Alzheimer DementiaPhase 1Allo-IMNCT03748303
Allopregnanolone Regenerative Therapeutic for Early Alzheimer's Disease: Intramuscular Study
A Phase 1 study testing Allopregnanolone in people with Alzheimer Dementia, run by University of Arizona at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Alzheimer Dementia
- What is being tested?
- Allopregnanolone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2019to 21 Dec 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Southern California - Alzheimer Disease Research Center - Healthcare Consultation Center II
Los Angeles, California, 90033
About this study
Official title: Allopregnanolone Regenerative Therapeutic for Early Alzheimer's Disease: IV to IM Bridging Study
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.
Read the detailed description
The purpose of this bridging study is to advance the therapeutic development of Allopregnanolone (Allo) by using the intramuscular (IM) route of administration as an alternative to the intravenous (IV) route. In order to identify the equivalent IM dose we will conduct pharmacokinetic (PK) analysis previously informed by simulations and modeling. We will recruit a total of 12 participants, both males and females equally distributed, into this single-arm, open-label study.
PK analysis and dose finding will take place for the initial 4 weeks; some participants may not require all 4 weeks of initial dosing to establish maintenance dose. Once maintenance dose is established all participants will receive weekly administration of Allo IM until they complete 12 weeks total of Allo exposure (5 or 6 clinic visits and 6 or 7 home-nurse visits).
- Conditions
- Alzheimer Dementia
- Being tested
- Allopregnanolone
- Keywords
- Alzheimer's disease, Mild cognitive impairment, Dementia, Regenerative therapeutic, Drug development
- Medical subject headings
- Alzheimer Disease, Cognitive Dysfunction, Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AllopregnanoloneDrug
Also called Allo
Administration of weekly IM injections of Allopregnanolone.
| Study group | Role | What they receive |
|---|---|---|
| Allo IM cohort | Experimental | Allopregnanolone 4-18mg IM, weekly, for 12 weeks. Receives: Drug: Allopregnanolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety - Adverse events
Incidence and severity of treatment emergent adverse events assessed weekly.
Measured: From baseline to visit 16 (14 weeks)
Safety - clinical laboratory measures
Proportion of subjects exceeding pre-established critical laboratory values.
Measured: From Baseline to visit 16 (14 weeks)
Safety - clinical assessment
Proportion of subjects with abnormal findings in physical/neurological exams, vital signs and electrocardiograms.
Measured: From Baseline to visit 16 (14 weeks)
Secondary outcomes (6)
Pharmacokinetic parameter - Cmax
Determine maximum serum concentration of Allo after IM administration of each dose.
Measured: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - AUC
Determine the area under the curve after each IM administration of Allo.
Measured: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - Tmax
Determine the time at which Cmax is attained.
Measured: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - Clearance
Pharmacokinetic measurement of the volume of plasma from which Allo is completely removed per unit time.
Measured: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - Volume of distribution
Determine the volume of distribution at steady state of Allo.
Measured: Visits 3 - 6 (up to 4 weeks)
Satisfaction and feasibility of home nurse survey
Standardized patient satisfaction questionnaire to assess the feasibility of home-health care visits to administer the study medication and its desirability by participants and caregivers. Levels of satisfaction measured on a 5-point scale (1 = lowest satisfaction, 5 = greatest).
Measured: Visits 8-9 and 11-15 (up to 8 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2018
- Study start1 Oct 2019
- Primary completion21 Dec 2022
- Study completion21 Dec 2022
Why it stopped: Study interrupted by COVID-19 pandemic and could not be restarted at current location. Study investigators determined that relocating and continuing the study under its original scope was not feasible given preliminary results from IM injections.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alzheimer DementiaStudies of the same condition, open and under way.
