In this study we will investigate how quickly and to what extent GBT021601 is absorbed, transported, and eliminated from the body. For this study, GBT021601 is radioactively labelled with carbon-14 (14C). In this way, GBT021601 can be traced in…
ID
Source
Brief title
Condition
- Red blood cell disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
- To determine the whole blood and plasma concentrations of [14C]-GBT021601
total radioactivity.
- To assess the mass balance by determining [14C]-GBT021601 total radioactivity
excreted in urine and feces.
- To determine the PK of GBT021601 in whole blood, plasma, and urine.
Secondary outcome
- To assess the safety and tolerability of GBT021601 administration in healthy
participants.
- To characterize and identify metabolites of [14C]-GBT021601 in whole blood,
plasma, urine, and feces.
Background summary
GBT021601 is a new compound that may potentially be used for the treatment of
sickle cell disease (also known as sickle cell anemia). This disease causes
severe tiredness and attacks of pain, and increases susceptibility to
infections. People with sickle cell disease generally have a lower life
expectancy.
Sickle cell disease is an inherited form of anemia, because red blood cells
have the shape of a sickle. Sickle cells are less able to carry oxygen around
the body than normal red blood cells.
GBT021601 is being developed with the aim of improving transport of oxygen
around the body and preventing the formation of sickle cells.
Study objective
In this study we will investigate how quickly and to what extent GBT021601 is
absorbed, transported, and eliminated from the body. For this study, GBT021601
is radioactively labelled with carbon-14 (14C). In this way, GBT021601 can be
traced in blood, urine, and feces.
We also investigate how safe the new compound GBT021601 is and how well it is
tolerated when it is used by healthy subjects. In addition, we look at
breakdown products (metabolites) of GBT021601 in blood, urine, and feces.
GBT021601 has been administered to humans before. In addition, it has been
extensively tested in the laboratory and on animals.
Study design
The study will take a maximum of 8 months from the screening until the
follow-up visit.
In total the volunteer will visit the research center up to 13 times:
- once for the screening.
- once for a long stay in the research center.
- Up to 11 times for a 24 hours visit to the research center.
The volunteer will once receive 200 mg 14C radioactively labeled GBT021601.
Intervention
The volunteer will be given 200 milligrams (mg) 14C labeled GBT021601 as an
oral solution of 400 milliliters (mL). This oral solution may have a bitter
taste. After administration of the oral solution, the vial will be rinsed twice
times with 50 mL of water, which the volunteer will also be required to drink.
Study burden and risks
Blood draw
Drawing blood may be painful or cause some bruising. The use of the indwelling
cannula (a tube in a vein in the arm) can sometimes lead to inflammation,
swelling, hardening of the vein, blood clotting, and bleeding in the
environment (bruising) of the puncture site. In some individuals, a blood draw
can sometimes cause pallor, nausea, sweating, low heart rate, or drop in blood
pressure with dizziness or fainting.
In total, we will take about 535 milliliters (mL) of blood from the volunteer
from screening to follow-up. This amount does not cause any problems in adults.
To compare: a blood donation involves 500 mL of blood being taken each time at
once. If the investigator thinks it is necessary for the safety of a subject,
extra samples might be taken for possible additional testing. If this happens,
the total amount of blood drawn may be more than the amount indicated above.
Heart tracing
To make a heart tracing, electrodes (small, plastic patches) will be placed on
the volunteers arms, chest and legs. Prolonged use of these electrodes can
cause skin irritation (rash and itching).
Fasting
If the volunteer have to fast for a prolonged time during the study, this may
lead to symptoms such as dizziness, headache, stomach upset, or fainting.
Coronavirus test
Samples for the coronavirus test will be taken from the back of the volunteers
nose and throat using swabs. Taking the samples only takes a few seconds, but
can cause discomfort and can give an unpleasant feeling. Taking a sample from
the back of the volunteers throat may cause the volunteer to gag. When the
sample is taken from the back of the volunteers nose, the volunteer may
experience a stinging sensation and the volunteers eyes may become watery.
Oyster Point Boulevard 181
South San Francisco CA 94080
US
Oyster Point Boulevard 181
South San Francisco CA 94080
US
Listed location countries
Age
Inclusion criteria
1. Sex: Male or female.
2. Age: 18 to 55 years, inclusive, at screening.
3. Body mass index (BMI): 18.0 to 27.0 kg/m2, inclusive, at screening.
4. Body weight: >=50 kg at screening.
5. Good physical and mental health on the basis of medical and surgical
history, physical examination, clinical laboratory (including clinical
chemistry, hematology, urinalysis, and coagulation), 12 lead electrocardiogram
(ECG), and vital signs, as judged by the Investigator.
Further criteria apply
Exclusion criteria
1. Employee of ICON or the Sponsor.
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 ADME 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 a history of
appendectomy are eligible for enrollment.
4. History of chronic constipation, or recent complaints of an irregular
defecation pattern (ie, less than once per day on average) in the opinion of
the Investigator.
5. History of surgery requiring general anesthesia (not including local
procedures) or major trauma within 12 weeks of screening, or a planned surgery
during participation in the study.
Further criteria apply
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2022-003108-34-NL |
| CCMO | NL82705.056.22 |