Try the modernized beta website. Learn more about the modernization effort.
Working… Menu

Phase IIb Trial to Evaluate Longeveron Mesenchymal Stem Cells to Treat Aging Frailty

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. Read our disclaimer for details. Identifier: NCT03169231
Recruitment Status : Active, not recruiting
First Posted : May 30, 2017
Last Update Posted : August 16, 2021
Information provided by (Responsible Party):
Longeveron Inc.

Brief Summary:
This is a randomized, placebo-controlled, double-blind, parallel arm, multi-center Phase 2b study.

Condition or disease Intervention/treatment Phase
Aging Frailty Biological: Longeveron Mesenchymal Stem Cells (LMSCs) Other: Placebo Phase 2

Detailed Description:
The objectives of this study are to assess safety and efficacy of Longeveron Mesenchymal Stem Cells (LMSCs) compared to placebo on 1) functional mobility and exercise tolerance, 2) patient-reported physical function, and 3) the inflammatory cytokine biomarker tumor necrosis factor (TNF-alpha).

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 150 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2b, Randomized, Blinded and Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Longeveron Allogenic Human Mesenchymal Stem Cells Infusion in Patients With Aging Frailty
Actual Study Start Date : July 6, 2017
Actual Primary Completion Date : April 30, 2021
Estimated Study Completion Date : September 2021

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Study Group A
Single peripheral IV infusion of 25 million Longeveron Mesenchymal Stem Cells (LMSCs)
Biological: Longeveron Mesenchymal Stem Cells (LMSCs)
Intravenously delivered

Experimental: Study Group B
Single peripheral IV infusion of 50 million Longeveron Mesenchymal Stem Cells (LMSCs)
Biological: Longeveron Mesenchymal Stem Cells (LMSCs)
Intravenously delivered

Experimental: Study Group C
Single peripheral IV infusion of 100 million Longeveron Mesenchymal Stem Cells (LMSCs)
Biological: Longeveron Mesenchymal Stem Cells (LMSCs)
Intravenously delivered

Experimental: Study Group D
Single peripheral IV infusion of 200 million Longeveron Mesenchymal Stem Cells (LMSCs)
Biological: Longeveron Mesenchymal Stem Cells (LMSCs)
Intravenously delivered

Placebo Comparator: Study Group E
Single peripheral IV infusion of placebo.
Other: Placebo
Intravenously delivered

Primary Outcome Measures :
  1. Change from baseline in 6 Minute Walk Test (6MWT) compared to placebo [ Time Frame: Baseline and 180 days post-infusion ]
    Change from baseline in 6MWT compared to placebo at 180 days post-infusion

Secondary Outcome Measures :
  1. Change in patient reported outcome of overall physical function capacity using the PROMIS-Physical Function-Short Form 20a compared to placebo [ Time Frame: 180 days post-infusion ]
    Change from baseline in physical function will be measured to assess Patient-Reported Outcome Measurement compared to placebo at 180 days post infusion.

  2. Change in TNF-alpha compared to placebo [ Time Frame: 180 days post-infusion ]
    Change in serum TNF-alpha compared to placebo

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   70 Years to 85 Years   (Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Be willing and able to provide written informed consent and comply with all procedures required by the Protocol.
  2. Be >70 and < 85 years of age at the time of signing the Informed Consent Form.
  3. Have a Canadian Study on Health and Aging (CSHA) Clinical Frailty Scale score of 5 "mildly frail" or 6 "moderately frail".
  4. Have a 6 minute walk distance of > 200m and < 400 m. Distances of two 6MWTs are to be within 15% of each other.
  5. Have a serum TNF-alpha level > 2.5 pg/mL

Exclusion Criteria:

