This grant builds upon the research from a prior grant: Anle138b Clinical Phase 1 Studies: Food Effect, Safety and Tolerability as well as Pharmacokinetics in Parkinson´s Disease Patients
Study Rationale:
Pathological alpha-synuclein aggregates, called Lewy bodies, are a hallmark of Parkinson’s disease (PD) and a promising target for therapeutics. Anle138b is a compound that inhibits alpha-synuclein aggregation and holds promise for slowing disease progression. Initial data from control volunteers who took anle138b capsules while fasting showed good blood levels and safety. Before testing anle138b in long-term studies, we want to learn if anle138b can be taken with food and if there is any difference in safety or in blood level profiles.
Hypothesis:
We hypothesize that (i) there will be no difference in terms of safety, tolerability or blood levels of anle138b between control volunteers and people with mild to moderate PD that could influence trial design or dosing and (ii) that anle138b can be taken with food.
Study Design:
Participants will take two dose levels in ascending order for seven days. Dosing will be double-blinded and placebo-controlled. To study the effect of food, on Day 8 volunteers will take anle138b with a standard breakfast. Potential changes in drug levels will be compared to those in control volunteers who take the drug first in a fasting condition and again after a standardized breakfast on a separate day.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
This study will provide us the basis for determining the best possible dosing scheme for a clinical study to test the efficacy of anle138b in people with PD, a crucial step toward determining whether anle138b might be suitable as a treatment to slow or stop the disease progression.
Next Steps for Development:
Based on the data from this trial, we will finalize the ongoing plans for disease-modification studies in synucleinopathies such as Multiple System Atrophy and PD.
Additional Support:
The Michael J. Fox Foundation would like to acknowledge the generous contributions of The Silverstein Foundation for Parkinson's with GBA and the Demoucelle Parkinson Charity as lead supporters providing funding for this project.