A Phase 2 interventional study of Allopregnanolone and Placebo in Alzheimer Dementia, Late Onset Alzheimer Disease and Neurodegenerative Diseases, sponsored by University of Arizona. Active, not recruiting at 9 sites in United States. Open to participants aged 55 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-06-30.
Sponsored by University of Arizona · Phase 2, Interventional, and Treatment
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, 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 6 months. After 6 months, all participants in the placebo group will be crossed-over to receive Allo for the remainder of the study (3 month open-label phase). Brain imaging to evaluate the primary endpoint will be conducted at baseline, 3 and 6 months.
3,675 studies on the registry are indexed under Alzheimer Disease; 869 are open to participants now.
This study's planned enrollment of 100 is above the median of 70 across 2,805 interventional studies indexed under Alzheimer Disease.
Browse Alzheimer Disease studies →University of Arizona is the lead sponsor of 466 studies on the registry; 87 are open to participants now.
Of its 47 completed or terminated interventional studies of FDA-regulated products, 29 (62%) have results posted.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria:
Main Exclusion Criteria:
Allopregnanolone 4mg IV 30-minute infusion once per week for 6 months.
Drug: Allopregnanolone
Placebo (normal saline) IV 30-minute infusion once per week for 6 months.
Other: Placebo
Allopregnanolone 4mg IV via 30-minute infusion, once per week.
Also known as: Allo
Normal saline solution IV via 30-minute infusion, once per week
Hippocampal volume
mm3
Time frame: Baseline to 6 months
Cambridge Cognition's Paired Associates Learning Test
Total errors score (adjusted) - number of errors made by the participant (range: 0 to \~120). Higher scores indicate poor performance.
Time frame: Baseline to 6 months
Cambridge Neuropsychological Test Automated Battery (CANTAB)
Composite score (higher score indicate better outcome)
Time frame: Baseline to 6 months
Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) 11
Total score (range 0 to 70); higher scores indicate poor performance.
Time frame: Baseline to 6 months
Alzheimer's Disease Cooperative Study (ADCS) Instrumental Activities of Daily (iADL) Living (iADL)
iADL subscore (range 0-56): Lower score indicates greater severity
Time frame: Baseline to 6 months
Safety and tolerability
Frequency of adverse events and serious adverse events
Time frame: Baseline to 6 months
Other regional brain volumes
Change in regional brain volumes (mm3)
Time frame: Baseline to 6 and 9 months
Diffusion tensor imaging (DTI)
Change in white matter tract diffusion (scalar values and/or mm2/sec)
Time frame: Baseline to 6 and 9 months
Resting state functional MRI
Change in functional connectivity (z transformed correlations)
Time frame: Baseline to 6 and 9 months
Arterial spin labeling (ASL)
Change in regional cerebral blood flow (mL/100g)
Time frame: Baseline to 6 and 9 months
Exploratory blood based biomarkers
Change from baseline in biomarkers of AD pathology, neurogenesis and inflammation
Time frame: Baseline to 6 and 9 months
Clinical Dementia Rating (CDR)
Sum of boxes score (CDR-SB): range 0-18
Time frame: Baseline to 6 and 9 months
Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) 14
Total score (range 0-90)
Time frame: Baseline to 6 and 9 months
Mini Mental State Examination (MMSE)
Total score (range 0-30)
Time frame: Baseline to 6 and 9 months
Neuropsychiatric Inventory-Questionnaire (NPI-Q)
Total score (range 0-36). Higher scores indicate greater symptom severity
Time frame: Baseline to 6 and 9 months
EuroQol 5-Dimension / 5-Level health-related quality of life scale scores (EQ-5D-5L)
Reported as frequency, percentage and index value.
Time frame: Baseline to 6 and 9 months
Quality of Life in Alzheimer's Disease scale (QoL-AD)
Total score (range 13-52). Higher score indicate better QoL
Time frame: Baseline to 6 and 9 months
Zarit Burden Interview (ZBI)
Total score (range 0-48). Higher scores indicate high burden
Time frame: Baseline to 6 and 9 months
This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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University of Arizona