The study will be performed in 2 parts, Part 1 and Part 2). The purpose of the study is to investigate how quickly and to what extent Olesoxime is absorbed and eliminated from the body. In addition, it will be investigated how safe Olesoxime is and…
ID
Source
Brief title
Condition
- Neuromuscular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The pharmacokinetic properties of 10mg/kg olesoxime as oral suspension and
20mg/kg olesoxime as oral solution
Secondary outcome
The safety and tolerability of 10mg/kg olesoxime as oral suspension and 20mg/kg
olesoxime as oral solution
Background summary
Olesoxime is a new investigational compound that may eventually be used for the
treatment of Spinal Muscular Atrophy (SMA), which is a genetic progressive
muscle disease.
The disease is caused by a defect in the so-called SMN1 gene. This genetic
defect can lead to dysfunction of nerve cells, which are needed to facilitate
muscle movement. Ultimately, muscles can become weakened or paralyzed.
Olesoxime is able to preserve the functioning of nerve cells. As a result,
Olesoxime protects nerve cells from death and not functioning properly.
Olesoxime is in development and is not registered as a drug but has been given
to humans before.
Study objective
The study will be performed in 2 parts, Part 1 and Part 2). The purpose of the
study is to investigate how quickly and to what extent Olesoxime is absorbed
and eliminated from the body. In addition, it will be investigated how safe
Olesoxime is and how well Olesoxime is tolerated.
This study is planned to be performed in 90 healthy male and female volunteers,
divided over 2 parts.
Part 1 will be performed in 30 healthy male and female volunteers divided over
2 groups. Each group will consist of 15 volunteers. Part 2 will be performed in
60 healthy male and female volunteers.
Study design
The study, conducted in the clinical research center in Groningen, consists of
a treatment period for which the volunteer is randomly assigned to one of the
two groups. Each group contains 15 volunteers. Group 1 receives olesoxime as an
oral suspension (10mg/kg), while group 2 receives olesoxime as an oral solution
(20mg/kg), in the fed state. Subjects will be dosed once and followed up in the
clinic for 48 hours after dosing. Follow-up visits to the clinic are planned
for day 5, 8, 12, 16 and 21.
Based on the pharmacokinetic results of this phase of the study, it will be
decided if phase 2 will be conducted. This optional part of the study consists
of 15 volunteers that will receive a dose of olesoxime that is smaller or equal
to 20mg/kg.
Part 2 of the study will consist of 1 period during which the volunteers will
receive Olesoxime once, either as a single administration, or divided over 2 or
3 administrations within a 24 hours period. Olesoxime is administered as a
drink. There are two different versions of this drink, one with sesame oil and
one without. Which version of the drink the volunteers receive and whether they
receive it as a single or divided administration will be decided by chance.
Regardless of which version of the drink they receive, or how it is
administered, they will have to drink between 4 and 40 milliliters in
volume.Part 2 of the study will be conducted in groups each containing at least
8 volunteers. They will only participate in one of these groups. The first two
clinic groups in Part 2 (Group 4 and Group 5 of the study) will receive the
version of the study compound which does not contain sesame oil. Half of the
volunteers will receive the drink as a single administration and half of the
volunteers will receive the drink divided over 2 administrations.
Intervention
Phase 1: 10mg/kg olesoxime as oral suspension, or 20mg/kg olesoxime as oral
solution (randomly assigned)
Study burden and risks
Olesoxime has been studied in a total of 17 completed studies in a total of
1468 healthy volunteers or patients (adults and children). From these subjects
or patients, 849 were exposed to daily doses ranging between 50 mg to 1000 mg
and treatment duration up to 33 months. In all studies Olesoxime has been shown
to be well tolerated at all test dose regimens. No risks were identified
following review of the currently available clinical trial data.
At the dose levels used in this study, no serious adverse reactions are to be
expected.
Grenzacherstrasse 124
Basel 4070
CH
Grenzacherstrasse 124
Basel 4070
CH
Listed location countries
Age
Inclusion criteria
1. Healthy male and female subjects, 18 to 64 years of age, inclusive. Healthy status is defined by absence of evidence of any active or chronic disease following a detailed medical and surgical history and a complete physical examination including vital signs, 12-lead ECG, hematology, blood chemistry, serology, and urinalysis.;2. For women of childbearing potential: agreement to remain abstinent or agreement to use an acceptable birth control method during the treatment period and for at least 90 days after the last dose of olesoxime.;3. A body mass index (BMI) between 18.0 to 30.0 kg/m2, inclusive, with a bodyweight < 100 kg on Day -1.;4. Able to participate and willing to give written informed consent and to comply with the study restrictions.
Exclusion criteria
1. Pregnant (or intending to become pregnant during the study) or lactating women, or positive urine pregnancy test at screening or day -1 (positive urine pregnancy test to be confirmed by a positive serum test).
2. History of any clinically significant gastrointestinal, renal, hepatic, broncho-pulmonary, neurological, psychiatric, cardiovascular, endocrinological, hematological or allergic disease (multiple allergies, seasonal allergy is acceptable), metabolic disorder, cancer, or cirrhosis.
3. In the opinion of the Investigator, any clinically significant major illness within one month before the screening examination or any febrile illness within one week prior to screening and up to first dose administration.
4. Clinically significant abnormalities in laboratory test results (including hepatic and renal panels, complete blood count, chemistry panel, and urinalysis) at screening. In the case of uncertain or questionable results, tests performed during screening may be repeated before randomization to confirm eligibility.
Design
Recruitment
Medical products/devices used
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 | EUCTR2016-002661-60-NL |
| CCMO | NL59229.056.16 |