Allopregnanolone Regenerative Therapeutic for Mild Alzheimer's Disease

Phase 2RecruitingNCT04838301

University of Arizona200 enrolled

Overview

A phase 2, double-blind, randomized, placebo-controlled clinical trial to evaluate the safety and efficacy of Allopregnanolone as a regenerative therapeutic for Alzheimer's disease.

This is a proof-of-concept phase 2 clinical trial to investigate the long-term safety and efficacy of Allo to function as a regenerative therapeutic to restore structural integrity and cognitive function of the brain in participants with mild Alzheimer's disease (AD) dementia. Study participants will be male and female, APOE ε4 positive diagnosed with probable AD, Mini-Mental State Exam (MMSE) 20 to 26, ages 55 to 80 years old. After a 2-4-week screening period, participants will be randomized to 4 mg Allo (administered intravenously over 30 minutes, once per week, in clinic) or matching placebo, 1:1 allocation, for a period of 12 months. After 12 months, all participants in the placebo group will be crossed-over to receive Allo for the remainder of the study (6 month open-label phase). Brain imaging to evaluate the primary endpoint will be conducted at baseline, 6 and 12 months.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

200

Conditions

Alzheimer Dementia Late Onset Alzheimer Disease Neurodegenerative Diseases

Interventions

AllopregnanoloneDRUG

Allopregnanolone 4mg IV via 30-minute infusion, once per week.

PlaceboOTHER

Normal saline solution IV via 30-minute infusion, once per week

Eligibility

Sex: ALLMin age: 55 YearsMax age: 80 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Men and postmenopausal women * Age 55 to 80 years old * Meets NIA-AA criteria for probable AD dementia * MMSE of 20-26 * APOE ε4 positive * Geriatric Depression Scale short form (GDS-S) score of ≤ 6 * No medical contraindications to participation * Capacity to provide informed consent at screening Exclusion Criteria: * Dementia other than probable AD * Use of benzodiazepines, anticonvulsants, antipsychotics, or other drugs that might interact with the GABA-A receptor complex * History of stroke with a modified Hachinski Ischemic Scale score \>4 * History of seizure disorder, focal brain lesion, traumatic brain injury * History within the last 5 years of a primary or recurrent malignant disease * Unstable or clinically significant cardiovascular, kidney or liver disease * MRI indicative of any other significant abnormality, including but not limited to one or more significant ARIA-E or macro-hemorrhage findings, or multiple microhemorrhages (\>8), or Fazekas score of 3; encephalomalacia, aneurysms, vascular malformations, subdural hematoma, or space occupying lesions * Any conditions that would contraindicate MRI studies.

Locations (8)

Perseverance Research Center

Scottsdale, Arizona, United States

RECRUITING

ATP Clinical Research

Costa Mesa, California, United States

RECRUITING

Syrentis Clinical Research

Santa Ana, California, United States