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Alzheimer DementiaPhase 2REGEN-BRAIN©NCT04838301

Allopregnanolone Regenerative Therapeutic for Mild Alzheimer's Disease

A Phase 2 study testing Allopregnanolone in people with Alzheimer Dementia and Late Onset Alzheimer Disease, run by University of Arizona at 9 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 55 to 80 years
What is being tested?
Allopregnanolone
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
100 participants
Where
9 sitesUnited States
Study dates
15 Aug 2023to 18 Mar 2027
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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

Main 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

Trial locations and contacts

9 sites in 1 country.

9 sites in 1 country

United States9 sites

  • ATP Clinical Research

    Costa Mesa, California, 92626

  • Optimus U Corporation

    Miami, Florida, 33135

  • Miami Jewish Health

    Miami, Florida, 33137

  • Combined Research Orlando

    Orlando, Florida, 32807

  • Syrentis Clinical Research

    Santa Ana, California, 92705

  • Perseverance Research Center

    Scottsdale, Arizona, 85253

  • University of Arizona / Clinical & Translational Sciences Research Center

    Tucson, Arizona, 85721

  • MedVadis Research

    Waltham, Massachusetts, 02451

  • Conquest Research

    Winter Park, Florida, 32789

Other trials in United States

About this study

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

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.

Read the detailed description

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.

Being tested
Allopregnanolone, Placebo
Keywords
Mild Alzheimer Disease, Regenerative Therapeutic, Neurogenesis, Allopregnanolone
Medical subject headings
Alzheimer Disease, Neurodegenerative Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AllopregnanoloneDrug

    Also called Allo

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

  • PlaceboOther

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

Study groups in NCT04838301
Study groupRoleWhat they receive
Allo groupExperimentalAllopregnanolone 4mg IV 30-minute infusion once per week for 6 months.
Receives: Drug: Allopregnanolone
Control groupPlacebo comparatorPlacebo (normal saline) IV 30-minute infusion once per week for 6 months.
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Hippocampal volume

    mm3

    Measured: Baseline to 6 months

Secondary outcomes (5)

  1. 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.

    Measured: Baseline to 6 months

  2. Cambridge Neuropsychological Test Automated Battery (CANTAB)

    Composite score (higher score indicate better outcome)

    Measured: Baseline to 6 months

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

    Total score (range 0 to 70); higher scores indicate poor performance.

    Measured: Baseline to 6 months

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

    iADL subscore (range 0-56): Lower score indicates greater severity

    Measured: Baseline to 6 months

  5. Safety and tolerability

    Frequency of adverse events and serious adverse events

    Measured: Baseline to 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Apr 2021
  2. Study start15 Aug 2023
  3. Primary completion18 Nov 2026 (estimated)
  4. Study completion18 Mar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Participants are assigned to the active intervention or placebo in parallel for 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).
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
100 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04838301
Sponsor's protocol ID
Allo-20-001
Other IDs
R01AG063826
Acronym
REGEN-BRAIN©
Status last verified
Jun 2026
First submitted
25 Mar 2021
Last updated
30 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Raikes AC, Hernandez GD, Matthews DC, Lukic AS, Law M, Shi Y, Schneider LS, Brinton RD. Exploratory imaging outcomes of a phase 1b/2a clinical trial of allopregnanolone as a regenerative therapeutic for Alzheimer's disease: Structural effects and functional connectivity outcomes. Alzheimers Dement (N Y). 2022 Mar 14;8(1):e12258. doi: 10.1002/trc2.12258. eCollection 2022.

    PubMed 35310526

  • Hernandez GD, Solinsky CM, Mack WJ, Kono N, Rodgers KE, Wu CY, Mollo AR, Lopez CM, Pawluczyk S, Bauer G, Matthews D, Shi Y, Law M, Rogawski MA, Schneider LS, Brinton RD. Safety, tolerability, and pharmacokinetics of allopregnanolone as a regenerative therapeutic for Alzheimer's disease: A single and multiple ascending dose phase 1b/2a clinical trial. Alzheimers Dement (N Y). 2020 Dec 16;6(1):e12107. doi: 10.1002/trc2.12107. eCollection 2020.

    PubMed 33344752

  • Brinton RD. Neurosteroids as regenerative agents in the brain: therapeutic implications. Nat Rev Endocrinol. 2013 Apr;9(4):241-50. doi: 10.1038/nrendo.2013.31. Epub 2013 Feb 26.

    PubMed 23438839

  • Brinton RD, Wang JM. Therapeutic potential of neurogenesis for prevention and recovery from Alzheimer's disease: allopregnanolone as a proof of concept neurogenic agent. Curr Alzheimer Res. 2006 Jul;3(3):185-90. doi: 10.2174/156720506777632817.

    PubMed 16842093

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Allopregnanolone in people with Alzheimer Dementia and Late Onset Alzheimer Disease, run by University of Arizona at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Hippocampal volume (time frame: Baseline to 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04838301, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jun 2026.

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