Adipose Stem Cells for Traumatic Spinal Cord Injury

NCT03308565 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 17-004621
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine if mesenchymal stem cells (MSC) derived from the fat tissue can be safely administered into the cerebrospinal fluid (CSF) of patients with spinal cord injury. Adipose-derived mesenchymal stem cells (AD-MSCs) have been used in previous research studies at the Mayo Clinic. All subjects enrolled in this study will receive AD-MSC treatment, which is still experimental and is not approved by the U.S. Food and Drug Administration (FDA) for large scale use. However, the FDA has allowed the use of this agent in this research study.

Conditions

Spinal Cord Injuries; Paralysis

Keywords

AIS grade A or B

Outcome Measures

Primary Outcomes (1)

• Incidence of acute adverse events

  An adverse event is defined any untoward or undesirable medical occurrence in the form of signs, symptoms, abnormal findings, or diseases that emerge or worsen relative to baseline (i.e., if present upon study entry) during the study regardless of causal relationship to the study drug

  up to 4 weeks post treatment

Secondary Outcomes (21)

• Incidence of delayed adverse events

  48 weeks post treatment

• Severity of adverse events

  4 weeks post-treatment

• Relationship of adverse events to study drug

  4 weeks post-treatment

• Incidence of serious adverse events (SAE)

  48 weeks post-treatment

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

  baseline, 96 weeks

Study Arms (1)

Phase I

EXPERIMENTAL

Patients will receive a single dose of 100 million autologous, adipose derived mesenchymal stem cells. The cells are isolated from patient's adipose tissue and expanded for intrathecal delivery.

Biological: Autologous, Adipose derived Mesenchymal Stem Cells

Interventions

Autologous, Adipose derived Mesenchymal Stem Cells BIOLOGICAL

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

Phase I

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
• SCI must be traumatic, blunt/non-penetrating in nature and not degenerative
• SCI must be within two weeks and up to 1 year after the event
• 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
• 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
• 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
• Participation in a study of an experimental drug or medical device within 3 months of study enrollment
• Known allergy to local anesthetics of other components of the study drug

Sponsors & Collaborators

• Mohamad Bydon: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Publications (2)

• Bydon M, Qu W, Moinuddin FM, Hunt CL, Garlanger KL, Reeves RK, Windebank AJ, Zhao KD, Jarrah R, Trammell BC, El Sammak S, Michalopoulos GD, Katsos K, Graepel SP, Seidel-Miller KL, Beck LA, Laughlin RS, Dietz AB. Intrathecal delivery of adipose-derived mesenchymal stem cells in traumatic spinal cord injury: Phase I trial. Nat Commun. 2024 Apr 1;15(1):2201. doi: 10.1038/s41467-024-46259-y.

  PMID: 38561341 DERIVED

• Bydon M, Dietz AB, Goncalves S, Moinuddin FM, Alvi MA, Goyal A, Yolcu Y, Hunt CL, Garlanger KL, Del Fabro AS, Reeves RK, Terzic A, Windebank AJ, Qu W. CELLTOP Clinical Trial: First Report From a Phase 1 Trial of Autologous Adipose Tissue-Derived Mesenchymal Stem Cells in the Treatment of Paralysis Due to Traumatic Spinal Cord Injury. Mayo Clin Proc. 2020 Feb;95(2):406-414. doi: 10.1016/j.mayocp.2019.10.008. Epub 2019 Nov 27.

  PMID: 31785831 DERIVED

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Spinal Cord Injuries; Paralysis

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

Study Officials

• Mohamad Bydon, MD

  Mayo Clinic, Rochester, MN

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Regulatory Sponsor

Study Record Dates

• First Submitted: October 9, 2017
• First Posted: October 12, 2017
• Study Start: December 5, 2017
• Primary Completion: October 22, 2019
• Study Completion: October 11, 2021
• Last Updated: August 30, 2022

Record last verified: 2022-08

Locations

US (1)