- More trials run by University of ArizonaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single-arm, open-label study
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Arizona
- Sponsor type
- University, hospital or other
- Collaborators
- University of Southern California, Alzheimer's Association
- Responsible party
- Principal investigator, Roberta Brinton, University of Arizona
- Investigators
- Roberta D Brinton, PhD, Principal investigator, University of ArizonaLon S Schneider, MD, MS, Principal investigator, University of Southern California
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03748303
- Sponsor's protocol ID
- AlloPhase1-IM
- Acronym
- Allo-IM
- Status last verified
- Mar 2026
- First submitted
- 12 Oct 2018
- Last updated
- 13 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allopregnanolone Regenerative Therapeutic for Early Alzheimer's Disease: IV to IM Bridging Study
- ClinicalTrials.gov ID
- NCT03748303
- Acronym / sponsor ID
- Allo-IM / AlloPhase1-IM
- Conditions
- Alzheimer Dementia
- Keywords
- Alzheimer's disease, Mild cognitive impairment, Dementia, Regenerative therapeutic, Drug development
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study interrupted by COVID-19 pandemic and could not be restarted at current location. Study investigators determined that relocating and continuing the study under its original scope was not feasible given preliminary results from IM injections.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- University of ArizonaUniversity, hospital or other
- Collaborators
- University of Southern California, Alzheimer's Association
- Study start
- 1 Oct 2019 (actual)
- Primary completion
- 21 Dec 2022 (actual)
- Study completion
- 21 Dec 2022 (actual)
- First posted
- 20 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The purpose of this bridging study is to advance the therapeutic development of Allopregnanolone (Allo) by using the intramuscular (IM) route of administration as an alternative to the intravenous (IV) route. In order to identify the equivalent IM dose we will conduct pharmacokinetic (PK) analysis previously informed by simulations and modeling. We will recruit a total of 12 participants, both males and females equally distributed, into this single-arm, open-label study.
PK analysis and dose finding will take place for the initial 4 weeks; some participants may not require all 4 weeks of initial dosing to establish maintenance dose. Once maintenance dose is established all participants will receive weekly administration of Allo IM until they complete 12 weeks total of Allo exposure (5 or 6 clinic visits and 6 or 7 home-nurse visits).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single-arm, open-label study
| Arm | Type | Receives |
|---|---|---|
| Allo IM cohort | Experimental | Drug: AllopregnanoloneAllopregnanolone 4-18mg IM, weekly, for 12 weeks. |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AllopregnanoloneAlso: AlloAdministration of weekly IM injections of Allopregnanolone. | Drug | Allo IM cohort |
Primary outcome
Safety - Adverse events
Time frame: From baseline to visit 16 (14 weeks)
Incidence and severity of treatment emergent adverse events assessed weekly.
Safety - clinical laboratory measures
Time frame: From Baseline to visit 16 (14 weeks)
Proportion of subjects exceeding pre-established critical laboratory values.
Safety - clinical assessment
Time frame: From Baseline to visit 16 (14 weeks)
Proportion of subjects with abnormal findings in physical/neurological exams, vital signs and electrocardiograms.
6 secondary outcomes
Pharmacokinetic parameter - Cmax
Time frame: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - AUC
Time frame: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - Tmax
Time frame: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - Clearance
Time frame: Visits 3 - 6 (up to 4 weeks)
Pharmacokinetic parameter - Volume of distribution
Time frame: Visits 3 - 6 (up to 4 weeks)
Satisfaction and feasibility of home nurse survey
Time frame: Visits 8-9 and 11-15 (up to 8 weeks)
Results and safety
The study was stopped early (reason given: Study interrupted by COVID-19 pandemic and could not be restarted at current location. Study investigators determined that relocating and continuing the study under its original scope was not feasible given preliminary results from IM injections.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Roberta D Brinton, PhD principal investigator
University of Arizona
Lon S Schneider, MD, MS principal investigator
University of Southern California
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Allopregnanolone in people with Alzheimer Dementia, run by University of Arizona at 1 site 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?
The sponsor lists 7 inclusion and 6 exclusion criteria. 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: stopped early, as of the sponsor's last update on 13 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Study interrupted by COVID-19 pandemic and could not be restarted at current location. Study investigators determined that relocating and continuing the study under its original scope was not feasible given preliminary results from IM injections.). Results are not available in the source record.
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?
Safety - Adverse events (time frame: From baseline to visit 16 (14 weeks)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 13 Mar 2026.