REGEN-BRAIN©

Active not recruitingPhase 2

Allopregnanolone Regenerative Therapeutic for Mild Alzheimer's Disease

Safety and Efficacy of Allopregnanolone (Allo) as a Regenerative Therapeutic for Alzheimer's Disease: Multicenter, Double-Blind, Randomized, Placebo-Controlled, Phase 2 Clinical Trial

Lead sponsor

University of Arizona

Asset

Allopregnanolone

Listed sites

Recruiting sites

Enrollment

100

estimated

Study population

Alzheimer’s disease

Key I/E criteria

•Alzheimer's disease•MMSE 20-26

Primary endpoint

•Hippocampal volume

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

Org study IDAllo-20-001

NCT IDNCT04838301

NihR01AG063826

Timeline

Milestones

Study first posted2021-04-09actual

Study start2023-08-15actual

Last update posted2026-06-30actual

Primary completion2026-11-18estimated

Study completion2027-03-18estimated

Assets

Drug assets

Allopregnanolone

Study populations

Who this study enrolls

Alzheimer’s disease

Eligibility

Who can enroll

Minimum age55 Years

Maximum age80 Years

SexAll

Healthy volunteersNot accepted

Inclusion criteria

Main Inclusion Criteria:

•Men and postmenopausal women

•Age 55 to 80 years old

•Meets NIA-AA criteria for probable AD dementia

•MMSE of 20-26

•Plasma p-Tau217 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.

•No evidence of AD-like pattern of brain atrophy

Endpoints (18)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Coverage by outcome category

Other (unclassified)

Global cognition

Neuroimaging

Caregiver / quality of life

Behavior / neuropsychiatric

Safety / tolerability / PK

Global cognition

4 endpoints

Secondary/protocol endpoint

Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) 11

Time frame:Baseline to 6 months

ADAS-Cog

descriptive

Other/protocol endpoint

Clinical Dementia Rating (CDR)

Time frame:Baseline to 6 and 9 months

Clinical Dementia Rating-Sum of Boxes (CDR-SB)

descriptive

Other/protocol endpoint

Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) 14

Time frame:Baseline to 6 and 9 months

ADAS-Cog

descriptive

Other/protocol endpoint

Mini Mental State Examination (MMSE)

Time frame:Baseline to 6 and 9 months

Mini-Mental State Examination (MMSE)

descriptive

Behavior / neuropsychiatric

1 endpoint

Other/protocol endpoint

Neuropsychiatric Inventory-Questionnaire (NPI-Q)

Time frame:Baseline to 6 and 9 months

Neuropsychiatric Inventory (NPI)

descriptive

Neuroimaging

4 endpoints

Primary/protocol endpoint

Hippocampal volume

Time frame:Baseline to 6 months

descriptive

Other/protocol endpoint

Diffusion tensor imaging (DTI)

Time frame:Baseline to 6 and 9 months

change from baseline, improvement

Other/protocol endpoint

Resting state functional MRI

Time frame:Baseline to 6 and 9 months

change from baseline, improvement

Other/protocol endpoint

Arterial spin labeling (ASL)

Time frame:Baseline to 6 and 9 months

change from baseline, improvement

Caregiver / quality of life

3 endpoints

Other/protocol endpoint

EuroQol 5-Dimension / 5-Level health-related quality of life scale scores (EQ-5D-5L)

Time frame:Baseline to 6 and 9 months

event count, event

Other/protocol endpoint

Quality of Life in Alzheimer's Disease scale (QoL-AD)

Time frame:Baseline to 6 and 9 months

descriptive

Other/protocol endpoint

Zarit Burden Interview (ZBI)

Time frame:Baseline to 6 and 9 months

descriptive

Safety / tolerability / PK

1 endpoint

Secondary/protocol endpoint

Safety and tolerability

Time frame:Baseline to 6 months

event count, event

Other (unclassified)

5 endpoints

Secondary/protocol endpoint/low confidence

Cambridge Cognition's Paired Associates Learning Test

Time frame:Baseline to 6 months

event count, event

Secondary/protocol endpoint/low confidence

Cambridge Neuropsychological Test Automated Battery (CANTAB)

Time frame:Baseline to 6 months

descriptive

Secondary/protocol endpoint/low confidence

Alzheimer's Disease Cooperative Study (ADCS) Instrumental Activities of Daily (iADL) Living (iADL)

Time frame:Baseline to 6 months

descriptive

Other/protocol endpoint/low confidence

Other regional brain volumes

Time frame:Baseline to 6 and 9 months

change from baseline, improvement

Other/protocol endpoint/low confidence

Exploratory blood based biomarkers

Time frame:Baseline to 6 and 9 months

change from baseline, improvement

Publications (4)

Bibliography

Records linked to this trial through ClinicalTrials.gov references, PubMed NCT search, and curated study seeds. 'Canonical' marks design/result papers; others are registry references or candidates.

Registry references + supporting bibliography

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableno registry results posted yet

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.