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

Ever Supreme Bio Technology

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Multiple Sclerosis

Treatments: Biological: Allogeneic umbilical cord mesenchymal stem cells; Biological: Control group

Study type: Interventional

Funder types: Industry

Identifiers

NCT05532943

ES-CMSC01-D1101

Details and patient eligibility

About

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.

Full 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.

Enrollment: 41 estimated patients

Sex: All

Ages: 20 to 65 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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41 participants in 2 patient groups

UMSC01

Experimental group

Description:

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

Treatment:

Biological: Allogeneic umbilical cord mesenchymal stem cells

Placebo

Sham Comparator group

Description:

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.

Treatment:

Biological: Control group