NCT06961383

Brief Summary

The goal of this clinical trial is to assess the safety and efficacy of repeated intrathecal (IT) injection of NG01, autologous bone marrow derived human stromal cells, in treating Secondary Progressive Multiple Sclerosis (SPMS), compared to placebo. The study will assess the proportion of participants demonstrating improvement in walking ability, defined as a reduction in the average time to complete the Timed 25-Foot Walk (T25FW) at 6, 9, and 12 months compared to baseline. This will be analyzed by the mean change in walking speed across these time points. The study will also evaluate the incidence and nature of treatment-emergent adverse events (AEs). Participants will receive intrathecal administrations of NG01, by lumbar puncture, and will be followed up for 6 months after their fourth administration.

Trial Health

67
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
45

participants targeted

Target at P25-P50 for phase_2

Timeline
22mo left

Started Oct 2025

Geographic Reach
2 countries

2 active sites

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress25%
Oct 2025Mar 2028

First Submitted

Initial submission to the registry

April 21, 2025

Completed
16 days until next milestone

First Posted

Study publicly available on registry

May 7, 2025

Completed
5 months until next milestone

Study Start

First participant enrolled

October 1, 2025

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2027

Expected
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2028

Last Updated

May 7, 2025

Status Verified

April 1, 2025

Enrollment Period

2 years

First QC Date

April 21, 2025

Last Update Submit

April 28, 2025

Conditions

Secondary Progressive Multiple Sclerosis (SPMS)

Keywords

Secondary progressive multiple sclerosisMultiple sclerosisAutologous cell therapy

Outcome Measures

Primary Outcomes (2)

  • Walking Ability

    Proportion of participants who achieved improvement in walking ability over baseline by mean change in walking speed based on the Timed 25-Foot Walk (T25FW) test, averaged over visits at month-6, month-9 and month-12. Baseline is defined as the change in average timed walk compared to baseline (average timed walk at 6-, 9-, 12-months minus baseline \< 0)

    12 months

  • Incidence of Treatment-Emergent Adverse Events (AEs)

    The occurrence of treatment-related AEs will be assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5 following enrollment, NG01 or placebo administrations (intrathecal), and during the 6 months of follow-up.

    15 months

Secondary Outcomes (10)

  • Walking Speed

    12 months

  • Neuroimaging Parameters - Change in T2-hyperintense Lesion

    12 months

  • Neuroimaging Parameters - Change in T1-hypointense Lesion

    12 months

  • Neuroimaging Parameters - Change in Brain and Thalamus

    12 months

  • Efficacy - Finger Dexterity

    12 months

  • +5 more secondary outcomes

Study Arms (3)

100x10^6 cells

EXPERIMENTAL

15 participants with SPMS will receive 4 IT administrations of NG01 (100×10\^6 cells), 3 months apart

Biological: NG01 - Autologous bone marrow derived human stromal cells

50x10^6 cells

EXPERIMENTAL

15 participants with SPMS will receive 4 IT administrations of NG01 (50×10\^6 cells), 3 months apart

Biological: NG01 - Autologous bone marrow derived human stromal cells

Placebo

PLACEBO COMPARATOR

15 participants with SPMS will receive 4 IT administrations of placebo solution, 3 months apart

Other: Sodium Chloride 0.9%

Interventions

NG01 - Autologous bone marrow derived human stromal cellsBIOLOGICAL

NG01 is a cellular therapy product of autologous stromal cells derived from the bone marrow of multiple sclerosis (MS) patients. NG01 is provided as a cell suspension for intrathecal injection.

100x10^6 cells50x10^6 cells
Sodium Chloride 0.9%OTHER

The placebo for use in the NG01 clinical trial is 0.9% Sodium Chloride Solution for Injection

Placebo

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Participants aged 18 to 65 years old.
  • Diagnosis of SPMS.
  • Documented EDSS worsening over the 2 years prior to study entry of ≥1 point for participants with EDSS \<6.0 at screening, and ≥0.5 point for participants with EDSS ≥6.0 at screening, or a documented worsening of at least 20% in the T25FW. If documented T25fW or EDSS is not available, a written summary of the clinical evidence of disability worsening over the previous 2 years and retrospective assessment of EDSS score from data up to 2 years prior to screening, must be submitted for central review by adjudication committee.
  • EDSS at the screening visit from 3.5 to 6.5 at screening.
  • T25FW at the screening visit of from 8.0 to 25 seconds.

