Study to Evaluate Investigational Allogeneic Cell Therapy Product hOMSC300 for Treatment of Early- to Moderate Stage MSA

Study Overview
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Brief Summary
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Purpose of this phase 1/2a study is to assess the safety and efficacy of intrathecal administration of allogeneic human oral mucosa stem cells (hOMSCs) in patients suffering from early to moderate stage Multiple System Atrophy (MSA) .

Official Title
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Phase 1/2a Randomized Controlled Study for Treatment of Early- to Moderate Stage Multiple System Atrophy Patients With the Investigational Allogeneic Cell Therapy Product, hOMSC300

Conditions
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Multiple System Atrophy, MSA - Multiple System Atrophy

Intervention / Treatment
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* Drug: hOMSC300
* Other: Sham Procedure


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient was diagnosed with probable MSA-C (cerebellar) or MSA-P (parkinsonian) variant within 60 months of symptom onset (excluding impotence) Subject can ambulate without the assistance of another person, defined as the ability to take at least 10 steps. Use of assistive devices is allowed Patient cognitive state permits him to sign informed consent, according to the PI's clinical judgement, and MoCA >= 24 Exclusion Criteria: Pregnant women and women before menopause Participants with a clinically significant or unstable medical or surgical condition that, in the opinion of the investigator, might preclude safe completion of the treatment or affect the treatment outcome. Patients with thrombocytopenia, other bleeding diathesis or taking anticoagulant therapy (not including Aspirin up to 100mg per day) Patients with known hypersensitivities to Plasmalyte, Gadolinium, Penicillin, and with general hypersensitivity to antibiotics Patients who fulfill the criteria of Parkinson's Disease History of electroconvulsive therapy History of brain surgery

Ages Eligible for Study
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Minimum Age: 50 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Patients will be allocated to one of the following arms: Low dose single intrathecal (IT) administration of hOMSC300 High dose single intrathecal (IT) administration of hOMSC300 Sham procedure (control)
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Low dose hOMSC300<br>Single IT administration of low dose hOMSC300 | Drug: hOMSC300<br>* Human Oral Mucosa Stem Cells<br>|
| Active Comparator: High dose hOMSC300<br>Single IT administration of high dose hOMSC300 | Drug: hOMSC300<br>* Human Oral Mucosa Stem Cells<br>|
| Other: Sham Procedure<br>Lumbar puncture only | Other: Sham Procedure<br>* Lumbar Puncture Only<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment related SAEs per dose group |  | 18 months | 
| Treatment related adverse events per dose group |  | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Clinically significant motor and behavioral change from baseline |  | 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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multiple system atrophy, MSA, stem cells, intrathecal