In this research study we will compare a new PTC857 formulation to the current PTC857 formulation. The aim of the new formulation is to improve ease of manufacturing and to reduce the effect of food on PTC857 uptake. Our aim is to study how quickly…
ID
Source
Brief title
Condition
- Neurological disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective of the study is to assess the relative bioavailability of
PTC857 following a single oral administration of the new (Phase 2) formulation
compared to current (Phase 1) formulation.
Secondary outcome
The secondary objective of the study is to assess the safety and tolerability
of PTC857 after administration of single doses of the new and current
formulations under fed (medium fat) conditions in healthy adult subjects.
Background summary
PTC857 is a new compound that is being studied for the treatment of
neurological disorders, such as Parkinson*s disease. Parkinson*s disease is a
brain disorder with symptoms such as arm or leg shaking and/or stiffness, and
difficulty with walking, balance, and coordination. It is caused by death of
specific nerve cells in the brain that are important for controlling movement.
There is currently no cure for Parkinson*s disease, and treatment is aimed at
reducing the symptoms. In Parkinson*s disease there is an overproduction of
highly reactive compounds that can damage cell structures, which in turn can
result in inflammation in the brain. PTC857 aims to reduce overproduction of
the highly reactive compounds by inhibiting a specific protein
(15-lipoxygenase).
PTC857 has been tested in humans before. In addition, it has been extensively
tested in the laboratory and on animals.
Study objective
In this research study we will compare a new PTC857 formulation to the current
PTC857 formulation. The aim of the new formulation is to improve ease of
manufacturing and to reduce the effect of food on PTC857 uptake.
Our aim is to study how quickly and to what extent both PTC857 formulations are
absorbed, transported, and eliminated from the body. Our other aim is to study
how safe both PTC857 formulations are and how well they are tolerated by
healthy participants.
Study design
For the study it is necessary that subjects stay in the research center for 1
period of 12 days (11 nights). In addition, we will call subjects approximately
27 days after leaving the research center.
Day 1 is the first day when subjects receive the study compound. Subjects will
leave the research center on Day 11 of the study.
Below is an overview of the days of the stay at the research center, or when
there's a visit at the research center.
Screening > Day -21 up to Day -2
Treatment period - Arrival > Day -1
Treatment period - In-house stay > Day -1 to Day 11
Treatment period - Departure > Day 11
Follow-up phone call > Day 38
Subjects will be given PTC857 as oral capsules with 240 milliliters (mL) of
water.
PTC857 will be given after they've consumed a medium-fat breakfast with a
standard composition. This breakfast must be started exactly on time and must
be finished within 25 minutes. The entire breakfast must be consumed. They have
to fast for at least 10 hours during the night before this breakfast.
One of the investigators will inspect the hands and mouth after subjects take
the study compound. This it to confirm they have taken the study compound.
Intervention
Subjects will receive PTC857 twice: the new formulation once and the current
formulation once. The order in which they receive the formulations will be
determined by chance. The first dose will be given on Day 1 and the second one
on Day 8. Each time they will receive a dose of 150 mg PTC857. Each capsule
contains 50 mg PTC857, meaning that they have to ingest 3 capsules for each
dosing. Each capsule is *size 0*, with a length of 21.7 mm and diameter of 7.6
mm.
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 (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 140 milliliters (mL) of blood. This amount does
not cause any problems in adults. To compare: a blood donation involves 500 mL
of blood being taken 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 will be
more than the amount indicated above, but the total amount of blood collected
will not be greater than 150 mL of blood.
Heart tracing
To make a heart tracing, electrodes will be placed on your arms, chest and
legs. Prolonged use of these electrodes can cause skin irritation (rash and
itching).
Meals/Fasting
The medium-fat breakfast is a study specific breakfast. Subjects must consume
the whole breakfast in 25 minutes. It can be difficult to consume the entire
breakfast, particularly for light eaters.
If they 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 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 subjects to gag. When the sample is taken from the back of
the nose, they may experience a stinging sensation and the eyes may become
watery.
100 Corporate Court
South Plainfield NJ 07080
US
100 Corporate Court
South Plainfield NJ 07080
US
Listed location countries
Age
Inclusion criteria
1. Males or females aged between 18 and 55 years old and have a body mass index
(BMI) between 18.5 and 30 kg/m2 (inclusive at Screening as well).
2. 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.
3. Females must be either postmenopausal for >=1 year or surgically sterile
(having undergone tubal ligation, hysterectomy, or bilateral oophorectomy) for
at least 6 months or, if of childbearing potential and not abstinent, willing
to use a highly effective method of contraception from Screening through 90
days after the last dose of study drug. For a detailed breakdown of the birth
control methods which may be considered as highly effective as well as the
acceptable birth control methods which may not be considered as highly
effective, refer to Section 11.4 and Section 11.5, respectively (Appendices).
4. Females who are abstinent will not be required to use a contraceptive method
unless they become sexually active.
5. Women of childbearing potential who are using hormonal contraception should
also employ, together with their male partner, at least one barrier method.
6. Females must refrain from ova (egg cell) donation during this time period.
Exclusion criteria
1. History of coagulopathy.
2. History of fat malabsorption.
3. Dietary restrictions that preclude participation.
4. History of allergies or adverse reactions to PTC857 or to any excipients in
the study drug formulation.
5. Females who are pregnant or nursing.
6. Subjects with a prior medical history of clinically significant
gastrointestinal, renal, hepatic, neurologic, hematologic, endocrine,
oncologic, pulmonary, immunologic, psychiatric, or cardiovascular disease or
any other condition which, in the opinion of the Investigator, would jeopardize
the safety of the subject or impact the validity of the study results.
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 | EUCTR2021-001948-10-NL |
| CCMO | NL77772.056.21 |