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

ImStem Biotechnology

Status and phase: Enrolling; Phase 1

Conditions: Multiple Sclerosis

Treatments: Biological: IMS001

Study type: Interventional

Funder types: Industry

Identifiers

NCT04956744

IMS001-01

Details and patient eligibility

About

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.

Enrollment: 30 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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30 participants in 3 patient groups

Low Dose

Experimental group

Description:

Low dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1.

Treatment:

Biological: IMS001

High Dose

Experimental group

Description:

High dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1.

Treatment:

Biological: IMS001

Optional Dose

Experimental group

Description:

High dose of cells/kg of intravenous (IV) IMS001 as a single dose infusion on Day 1 and at Month 6.

Treatment:

Biological: IMS001