Using stem cells to treat multiple system atrophy

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

This study is testing whether a new stem cell treatment can help people with multiple system atrophy feel better and slow down the progression of their disease.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 76 (estimated)
• Ages: 30 Years to 70 Years
• Sex: All
• Sponsor: Mayo Clinic (Academic / other)
• Locations: 1 site (Rochester, Minnesota)
• Trial ID: NCT05167721 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and effectiveness of intrathecally administered autologous mesenchymal stem cells in patients diagnosed with multiple system atrophy (MSA). The study employs a randomized, double-blind, placebo-controlled adaptive design to determine optimal dosing frequency and assess the impact on disease progression. Participants will receive either the stem cell treatment or a placebo, with outcomes measured using the Unified MSA Rating Scale. The trial builds on previous findings that suggest potential benefits of stem cell therapy in slowing disease progression.

Who should consider this trial

Good fit: Ideal candidates for this study are males and females aged 30-70 with a clinical diagnosis of MSA who can walk unaided and have normal cognitive function.

Not a fit: Patients who are pregnant, breastfeeding, or have cognitive impairments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could slow or halt the progression of multiple system atrophy, significantly improving patient quality of life.

How similar studies have performed: Previous studies have shown promising results with similar approaches using mesenchymal stem cells in neurodegenerative diseases, indicating potential for success in this trial.

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.

Where this trial is running

Rochester, Minnesota

• Mayo Clinic — Rochester, Minnesota, United States (Recruiting)

Study contacts

• Principal investigator: Wolfgang Singer, MD — Mayo Clinic
• Study coordinator: Tonette Gehrking

Conditions Multiple System Atrophy; mesenchymal stem cells; treatment trial