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

Launched by MAYO CLINIC · Dec 9, 2021

Keywords

ClinConnect Summary

This clinical trial is investigating a potential new treatment for Multiple System Atrophy (MSA), a rare and serious neurological condition that currently has no available treatments to slow its progression. The study aims to test the safety and effectiveness of using a patient’s own fat-derived stem cells, which will be delivered into the spinal fluid. Researchers will also look at how often these stem cells should be given to achieve the best results.

To participate in this trial, individuals need to be between 30 and 70 years old and have a confirmed diagnosis of MSA. They should be able to walk at least 50 yards without assistance and have normal cognitive function. Participants can expect to undergo various assessments and treatments throughout the study, and their safety will be closely monitored. It’s important to note that certain medical conditions and medications may exclude someone from participating, so potential volunteers should discuss their eligibility with their healthcare provider.

Gender

ALL

Eligibility criteria

• 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.