MAD study in patients with Parkinson’s Disease for LY3962681
Research type
Research Study
Full title
A randomized, double-blind (sponsor-unblinded), placebo-controlled, single-ascending-dose study in healthy volunteers and a double-blind (sponsor-unblinded), placebo-controlled, multiple-ascending-dose study in patients with Parkinson’s disease to evaluate the safety, tolerability, and PK/PD of LY3962681.
IRAS ID
1011003
Contact name
Patricia Sondergaard
Contact email
Sponsor organisation
Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company
Clinicaltrials.gov Identifier
Research summary
The purpose of this double-blind, placebo-controlled, randomised single-ascending-dose (SAD) and multiple-ascending-dose (MAD) Phase 1 study is to assess the safety and tolerability of LY3962681, pharmacokinetics (PK)/pharmacodynamics (PD) and exploration of biomarkers in healthy volunteers and participants (ppts) with Parkinson’s disease.
Parkinson’s Disease is a progressive neurodegenerative disorder affecting more than 10million people worldwide. No treatment is available that modifies the progressive underlying disease process.
Double blind means, neither the participant (ppt) nor the study doctor will know if the ppt is receiving the drug (called LY3962681) or the placebo. The placebo is artificial Cerebral Spinal Fluid (CSF) with no active drug. PK means what the body does to the drug, and PD means what the drug does to the body.
In the UK only the MAD part of the study will take place in patients with Parkinson’s disease, no healthy volunteers will be recruited. Multiple ascending dose, MAD, means different participants will receive different amounts of the study drug, participants will start on lower dose, if the safety and tolerability are favorable then the next set of participants will be given a higher dose, three dose levels will be given. Approximately 60 participants with Parkinson’s disease will be recruited worldwide.
In Parkinson’s disease a protein called alpha-synuclein builds up in aggregates (clumps) in the brain. LY3962681 is a drug that acts to stop the synthesis of -synuclein, and therefore its continued aggregation. The sponsor hypothesises that this will stop the downstream consequences that result in neuronal (brain cell) toxicity and clinical progression.
Participants will have blood tests, questionnaires, lumbar punctures, brain MRIs, spine X-ray, DaT/SPECT Radiography Scan, as well as vital sign and physical examinations. Time on the study is 54 weeks, plus an additional 28 weeks for the monitoring follow up study.
REC name
London - Chelsea Research Ethics Committee
REC reference
25/LO/0132
Date of REC Opinion
25 Mar 2025
REC opinion
Further Information Favourable Opinion