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

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)

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.

Study Overview

• Status: Recruiting
• Conditions: Multiple Sclerosis
• Intervention / Treatment: Biological: IMS001

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Richard Kim, MD; Phone Number: 860-281-7836; Email: richard.kim@imstem.com

Study Locations

• United States
  • Georgia
    • Atlanta, Georgia, United States, 30309
      • Recruiting
      • Shepherd Center
  • Massachusetts
    • Worcester, Massachusetts, United States, 01605
      • Recruiting
      • UMASS Memorial Medical Center
  • Utah
    • Salt Lake City, Utah, United States, 84103
      • Recruiting
      • Rocky Mountain MS Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 63 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: ImStem Biotechnology
• Collaborators: Rho, Inc.
• Investigators: Study Director: Richard Kim, MD, ImStem Biotechnology

Publications and helpful links

Helpful Links

• Imstem Biotechnology

Study record dates

Study Major Dates

• Study Start (Actual): August 31, 2021
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: June 22, 2021
• First Submitted That Met QC Criteria: July 8, 2021
• First Posted (Actual): July 9, 2021

Study Record Updates

• Last Update Posted (Actual): September 28, 2023
• Last Update Submitted That Met QC Criteria: September 26, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Multiple Sclerosis; Sclerosis
• Other Study ID Numbers: IMS001-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No