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MSAPhase 1/2NCT02315027

Mesenchymal Stem Cell Therapy in Multiple System Atrophy

A Phase 1/2 study testing Autologous Mesenchymal Stem Cells in people with MSA, run by Mayo Clinic at 1 site in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

The purpose of this study is to determine whether mesenchymal stem cells (MSCs) can be safely delivered to the cerebrospinal fluid (CSF) of patients with multiple system atrophy (MSA). Funding Source - FDA OOPD. Read more about this study

Who is it for?
Ages 30 to 80 years
Conditions the study is for
MSA
What is being tested?
Autologous Mesenchymal Stem Cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
1 siteUnited States
Study dates
Oct 2012to Mar 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 30 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Participants aged 30-80 years old with a diagnosis of MSA based on clinical criteria and standardized autonomic testing. This approach allows for identification of patients with MSA with very high specificity and is yet sensitive enough to allow for enrollment of patients at a disease stage at which an intervention on the natural disease course has a meaningful impact on patient outcome. Patients therefore have to fulfill Gilman Criteria (2000) for probable MSA of the parkinsonian subtype (MSA-P) or cerebellar subtype (MSA-C) and have findings on autonomic function testing suggestive of MSA (CASS ≥5 or a TST% ≥25%).
  • Participants who are less than 4 years from the time of documented MSA diagnosis.
  • Participants with an anticipated survival of at least 3 years in the opinion of the investigator.
  • Participants who are willing and able to give informed consent.
  • "Normal" cognition as assessed by Mini-Mental State Examination (MMSE). We will require a value >24.

Who cannot join

  • Any of the following conditions will exclude the participant from entering the study:
  • Women of childbearing potential who do not 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 the results of the study. These include conditions causing significant central nervous system (CNS) or autonomic dysfunction, including congestive heart failure, recent (<6 months) myocardial infarct, cardiopulmonary disease, severe, uncontrolled hypertension, thrombocytopenia (<50 x 10(9)/L), severe anemia (<8g/dl), immunocompromised state, liver or kidney disease (creatinine >2.3mg/dl), uncontrolled diabetes mellitus (HbA1c >10g%), alcoholism, amyloidosis, uncontrolled hypothyroidism, sympathectomy, unstable peripheral neuropathies, concurrent infections, orthopedic problems that compromise mobility and activity of daily living, cerebrovascular accidents, neurotoxin or neuroactive drug exposure, parkinsonism due to drugs (including neuroleptics, alpha-methyldopa, reserpine, metoclopramide).
  • Participants with malignant neoplasms.
  • Participants who have taken any investigational products within 60 days prior to baseline.
  • Medications that could affect autonomic function. If patients are taking those medications, those will be suspended prior to autonomic testing. Therapy with midodrine, anticholinergic, alpha and beta adrenergic antagonists or other medications that affect autonomic function will be withdrawn 48 hours prior to autonomic evaluations. Fludrocortisone doses up to 0.2 mg per day will be permitted.
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  • Diseases with features of Parkinsons Disease; e.g., diffuse Lewy body disease, progressive supranuclear palsy, essential tremor, hereditary olivopontocerebellar atrophy, or postencephalitic parkinsonism.
  • Dementia (DSM-IV criteria - American Psychiatric Association 1994). The score on the Mini-Mental State Examination must be >24.
  • History of electroconvulsive therapy.
  • History of brain surgery for Parkinsons disease.
  • Patients with contraindication for MRI scanning, including those with MRI-incompatible pacemakers
  • Patients with active systemic infection or local infection, which is close to the spinal injection site

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

Other trials in United States

About this study

Official title: Intrathecal Autologous Mesenchymal Stem Cell Therapy in Multiple System Atrophy (MSA) - Effect of Dose and Natural History

The purpose of this study is to determine whether mesenchymal stem cells (MSCs) can be safely delivered to the cerebrospinal fluid (CSF) of patients with multiple system atrophy (MSA). Funding Source - FDA OOPD.

Read the detailed description

The primary aim is to evaluate the safety and tolerability of intrathecal injection of autologous MSCs in a dose escalation study in patients with MSA. Safety secondary goals include to monitor changes in peripheral blood and in components of CSF, and monitor for any changes of nervous system structures using MRI. Efficacy secondary goals include evaluating potential efficacy by providing a number of studies and instruments that will detect changes in the course of the disease in terms of autonomic and neurologic symptoms and deficits.

