Evaluate the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells in Patients With Multiple Sclerosis

NCT05532943 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ES-CMSC01-D1101
• Study Type: interventional
• Target: 41
• Locations: 1 country
• Sites: 1

Brief Summary

This study is to identify the safety and efficacy of repeat IV(Intravenous) and IT(Intrathecal) administrations of UMSC01 in patients with MS. While anti-inflammatory drugs are routinely used for the treatment of MS by inhibiting immune responses, their effects on axon remyelination or neuroregeneration are limited. The combined systemic delivery of UCMSCs via intravenous injection and local administration of the cells by IT was to have safety and therapeutic efficacy for patients with MS.

Conditions

Multiple Sclerosis

Outcome Measures

Primary Outcomes (2)

• Primary Endpoint for Phase I portion

  SAE, SUSAR, and AE incidences over the study period

  from visit 2 to 12-month follow-up period

• Primary Endpoint for Phase IIa portion

  CFB of EDSS to Visit 10

  from visit 2 to 12-month follow-up period

Secondary Outcomes (25)

• Efficacy endpoint for phase I portion

  from visit 2 to 12-month follow-up period

• Efficacy endpoint for phase I portion

  from visit 2 to 12-month follow-up period

• Efficacy endpoint for phase I portion

  from visit 2 to 12-month follow-up period

• Efficacy endpoint for phase I portion

  from visit 2 to 12-month follow-up period

• Efficacy endpoint for phase I portion

  from visit 2 to 12-month follow-up period

Other Outcomes (1)

• The exploratory endpoints are listed below for both phase I and IIa portions

  from visit 2 to 12-month follow-up period

Study Arms (2)

UMSC01

EXPERIMENTAL

UMSC01 cells mixed with normal saline will be administered to patients after the onset of diagnosis of multiple sclerosis.

Biological: Allogeneic umbilical cord mesenchymal stem cells

Placebo

SHAM COMPARATOR

For IV administration, normal saline will be administered to patients after the onset of diagnosis of multiple sclerosis. For IT administration, sham puncture procedure will partially penetrate without reaching subarachnoid space, and no spinal fluid will be drawn.

Biological: Control group

Interventions

Allogeneic umbilical cord mesenchymal stem cells BIOLOGICAL

UMSC01 cells will be IV infusion followed by IT infusion with 12 months of follow up after treatment.

UMSC01

Control group BIOLOGICAL

Normal saline will be IV infusion followed by sham-IT infusion with 12 months of follow up after treatment.

Placebo

Eligibility Criteria

• Age: 20 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients are willing to sign informed consent.
• Male or female are age between 20 to 65 years old on date of consent.
• Diagnosis of Relapsing-Remitting MS (RRMS) (≥1 clinically documented relapse in the past 12 months, ≥2 clinically documented relapses in the last 24 months or ≥ 1 gadolinium enhanced lesion or T2 new lesion in the last 12 months) or Secondary Progressive MS (SPMS) (EDSS increase ≥1.0 point (baseline EDSS ≤ 5.0) or ≥ 0.5 point (baseline EDSS ≥5.5), and ≥1 clinical relapse or ≥1 gadolinium enhanced lesion in the last 12 months)
• MS diagnosis established between 2 to 15 years and EDSS score between 2.0 to 6.5 before enrollment
• Patient has appropriated blood clotting function as assessed by the following laboratory requirements: PT, APTT ≤ 1.5X upper limit of normal (ULN).
• Treatment failure (either ≥ 1 relapse, ≥ 1 new T2 lesion, ≥ one gadolinium enhanced lesion or EDSS deterioration) with at least one of MS disease modifying therapy as Interferon-β, Glatiramer acetate (Copaxone), Dimethyl fumarate (Tecfidera), Teriflunomide (Aubagio), Fingolimod (Gilenya), Ozanimod (Zeposia), Cladribine (Mavenclad), Siponimod (Mayzent), Ofatumumab (Kesimpta), or Natalizumab (Tysabri) for more than 6 months
• All male patients and female patients with child-bearing potential (between puberty and 2 years after menopause) should use appropriate contraception method(s) for at least 4 weeks after UMSC01 treatment

You may not qualify if:

• Pregnancy, lactation, and those who are not pregnant but did not, or unwilling to, take effective contraceptives measures 4 weeks before and after the treatment.
• Patients with uncontrolled diabetes (fasting blood glucose \> 250 mg/dL)
• Patients with inadequate hepatic and renal function: AST and ALT \> 5X ULN; eGFR \< 30 mL/min.
• Patients who are unable to undergo Brain MRI examination for any reason.
• Patients who have medical history or current clinically active malignant tumor, peripheral neuropathy, myopathy or other clinically significant neurological diseases that will confound the evaluation of this study.
• Patients who have immuno-compromised condition or is with known clinically significantly autoimmune conditions other than MS or is receiving immunosuppressive treatments other than MS treatment within 6 months.
• With active infection that required systemic treatment
• Patients who are participating in other clinical trials with an investigational product within 1 month.
• Patients who were treated with cytotoxic medications during the last 1 month prior to the infusion.
• Relapse of MS within1 month before UMSC01 infusion.
• With anti-CD20 therapy, such as rituximab
• Patients not suitable to participate the trial as judged by the Investigator(s)

Sponsors & Collaborators

• Ever Supreme Bio Technology Co., Ltd.: lead

Study Sites (1)

China Medical University Hospital

Taichung, Non-US, 404, Taiwan

RECRUITING

MeSH Terms

Conditions

Multiple Sclerosis

Interventions

Control Groups

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Research Design; Epidemiologic Methods; Investigative Techniques; Research Design; Methods

Study Officials

• Woei C Shyu

  Ever Supreme Bio Technology Co., Ltd.

  STUDY CHAIR

Central Study Contacts

Sammi Hsu

CONTACT

886-4-2325-288; cthsu@ever-supreme.com.tw

Jack Tsai

CONTACT

886-4-2325-288; cktsai@ever-supreme.com.tw

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 5, 2022
• First Posted: September 8, 2022
• Study Start: September 8, 2023
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2028
• Last Updated: August 7, 2025

Record last verified: 2025-08

Locations

TW (1)