Allopregnanolone Regenerative Therapeutic for Mild Alzheimer’s Disease

Brief Title

Allopregnanolone Regenerative Therapeutic for Mild Alzheimer's Disease

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

Brief 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.
    

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, 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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

Hippocampal volume

Secondary Outcome

 Cambridge Cognition's Paired Associates Learning Test

Condition

Alzheimer Dementia

Intervention

Allopregnanolone

Study Arms / Comparison Groups

 Allo group
Description:  Allopregnanolone 4mg IV 30-minute infusion once per week for 12 months.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

200

Start Date

May 2021

Completion Date

June 2024

Primary Completion Date

January 2024

Eligibility Criteria

        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.
      

Gender

All

Ages

55 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

, , 



Administrative Informations


NCT ID

NCT04838301

Organization ID

Allo-20-001

Secondary IDs

R01AG063826

Responsible Party

Principal Investigator

Study Sponsor

University of Arizona

Collaborators

 National Institute on Aging (NIA)

Study Sponsor

, , 


Verification Date

April 2021