019
History: 19/02/2019
History: 19/02/2019
Current: 19/02/2019
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2019090195
Protocol number
GBT440-007
MOH registration number
1288/ص
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Retrospective
Type of registration: Justify
Requested by Sponsor- Registry not in place upon study initiation
Date of registration in national regulatory agency
11/06/2014
Primary sponsor
Global Blood Therapeutics Inc.
Primary sponsor: Country of origin
United States of America
Public title
Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
Acronym
Scientific title
A Phase 2a, Open-label, Single and Multiple Dose Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Exploratory Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease
Acronym
Brief summary of the study: English
This study consists of three parts, Parts A, B, and C. - Part A is a single dose PK study in pediatric participants with Sickle Cell Disease. (Closed on 07 Aug 2017 (LPLV)) - Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years of age (Closed on 04 Jan 2019 (LPLV)) - Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of GBT440 is pediatric participants with Sickle Cell Disease who are 4 to 17 years of age.
Brief summary of the study: Arabic
عند الأطفال GBT440 دراسة لتقييم تأثير المصابين بمرض الخلايا المنجلية
Health conditions/problem studied: Specify
Sickle Cell Disease
Interventions: Specify
Drug: GBT440 administered as oral capsules, tablets or dispersible tablets
Key inclusion and exclusion criteria: Inclusion criteria
1. Male or female participants with homozygous hemoglobin SS (HbSS) or hemoglobin S beta0 thalassemia (HbS β0 thal). 2. Age: • Part A – 6 to 17 years of age. (Cohort 1 [12 to 17] and Cohort 2 [6 to 11] as defined in the Study Design) • Part B – 12 to 17 years of age • Part C – 4 to 17 years of age 3. A participant taking hydroxyurea (HU) may be enrolled if the dose has been stable for at least 3 months with no anticipated need for dose adjustments during the study and no sign of hematological toxicity. 4. Hemoglobin (Hb): • Part A – No restriction • Part B – Hb ≤10.5 g/dL • Part C – Hb ≤10.5 g/dL 5. Written informed parental/guardian consent and participant assent has been obtained per institutional review board (IRB)/Ethics Committee (EC) policy and requirements, consistent with ICH guidelines.
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
4
Key inclusion and exclusion criteria: Age maximum
17
Key inclusion and exclusion criteria: Exclusion criteria
1. Any one of the following requiring medical attention within 14 days prior to signing the Informed Consent Form (ICF): • Vaso-occlusive crisis (VOC) • Acute chest syndrome (ACS) • Splenic sequestration crisis • Dactylitis 2. Requires chronic transfusion therapy 3. History of stroke or meeting criteria for primary stroke prophylaxis (history of two TCD measurements ≥200 cm/sec) 4. Transfusion within 30 days prior to signing the ICF
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
N/A: Single arm study
Study design: Masking
Open (masking not used)
Study design: Control
Dose comparison
Study phase
2
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
No
Name of IMP
GBT440 (Voxelotor)
Type of IMP
Others
Type of IMP: Specify
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
N/A
Target sample size
24
Actual enrollment target size
32
Date of first enrollment: Type
Actual
Date of first enrollment: Date
21/07/2016
Date of study closure: Type
Actual
Date of study closure: Date
31/01/2021
Recruitment status
Recruiting
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
N/A
Additional data URL
https://clinicaltrials.gov/ct2/show/NCT02850406
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Clinicaltrials.gov
NCT02850406
EU Clinical Trials Registry
EudraCT: 2016-004209-15
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
Dan
Rudin
171 Oyster Point Blvd., Suite 300 South San Francisco, CA 94080
United States of America
(650) 534-2574
drudin@gbt.com
Global Blood Therapeutics
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-Oncology
Approved
Rafik Hariri University Hospital
Dr. Adlette Inati
Pediatric Hematology-Oncology
Approved
Nini Hospital
Dr. Adlette Inati
Pediatric Hematology-Oncology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
American University of Beirut Medical Center
09/07/2018
Dr. Fuad Ziyadeh
irb@aub.edu.lb
9611738025
Rafic Hariri University Hospital
31/08/2018
Dr. Iyad Issa
NA
9611830000
Nini Hospital
31/08/2018
Dr. Nabil Kabbara
NA
9616431400
Countries of Recruitment
Name
Lebanon
United States of America
United Kingdom
Health Conditions or Problems Studied
Condition
Code
Keyword
Sickle Cell Disease
Sickle-cell disorders (D57)
Sickle Cell, Anemia, Hemolytic, Congenital, Hematologic Diseases
Interventions
Intervention
Description
Keyword
Drug
GBT440
Oral Capsule, Tablet or Dispersible Tablet
Primary Outcomes
Name
Time points
Measure
Part A: Pharmacokinetic profile of GBT440 including maximum concentration
Pre-dose to Day 15
Pharmacokinetic profile
Part A: Pharmacokinetic profile of GBT440 including the time taken to reach the maximum concentration
Pre-dose to Day 15
Pharmacokinetic profile
Part A: Pharmacokinetic profile of GBT440 including the total drug concentration over time
Pre-dose to Day 15
Pharmacokinetic profile
Part B: Change in hemoglobin
Baseline to Week 24
Hemoglobin in Blood
Part C: Change in cerebral blood flow
Baseline to Week 48
TAMM TCD velocity
Key Secondary Outcomes
Name
Time points
Measure
Part A: Number of participants with treatment-related adverse events
Days 1 - 15
Assessed by CTCAE v4.03
Part B: Multiple Dose effect on Clinical Measures of Hemolysis
Day 1 - Week 24
Clinical Measures of Hemolysis
Part B: Pharmacokinetic profile of GBT440 including maximum concentration
Pre-dose to Week 24
Pharmacokinetic profile
Part B: Pharmacokinetic profile of GBT440 including the time taken to reach the maximum concentration
Pre-dose to Week 24
Pharmacokinetic profile
Part B: Pharmacokinetic profile of GBT440 including the total drug concentration over time
Pre-dose to Week 24
Pharmacokinetic profile
Part C: Multiple dose effect on clinical measures of hemolysis
Baseline to Week 24 and Week 48
Clinical Measures of Hemolysis
Part C: Change in cerebral blood flow
Baseline to Week 24
Measured by the TAMM TCD velocity
Part C: Pharmacokinetic profile of GBT440 including maximum concentration
Pre-Dose to Week 48
Pharmacokinetic profile
Part C: Pharmacokinetic profile of GBT440 including the time taken to reach the maximum concentration
Pre-Dose to Week 48
Pharmacokinetic profile
Part C: Pharmacokinetic profile of GBT440 including the total drug concentration over time
Pre-Dose to Week 48
Pharmacokinetic profile
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