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

NCT03169231 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: 001-03
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 7

Brief Summary

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

Conditions

Aging Frailty

Outcome Measures

Primary Outcomes (1)

• Change from baseline in 6 Minute Walk Test (6MWT) compared to placebo

  Change from baseline in 6MWT compared to placebo at 180 days post-infusion

  Baseline and 180 days post-infusion

Secondary Outcomes (2)

• Change in patient reported outcome of overall physical function capacity using the PROMIS-Physical Function-Short Form 20a compared to placebo

  180 days post-infusion

• Change in TNF-alpha compared to placebo

  180 days post-infusion

Study Arms (5)

Study Group A

EXPERIMENTAL

Single peripheral IV infusion of 25 million Longeveron Mesenchymal Stem Cells (LMSCs)

Biological: Longeveron Mesenchymal Stem Cells (LMSCs)

Study Group B

EXPERIMENTAL

Single peripheral IV infusion of 50 million Longeveron Mesenchymal Stem Cells (LMSCs)

Biological: Longeveron Mesenchymal Stem Cells (LMSCs)

Study Group C

EXPERIMENTAL

Single peripheral IV infusion of 100 million Longeveron Mesenchymal Stem Cells (LMSCs)

Biological: Longeveron Mesenchymal Stem Cells (LMSCs)

Study Group D

EXPERIMENTAL

Single peripheral IV infusion of 200 million Longeveron Mesenchymal Stem Cells (LMSCs)

Biological: Longeveron Mesenchymal Stem Cells (LMSCs)

Study Group E

PLACEBO COMPARATOR

Single peripheral IV infusion of placebo.

Other: Placebo

Interventions

Longeveron Mesenchymal Stem Cells (LMSCs) BIOLOGICAL

Intravenously delivered

Study Group A; Study Group B; Study Group C; Study Group D

Placebo OTHER

Intravenously delivered

Study Group E

Eligibility Criteria

• Age: 70 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Be unwilling or unable to perform any of the assessments required by the protocol.
• 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.
• Have a score of 24 or lower on the Mini Mental State Examination (MMSE)
• Have poorly controlled blood glucose levels (HbA1c \>8.0%).
• 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.
• Have any condition that in the opinion of the Principal Investigator limits lifespan to \< 1 year.
• Have autoimmune disease (e.g. rheumatoid arthritis, systemic lupus erythematosus).
• Be using chronic immunosuppressant therapy such as high-dose corticosteroids or TNF-alpha antagonists (prednisone use at doses of \< 5 mg daily is allowed).
• 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.
• Test positive for verimic Hepatitis C virus, HIV1/2, or syphilis
• Have a resting blood oxygen saturation of \<93% (measured by pulse oximetry).
• Known or suspected alcohol or drug abuse within three years preceding Screening
• Have a known hypersensitivity to dimethyl sulfoxide (DMSO).
• Be an organ transplant recipient (other than transplantation for corneal, bone, skin, ligament, or tendon).

Sponsors & Collaborators

• Longeveron Inc.: lead

Study Sites (7)

Soffer Health Institute

Aventura, Florida, 33180, United States

Location

Clinical Research of South Florida

Coral Gables, Florida, 33134, United States

Location

Clinical Physiology Associates

Fort Myers, Florida, 33912, United States

Location

Miami VA Healthcare System

Miami, Florida, 33125, United States

Location

Vista Health Research

Miami, Florida, 33176, United States

Location

Panax Clinical Research

Miami Lakes, Florida, 33014, United States

Location

Advanced Research for Health Improvement, LLC

Naples, Florida, 34102, United States

Location

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 24, 2017
• First Posted: May 30, 2017
• Study Start: July 6, 2017
• Primary Completion: September 30, 2021
• Study Completion: September 30, 2021
• Last Updated: March 7, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)