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Allogeneic Human Mesenchymal Stem Cell Infusion Versus Placebo in Patients With Alzheimer's Disease

This study is currently recruiting participants.
Verified March 2017 by Longeveron LLC
Sponsor:
ClinicalTrials.gov Identifier:
NCT02600130
First Posted: November 9, 2015
Last Update Posted: March 17, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Longeveron LLC
  Purpose
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.

Condition Intervention Phase
Alzheimer's Disease Biological: Longeveron Mesenchymal Stem Cells Biological: Placebo Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase, I Prospective, Randomized, Double-Blinded, Placebo-controlled Trial to Evaluate the Safety and Potential Efficacy of Longeveron Allogeneic Human Mesenchymal Stem Cell (LMSCs) Infusion Versus Placebo in Patients With Alzheimer's Disease

Resource links provided by NLM:


Further study details as provided by Longeveron LLC:

Primary Outcome Measures:
  • 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 Outcome Measures:
  • Preliminary efficacy will be determined by examining for changes in AD status and rate decline as assessed by the following. [ Time Frame: At Baseline, 2, 4, 13,26, 39, and 52 weeks ]
    • Neurological/neurocognitive assessments. ADAS-Cog 11 (Alzheimer's Disease Assessment Scale-cognitive subscale 11) MMSE (Mini Mental State Examination) NPI (Neuropsychiatric Inventory) UPSIT (University of Pennsylvania Smell Identification Test) GDS (Geriatric Depression Scale)
    • Quality of life assessments. ADCS-ADL (Alzheimer's Disease Cooperative Study Activities of Daily Living) QOL-AD (Quality of Life-Alzheimer's Disease)
    • Blood inflammatory and AD biomarkers. IL-1 IL-6 TGF-β1 TNF-α CRP D-Dimer Fibrinogen ApoE
    • Cerebrospinal fluid (CSF) inflammatory biomarkers. IL-1 IL-6 TGF-β1 TNF-α
    • CSF biomarkers of AD. Tau. Phosphorylated tau. Beta-amyloid.
    • Brain volumetry calculated using MRI, including:

    Hippocampal volume. Ventricular volume. Whole-brain volume.



Estimated Enrollment: 30
Study Start Date: August 2016
Estimated Study Completion Date: October 2019
Estimated Primary Completion Date: August 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1
Cohort 1 (10 subjects) Target dose 20 million Longeveron Mesenchymal Stem Cells (LMSCs) via peripheral intravenous infusion.
Biological: Longeveron Mesenchymal Stem Cells
via peripheral intravenous infusion
Experimental: Cohort 2
Cohort 2 (10 subjects) Target dose 100 million Longeveron Mesenchymal Stem Cells (LMSCs)via peripheral intravenous infusion.
Biological: Longeveron Mesenchymal Stem Cells
via peripheral intravenous infusion
Placebo Comparator: Cohort 3
Cohort 3 (5 subjects) Placebo (Plasmalyte A and 1% human serum albumin (HSA)) via peripheral intravenous infusion.
Biological: Placebo
via peripheral intravenous infusion

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 2,4,13,39 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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   50 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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;
  • have normal levels of thyroid hormone (free T4) and thyroid-stimulating hormone (TSH);
  • have normal levels of B12 and folate;
  • be using an acetylcholinesterase inhibitor and/or Memantine treatment on a stable dosage for at least 4 months prior to screening;
  • have a designated study partner who will accompany the subject to all clinic visits and participate in the subject's clinical assessments;
  • be living in the community, including in an assisted living facility, but excluding long-term care nursing facilities.
  • be negative for hepatitis B HsAg, viremic hepatitis C, and HIV.

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);
  • 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.
  • have been diagnosed with malignancy within the past 5 years, with the exception of curatively-treated basal cell carcinoma, squamous cell carcinoma, melanoma in situ or cervical carcinoma;
  • be female with an FSH < 25.8 IU/L;
  • be a female who is pregnant, nursing, or of childbearing potential (defined in Section 9.2.2) while not practicing effective contraception (female subjects must undergo a urine pregnancy test at screening and on the infusion day prior to infusion);
  • have any serious illness or any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the subject or preclude successful completion of the study;
  • have any serious illness or any other condition that, in the opinion of the investigator, may compromise the validity of the study (e.g., signs of stroke, traumatic brain injury (TBI), multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), and Parkinsonism);
  • have participated in any investigational therapeutic or device trial within the past 5 years that the investigator feels would influence or affect the outcome of the study;
  • Be currently participating (or participated within the previous 30 days) in an investigational therapeutic or device trial.
  Contacts and Locations
Information from the National Library of Medicine

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): NCT02600130


Contacts
Contact: Suzanne Page, JD 305-342-9590 spage@longeveron.com
Contact: Ann Glasse, RN, BSN, MBA 786-769-4935 aglasse@longeveron.com

Locations
United States, Florida
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33136
Contact: Carmen Perez    305-243-0184    c.perez71@med.miami.edu   
Principal Investigator: Bernard S. Baumel, MD         
Sponsors and Collaborators
Longeveron LLC
Investigators
Principal Investigator: Bernard S. Baumel, MD Principal Investigator
  More Information

Responsible Party: Longeveron LLC
ClinicalTrials.gov Identifier: NCT02600130     History of Changes
Other Study ID Numbers: 00-0000-01
First Submitted: November 2, 2015
First Posted: November 9, 2015
Last Update Posted: March 17, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Alzheimer Disease
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Tauopathies
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders