NCT06263010

Allopregnanolone as a Regenerative Treatment for Parkinson's Disease

Allopregnanolone as a Regenerative Treatment for Parkinson's Disease: An Open-label, Pilot Clinical Trial

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Roberta Brinton · Academic / Other
• Sex: All
• Age: 40 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The goal of this open-label, pilot clinical trial is to test allopregnanolone as a regenerative treatment in patients with Parkinson's disease (PD). The main questions this study aims to answer are: 1. Is a large-scale clinical trial testing how well it works in patients with PD feasible? 2. Is allopregnanolone safe and well-tolerated in patients with PD. 3. Can we see any signals of changes in imaging and clinical scales? Participants will receive a weekly infusion in their vein of allopregnanolone for a total of 12 weeks. There is no placebo so everyone receives allopregnanolone and "Open-label" means the study is not blinded so both the participant and investigators know the assigned treatment.

Detailed description

This is an open-label, pilot clinical trial of allopregnanolone (Allo) as a regenerative treatment for Parkinson's disease. A total of 10 study participants will receive weekly infusions of Allo for 12 weeks. Participants will be male and female, age 40-80 years with a history of idiopathic, sporadic PD who have a Hoehn \& Yahr stage 1-4. Allo is a potent neuroregenerative agent that promotes proliferation of human neural stem cells and has the potential to function as a regenerative therapeutic to restore motor function in persons with PD. Results from several preclinical studies in rodent models of PD confirm improved motor function after Allo treatment. Primary Objective: To assess the feasibility of a large-scale trial to determine the efficacy of weekly infusions of Allo in in patients with Idiopathic sporadic PD. Secondary Objectives: To evaluate the safety and tolerability of weekly infusions of Allo in participants with idiopathic sporadic PD, and assess single-dose pharmacokinetics of allopregnanolone. Tertiary / Exploratory Objectives: To assess efficacy signals of weekly infusions of Allo in participants with idiopathic sporadic PD. * Determine target engagement of Allo using dopamine transporter (DaT) imaging and magnetic resonance imaging (MRI). * Evaluate effect of Allo on motor function tests. * Evaluate the effect of Allo on cognitive function tests and clinical ratings. * Compare response to Allo administration between APOE4 carriers and non-carriers.

Conditions

• Parkinson Disease

Interventions

Type	Name	Description
DRUG	Allopregnanolone	Allopregnanolone is a neurosteroid ( 3α,5α-tetrahydroprogesterone, 3α-hydroxy-5α-pregnan-20-one) and by-product of the metabolism of the hormone progesterone.

Timeline

• Start date: 2024-01-12
• Primary completion: 2025-07-15
• Completion: 2025-07-15
• First posted: 2024-02-16
• Last updated: 2026-03-13

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study