Could stem cells slow a fatal brain disease?
NCT ID NCT05167721
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 2 trial tests whether a patient's own stem cells, injected into the spinal fluid, can slow the progression of multiple system atrophy (MSA), a rare and fatal brain disease. The study involves 71 adults aged 30-70 with early-stage MSA. Researchers will measure changes in symptoms over time to see if the treatment is safe and effective.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- autologous mesenchymal stem cells (from the patient's own fat tissue)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops the progression of multiple system atrophy, a currently untreatable fatal disease.
- What could go wrong
- This is an early Phase 2 trial with only 71 participants, so results may not apply to all patients. The treatment involves spinal injections, which carry risks like infection or nerve damage, and it may not slow the disease at all.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
71 people
The number who actually took part.
- Started
-
Dec 2021
- Expected to finish
-
Dec 2026
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
30 to 70 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Mayo Clinic
Rochester, Minnesota, 55905, United States
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