Allopregnanolone treatment 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
This study is testing if a new treatment called Allopregnanolone can help improve brain health in people aged 55 to 80 with mild Alzheimer's disease.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | 55 Years to 80 Years |
| Sex | All |
| Sponsor | University of Arizona Academic / other |
| Locations | 10 sites (Scottsdale, Arizona and 9 other locations) |
| Trial ID | NCT04838301 on ClinicalTrials.gov |
What this trial studies
This phase 2 clinical trial aims to evaluate the safety and efficacy of Allopregnanolone as a regenerative therapeutic for individuals with mild Alzheimer's disease. Participants, aged 55 to 80 and APOE ε4 positive, will be randomly assigned to receive either Allopregnanolone or a placebo over a 12-month period. The study will assess cognitive function and brain imaging at baseline, 6 months, and 12 months to determine the treatment's impact on brain health. After the initial phase, all participants in the placebo group will have the opportunity to receive Allopregnanolone in an open-label phase.
Who should consider this trial
Good fit: Ideal candidates are men and postmenopausal women aged 55 to 80 with a diagnosis of probable Alzheimer's disease and an MMSE score of 20 to 26.
Not a fit: Patients with dementia other than probable Alzheimer's disease or those with significant medical contraindications will not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve cognitive function and restore brain integrity in patients with mild Alzheimer's disease.
How similar studies have performed: While this approach is novel, previous studies have explored neuroregenerative therapies, but the specific use of Allopregnanolone in this context is less established.
Eligibility criteria
Show full inclusion / exclusion 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 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
Where this trial is running
Scottsdale, Arizona and 9 other locations
- Perseverance Research Center — Scottsdale, Arizona, United States (Recruiting)
- University of Arizona / Clinical & Translational Sciences Research Center — Tucson, Arizona, United States (Recruiting)
- ATP Clinical Research — Costa Mesa, California, United States (Recruiting)
- Wake Research-PRI, LLC — Los Alamitos, California, United States (Withdrawn)
- Syrentis Clinical Research — Santa Ana, California, United States (Terminated)
- Optimus U Corporation — Miami, Florida, United States (Recruiting)
- Miami Jewish Health — Miami, Florida, United States (Recruiting)
- Combined Research Orlando — Orlando, Florida, United States (Terminated)
- Conquest Research — Winter Park, Florida, United States (Recruiting)
- MedVadis Research — Waltham, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: Roberta D Brinton, PhD — University of Arizona
- Study coordinator: Claudia M Lopez, BS
- Email: claudiml@arizona.edu
- Phone: 520-626-6276
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.