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Spinal Cord InjuriesPhase 2NCT04520373

Autologous Adipose Derived Mesenchymal Stem Cells for Spinal Cord Injury Patients

A Phase 2 study testing Best Medical Management: Occupational and Physical Therapy and Autologous, Adipose Derived Mesenchymal Stem Cells in people with Spinal Cord Injuries and Paralysis, run by Mayo Clinic at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Spinal Cord Injuries, Paralysis
What is being tested?
Best Medical Management: Occupational and Physical Therapy and Autologous, Adipose Derived Mesenchymal Stem Cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
1 siteUnited States
Study dates
25 Jun 2020to Jul 2026
Phase
Phase 2
Are there results?
Not yet: the study is under way

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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 22Show fewer
  • 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
  • Any contraindication to MRI scan according to MRI guidelines, or unwillingness to undergo MRI procedures
  • History of or current evidence of alcohol or drug abuse or dependence, recreational use of illicit drug or prescription medications, or use of medical marijuana within 30 days of study entry
  • Diagnosis of schizophrenia or bipolar disorder
  • Patients with baseline depression, diagnosed by the Beck Depression Inventory Assessment
  • Currently taking riluzole for treatment of amyotrophic lateral sclerosis (ALS)

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

Other trials in United States

About this study

Official title: CELLTOP Part II: A Phase II Clinical Trial of Autologous Adipose Derived Mesenchymal Stem Cells in the Treatment of Paralysis Due to Traumatic Spinal Cord Injury

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.

Being tested
Best Medical Management: Occupational and Physical Therapy, Autologous, Adipose Derived Mesenchymal Stem Cells
Medical subject headings
Spinal Cord Injuries, Paralysis

What participants receive

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

  • Best Medical Management: Occupational and Physical TherapyOther

    Observation while undergoing Occupational and Physical Therapy for 6 months

  • Autologous, Adipose Derived Mesenchymal Stem CellsBiological

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

Study groups in NCT04520373
Study groupRoleWhat they receive
Treatment Group 1: AD-MSC InjectionExperimentalPatients 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.
Receives: Biological: Autologous, Adipose Derived Mesenchymal Stem Cells
Treatment Group 2: Best Medical ManagementActive comparatorPatients 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.
Receives: Other: Best Medical Management: Occupational and Physical Therapy; Biological: Autologous, Adipose Derived Mesenchymal Stem Cells

What is being measured

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

  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).

    Measured: up to 12 months post injection

Secondary outcomes (6)

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

    Somatosensory Evoked Potentials (SSEPs) are electrical responses recorded from the nervous system following electrical stimulation of a peripheral nerve. For example, stimulation of the median nerve at the wrist produces electrical activity that travels along the sensory pathway on its way to the brain. This activity can be recorded with electrodes positioned along that pathway.

    Measured: baseline, up to 12 months post injection

  2. change in Neurogenic Bladder Symptom Score (NBSS)

    Neurogenic Bladder Symptom Score (NBSS) The NBSS was designed as an objective and validated way to assess bladder symptoms in patients with neurogenic bladder dysfunction as a result of spinal cord injury, multiple sclerosis, and spinal bifida. The NBSS consists of 24 questions. The first question classifies patients by bladder management, but does not make up part of the numeric score. The remaining questions address 3 domains: incontinence, storage and voiding, and consequences. The final question is an overall quality of life question. The total score can range from 0 (no symptoms at all) to 74 (maximum symptoms).

    Measured: up to 24 months post injection

  3. change in Neurogenic Bowel Symptom Score

    The Adult Neurogenic Bowel Dysfunction Score¹, also known as the Adult NBD score has been designed to help healthcare professionals evaluate the effectiveness of their patient's current bowel management routine by assessing the impact it has on the patient's quality of life. By having your patients answer 10 simple questions, the Adult NBD Score can help identify and quantify severity of bowel dysfunction in the adult patients. The Adult NBD score consists of 10 questions and is a symptom-score where each symptom is weighted based on its impact on quality of life. The scores from each question are added together and a total score is calculated. The maximum score is 47 while minimum is 0. Score Severity of bowel dysfunction 0-6 Very minor 7-9 Minor 10-13 Moderate 14+ Severe

    Measured: up to 24 months post injection

  4. Incidence of abnormal CSF composition

    Composition in reference to normal laboratory values.

    Measured: up to 4 weeks post injection

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

    Patients will undergo Magnetic Resonance Imaging of the spine and the spinal cord with and without contrast.

    Measured: up to 12 months post injection

  6. Correlation of adverse events to study drug

    The relationship of adverse events to study drug will be graded into the following categories: probable, possible, unlikely, unrelated.

    Measured: up to 4 weeks post injection

Timeline

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

  1. First posted20 Aug 2020
  2. Study start25 Jun 2020
  3. Primary completionJul 2026 (estimated)
  4. Study completionJul 2026 (estimated)

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
None (open label)
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04520373
Sponsor's protocol ID
19-011706
Status last verified
Jul 2025
First submitted
14 Jul 2020
Last updated
14 Oct 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Best Medical Management: Occupational and Physical Therapy and Autologous, Adipose Derived Mesenchymal Stem Cells in people with Spinal Cord Injuries and Paralysis, run by Mayo Clinic at 1 site 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?

The sponsor lists 8 inclusion and 22 exclusion criteria. 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: under way, not looking for participants, as of the sponsor's last update on 14 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

•Change in sensory and motor function following completion of treatment as measured by the American Spinal Injury Association (ASIA) Impairment Scale (AIS) (time frame: up to 12 months post injection). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04520373.

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 Oct 2025.

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