Clinical trial NCT02034188: Feasibility Study of Human Umbilical Cord Tissue-Derived Mesenchymal Stem Cells in Patients With Multiple Sclerosis
Official title: Feasibility Study of Human Umbilical Cord Tissue-Derived Mesenchymal Stem Cells in Patients With Multiple Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Multiple Sclerosis
Keywords: multiple sclerosis
umbilical cord
mesenchymal
stem cells
Lead sponsor: Translational Biosciences
Sponsor class: INDUSTRY
First posted: 2014-01-13
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Allogeneic human umbilical cord tissue-derived stem cells injected intravenously (IV) once per day for 7 days is a safe and will induce a therapeutic effect in multiple sclerosis (MS) patients.

Detailed description: The proposed study will assess primarily safety and secondary efficacy endpoints of allogeneic umbilical cord mesenchymal stem cells (UC-MSC) administered to 20 patients with MS.

The primary objective of the trial is freedom from treatment associated adverse events at 4,12 and 52 weeks post treatment. Secondary objective will be efficacy as assessed at baseline, week 12 and 52 and will be quantified based on the following: Neurological assessment of the MS functional composite assessment which comprises of Expanded Disability Status Scale (EDSS), the expanded EDSS (Rating Neurologic Impairment in Multiple Sclerosis, the Scripps neurological rating scale (NRS), paced auditory serial addition test (PASAT), the nine-hole peg test, and 25-foot walking time. Short-form 36 (SF-36) quality of life questionnaire and gadolinium enhanced MRI scans of the brain and cervical spinal cord will also be performed at the indicated time points.

Eligibility criteria: Inclusion Criteria:

* Patients willing to sign informed consent and capable of understanding the features of this clinical trial.
* Willing to keep a weekly diary and undergo observation for 12 months
* Non-pregnant patients 18-55 years of age with MS according to the revised McDonald criteria and meeting the Poser criteria for clinically defined MS.
* EDSS scores of 2·0 to 5·5 points assessed at least 3 months after the last acute attack of MS.
* Must have proof of health insurance in country of residence.

Exclusion Criteria:

* Patients with evidence of active proliferative retinopathy.
* Patients with poorly controlled diabetes mellitus (glycated hemoglobin: HbA1C \> 8.5%).
* Patients with renal insufficiency (Creatinine\> 2.5) or failure.
* Infection as evidenced by white blood cell (WBC) count of \>15,000 k/cumm and/or temperature \> 38 Celsius.
* History of organ transplant.
* History of previous or active malignancy, except for localised cutaneous basal or squamous cell carcinoma or carcinoma in situ of the cervix
* Exercise limiting angina ( Canadian Cardiovascular Society Class 3
* Congestive heart failure (New York Heart Association class 3
* Unstable angina
* Acute ST elevation myocardial infarction (MI) within 1month
* Transient ischemic heart attack or stroke within 1 month
* Severe valvular heart disease

Primary outcomes:
measure: Number of participants with adverse events
timeFrame: 12 months
Secondary outcomes:
measure: Number of participants with a change in disability as measured by Expanded Disability Status Scale (EDSS)
timeFrame: 12 months
measure: Number of participants with a change in neurological impairment as measured by Scripps Neurological Rating Scale
timeFrame: 12 months
measure: Number of participants with a change in cognitive function as measured by the • Paced Auditory Serial Addition Test (PASAT)
timeFrame: 12 months
measure: Number of participants with a change in upper extremity function as measured by the Nine Hole Peg Test
timeFrame: 12 months
measure: Number of participants with a change in mobility and leg function as measured by the 25 foot walking test
timeFrame: 12 months
measure: Number of participants with a change in quality of life as measured by the Short form 36 (SF-36) quality of life questionnaire
timeFrame: 12 months
measure: Number of participants experiencing pulmonary edema as measured by 12-lead electrocardiogram (ECG)
timeFrame: 1 month, 3 months
measure: Number of participants with a change in brain or spinal cord lesions as measured by gadolinium-enhanced magnetic resonance imaging (MRI)
timeFrame: 12 months
Other outcomes:
Locations:
city: Panama City
country: Panama
facility: Stem Cell Institute
Study officials:
affiliation: Translational Biosciences / Stem Cell Institute
name: Jorge Paz-Rodriguez, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Riordan NH, Morales I, Fernandez G, Allen N, Fearnot NE, Leckrone ME, Markovich DJ, Mansfield D, Avila D, Patel AN, Kesari S, Paz Rodriguez J. Clinical feasibility of umbilical cord tissue-derived mesenchymal stem cells in the treatment of multiple sclerosis. J Transl Med. 2018 Mar 9;16(1):57. doi: 10.1186/s12967-018-1433-7.