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

NCT05167721 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 21-005569FD-R-07290-01
• Study Type: interventional
• Target: 71
• Locations: 1 country
• Sites: 1

Brief Summary

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

Keywords

mesenchymal stem cells; treatment trial

Outcome Measures

Primary Outcomes (1)

• Change in UMSARS total (= UMSARS I + UMSARS II) score

  Rate of disease progression assessed using the change in the UMSARS total (= UMSARS I + UMSARS II) score

  12 months

Secondary Outcomes (6)

• Change in UMSARS II score

  12 months

• Change in UMSARS I score

  12 months

• Change in modified UMSARS scale

  12 months

• Change in NfL

  9 - 12 months

• Rate of atrophy of selected brain regions

  12 months

Study Arms (3)

Arm 1

ACTIVE COMPARATOR

25 million mesenchymal stem cells administered intrathecally every 3 months for 4 injections

Biological: Autologous Mesenchymal Stem Cells

Arm 2

ACTIVE COMPARATOR

25 million mesenchymal stem cells administered intrathecally every 6 months for 2 injections (placebo injections at 3 month and 9 month timepoints)

Biological: Autologous Mesenchymal Stem Cells; Other: Placebo

Arm 3

PLACEBO COMPARATOR

Placebo (lactated Ringer's) administered intrathecally every 3 months for 4 injections

Other: Placebo

Interventions

Autologous Mesenchymal Stem Cells BIOLOGICAL

Autologous Mesenchymal Stem Cells administered intrathecally

Arm 1; Arm 2

Placebo OTHER

Placebo administered intrathecally

Arm 2; Arm 3

Eligibility Criteria

• Age: 30 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females aged 30-70 years, who are willing and able to give informed consent.
• Clinical diagnosis of MSA, fulfilling consensus criteria for probable MSA.
• 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).
• Anticipated survival of at least 3 years in the opinion of the investigator.
• Normal cognition as assessed by the Montreal Cognitive Assessment (MOCA). We will require a value ≥26.

You may not qualify if:

• 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.
• 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.
• Participants who have taken any investigational products within 90 days prior to baseline, or with expected effects lasting beyond 60 days prior to baseline.
• 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.
• Patients with contraindication to any of the study procedures, in particular MRI scanning.

Sponsors & Collaborators

• Mayo Clinic: lead

Study Sites (1)

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Multiple System Atrophy

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Wolfgang Singer, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: Randomized Double-Blind Placebo-Controlled Adaptive Design Trial

Study Record Dates

• First Submitted: November 30, 2021
• First Posted: December 22, 2021
• Study Start: December 15, 2021
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: May 6, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)