Intrathecal Amniotic Fluid Stem Cells for Progressive Multiple Sclerosis

February 5, 2026 updated by: Wake Forest University Health Sciences

A Phase I/II Trial of Intrathecal Delivery of Amniotic Fluid Stem Cells for Primary and Secondary Progressive Multiple Sclerosis

In this clinical trial, researchers are exploring a novel approach to delivering therapy directly into the spinal fluid, which surrounds and nourishes the brain and spinal cord. The study focuses on patients with progressive multiple sclerosis (MS), a form of the disease that leads to worsening disability without the typical relapses seen in other MS subtypes.

This investigational therapy involves the use of stem cells derived from amniotic fluid-the protective liquid surrounding a developing baby in the womb. To the best of the researchers' knowledge, these specific stem cells have never been tested in MS patients before. Amniotic fluid is ethically sourced from routine medical procedures during pregnancy, and similar stem cells can also be obtained from placentas that are typically discarded after childbirth.

Participants in the trial will receive multiple injections of these stem cells into their spinal fluid over the course of a year. Researchers will closely monitor for the safety of this therapy, as well as monitor the participants' walking ability and other neurological functions to assess potential improvements.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Atrium Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Adults aged 18-60 years.
  • Diagnosed with primary or secondary progressive MS according to the 2017 revised McDonald criteria.
  • Expanded Disability Status Scale (EDSS) of 3.0 to 8.0 with at least one functional scale that is not vision or sensory or brainstem or cognitive or bowel/bladder that is scoring 3.0 or greater.
  • Clinically stable as determined by their neurologist for the past 6 months.
  • On no disease-modifying therapy (DMT) for at least 6 months, or on the same DMT for at least 6 months before study entry.
  • Can give informed consent.
  • Women of child-bearing age must practice adequate contraception techniques in the eye of the investigator and continue to do so during the study period.
  • Must be a good candidate, known for compliance for example, in the opinion of the investigators.

Exclusion Criteria:

  • Participation in another clinical trial within the last 30 days.
  • Severe allergic reactions to any component of the study treatments.
  • Significant comorbidities.
  • Active infections or malignancies.
  • History of a malignancy within the past two years except for skin cancer that has been excised and controlled with only surgical treatment, not requiring chemotherapy or radiotherapy.
  • Any medical condition that the investigator deems as unsuitable with therapy.
  • Prior organ, tissue, or stem cell transplant or cell therapy within 3 years of study entry
  • A diagnosis of a progressive neurological disorder other than multiple sclerosis.
  • Inability to have an MRI scan.
  • Inability to have a lumbar puncture, for example severe bleeding diathesis.
  • Pregnant or breastfeeding or intention to become pregnant during the study.
  • The results of the following laboratory results deemed suitable by the investigators: CBC with diff, CMP, INR, aPTT, Hepatitis C serology, HIV serology, RPR.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Open label stem cell arm

The first 5 participants will receive 4 intrathecal injections of c-Kit stem cells at a dose of 25 x 106 cells per injection.

The next 5 patients will receive 4 intrathecal injections of c-Kit stem cells at a dose of 50 x 106cells per injection.

The next 10 patients will receive 4 intrathecal injections of c-Kit stem cells at a dose of 80 x 106cells per injection.

Only if a dose is deemed safe by the investigators after 1 month of its first injection, will we advance to the next dose.
Biological: Amniotic fluid or placental CD117 stem cells
CD117 (c-Kit) positive stem cells, extracted from ethical fetal sources such as amniotic fluid and placental tissue.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Time Frame: From enrollment to the end of treatment at 12 months
The primary safety outcome will be the incidence of adverse events (AEs) and serious adverse events (SAEs) related to the treatment, including their severity and duration. Adverse events will be classified according to the NIH Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
From enrollment to the end of treatment at 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Multiple Sclerosis Functional Composite (MSFC) score
Time Frame: From enrollment to the end of treatment at 12 months
The Multiple Sclerosis Functional Composite (MSFC) is a three-part quantitative objective measure of neurologic function, measuring leg (timed 25-foot walk [25FTW] measured in seconds), arm (nine-hole peg test [9HPT] measured in seconds), and cognitive (three-second paced auditory serial addition test [PASAT3] measured in z-score) functions. The MSFC is a continuous scale that is a composite of the scores of its three parts. A change of 20% or more of the total score (the MSFC composite) or any of its individual component parts (25FTW, 9HPT, PASAT3) is considered clinically significant.
From enrollment to the end of treatment at 12 months
The Six-minute Walk Test
Time Frame: From enrollment to the end of treatment at 12 months
The maximum distance walked in 6 minutes is measured. If the patient cannot complete the 6-minute walk then the measurement becomes the total time walked. A 20% change in the distance walked in 6 minutes for those who complete the 6-minute walk, or a 20% change in the total time walked for those who cannot, will be considered clinically significant.
From enrollment to the end of treatment at 12 months
The Expanded Disability Status Scale (EDSS)
Time Frame: From enrollment to the end of treatment at 12 months
The Expanded Disability Status Scale (EDSS) score ranges from 0 to 10.0, with higher scores indicating a greater degree of disability.25 Participants will undergo EDSS assessments at baseline, 6 months, and 12 months. A reduction in EDSS score by 1 point for baseline scores ≤5.5 and by 0.5 points for baseline scores ≤6.0 will indicate a reduction in disability, and vice versa for disability progression.
From enrollment to the end of treatment at 12 months
The Multiple Sclerosis Quality of Life-54 (MSQoL-54)
Time Frame: From enrollment to the end of treatment at 12 months

The Multiple Sclerosis Quality of Life-54 (MSQoL-54) is a health-related quality of life (HRQoL) questionnaire specifically designed for people with multiple sclerosis (MS). The MSQoL-54 generates 12 subscales and 2 composite scores.

Scoring:

  1. Item scores range from 1 to 6, depending on the question format.
  2. Raw scores for each subscale are calculated by averaging item responses.
  3. Transformed scores: These raw scores are converted to a 0-100 scale, where 0 represents the worst possible health and 100 represents the best possible health.
  4. Composite scores for physical and mental health are calculated by averaging the relevant subscale scores.

Interpretation:

  • Higher scores indicate better quality of life.
  • The physical health composite reflects mobility, energy, and physical pain.
  • The mental health composite covers emotional well-being, cognitive function, and social interaction.
From enrollment to the end of treatment at 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Wake Forest University Health Sciences

Investigators

  • Principal Investigator: Mahmoud Abdelrazek, MD, Wake Forest University Health Sciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 1, 2026

Primary Completion (Estimated)

August 1, 2026

Study Completion (Estimated)

November 1, 2026

Study Registration Dates

First Submitted

February 16, 2025

First Submitted That Met QC Criteria

February 19, 2025

First Posted (Actual)

February 24, 2025

Study Record Updates

Last Update Posted (Actual)

February 6, 2026

Last Update Submitted That Met QC Criteria

February 5, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AFSC-PMS-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

The clinical trial study protocol will be published as a separate paper. The individual participant data will be shared in the appendix of the peer-reviewed study manuscript.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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