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Alzheimer's DiseasePhase 1NCT02600130

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

A Phase 1 study testing Longeveron Mesenchymal Stem Cells in people with Alzheimer's Disease, run by Longeveron Inc. at 3 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 50 to 80 years
Conditions the study is for
Alzheimer's Disease
What is being tested?
Longeveron Mesenchymal Stem Cells
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
33 participants
Where
3 sitesUnited States
Study dates
10 Oct 2016to Sep 2021
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 50 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

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;
  • 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.

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 a female who is pregnant, nursing, or of childbearing potential 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;
  • be positive for HIV, Syphilis and Hepatitis C; or
  • be positive for Hepatitis B. If the subject tests positive for anti-HBc or anti-HBs, subject must be currently receiving treatment for hHepatitis B prior to infusion and remain on treatment throughout the study.

Trial locations and contacts

3 sites in 1 country.

United States3 sites
  • Brain Matters Research

    Delray Beach, Florida, 33445

  • University of Miami Miller School of Medicine

    Miami, Florida, 33136

  • Miami Jewish Health

    Miami, Florida, 33137

Other trials in United States

About this study

Official title: 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

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.

Read the 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.

Being tested
Longeveron Mesenchymal Stem Cells, Placebo
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Longeveron Mesenchymal Stem CellsBiological

    via peripheral intravenous infusion

  • PlaceboBiological

    via peripheral intravenous infusion

Study groups in NCT02600130
Study groupRoleWhat they receive
Cohort 1ExperimentalCohort 1 (10 subjects) Target dose 20 million Longeveron Mesenchymal Stem Cells (LMSCs) via peripheral intravenous infusion.
Receives: Biological: Longeveron Mesenchymal Stem Cells
Cohort 2ExperimentalCohort 2 (10 subjects) Target dose 100 million Longeveron Mesenchymal Stem Cells (LMSCs)via peripheral intravenous infusion.
Receives: Biological: Longeveron Mesenchymal Stem Cells
Cohort 3Placebo comparatorCohort 3 (5 subjects) Placebo (Plasmalyte A and 1% human serum albumin (HSA)) via peripheral intravenous infusion.
Receives: Biological: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: 30 days post infusion

Secondary outcomes (1)
  1. Preliminary efficacy will be determined by examining for changes in AD status and rate decline as assessed by the following.

    • 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.

    Measured: At Baseline, 2, 4, 13, 26, 39, and 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Nov 2015 (estimated)
  2. Study start10 Oct 2016
  3. Primary completionSep 2020
  4. Study completionSep 2021

Resources

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
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
33 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02600130
Sponsor's protocol ID
00-0000-01
Status last verified
Dec 2021
First submitted
2 Nov 2015
Last updated
14 Dec 2021
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Longeveron Mesenchymal Stem Cells in people with Alzheimer's Disease, run by Longeveron Inc. at 3 sites 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?

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: finished, as of the sponsor's last update on 14 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

To demonstrate the safety of LMSCs administered to subjects with Alzheimer's disease. (time frame: 30 days post infusion). 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 14 Dec 2021.

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