Allogeneic Mesenchymal Stem Cell-Derived Exosome Therapy for Progressive Multiple Sclerosis

NCT07146087 | Suspended Early Phase 1

• 1 other identifier: Bio120002
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to learn if intravenous infusion of allogeneic mesenchymal stem cell (MSC)-derived exosomes can slow disability progression and improve neurological function in adults with progressive multiple sclerosis (MS). The trial will also evaluate the safety and tolerability of repeated exosome infusions. The main questions it aims to answer are: Does MSC-derived exosome therapy reduce disability progression as measured by the Expanded Disability Status Scale (EDSS)? Does MSC-derived exosome therapy decrease neuroinflammation and brain lesion burden compared with placebo? Researchers will compare participants who receive MSC-derived exosome therapy to those who receive placebo (saline infusion) to see if the treatment improves clinical and biological outcomes. Participants will: Receive either MSC-derived exosome infusions or placebo infusions every 3 months for 1 year (4 total infusions) Undergo clinical assessments (neurological exams, EDSS scoring, neurocognitive testing) Provide blood and cerebrospinal fluid samples for biomarker analysis Have MRI scans to evaluate lesion load and brain volume Complete questionnaires on quality of life and daily functioning

Conditions

Multiple Sclerosis

Keywords

sclerosis; multiple sclerosis; MSC

Outcome Measures

Primary Outcomes (1)

• Change in Expanded Disability Status Scale (EDSS) score

  Unit of measure: points (range: 0-10; higher scores indicate worse disability).

  24 months from the day of administration

Secondary Outcomes (2)

• Change in T2 lesion load and brain atrophy

  24 months after the first administration

• Change in neurocognitive performance

  24 months after first administration

Study Arms (2)

MSC-Derived Exosomes

EXPERIMENTAL

Participants receive intravenous infusions of allogeneic mesenchymal stem cell (MSC)-derived exosomes every 12 weeks for 48 weeks (total of 4 infusions) in addition to their stable background MS therapy (if any).

Biological: MSC-Derived Exosomes

Normal Saline

PLACEBO COMPARATOR

Participants receive intravenous infusions of 0.9% sodium chloride (saline) matched in volume, appearance, and schedule to the experimental arm, in addition to their stable background MS therapy (if any).

Biological: Placebo (Normal Saline)

Interventions

MSC-Derived Exosomes BIOLOGICAL

Purified, sterile, acellular extracellular vesicles (exosomes) isolated from culture supernatant of GLP-manufactured allogeneic human bone-marrow-derived MSCs. Release criteria include particle size distribution 30-150 nm (nanoparticle tracking analysis), particle count, sterility/endotoxin testing, mycoplasma negative, and predefined protein/marker profile (e.g., CD9/CD63/CD81 positive).

MSC-Derived Exosomes

Placebo (Normal Saline) BIOLOGICAL

0.9% sodium chloride solution matched to the experimental product in volume, appearance, infusion set-up, and administration procedures.

Normal Saline

Eligibility Criteria

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

You may qualify if:

• Age 18-65 years.
• Diagnosis of progressive multiple sclerosis (primary or secondary) confirmed
• EDSS score 3.0-6.5.

You may not qualify if:

• Relapse or corticosteroid treatment within 3 months before screening.
• Other significant neurological or autoimmune disorders.
• Active infection, malignancy, or uncontrolled systemic disease.

Sponsors & Collaborators

• Biocells Medical: lead

Study Sites (1)

Biocells Medical

Warsaw, Poland

Location

MeSH Terms

Conditions

Multiple Sclerosis; Sclerosis

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Specialist in regenerative medicine

Study Record Dates

• First Submitted: August 20, 2025
• First Posted: August 28, 2025
• Study Start: July 1, 2020
• Primary Completion: June 30, 2023
• Study Completion (Estimated): December 31, 2027
• Last Updated: August 28, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1)