Lomecel-B Infusion Versus Placebo in Patients With Alzheimer's Disease

NCT02600130 | Completed Phase 1 DMC

• 1 other identifier: 00-0000-01
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 3

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.

Conditions

Alzheimer's Disease

Outcome Measures

Primary Outcomes (1)

• To demonstrate the safety of LMSCs administered to subjects with Alzheimer's disease.

  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).

  30 days post infusion

Secondary Outcomes (1)

• 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

Study Arms (3)

Cohort 1

EXPERIMENTAL

Cohort 1 (10 subjects) Target dose 20 million Longeveron Mesenchymal Stem Cells (LMSCs) via peripheral intravenous infusion.

Biological: Longeveron Mesenchymal Stem Cells

Cohort 2

EXPERIMENTAL

Cohort 2 (10 subjects) Target dose 100 million Longeveron Mesenchymal Stem Cells (LMSCs)via peripheral intravenous infusion.

Biological: Longeveron Mesenchymal Stem Cells

Cohort 3

PLACEBO COMPARATOR

Cohort 3 (5 subjects) Placebo (Plasmalyte A and 1% human serum albumin (HSA)) via peripheral intravenous infusion.

Biological: Placebo

Interventions

Longeveron Mesenchymal Stem Cells BIOLOGICAL

via peripheral intravenous infusion

Cohort 1; Cohort 2

Placebo BIOLOGICAL

via peripheral intravenous infusion

Cohort 3

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;
• have normal levels of thyroid hormone (free T4) and thyroid-stimulating hormone (TSH);
• have normal levels of B12 and folate;
• have a designated study partner who will accompany the subject to all clinic visits and participate in the subject's clinical assessments; or
• be living in the community, including in an assisted living facility, but excluding long-term care nursing facilities.

You may not qualify if:

• 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);
• be active listed (or expected to be listed) for transplant of any organ;
• be an organ transplant recipient;
• have a known hypersensitivity to dimethyl sulfoxide (DMSO).
• have a condition that is projected to limited life expectancy to \< 1 year.
• have a sitting or resting systolic blood pressure \>180 mm Hg or diastolic blood pressure \>110 mm Hg at Screening;
• have a history of alcohol or drug abuse within the past 5 years.

Sponsors & Collaborators

• Longeveron Inc.: lead

Study Sites (3)

Brain Matters Research

Delray Beach, Florida, 33445, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Miami Jewish Health

Miami, Florida, 33137, United States

Location

MeSH Terms

Conditions

Alzheimer Disease

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 2, 2015
• First Posted: November 9, 2015
• Study Start: October 10, 2016
• Primary Completion: September 1, 2020
• Study Completion: September 1, 2021
• Last Updated: December 14, 2021

Record last verified: 2021-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)