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Evaluate the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells in Patients With Multiple Sclerosis (UMSC01)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05532943
Recruitment Status : Not yet recruiting
First Posted : September 8, 2022
Last Update Posted : September 8, 2022
Sponsor:
Information provided by (Responsible Party):
Ever Supreme Bio Technology Co., Ltd.

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. We hypothesize that 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.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Biological: Allogeneic umbilical cord mesenchymal stem cells Biological: Control group Phase 1 Phase 2

Detailed Description:
There is single arm in Phase I part: 6 patients will be enrolled sequentially for safety considerations. The patient will receive UMSC01 via IV followed by IT at day 28 as described in above. After all patients in Phase I complete the safety assessment by SMC without any major safety issue 4 weeks after the last UMSC01 administration, the Phase IIa part will be initiated. There are 2 arms in Phase IIa part: Sham-controlled with conventional treatment control and administration of UMSC01 with conventional treatment.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Seamless Phase I/IIa Clinical Study to Evaluate the Safety and Efficacy of Allogeneic Umbilical Cord Mesenchymal Stem Cells in Patients With Multiple Sclerosis
Estimated Study Start Date : December 31, 2022
Estimated Primary Completion Date : December 31, 2026
Estimated Study Completion Date : December 31, 2026

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: UMSC01
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
UMSC01 cells will be IV infusion followed by IT infusion with 12 months of follow up after treatment.

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




Primary Outcome Measures :
  1. Primary Endpoint for Phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    SAE, SUSAR, and AE incidences over the study period

  2. Primary Endpoint for Phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of EDSS to Visit 10


Secondary Outcome Measures :
  1. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB for EDSS of follow up visits (Visit 6-10)

  2. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB for brain MRI parameters of follow-up visits (Visit 6 -10)

  3. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    Quality of life: CFB for MSQoL-54 questionnaire score of follow-up visits (Visit 6 -10)

  4. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of T25FW scores of follow-up visits (Visit 6-10)

  5. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of 9-HPT scores of follow-up visits (Visit 6-10)

  6. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of PASAT scores of follow-up visits (Visit 6-10)

  7. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of SDMT scores of follow-up visits (Visit 6-10)

  8. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of RNFL thickness, measured by OCT of follow-up visits (Visit 6-10)

  9. Efficacy endpoint for phase I portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of MSFC of follow-up visits (Visit 6-10)

  10. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 6-month follow-up period ]
    Time to onset of CDW confirmed by EDSS at least 6 months

  11. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    ARR (Annualized relapse rate), where relapse is defined as new or worsening neurological symptoms lasting for >24 hours

  12. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow-up visits (Visit 6 -10) for EDSS

  13. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for brain MRI parameters

  14. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    Quality of life: CFB of follow up visits (Visit 6-10) for MSQoL-54 questionnaire score

  15. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for T25FW scores

  16. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for 9-HPT scores

  17. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for PASAT scores

  18. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for SDMT scores

  19. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for RNFL thickness, measured by OCT

  20. Efficacy endpoints for phase IIa portion [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of follow up visits (Visit 6-10) for MSFC

  21. The safety endpoints are listed below for both phase I and IIa portions [ Time Frame: from visit 2 to 12-month follow-up period ]
    SAE, SUSAR, and AE incidences over the study period

  22. The safety endpoints are listed below for both phase I and IIa portions [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of laboratory data to subsequent visits

  23. The safety endpoints are listed below for both phase I and IIa portions [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of physical examination to subsequent visits

  24. The safety endpoints are listed below for both phase I and IIa portions [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of vital signs to subsequent visits

  25. The safety endpoints are listed below for both phase I and IIa portions [ Time Frame: from visit 2 to 12-month follow-up period ]
    CFB of AFP, CEA, CA199, SCC, IgA, anti-EBV, β-HCG, CA125, CA153, and PSA to Visit 6 (Phase I) or Visit 10 (Phase IIa)


Other Outcome Measures:
  1. The exploratory endpoints are listed below for both phase I and IIa portions [ Time Frame: from visit 2 to 12-month follow-up period ]
    Immunological markers, including CD3, CD4, CD8 surface markers, IgG, IgM, anti-HLA antibodies and Panel Reactive Antibody Assay in whole blood



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   20 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients are willing to sign informed consent.
  2. Male or female are age between 20 to 65 years old on date of consent.
  3. 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)
  4. MS diagnosis established between 2 to 15 years and EDSS score between 2.0 to 6.5 before enrollment
  5. Patient has appropriated blood clotting function as assessed by the following laboratory requirements: PT, APTT ≤ 1.5X upper limit of normal (ULN).
  6. 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
  7. 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

Exclusion Criteria:

  1. 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.
  2. Patients with uncontrolled diabetes (fasting blood glucose > 250 mg/dL)
  3. Patients with inadequate hepatic and renal function: AST and ALT > 5X ULN; eGFR < 30 mL/min.
  4. Patients who are unable to undergo Brain MRI examination for any reason.
  5. 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.
  6. 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.
  7. With active infection that required systemic treatment
  8. Patients who are participating in other clinical trials with an investigational product within 1 month.
  9. Patients who were treated with cytotoxic medications during the last 1 month prior to the infusion.
  10. Relapse of MS within1 month before UMSC01 infusion.
  11. With anti-CD20 therapy, such as rituximab
  12. Patients not suitable to participate the trial as judged by the Investigator(s)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05532943


Contacts
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Contact: Sammi Hsu 886-4-2325-288 ext 507 cthsu@ever-supreme.com.tw
Contact: Woei C Shyu shyu9423@gmail.com

Locations
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Taiwan
China Medical University Hospital
Taichung, Non-US, Taiwan, 404
Contact: Sammi Hsu       cthsu@ever-supreme.com.tw   
Principal Investigator: Yuh C Guo, MD         
Sponsors and Collaborators
Ever Supreme Bio Technology Co., Ltd.
Investigators
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Study Chair: Woei C Shyu Ever Supreme Bio Technology Co., Ltd.
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Responsible Party: Ever Supreme Bio Technology Co., Ltd.
ClinicalTrials.gov Identifier: NCT05532943    
Other Study ID Numbers: ES-CMSC01-D1101
First Posted: September 8, 2022    Key Record Dates
Last Update Posted: September 8, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Multiple Sclerosis
Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases