Autologous Adipose Derived Mesenchymal Stem Cells for Spinal Cord Injury Patients

NCT04520373 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 19-011706
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to investigate the safety and potential therapeutic effects of autologous, culture-expanded, adipose derived mesenchymal stem cell intrathecal injections in the treatment of spinal cord injury.

Conditions

Spinal Cord Injuries; Paralysis

Outcome Measures

Primary Outcomes (1)

• •Change in sensory and motor function following completion of treatment as measured by the American Spinal Injury Association (ASIA) Impairment Scale (AIS)

  The ASIA Impairment Scale describes a person's functional impairment (both motor and sensory) as a result of their spinal cord injury. The scale has 5 levels, ranging from A (complete) to E (normal).

  up to 12 months post injection

Secondary Outcomes (6)

• Change in sensory and motor function following completion of treatment as measured by Somatosensory Evoked Potentials (SSEPs)

  baseline, up to 12 months post injection

• change in Neurogenic Bladder Symptom Score (NBSS)

  up to 24 months post injection

• change in Neurogenic Bowel Symptom Score

  up to 24 months post injection

• Incidence of abnormal CSF composition

  up to 4 weeks post injection

• Number of subjects who develop a new pathologic mass at the spinal cord area of injection or anywhere along the spinal cord.

  up to 12 months post injection

Study Arms (2)

Treatment Group 1: AD-MSC Injection

EXPERIMENTAL

Patients will receive a single dose of autologous, adipose derived mesenchymal stem cells one time. The cells are isolated from patient's adipose tissue and expanded for intrathecal delivery.

Biological: Autologous, Adipose Derived Mesenchymal Stem Cells

Treatment Group 2: Best Medical Management

ACTIVE COMPARATOR

Patients will be observed over six months while attending physical and occupational therapy. After six months, patients will receive a single dose of autologous, adipose derived mesenchymal stem cells one time. The cells are isolated from patient's adipose tissue and expanded for intrathecal delivery.

Other: Best Medical Management: Occupational and Physical Therapy; Biological: Autologous, Adipose Derived Mesenchymal Stem Cells

Interventions

Best Medical Management: Occupational and Physical Therapy OTHER

Observation while undergoing Occupational and Physical Therapy for 6 months

Treatment Group 2: Best Medical Management

Autologous, Adipose Derived Mesenchymal Stem Cells BIOLOGICAL

The mesenchymal stem cells will be collected and expanded from the patients' adipose tissue.

Treatment Group 1: AD-MSC Injection; Treatment Group 2: Best Medical Management

Eligibility Criteria

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

You may qualify if:

• Male or female aged 18 years and older
• Females of childbearing potential must have a negative pregnancy test prior to receiving the study drug and will agree to use adequate contraception (hormonal/barrier method or abstinence) from the time of screening to a period of 1 year following completion of the drug treatment cycle. Females of childbearing potential are defined as premenopausal and not surgically sterilized, or post-menopausal for fewer than 2 years. If the urine pregnancy test is positive, the study drug will not be administered and the result will be confirmed by a serum pregnancy test. Serum pregnancy tests will be performed at a central clinical laboratory, whereas urine pregnancy tests will be performed by qualified personnel using kit.
• Females becoming pregnant during the study will continue to be monitored for the duration of the study or completion of the pregnancy, whichever is longer. Monitoring will include perinatal and neonatal outcome. Any SAEs associated with pregnancy will be recorded.
• AIS grade A or B of SCI at the time of injury with or without subsequent improvement within 1 year of injury that has progressed to a higher AIS grade with a plateau in functional improvement
• SCI must be traumatic, blunt/non-penetrating in nature and not degenerative
• Full understanding of the requirements of the study and willingness to comply with the treatment plan, including fat harvesting, laboratory tests, diagnostic imaging, complete physical and neurologic examination and follow-up visits and assessments
• Full understanding of the requirements of home exercise program prescribed by physical and occupational therapists.
• Once the nature of the study is fully explained and prior to any study-related procedure is initiated the subject is willing to provide written, informed consent and complete HIPAA documentation

You may not qualify if:

• Pregnant or nursing, or planning on becoming pregnant during the study period
• AIS grade of SCI other than A or B at the time of injury
• Non-traumatic SCI
• History of receiving mesenchymal stem cell, gene or exosome therapy for any indications
• History of intra-spinal infection
• History of superficial infection in the index spinal level within 6 months of study
• Evidence of current superficial infection affecting the index spinal level at the time of enrollment
• On chronic, immunosuppressive transplant therapy or having a chronic, immunosuppressive state, including use of systemic steroids/corticosteroids
• Taking anti-rheumatic disease medication (including methotrexate or other antimetabolites) within 3 months prior to study enrollment
• Ongoing infectious disease, including but not limited to tuberculosis, HIV, hepatitis, and syphilis
• Fever, defined as temperature above 100.4 F/38.0 Celsius, or mental confusion at baseline
• Significant improvement between the time of adipose tissue harvest and the time of injection, defined as improvement from AIS grade A or B to AIS grade C or greater.
• Clinically significant cardiovascular (e.g. history of myocardial infarction, congestive heart failure or uncontrolled hypertension \> 90 mmHg diastolic and/or 180 mmHg systolic), neurological (e.g. stroke, TIA) renal, hepatic or endocrine disease (e.g. diabetes, osteoporosis).
• History of malignancy including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline). Any other malignancy will not be allowed.
• History of blood dyscrasia, including but not limited to anemia, thrombocytopenia, and monoclonal gammopathy

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Spinal Cord Injuries; Paralysis

Interventions

Physical Therapy Modalities

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Trauma, Nervous System; Wounds and Injuries; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Therapeutics; Rehabilitation

Study Officials

• Wenchun Qu, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 14, 2020
• First Posted: August 20, 2020
• Study Start: June 25, 2020
• Primary Completion (Estimated): July 1, 2026
• Study Completion (Estimated): July 1, 2026
• Last Updated: October 14, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)