You may not qualify if:

  • Documented clinical relapse during the 24 months prior to enrollment and/or evidence of enhancing lesions on an MRI obtained at screening.
  • Pregnancy, breast feeding or women with childbearing potential without an acceptable form of contraception.
  • History of a general chronic handicapping/incapacitating disease other than MS.
  • Participants with clotting disorders
  • Participants unable to undergo an MRI scan.
  • Participants with uncontrolled hepatic disorders, renal or cardiovascular disease, or cancer.
  • Laboratory tests out of normal ranges considered by the investigator as clinically significant.
  • Participants with history or current alcohol abuse or drug addiction.
  • Untreated or uncontrolled psychiatric disorders, or positive suicidal risk assessed by Columbia-Suicide Severity Rating Scale (C-SSRS).
  • Participants who have ever received NG01/MSCs treatment.
  • Participants who, in the opinion of the investigator, are unable to fully comprehend the consenting process or likely to be non-compliant with the study procedures or for whom long-term follow-up seems difficult to achieve.
  • Relapse occurring between screening and randomization.
  • Less than 6 months of the current disease-modifying therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

University of Miami Hospital & Clinics - Sylvester Comprehensive Cancer Center - Neurology

Miami, Florida, 33136, United States

Location

Hadassah University Hospital

Jerusalem, Israel

Location

Related Publications (4)

  • Petrou P, Gothelf Y, Argov Z, Gotkine M, Levy YS, Kassis I, Vaknin-Dembinsky A, Ben-Hur T, Offen D, Abramsky O, Melamed E, Karussis D. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016 Mar;73(3):337-44. doi: 10.1001/jamaneurol.2015.4321.

    PMID: 26751635BACKGROUND

  • Petrou P, Kassis I, Ginzberg A, Halimi M, Yaghmour N, Abramsky O, Karussis D. Long-Term Clinical and Immunological Effects of Repeated Mesenchymal Stem Cell Injections in Patients With Progressive Forms of Multiple Sclerosis. Front Neurol. 2021 May 31;12:639315. doi: 10.3389/fneur.2021.639315. eCollection 2021.

    PMID: 34135843BACKGROUND

  • Petrou P, Kassis I, Levin N, Paul F, Backner Y, Benoliel T, Oertel FC, Scheel M, Hallimi M, Yaghmour N, Hur TB, Ginzberg A, Levy Y, Abramsky O, Karussis D. Beneficial effects of autologous mesenchymal stem cell transplantation in active progressive multiple sclerosis. Brain. 2020 Dec 1;143(12):3574-3588. doi: 10.1093/brain/awaa333.

    PMID: 33253391BACKGROUND

  • Petrou P, Kassis I, Yaghmour NE, Ginzberg A, Karussis D. A phase II clinical trial with repeated intrathecal injections of autologous mesenchymal stem cells in patients with amyotrophic lateral sclerosis. Front Biosci (Landmark Ed). 2021 Oct 30;26(10):693-706. doi: 10.52586/4980.

    PMID: 34719198BACKGROUND

MeSH Terms

Conditions

Multiple Sclerosis, Chronic ProgressiveMultiple Sclerosis

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNSAutoimmune Diseases of the Nervous SystemNervous System DiseasesDemyelinating DiseasesAutoimmune DiseasesImmune System DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

ChloridesHydrochloric AcidChlorine CompoundsInorganic ChemicalsSodium Compounds

Central Study Contacts

Flavia Nelson, MD

CONTACT

305-243-4015fxn133@med.miami.edu

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
CRO Monitors, Sponsor
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 21, 2025

First Posted

May 7, 2025

Study Start

October 1, 2025

Primary Completion (Estimated)

October 1, 2027

Study Completion (Estimated)

March 1, 2028

Last Updated

May 7, 2025

Record last verified: 2025-04

Data Sharing

IPD Sharing
Will not share

Locations

IL(1)US(1)