No registrations found.
ID
Source
Health condition
Parkinson's disease
Sponsors and support
Intervention
Outcome measures
Primary outcome
To evaluate the safety and tolerability of two oral dose levels of LTI-291 (10 and 60 mg) following 28 days of treatment in patients with GBA-PD.
Secondary outcome
To characterize the plasma PK of LTO-291 (10 and 60 mg) in patients with GBA-PD.
Background summary
Background of the study: LTI-291 is a new compound that may eventually be used for the treatment of patients with Parkinson’s disease who have a GBA1 mutation. LTI-291 is an activator of an enzyme named glucocerebrosidase (GCase). Everyone has this enzyme, but
studies have shown that in a subgroup of patients with Parkinson’s disease who have a mutation in a specific gene called
glucocerebrosidase 1 (GBA1), there is a deficiency of GCase which results in build-up of certain substrates of this enzyme
in the cells. LTI-291 acts by increasing the activity of the enzyme GCase to a normal level and thus leads to a decrease in
the build-up of substrates.
Objective of the study:
This study will be performed in patients with Parkinson’s disease who have a GBA1 mutation.
The purpose of this study is to investigate how safe the new compound LTI-291 is and how well it is tolerated when it is
administered to patients with Parkinson’s disease. It will also be investigated how quickly and to what extent LTI-291 is
absorbed and eliminated from the body (this is called pharmacokinetics). In addition, the effect of LTI-291 on the body (on
the brain and on certain biomarkers in the blood) will be investigated (this is called pharmacodynamics). LTI-291 will be compared with a placebo.
Study design
28 days.
Intervention
Multiple doses of LTI-291 10 or 60 mg once daily for 28 days.
Inclusion criteria
Mannelijke of vrouwelijke vrijwilliger; ziekte van Parkinson met een GBA1 mutatie; minimaal 18 jaar; BMI 18.0 - 35.0 (kilogram/meter2); gewicht minstens 45 kilogram.
Exclusion criteria
Lijdend aan hepatitis B, diabetes, hepatitis C, kanker of HIV/AIDS. Indien gedurende de 90 dagen voorafgaand aan de start van dit onderzoek aan een ander geneesmiddelenonderzoek is deelgenomen. Indien gedurende de 60 dagen voor start van dit onderzoek bloed is gegeven.
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 |
|---|---|
| NTR-new | NL7061 |
| NTR-old | NTR7299 |
| Other | LSO882EC-178821 : LTI-291-004 |