A Single and Multiple Dose Study to Determine the Safety and Tolerability of GBT021601 in Healthy Adult Volunteers - Part A
- Conditions
- Sickle Cell DiseaseBlood - Haematological diseasesHuman Genetics and Inherited Disorders - Other human genetics and inherited disorders
- Registration Number
- ACTRN12620001324909
- Lead Sponsor
- inear Clinical Research Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 64
1. Healthy males or females between 18 and 55 years inclusive at screening.
2. No clinically significant findings as assessed by review of medical and surgical history,
vital signs assessments, 12-lead ECG, physical examination, and clinical laboratory
evaluations conducted at screening and on day of admission. In the event of clinically
significant abnormalities, a single repeat measurement/test may be performed at the
discretion of the investigator to confirm eligibility based upon initial vital signs, ECG, or
clinical laboratory tests.
3. Body mass index (BMI) between 18.0 and 30.0 kg/m^2 inclusive, and body weight more than or equal to50 kg at screening and Day -1. BMI = weight (kg)/(height[m])^2
4. Females must be of non-childbearing potential (eg, postmenopausal [defined as
amenorrhoeic for at least one year with a follicle stimulating hormone level >40 IU/L] or
surgically sterile by hysterectomy, bilateral tubal ligation, bilateral salpingectomy, or
bilateral oophorectomy [documentation required]).
5. Males who are not surgically sterile with partners of childbearing potential must agree to
use a highly effective method of birth control during the study and for 120 days following
study drug dose. A highly effective method of contraception is defined as one that results
in a low documented failure rate when used consistently and correctly such as: condom
plus use of an intrauterine device; intrauterine system or hormonal method of
contraception (oral, injected, implanted, or transdermal) for your female partner; or
sexual abstinence.
6. Males must agree to not donate sperm during the study and for 120 days following study
drug dosing.
7. Participants must be able to communicate effectively in English with the study personnel.
8. Participants must be nonsmokers, defined as having abstained from tobacco- or nicotine- containing products (eg, cigarettes, cigars, chewing tobacco, snuff, nicotine patches, and electronic cigarettes) in the 6 months prior to screening.
9. Participants must be informed of the nature and risks of the study and give written
consent prior to screening.
1. Positive pregnancy test or currently breastfeeding.
2. History or presence of clinically significant allergic diseases (except for untreated,
asymptomatic, seasonal allergies) at time of dosing, in the opinion of the investigator.
3. History or presence of conditions which, in the opinion of the investigator, are known to
interfere with the absorption, distribution, metabolism, or excretion of drugs, such as
previous surgery on the gastrointestinal tract (including removal of parts of the stomach,
bowel, liver, gall bladder, or pancreas). Participants who have had an appendectomy are
eligible for enrollment.
4. History or presence of conditions that may place the participant at increased safety risk as determined by the investigator.
5. History of surgery or major trauma within 12 weeks of screening, or surgery planned during the study.
6. Any signs and/or symptoms of acute illness, including COVID-19, at screening, Day -1, or Day 1.
7. Abnormal ECGs collected at screening or Day -1, including QT interval corrected for
heart rate (HR) using Fridericia’s formula (QTcF) >450 msec for males and >470 msec
for females, or any cardiac rhythm other than sinus rhythm that is interpreted by the
investigator to be clinically significant. An additional triplicate ECG may be performed
to confirm ECG abnormalities (ie, to confirm that a participant is eligible).
8. Known personal or family history of congenital long QT syndrome or known family
history of sudden death.
9. Resting bradycardia (HR <45 beats per minute [bpm]) or resting tachycardia
(HR >100 bpm) at screening or Day -1 based on vital signs measurements. A single
repeat measurement may be performed to confirm vital sign abnormalities (ie, to confirm
that a participant is eligible). Each of the readings must be not clinically significant to
qualify for enrollment into the study.
10. Hypertension, defined as resting systolic blood pressure (BP) >140 mmHg or resting
diastolic BP >90 mmHg at screening or Day -1. A single repeat measurement may be
performed to confirm vital sign abnormalities (ie, to confirm that a participant is eligible).
Each of the readings must be not clinically significant per the investigator’s discretion to
qualify for enrollment into the study.
11. History of alcohol abuse or use of drugs of abuse in the 12 months prior to the date of
consent. Excess alcohol is defined as more than 14 drinks/week for women and 21 drinks/week for men. A drink is defined as 360 mL of beer 150 mL of wine, or 45 mL of spirits.
12. Use of prescription medications within 14 days or 5 half-lives, whichever is longer, or
any over-the-counter (OTC) drugs, including herbal preparations or dietary supplements
within 7 days or 5 half-lives, whichever is longer, prior to Day -1 or requiring continued use during study participation. Occasional use of acetaminophen/paracetamol (e.g. up to 2g/day) is permitted up to 24 hours before the first dose.
13. Consumption of more than 400 mg of caffeine (approximately 4 cups of coffee) per day
within 30 days of screening or unwillingness to abstain from consumption of caffeine- or
xanthine-containing products (eg, tea, coffee, chocolate, cola, etc.) from 48 hours prior to
dosing (Day 1) through Day 7 (Part A) and from 48 hours prior to dosing and through
Day 21 (Part B).
14. Unwillingness to abstain from alcohol from 48 hours prior to dosing (Day 1) through
Day 29.
15. Consumption of grapefruit and/or grapefruit juic
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Part A:<br>To evaluate the safety and tolerability of single ascending doses of GBT021601 in healthy participants as assessed by the collection of safety data, such as adverse events, clinical laboratory tests, vital signs, Physical Exams and various ECGs.[Daily assessments during the inpatient period (up to 7 days post dose) and during the follow up period, up to Day 98 (9 visits 4-14 days apart)]
- Secondary Outcome Measures
Name Time Method