Conditions
MSA
Being tested
Autologous Mesenchymal Stem Cells

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Autologous Mesenchymal Stem CellsBiological

    single dose of 1 × 10(7) cells intrathecally

  • Autologous Mesenchymal Stem CellsBiological

    2 doses of 5 × 10(7) cells intrathecally each 1 month (±4 days) apart

  • Autologous Mesenchymal Stem CellsBiological

    2 doses of 1 × 10(8) cells intrathecally each 1 month apart

  • Autologous Mesenchymal Stem CellsBiological

    Ten doses of 5 x 10(7) (±20%) cells intrathecally six months (±1 month) apart

  • Autologous Mesenchymal Stem CellsBiological

    Ten doses of 2.5 x 10(7) (±20%) cells intrathecally six months (±1 month) apart

Study groups in NCT02315027
Study groupRoleWhat they receive
1 dose of 1 × 10(7) MSCsExperimentalGroup 1: Participants will receive a single intrathecal dose of 1 × 10(7) mesenchymal stem cells (MSCs)
Receives: Biological: Autologous Mesenchymal Stem Cells
2 doses of 5 × 10(7) MSCsExperimentalGroup 2: Participants will receive one intrathecal dose of 5 × 10(7) mesenchymal stem cells (MSCs), followed by a second intrathecal dose of 5 × 10(7) MSCs approximately one month later
Receives: Biological: Autologous Mesenchymal Stem Cells
2 doses of 1 × 10(8) MSCsExperimentalGroup 3: Participants will receive one intrathecal dose of 1 × 10(8) mesenchymal stem cells (MSCs), followed by a second intrathecal dose of 1 × 10(8) MSCs approximately one month later
Receives: Biological: Autologous Mesenchymal Stem Cells
10 doses of 5 x 10(7) (±20%) MSCsExperimentalGroup 4: Participants will receive up to 10 doses of 5 x 10(7) (±20%) mesenchymal stem cells (MSCs) approximately 6 months apart.
Receives: Biological: Autologous Mesenchymal Stem Cells
10 doses of 2.5 x 10(7) (±20%) MSCsExperimentalGroup 5: Participants will receive up to 10 doses of 2.5 x 10(7) (±20%) mesenchymal stem cells (MSCs) approximately 6 months apart.
Receives: Biological: Autologous Mesenchymal Stem Cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse event frequency (by severity, type, attribution, and intervention dose).

    Measured: 14 months

Secondary outcomes (7)

  1. Rate of change of Unified Multiple System Atrophy Rating Scale (UMSARS) I score from baseline to 12 months (or last available date), compared with placebo limb of Rifampicin trial (historical control cohort).

    Measured: 12 months

  2. Rate of change from baseline to 12 months (or last available date) in UMSARS II score.

    Measured: 12 months

  3. Rate of change from baseline to 12 months (or last available date) in UMSARS total score.

    Measured: 12 months

  4. Rate of change in COMPASS-select score from baseline to 12 months.

    Measured: 12 months

  5. Change in CASS score and thermoregulatory sweat test (TST) % from baseline to 12 months.

    Measured: 12 months

  6. MRI morphometric changes using dedicated algorithms to evaluate rate of atrophy of defined areas of brain from baseline to 12 months.

    Measured: 12 months

  7. Change in CSF biomarkers from baseline to 2 months.

    Measured: 2 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Dec 2014 (estimated)
  2. Study startOct 2012
  3. Primary completionMar 2027 (estimated)
  4. Study completionMar 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02315027
Sponsor's protocol ID
12-005950
Other IDs
R01FD004789, R01NS092625
Status last verified
Apr 2026
First submitted
31 Oct 2014
Last updated
8 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Camilleri ET, Gustafson MP, Dudakovic A, Riester SM, Garces CG, Paradise CR, Takai H, Karperien M, Cool S, Sampen HJ, Larson AN, Qu W, Smith J, Dietz AB, van Wijnen AJ. Identification and validation of multiple cell surface markers of clinical-grade adipose-derived mesenchymal stromal cells as novel release criteria for good manufacturing practice-compliant production. Stem Cell Res Ther. 2016 Aug 11;7(1):107. doi: 10.1186/s13287-016-0370-8.

    PubMed 27515308

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Autologous Mesenchymal Stem Cells in people with MSA, run by Mayo Clinic at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse event frequency (by severity, type, attribution, and intervention dose). (time frame: 14 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02315027, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2026.

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