Allopregnanolone Regenerative Therapeutic for Early Alzheimer's Disease: Intramuscular Study

NCT03748303 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: AlloPhase1-IM
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to identifying the intramuscular dose equivalent to the 4mg intravenous dose and assess its safety and tolerability as a weekly injection.

Conditions

Alzheimer Dementia

Keywords

Alzheimer's disease; Mild cognitive impairment; Dementia; Regenerative therapeutic; Drug development

Outcome Measures

Primary Outcomes (3)

• Safety - Adverse events

  Incidence and severity of treatment emergent adverse events assessed weekly.

  From baseline to visit 16 (14 weeks)

• Safety - clinical laboratory measures

  Proportion of subjects exceeding pre-established critical laboratory values.

  From Baseline to visit 16 (14 weeks)

• Safety - clinical assessment

  Proportion of subjects with abnormal findings in physical/neurological exams, vital signs and electrocardiograms.

  From Baseline to visit 16 (14 weeks)

Secondary Outcomes (6)

• Pharmacokinetic parameter - Cmax

  Visits 3 - 6 (up to 4 weeks)

• Pharmacokinetic parameter - AUC

  Visits 3 - 6 (up to 4 weeks)

• Pharmacokinetic parameter - Tmax

  Visits 3 - 6 (up to 4 weeks)

• Pharmacokinetic parameter - Clearance

  Visits 3 - 6 (up to 4 weeks)

• Pharmacokinetic parameter - Volume of distribution

  Visits 3 - 6 (up to 4 weeks)

Other Outcomes (12)

• MRI brain volumes

  Baseline to visit 16 (14 weeks)

• Cambridge Neuropsychological Test Automated Battery (CANTAB) Paired Associates Learning (PAL)

  Baseline to visit 16 (14 weeks)

• Mini-Mental State Exam (MMSE)

  Baseline to visit 16 (14 weeks)

Study Arms (1)

Allo IM cohort

EXPERIMENTAL

Allopregnanolone 4-18mg IM, weekly, for 12 weeks.

Drug: Allopregnanolone

Interventions

Allopregnanolone DRUG

Administration of weekly IM injections of Allopregnanolone.

Also known as: Allo

Allo IM cohort

Eligibility Criteria

• Age: 55 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Provision of signed and dated informed consent form
• Stated willingness to comply with all study procedures and availability for the duration of the study
• Men or postmenopausal women, aged 55 years or older
• Diagnosis of MCI due to AD or mild AD
• In good general health as evidenced by medical history and with no medical contraindications to participation
• MMSE \> 20 at screen
• Caregiver willing and capable to accompany the patient to clinic visits

You may not qualify if:

• Daily use of benzodiazepines, sedative/hypnotics, anticonvulsants, antipsychotics, and other drugs that might interact with the GABA-A receptor complex.
• Seizure disorder, history of stroke, focal brain lesion, traumatic brain injury, substance abuse, malignancy.
• Clinically significant laboratory or ECG abnormality obtained at screening visit.
• MRI indicative of significant abnormality, including but not limited to evidence of a single prior hemorrhage or infarct \>1 cm3, multiple lacunar infarcts (\>1) or evidence of a single prior infarct \>1cm3, evidence of a cerebral contusion, encephalomalacia, aneurysms, vascular malformations, subdural hematoma, or space occupying lesions (e.g. abscess or tumor).
• Has any contraindications for MRI studies, including claustrophobia, the presence of metal (ferromagnetic) implants, or a cardiac pacemaker that is not compatible with MRI.
• Is currently enrolled in a clinical trial involving an off-label use of an investigational drug or device, or concurrently enrolled in any other type of medical research or observational study judged not to be scientifically or medically compatible with this study.

Sponsors & Collaborators

• University of Southern California: collaborator
• University of Arizona: lead
• Alzheimer's Association: collaborator

Study Sites (1)

University of Southern California - Alzheimer Disease Research Center - Healthcare Consultation Center II

Los Angeles, California, 90033, United States

Location

MeSH Terms

Conditions

Alzheimer Disease; Cognitive Dysfunction; Dementia

Interventions

Pregnanolone

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Cognition Disorders

Intervention Hierarchy (Ancestors)

Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Roberta D Brinton, PhD

  University of Arizona

  PRINCIPAL INVESTIGATOR

• Lon S Schneider, MD, MS

  University of Southern California

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Single-arm, open-label study

Study Record Dates

• First Submitted: October 12, 2018
• First Posted: November 20, 2018
• Study Start: October 1, 2019
• Primary Completion: December 21, 2022
• Study Completion: December 21, 2022
• Last Updated: March 13, 2026

Record last verified: 2026-03

Locations

US (1)