Primary objective: To investigate the pharmacokinetics (PK) of multiple doses arimoclomol in plasma en urine.To investigate and quantify metabolites of arimoclomol in plasma and urine (reported seperately)Secondary objective: To evaluate the safety…
ID
Source
Brief title
Condition
- Other condition
Synonym
Health condition
diseases regarding conditions that involve misfolded proteins or protein aggregation disorders
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Pharmacokinetic parameters of arimoclomol including Cmax, tmax, AUC0-t, AUC0-8,
AUC0-inf, CL/F, Vz/F, and t*, Cavg, AR, Ae, fe and CLR after multiple dose
administration in healthy young male subjects.
Secondary outcome
Safety parameters include: adverse events (AEs), clinical laboratory
assessments, vital signs, weight, ECG, physical and neurological examination
and C-SSRS scores after multiple dose administration in healthy young male
subjects.
Background summary
The heat shock response is a natural defense mechanism in all cells. It
protects the cells from accumulation of misfolded proteins or other waste
products, which would otherwise lead to toxicity and disease. Arimoclomol is an
orally available small molecule that readily crosses the blood brain barrier to
stimulate an increased production of HSP, in particular HSP70, in stressed
cells. Arimoclomol may therefore be used to treat conditions that involve
misfolded proteins, including lysosomal storage disease (LSD) (for example
Niemann Pick disease, type C (NPC) and Gaucher's disease (GD)) and protein
aggregation disorders (for example amyotrophic lateral sclerosis (ALS) and
sporadic inclusion body myositis (sIBM)).
Study objective
Primary objective:
To investigate the pharmacokinetics (PK) of multiple doses arimoclomol in
plasma en urine.
To investigate and quantify metabolites of arimoclomol in plasma and urine
(reported seperately)
Secondary objective:
To evaluate the safety and tolerability of multiple doses arimoclomol.
Study design
This study is an interventional, open-label, multiple-dose study in healthy
young men.
Intervention
All subjects will receive multiple doses of 400 mg arimoclomol three times a
day (TID) for 5 consecutive days and a single morning dose on Day 6.
Study burden and risks
Since this study is being executed in healthy volunteers. There are no
anticipated benefits of the IMP. Please see the IMPD for further information.
Ole Maaloes Vej 3
Copenhagen DK-2200 N
DK
Ole Maaloes Vej 3
Copenhagen DK-2200 N
DK
Listed location countries
Age
Inclusion criteria
subject is a man
subject is older than or 18 and younger than or 45 years of age at Screening
For more inclusion criteria, please refer to the protocol
Exclusion criteria
The subject has a history of severe drug allergy or hypersensitivity.
The subject has taken any investigational medicinal product within 3 months prior to the first dose of IMP.
For more exclusion criteria, please refer to the protocol
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 | EUCTR2018-002517-34-NL |
| CCMO | NL66925.056.18 |