Allopregnanolone as a Regenerative Treatment for Parkinson's Disease

NCT06263010 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: CIBS-2023-01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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. 1.Is a large-scale clinical trial testing how well it works in patients with PD feasible?
2. 2.Is allopregnanolone safe and well-tolerated in patients with PD.
3. 3.Can we see any signals of changes in imaging and clinical scales?

Conditions

Parkinson Disease

Keywords

Parkinson; Allopregnanolone; Regenerative therapeutic

Outcome Measures

Primary Outcomes (1)

• Study completion

  Proportion of participant progression to study completion.

  Week 13

Secondary Outcomes (6)

• Adverse Events

  Weekly from Baseline to Week 16

• Infusion Reactions

  Weekly from Week 1 to Week 16

• Pharmacokinetics: Peak Plasma Concentration (Cmax)

  Week 1

• Pharmacokinetics: Time of peak concentration (tmax)

  Week 1

• Pharmacokinetics: Half-life (t1/2)

  Week 1

Other Outcomes (16)

• Dopamine transporter (DaT) SPECT imaging

  Baseline to week 13

• MRI: Regional brain volumes

  Screening to week 13

• MRI: Fractional Anisotropy

  Screening to week 13

Study Arms (2)

Allo APOE4 carriers

EXPERIMENTAL

Group of 5 participants who are carriers of the APOE4 gene and will receive allopregnanolone 4mg administered weekly via a 30-min IV infusion for a duration of 12 weeks.

Drug: Allopregnanolone

Allo APOE4 none-carriers

ACTIVE COMPARATOR

Group of 5 participants who are none-carriers of the APOE4 gene and will receive allopregnanolone 4mg administered weekly via a 30-min IV infusion for a duration of 12 weeks.

Drug: Allopregnanolone

Interventions

Allopregnanolone DRUG

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

Allo APOE4 carriers; Allo APOE4 none-carriers

Eligibility Criteria

• Age: 40 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• History of Idiopathic sporadic Parkinson disease
• Hoehn \& Yahr stage 1-4
• Have been on stable doses of all anti-Parkinson's medications for 30 days prior to screening
• Provision of signed and dated informed consent form

You may not qualify if:

• Evidence of Parkinsonian syndrome.
• Any conditions that would contraindicate MRI studies.
• Undergone deep brain stimulation (DBS) surgery as treatment for PD.
• Iodine allergy, known serious hypersensitivity to ioflupane I-123, or other inability to undergo DaTscan.
• Clinically significant abnormal laboratory value and/or clinically significant unstable medical or psychiatric illness.
• History within the last 5 years of a primary or recurrent malignant disease, with the exception of resected cutaneous squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ, or prostate cancer in situ with a post-treatment prostatic-specific antigen within normal range.
• Serious or unstable illnesses including cardiovascular, hepatic, renal, gastroenterologic, respiratory, endocrinologic, neurologic (other than PD), psychiatric, immunologic, or hematologic disease, and any other conditions that, in the investigator's opinion, could interfere with the safety and efficacy analyses in this study.
• History of chronic alcohol or substance abuse/dependence within the past 3 years.
• Current use of benzodiazepines, anticonvulsants, antipsychotics, or other drugs that might interact with the gamma-aminobutyric acid-A (GABA-A) receptor complex; use of calcium-channel blockers (e.g., amlodipine); use of dietary supplements containing Pregnenolone.
• Treatment with another investigational drug within 3 months of screening.

Sponsors & Collaborators

• Roberta Brinton: lead

Study Sites (1)

The University of Arizona Clinical & Translational Science Research Center

Tucson, Arizona, 85721, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

Pregnanolone

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Intervention Hierarchy (Ancestors)

Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Scott Sherman, MD

  University of Arizona

  PRINCIPAL INVESTIGATOR

• Roberta D Brinton, PhD

  University of Arizona

  STUDY CHAIR

• Gerson D Hernandez, MD, MPH

  University of Arizona

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 8, 2024
• First Posted: February 16, 2024
• Study Start: January 12, 2024
• Primary Completion: July 15, 2025
• Study Completion: July 15, 2025
• Last Updated: March 13, 2026

Record last verified: 2026-03

Locations

US (1)