Intrathecal Amniotic Fluid Stem Cells for Progressive Multiple Sclerosis

NCT06841068 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: AFSC-PMS-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Multiple Sclerosis, Progressive

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)

  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 Outcomes (4)

• The Multiple Sclerosis Functional Composite (MSFC) score

  From enrollment to the end of treatment at 12 months

• The Six-minute Walk Test

  From enrollment to the end of treatment at 12 months

• The Expanded Disability Status Scale (EDSS)

  From enrollment to the end of treatment at 12 months

• The Multiple Sclerosis Quality of Life-54 (MSQoL-54)

  From enrollment to the end of treatment at 12 months

Study Arms (1)

Open label stem cell arm

EXPERIMENTAL

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

Interventions

Amniotic fluid or placental CD117 stem cells BIOLOGICAL

CD117 (c-Kit) positive stem cells, extracted from ethical fetal sources such as amniotic fluid and placental tissue.

Open label stem cell arm

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead

Study Sites (1)

Atrium Health

Charlotte, North Carolina, 28204, United States

Location

Related Publications (2)

• Rosner M, Horer S, Feichtinger M, Hengstschlager M. Multipotent fetal stem cells in reproductive biology research. Stem Cell Res Ther. 2023 Jun 7;14(1):157. doi: 10.1186/s13287-023-03379-4.

  PMID: 37287077 BACKGROUND

• Lassmann H. Pathogenic Mechanisms Associated With Different Clinical Courses of Multiple Sclerosis. Front Immunol. 2019 Jan 10;9:3116. doi: 10.3389/fimmu.2018.03116. eCollection 2018.

  PMID: 30687321 BACKGROUND

MeSH Terms

Conditions

Multiple Sclerosis, Chronic Progressive

Condition Hierarchy (Ancestors)

Multiple Sclerosis; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Mahmoud Abdelrazek, MD

  Wake Forest University Health Sciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 16, 2025
• First Posted: February 24, 2025
• Study Start: February 1, 2026
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: February 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)