NCT03608371

BTRX-246040 Study in Participants With Parkinson's Disease With Motor Fluctuations

Phase 2A, Double-blind, Placebo-controlled Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Efficacy of BTRX-246040 in Parkinson's Disease Subjects With Motor Fluctuations

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: BlackThorn Therapeutics, Inc. · Industry
• Sex: All
• Age: 30 Years – 76 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetics and efficacy of BTRX-246040 in participants with Parkinson's Disease who have motor fluctuations and predictable early morning off periods.

Detailed description

Study treatment is 1 day and total duration of the study is up to 36 days, including an approximate 28-day screening period. The study consists of 3 sequential, ascending dose cohorts of 8 participants each with a 6:2 randomization to BTRX-246040 or placebo. The planned dosing for each cohort is 40, 80, and 120 mg. After enrollment of the first cohort is completed, doses for subsequent cohorts may be modified based on review of the available data (safety, tolerability, efficacy, and pharmacokinetics) by an unblinded Dosing Review Committee (DRC). A similar review and determination of dosing for the subsequent cohort is to be performed after completion of each cohort and based on all data available from previous cohorts. Participants who meet entry criteria assessed at the screening visit (up to 28 days prior to Day 1) present to the clinic on the morning of Day 1 (treatment day) in the practically defined OFF state (having withheld anti-parkinsonian medications after 10:00 PM the evening prior). Participants are to be dosed with study drug and remain on site for an 8-hour observation period with Unified Parkinson's Disease Rating Scale (UPDRS) Part III motor response, dyskinesia rating and ON/OFF status assessed pre-dose, every 30 minutes for 4 hours post-dose, and then hourly for 4 additional post-dose hours (i.e., 8 hours total post-dose) prior to being discharged. Blood for pharmacokinetics are to be collected 6 times at scheduled intervals within the 8-hour observation period. A follow-up safety visit is scheduled 7 days later.

Conditions

• Parkinson Disease
• Motor Disorder

Interventions

Type	Name	Description
DRUG	BTRX-246040	oral capsule
DRUG	Placebo	oral capsule matching BTRX-246040 capsule

Timeline

• Start date: 2018-08-31
• Primary completion: 2019-04-16
• Completion: 2019-04-23
• First posted: 2018-07-31
• Last updated: 2025-06-24
• Results posted: 2025-06-24

Locations

4 sites across 1 country: United States

Regulatory

• FDA-regulated drug study