A Phase, I Prospective, Randomized, Double-Blinded, Placebo-controlled, Trial to Evaluate the Safety and Potential Efficacy of Lomecel-B Infusion Versus Placebo in Patients With Alzheimer's Disease
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Alzheimer's Disease
- Sponsor
- Longeveron Inc.
- Enrollment
- 33
- Locations
- 3
- Primary Endpoint
- To demonstrate the safety of LMSCs administered to subjects with Alzheimer's disease.
- Status
- Completed
- Last Updated
- 4 years ago
Overview
Brief Summary
This is a Phase I, prospective, randomized, placebo-controlled, double-blinded study designed to test the safety and efficacy of LMSCs (Longeveron Mesenchymal Stem Cells) for the treatment of subjects with clinically diagnosed Alzheimer's disease.
Detailed Description
This is a randomized, placebo-controlled clinical trial designed to evaluate the safety and efficacy of LMSCs (Longeveron Mesenchymal Stem Cells) or placebo in subjects with Alzheimer's Disease. Following a successful Safety Run-In Phase, a total of twenty-five (25) subjects will be randomized to (2:2:1) to receive low-dose LMSCs, high-dose LMSCs or placebo. After randomization, baseline imaging, and study product infusion, subjects will be followed up at 2,4,13, 26, 39 and 52 week post study product infusion. Intention-to-treat study population will be used for the purpose of the endpoint analysis and safety evaluations.
Investigators
Eligibility Criteria
Inclusion Criteria
- •All subjects enrolled in this trial must:
- •provide written informed consent;
- •be 50 - 80 years of age at the time of signing the Informed Consent form;
- •have a body mass of 45 - 150 kg;
- •at the time of enrollment, be diagnosed with AD in accordance with the NINCDS-AA criteria;
- •score between 18 and 24 on the Mini Mental State Examination (MMSE);
- •has one (or more) identified adult caregiver who is willing to provide written informed consent for his/her own participation; is able to read, understand, and speak the designated language at the study site; either lives with the subject or sees the subject for ≥2 hours/day ≥3 days/week; and agrees to accompany the subject to each study visit;
- •blood oxygen saturation ≥93% determined via pulse oximetry;
- •have a brain MRI consistent with AD;
- •have a PET scan using an FDA-approved tracer (e.g., AMYViD, Vizamyl, or Neuraceq), and which indicates the presence of beta-amyloid plaques in the cerebral cortex, within 5 years of enrollment;
Exclusion Criteria
- •All subjects enrolled in this trial must not:
- •be unable to perform any of the assessments required for endpoint analysis;
- •show signs of dementia other than AD, such as from AIDS (Acquired Immunodeficiency Syndrome), CJD (Creutzfeldt-Jakob disease), LBD (Lewy Bodies dementia), CVD (Cerebrovascular dementia), PSP (Progressive Supranuclear Palsy), MCI (multiple cerebral infarctions) or NPH (normal pressure hydrocephalus);
- •have any other neurodegenerative disease;
- •have a history of a seizure disorder;
- •have clinically important abnormal screening laboratory values beyond AD;
- •have any conditions that would contraindicate an MRI, such as the presence metallic objects in the eyes, skin, or heart;
- •have any conditions that would contraindicate a PET scan;
- •have \> 4 cerebral microhemorrhages (regardless of their anatomical location or diagnostic characterization as "possible" or "definite"), a single area of superficial siderosis, or evidence of a prior macrohemorrhage as assessed by MRI;
- •be currently using corticosteroids or similar powerful steroidal anti-inflammatory medication (e.g., Prednisone) on a regular basis (exceptions allowed include regular use of steroidal nasal sprays, topical steroids, and estrogen-replacement therapy);
Outcomes
Primary Outcomes
To demonstrate the safety of LMSCs administered to subjects with Alzheimer's disease.
Time Frame: 30 days post infusion
Incidence of any treatment-emergent serious adverse event (TE-SAE), defined as one or more of the following untoward medical occurrences happening within the first 30 days after infusion. * Life-threatening event (e.g., stroke or non-fatal pulmonary embolism). * Requires inpatient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Results in death. * Leads to other clinically significant untoward laboratory test result(s) or medical condition(s), determined per Investigator's judgement (e.g., new clinically asymptomatic brain microhemorrhages).
Secondary Outcomes
- Preliminary efficacy will be determined by examining for changes in AD status and rate decline as assessed by the following.(At Baseline, 2, 4, 13, 26, 39, and 52 weeks)