In this study we will investigate what the effect of food is on how the study compound PTC518 is absorbed, transported, and eliminated from the body.We will also investigate how safe the new compound PTC518 is and how well it is tolerated when it is…
ID
Source
Brief title
Condition
- Neurological disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Characterize the effect of food on the PK of PTC518 administered to healthy
subjects under fed and fasted conditions.
Secondary outcome
Characterize the safety and tolerability of a single dose of PTC518
administered to healthy subjects under fed and fasted conditions.
Background summary
PTC518 is a new compound that may potentially be used for the treatment of
Huntington*s disease. Huntington*s disease is an inherited disease in the brain
where nerve cells are broken down. This causes problems with movement and
thinking. Huntington*s disease is caused by an error in the genetic information
(DNA) that results in a harmful version of a protein called huntingtin. This
version of the huntingtin protein damages brain cells. Reducing the amount of
huntingtin is a potential way to treat Huntington*s disease. The study compound
PTC518 aims to reduce the production of the huntingtin protein.
Study objective
In this study we will investigate what the effect of food is on how the study
compound PTC518 is absorbed, transported, and eliminated from the body.
We will also investigate how safe the new compound PTC518 is and how well it is
tolerated when it is used by healthy participants.
PTC518 has been used by humans before. In addition, it has been extensively
tested in the laboratory and on animals.
Study design
For the study it is necessary that the volunteer stays in the research center
for 2 periods of 5 days (4 nights). The volunteer will also come back to the
research center for a visit after leaving each period (Day 11 [±3 days] and Day
32 [±3 days]).
Day 1 and Day 22 are the days that the volunteer will receive the study
compound. The volunteer will be expected at the research center the day before
the day of intake of the study compound. The volunteer will leave the research
center after 4 nights in the research center (Day 4 and Day 25).
There will be 3 weeks between dosings.
Below is an overview of the days the volunteer stays at the research center, or
when they visit the research center.
Screening:
Day -28 up to Day -2
Period 1:
Arrival on Day -1
In house stay from Day -1 up to Day 4
Departure on Day 4
Visit on Day 11 (±3 days)
Period 2:
Arrival on Day 21
In house stay from Day 21 up to Day 25
Departure on Day 25
Visit on Day 32 (±3 days)
Follow-up phone call:
Day 35
The volunteer will be given PTC518 as an oral tablet with 240 milliliters (mL)
of (tap) water.
Prior to dosing the volunteer has to fast for at least 10 hours. During
fasting, water is allowed, except 1 hour prior to until 1 hour after each
intake of the study compound. Depending on what group the volunteer is in, they
will either get a high-fat breakfast, low-fat breakfast, or no breakfast prior
to dosing in each period. The volunteers will get 2 of these 3 possibilities.
There are 6 treatment sequences possible. Which sequence you will follow will
be determined by drawing lots:
Sequence 1 = during period 1 a low-fat breakfast and during period 2 a high-fat
breakfast
Sequence 2 = during period 1 a high-fat breakfast and during period 2 a low-fat
breakfast
Sequence 3 = during period 1 a low-fat breakfast and during period 2 fasting
Sequence 4 = during period 1 fasting and during period 2 a low-fat breakfast
Sequence 5 = during period 1 a high-fat breakfast and during period 2 fasting
Sequence 6 = during period 1 fasting and during period 2 a high-fat breakfast
If dosing is done after breakfast, the breakfast must be started exactly on
time and must be finished within 20 minutes. The entire breakfast must be
consumed.
Hands and mouth of the volunteers will be inspected after the study compound
intake. This it to check if they have taken the study compound.
Intervention
The volunteer will receive 20 milligram of PTC518 once per period, so in total
2 times.
Study burden and risks
Blood draw:
Drawing blood may be painful or cause some bruising. The use of the indwelling
cannula can sometimes lead to inflammation, swelling, hardening of the vein,
blood clotting, and bleeding in the environment 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 109 mL of blood 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 at once each time. If the investigator
thinks it is necessary for the safety of a participant, 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 will be placed on arms, chest and legs.
Prolonged use of these electrodes can cause skin irritation.
Fasting:
If someone has 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 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 throat may cause gagging. When the sample is taken from the back of the
nose, The volunteer may experience a stinging sensation and eyes may become
watery.
Corporate Court 100
South Plainfield 07080 NJ
US
Corporate Court 100
South Plainfield 07080 NJ
US
Listed location countries
Age
Inclusion criteria
1. Subjects must understand the nature of the study and must provide signed and
dated written informed consent before the conduct of any study-related
procedures and comply with all protocol requirements.
2. Sex: Male or female
3. Age: 18 to 65 years, inclusive, at Screening.
4. Body mass index (BMI): >=18.0 to 30.0 kg/m2 with a body weight >=50.0 kg for
male subjects and a body weight >=45.0 kg for female subjects at Screening.
5. Healthy as determined by the investigator or designee, based upon a medical
evaluation including medical history, physical examination, laboratory test
results, ECG recording (eg, a corrected QT interval using Fridericia*s formula
[QTcF] <=450 msec for males and QTcF <=470 msec for females), and vital signs at
Screening. Out-of-range values can be repeated once.
Further criteria apply, see protocol.
Exclusion criteria
1. Employee of PRA Health Sciences (PRA) or PTC.
2. History of hypersensitivity reactions to any excipients in the study drug
and/or food allergies.
3. History of malignancy within 5 years, unless one of the following, treated
and considered cured: basal cell carcinoma, in situ cervical cancer, or breast
ductal carcinoma in situ.
4. Previously randomized in this clinical trial.
5. Veins unsuitable for repeated venipuncture or for cannulation.
Further criteria apply, see protocol.
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 | EUCTR2021-006463-23-NL |
| CCMO | NL80261.000.22 |