  1. Be unwilling or unable to perform any of the assessments required by the protocol.
  2. Have a diagnosis of any disabling neurologic disorder, including, but not limited to, Parkinson's disease, Amyotrophic Lateral Sclerosis, multiple sclerosis, cerebrovascular accident with residual deficits (e.g., muscle weakness or gait disorder), or diagnosis of dementia.
  3. Have a score of 24 or lower on the Mini Mental State Examination (MMSE)
  4. Have poorly controlled blood glucose levels (HbA1c >8.0%).
  5. Have a clinical history of malignancy within 2.5 years (i.e., subjects with prior malignancy must be cancer free for 2.5 years) except curatively-treated basal cell carcinoma, melanoma in situ or cervical carcinoma.
  6. Have any condition that in the opinion of the Principal Investigator limits lifespan to < 1 year.
  7. Have autoimmune disease (e.g. rheumatoid arthritis, systemic lupus erythematosus).
  8. Be using chronic immunosuppressant therapy such as high-dose corticosteroids or TNF-alpha antagonists (prednisone use at doses of < 5 mg daily is allowed).
  9. Test positive for hepatitis B virus

    a. If the subject tests positive for anti-hepatitis B core antigen (HBc) or anti-HBs, they must be currently receiving treatment for Hepatitis B prior to infusion and remain on treatment throughout the study.

  10. Test positive for verimic Hepatitis C virus, HIV1/2, or syphilis
  11. Have a resting blood oxygen saturation of <93% (measured by pulse oximetry).
  12. Known or suspected alcohol or drug abuse within three years preceding Screening
  13. Have a known hypersensitivity to dimethyl sulfoxide (DMSO).
  14. Be an organ transplant recipient (other than transplantation for corneal, bone, skin, ligament, or tendon).
  15. Be actively listed (or expected future listing) for transplant of any organ (other than corneal transplant).
  16. Have any clinically important abnormal screening laboratory values, including, but not limited to:

    1. Hemoglobin <10.0 g/dL,
    2. White blood cell <2,500/ul, or platelet count <100,000/ul
    3. Liver dysfunction evidenced by enzymes (AST and ALT) > 3 times the upper limit of normal (ULN)
    4. Coagulopathy (INR>1.3) not due to a reversible cause (e.g. warfarin and/or Factor Xa inhibitors).
  17. Uncontrolled hypertension (resting systolic blood pressure >180 mm Hg or diastolic blood pressure of > 110 mm Hg at Screening)
  18. Have unstable angina pectoris, uncontrolled or severe peripheral artery disease within the previous 3 months.
  19. Have congestive heart failure defined by NYHS (New York Heart Association) Class III or IV, or an ejection fraction of <25%.
  20. Have a coronary artery bypass surgery, angioplasty, or peripheral vascular disease revasculation or a myocardial infarction within previous 3 months.
  21. Have severe pulmonary dysfunction: acute exacerbation of chronic obstructive lung disease stage III or IV (Gold classification), and/or PaO2 levels <60 mmHg.
  22. Have a partial ileal gastric bypass, or other significant intestinal malabsorption.
  23. Have advanced liver or renal disease
  24. Have cognitive or language barriers that prohibit obtaining informed consent or any study elements.
  25. Be currently hospitalized, or living in an assisted living facility or a long-term care facility.
  26. Be currently participating (or participated within the previous 30 days of consent) in an investigational therapeutic or device trial.
  27. Have a history or current evidence of any condition, therapy, laboratory abnormality, or other circumstance that might confound the results of the study, or interfere with the patient's participation for the full duration of the study.

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 identifier (NCT number): NCT03169231

Layout table for location information
United States, Florida
Soffer Health Institute
Aventura, Florida, United States, 33180
Clinical Research of South Florida
Coral Gables, Florida, United States, 33134
Clinical Physiology Associates
Fort Myers, Florida, United States, 33912
Panax Clinical Research
Miami Lakes, Florida, United States, 33014
Miami VA Healthcare System
Miami, Florida, United States, 33125
Vista Health Research
Miami, Florida, United States, 33176
Advanced Research for Health Improvement, LLC
Naples, Florida, United States, 34102
Sponsors and Collaborators
Longeveron Inc.
Layout table for additonal information
Responsible Party: Longeveron Inc. Identifier: NCT03169231    
Other Study ID Numbers: 001-03
First Posted: May 30, 2017    Key Record Dates
Last Update Posted: August 16, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Pathologic Processes