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

Who is this study for? Patients with Multiple System Atrophy
What treatments are being studied? Autologous Mesenchymal Stem Cells
Status: Active, not recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 30
Maximum Age: 80
Healthy Volunteers: No
View:

• 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.

Locations
United States
Minnesota
Mayo Clinic in Rochester
Rochester
Time Frame
Start Date: October 2012
Estimated Completion Date: March 2024
Participants
Target number of participants: 30
Treatments
Experimental: 1 dose of 1 × 10(7) MSCs
Group 1: Participants will receive a single intrathecal dose of 1 × 10(7) mesenchymal stem cells (MSCs)
Experimental: 2 doses of 5 × 10(7) MSCs
Group 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
Experimental: 2 doses of 1 × 10(8) MSCs
Group 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
Experimental: 10 doses of 5 x 10(7) (±20%) MSCs
Group 4: Participants will receive up to 10 doses of 5 x 10(7) (±20%) mesenchymal stem cells (MSCs) approximately 6 months apart.
Experimental: 10 doses of 2.5 x 10(7) (±20%) MSCs
Group 5: Participants will receive up to 10 doses of 2.5 x 10(7) (±20%) mesenchymal stem cells (MSCs) approximately 6 months apart.
Sponsors
Leads: Mayo Clinic
Collaborators: National Institute of Neurological Disorders and Stroke (NINDS), Food and Drug Administration (FDA)

This content was sourced from clinicaltrials.gov

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