Clinical trial NCT04520373: Autologous Adipose Derived Mesenchymal Stem Cells for Spinal Cord Injury Patients
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Spinal Cord Injuries
Paralysis
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
First posted: 2020-08-20
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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
* 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)

Primary outcomes:
measure: •Change in sensory and motor function following completion of treatment as measured by the American Spinal Injury Association (ASIA) Impairment Scale (AIS)
timeFrame: up to 12 months post injection
Secondary outcomes:
measure: Change in sensory and motor function following completion of treatment as measured by Somatosensory Evoked Potentials (SSEPs)
timeFrame: baseline, up to 12 months post injection
measure: change in Neurogenic Bladder Symptom Score (NBSS)
timeFrame: up to 24 months post injection
measure: change in Neurogenic Bowel Symptom Score
timeFrame: up to 24 months post injection
measure: Incidence of abnormal CSF composition
timeFrame: up to 4 weeks post injection
measure: Number of subjects who develop a new pathologic mass at the spinal cord area of injection or anywhere along the spinal cord.
timeFrame: up to 12 months post injection
measure: Correlation of adverse events to study drug
timeFrame: up to 4 weeks post injection
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic in Rochester
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Mohamad Bydon, MD
role: PRINCIPAL_INVESTIGATOR
References: