Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy

Trial ID: NCT05167721

Interventional - Phase 2 - Active, not recruiting

Multiple system atrophy (MSA) is a rare, rapidly progressive, and invariably fatal neurological condition characterized by autonomic failure, parkinsonism, and/or ataxia. There is no available treatment to slow or halt disease progression. The purpose of this study is to assess optimal dosing frequency, effectiveness and safety of adipose-derived autologous mesenchymal stem cells delivered into the spinal fluid of patients with MSA. Funding source: FDA Office of Orphan Product Development (OOPD), Mayo Clinic Executive Dean for Research Transformational Award, Mayo Clinic Regenerative Medicine, and Mayo Clinic Department of Neurology.

Conditions

• Multiple System Atrophy

Interventions

• Autologous Mesenchymal Stem Cells
• Placebo

Eligibility

Inclusion criteria

• 1. Males or females aged 30-70 years, who are willing and able to give informed consent.
• 2. Clinical diagnosis of MSA, fulfilling consensus criteria for probable MSA.
• 3. UMSARS I (omitting question 11) between 5 and 17, and able to walk unaided (i.e. able to walk at least 50 yards without the use of a cane or walker, and without other support such as holding on to an arm or touching walls).
• 4. Anticipated survival of at least 3 years in the opinion of the investigator.
• 5. Normal cognition as assessed by the Montreal Cognitive Assessment (MOCA). We will require a value ≥26.

Exclusion criteria

• 1. Pregnant or breastfeeding women, and women of childbearing potential who do not agree to practice an acceptable method of birth control. Acceptable methods of birth control in this study are: surgical sterilization, intrauterine devices, partner's vasectomy, a double-protection method (condom or diaphragm with spermicide), hormonal contraceptive drug (i.e., oral contraceptive, contraceptive patch, long-acting injectable contraceptive) with a required second mode of contraception.
• 2. Participants with a clinically significant or unstable medical or surgical condition that, in the opinion of the investigator, might preclude safe completion of the study or might affect study results. These include conditions causing significant CNS or autonomic dysfunction, clinically significant peripheral neuropathy, active malignant neoplasm, amyloidosis, active autoimmune disease, immunocompromised state, active infection, congestive heart failure (NYHA III or IV), recent (\<6 months) myocardial infarction, history of stoke with residual deficits, uncontrolled diabetes mellitus, alcoholism, orthopedic problems that compromise mobility and activity of daily living, significant liver or kidney disease, thrombocytopenia (\<50 x 109/L), disorders affecting coagulation, and patients on active anticoagulation.
• 3. Participants who have taken any investigational products within 90 days prior to baseline, or with expected effects lasting beyond 60 days prior to baseline.
• 4. Medications that could affect clinical evaluations are permitted but need to be withdrawn at least four half-lives prior to study visits. Those include medications used to treat motor symptoms, such as levodopa and other anti-Parkinsonian medications.
• 5. Patients with contraindication to any of the study procedures, in particular MRI scanning.

Sponsor

Mayo Clinic

Source: ClinicalTrials.gov

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