A Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of IMS001 in Subjects With Multiple Sclerosis

Study Overview
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Brief Summary
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This is a Phase 1 study of IMS001, given as a single dose to subjects with Multiple Sclerosis who experience inadequate response and/or intolerability to disease modifying treatments. IMS001 is a human embryonic cell derived (hESC) mesenchymal stem cell (MSC). MSCs have the potential to modulate disease course.

Official Title
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A Phase 1, Dose-Escalating, Open-label Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Single Dose of IV IMS001 in Subjects With Multiple Sclerosis and Treatment Failure to Prior Disease Modifying Treatments (DMTs)

Conditions
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Multiple Sclerosis

Intervention / Treatment
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* Biological: IMS001


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Provides signed and dated informed consent in accordance with local regulations. 18 to 65 years of age. Diagnosis of MS. Has had an inadequate response DMTs. EDSS within protocol parameters. Able and willing to undergo MRIs. Must be clinically stable for 1 month prior to Day 1. Exclusion Criteria: Has a known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001. Has history of excluded medications, per protocol, prior to Day 1. Has a history of neoplastic disease except for basal cell carcinoma, non-metastatic squamous cell carcinoma of the skin that has been excised with clean margins, or adequately treated in-situ carcinoma of the cervix. Prior history of any other autoimmune disease, myelodysplasia, or hematologic disease. Prior treatment with any allogeneic cell therapy or tissue transplant. Prior history of smoking equivalent to ˃20 cumulative pack years of cigarettes. Recent clinically significant infection during the Screening Phase. Has any medical or psychiatric condition that would impact outcome or participation in the study. Has clinically significant abnormal findings on the electrocardiogram (ECG) during the Screening Phase. Has known or documented human immunodeficiency virus (HIV) infection or active Hepatitis B, or C. Has an elevated liver function test abnormality during the Screening Phase. Has abnormalities of blood count during the Screening Phase. Has laboratory abnormalities of renal function during the Screening Phase. Has other clinically significant laboratory abnormalities during Screening Phase. Body weight ≥120 kg. Women pregnant, breast feeding, or planning to become pregnant during the study. Is unavailable for the duration of the trial, is likely to be noncompliant with the protocol, or is generally felt to be unsuitable by the principal investigator. Current or recent participation in any other clinical or device trials within 3 months prior to Day 1.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 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: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Low Dose<br>Low dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1. | Biological: IMS001<br>* IMS001 is a human embryonic cell derived (hESC) mesenchymal stem cell (MSC).<br>|
| Experimental: High Dose<br>High dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1. | Biological: IMS001<br>* IMS001 is a human embryonic cell derived (hESC) mesenchymal stem cell (MSC).<br>|
| Experimental: Optional Dose<br>High dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1 and at Month 6. | Biological: IMS001<br>* IMS001 is a human embryonic cell derived (hESC) mesenchymal stem cell (MSC).<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability | Frequency of treatment-emergent adverse events (TEAEs). | Day 1 to Month 60 | 
| Safety and tolerability | Clinically significant laboratory abnormalities. | Day 1 to Month 60 |