Allopregnanolone for Mild Cognitive Impairment Due to Alzheimer's Disease or Mild AD (Allo)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02221622 |
Recruitment Status :
Completed
First Posted : August 20, 2014
Last Update Posted : July 5, 2019
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Condition or disease | Intervention/treatment | Phase |
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Mild Cognitive Impairment Alzheimer Disease | Drug: Allopregnanolone injection (intravenous solution) Drug: Placebo injection (intravenous solution) | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 24 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | Allopregnanolone Regenerative Therapeutic for MCI/AD: Dose Finding Phase 1 |
Study Start Date : | August 2014 |
Actual Primary Completion Date : | February 2018 |
Actual Study Completion Date : | February 2018 |

Arm | Intervention/treatment |
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Experimental: Allopregnanolone 2 mg
Drug: Allopregnanolone injection (intravenous solution) once per week for 12 weeks
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Drug: Allopregnanolone injection (intravenous solution)
Allopregnanolone intravenous infusion
Other Names:
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Experimental: Allopregnanolone 4 mg
Drug: Allopregnanolone injection (intravenous solution) once per week for 12 weeks
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Drug: Allopregnanolone injection (intravenous solution)
Allopregnanolone intravenous infusion
Other Names:
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Experimental: Allopregnanolone 6-18 mg
Drug: Allopregnanolone injection (intravenous solution) once per week for 12 weeks
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Drug: Allopregnanolone injection (intravenous solution)
Allopregnanolone intravenous infusion
Other Names:
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Placebo Comparator: Placebo
Drug: Placebo injection (intravenous solution) once per week for 12 weeks
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Drug: Placebo injection (intravenous solution)
Placebo intravenous infusion |
- Safety profile: Adverse events [ Time Frame: From Baseline to week 16 ]Incidence and severity of treatment emergent adverse events assessed weekly per treatment arm.
- Safety profile: Clinical laboratory measurements [ Time Frame: From Baseline to week 13 ]
Evaluating the proportion of subjects exceeding pre-established critical values per treatment arm:
Alanine aminotransferase (ALT, U/L) > 5 times upper normal limit Aspartate aminotransferase (AST, U/L) > 5 times upper normal limit Total serum bilirubin (mg/dl) > 2 times upper normal limit Serum creatinine (mg/dl) > 2 times upper normal limit Serum creatine phosphokinase (U/L) > 5 times upper normal limit
- Safety profile: ARIA [ Time Frame: From Baseline to week 13 ]MRI based assessment of amyloid related imaging abnormalities (ARIA); proportion of subjects with ARIA
- Safety profile: Physical and neurological examination [ Time Frame: From Baseline to week 16 ]To evaluate the proportion of abnormal examination findings of subjects in each treatment arm.
- Tolerability - Maximum tolerated dose (MTD) [ Time Frame: From Baseline to week 12 ]Onset of sedation will define the upper most limit of drug dose
- Pharmacokinetic profile after single and multiple doses: Maximum Concentration (Cmax) [ Time Frame: Weeks: 1 and 12 ]Measurement of maximum concentration
- Pharmacokinetic profile after single and multiple doses: time attain to Cmax (Tmax) [ Time Frame: Weeks: 1 and 12 ]Time to attain maximum concentration.
- Pharmacokinetic profile after single and multiple doses: Area under the curve (AUC) [ Time Frame: Weeks: 1 and 12 ]Pharmacokinetic parameter.
- Pharmacokinetic profile after single and multiple doses: Drug Clearance (CL) [ Time Frame: Weeks: 1 and 12 ]Pharmacokinetic parameter.
- Pharmacokinetic profile after single and multiple doses: apparent volume of distribution at steady state (Vss) [ Time Frame: Weeks: 1 and 12 ]Pharmacokinetic parameter.
- Cognitive tests (ADAS-Cog; MMSE/MoCA; ADCS-CGIC; CogState) [ Time Frame: Baseline to Week 13 ]Alzheimer's disease Assessment Scale Cognitive Subscale 14 (ADAS-Cog); Mini-Mental State Exam (MMSE); Montreal Cognitive Assessment (MoCA); Alzheimer's disease Cooperative Study Clinical Global Impression of Change (ADCS-CGIC); CogState 12-min battery (CogState)
- Brain MRI volumetrics [ Time Frame: Baseline and Week 13 ]Gray matter, white matter and hippocampal volume measurements, including subfield analysis.

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Ages Eligible for Study: | 55 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Men or postmenopausal women
- 55 years of age or older
- Diagnosis of MCI due to AD or mild AD
- MMSE > 20 at screen
- Capacity to provide informed consent
- Residing in the community with a caregiver able to accompany the patient to clinic visits
- No medical contraindications to participation
- Willingness to comply with study procedures
Exclusion Criteria:
- 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
- MRI indicative of any other significant abnormality, including but not limited to evidence of a cerebral contusion, encephalomalacia, aneurysms, vascular malformations, subdural hematoma, or space occupying lesions
- Any condition that would contraindicate an MRI such as the presence of metallic objects in the eyes, skin, heart, or body

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02221622
United States, California | |
University of Southern California - Alzheimer Disease Research Center - Healthcare Consultation Center II | |
Los Angeles, California, United States, 90033 |
Principal Investigator: | Roberta D Brinton, Ph.D. | University of Southern California | |
Principal Investigator: | Lon S Schneider, M.D. | University of Southern California |
Responsible Party: | University of Southern California |
ClinicalTrials.gov Identifier: | NCT02221622 |
Other Study ID Numbers: |
AlloPhase1 1UF1AG046148 ( U.S. NIH Grant/Contract ) |
First Posted: | August 20, 2014 Key Record Dates |
Last Update Posted: | July 5, 2019 |
Last Verified: | May 2018 |
Alzheimer's disease Mild Cognitive Impairment Dementia Regenerative therapeutic |
Alzheimer Disease Cognitive Dysfunction Dementia Brain Diseases Central Nervous System Diseases Nervous System Diseases Tauopathies Neurodegenerative Diseases |
Neurocognitive Disorders Mental Disorders Cognition Disorders Pregnanolone Pharmaceutical Solutions Anesthetics Central Nervous System Depressants Physiological Effects of Drugs |