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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT05036512




Registration number
NCT05036512
Ethics application status
Date submitted
13/08/2021
Date registered
5/09/2021
Date last updated
5/09/2021

Titles & IDs
Public title
A Study to Evaluate GBT021601 in Single and Multiple Doses in Healthy Participants
Scientific title
A Double-Blind, Randomized, Placebo-Controlled, Single and Multiple Ascending Dose (SAD/MAD) Study to Evaluate the Safety, Tolerability, PK, and Food Effect of GBT021601, a Hemoglobin S Polymerization Inhibitor, in Healthy Participants.
Secondary ID [1] 0 0
GBT021601-011
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Sickle Cell Disease 0 0
Condition category
Condition code
Blood 0 0 0 0
Anaemia

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GBT021601

Placebo Comparator: Placebo - Placebo as a tablet or capsule with dose based off of preceding cohort's data.

Experimental: GBT021601 - GBT021601 as a tablet or capsule with dose based off of preceding cohort's data.


Treatment: Drugs: GBT021601
Administered orally with water as a single dose in the morning.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety, as assessed by frequency and severity of adverse events (AEs) - AEs will be coded to system organ class and preferred term using the Medical Dictionary for Regulatory Activities (MedDRA) and summarized.
Timepoint [1] 0 0
119 days from screening Part A, 134 days from screening Part B
Primary outcome [2] 0 0
Safety, as assessed by changes in Heart Rate. - Number of participants with changes in heart rate (bpm) as compared to baseline.
Timepoint [2] 0 0
119 days from screening Part A, 134 days from screening Part B
Primary outcome [3] 0 0
Safety, as assessed by changes in eGFR - Number of participants with changes in eGFR from baseline
Timepoint [3] 0 0
119 days from screening Part A, 134 days from screening Part B
Primary outcome [4] 0 0
Safety, as assessed by changes in alanine aminotransferase (ALT) - Number of participants with changes in alanine aminotransferase (ALT)
Timepoint [4] 0 0
119 days from screening Part A, 134 days from screening Part B
Primary outcome [5] 0 0
Safety, as assessed by changes in Blood pressure - Number of participants with changes in systolic (mmHg) and diastolic (mmHg) blood
Timepoint [5] 0 0
119 days from screening Part A, 134 days from screening Part B
Primary outcome [6] 0 0
Plasma concentration - Time of Cmax
Timepoint [6] 0 0
119 days from screening Part A
Primary outcome [7] 0 0
Plasma concentration - Cmax on D1-D15
Timepoint [7] 0 0
134 days from screening Part B
Secondary outcome [1] 0 0
Determine whole blood concentration of GBT021601 - Hemoximetry will be used to assess oxygen saturation in whole blood by generating oxygen equilibrium curves (OECs) which relate the extent of Hb-O2 saturation to the partial pressure of O2 (pO2) and measure the binding affinity of O2 to Hb.
Timepoint [1] 0 0
119 days from screening Part A
Secondary outcome [2] 0 0
Determine plasma concentration of GBT021601. - With dosing data from each cohort determine the steady-state maximum plasma/whole blood concentration (Cmax).
Timepoint [2] 0 0
134 days from screening Part B
Secondary outcome [3] 0 0
Safety, as assessed by changes in QTcF - Number of participants with changes in the QTcF interval from baseline
Timepoint [3] 0 0
119 days from screening Part A, 134 days from screening Part B

Eligibility
Key inclusion criteria
- Healthy males and females = 18 to = 55 years of age

- Body mass index = 18.0 to = 30.0 kg/m2

- Body weight = 50 kg at screening and Day -1
Minimum age
18 Years
Maximum age
55 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Positive pregnancy test or currently breastfeeding.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Linear Clinical Research - Nedlands
Recruitment postcode(s) [1] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Global Blood Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This first in human study is designed to evaluate the safety, tolerability, pharmacokinetics
(PK), and food effect of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in healthy
participants.
Trial website
https://clinicaltrials.gov/show/NCT05036512
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Eleanor Lisbon, MD, MPH
Address 0 0
GBT
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Jessica Guider, MS, MPH, MBA
Address 0 0
Country 0 0
Phone 0 0
630-364-3275
Fax 0 0
Email 0 0
jguider@gbt.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT05036512