Clinical trial NCT06841068: Intrathecal Amniotic Fluid Stem Cells for Progressive Multiple Sclerosis
Official title: A Phase I/II Trial of Intrathecal Delivery of Amniotic Fluid Stem Cells for Primary and Secondary Progressive Multiple Sclerosis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Multiple Sclerosis, Progressive
Lead sponsor: Wake Forest University Health Sciences
Sponsor class: OTHER
First posted: 2025-02-24
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
timeFrame: From enrollment to the end of treatment at 12 months
Secondary outcomes:
measure: The Multiple Sclerosis Functional Composite (MSFC) score
timeFrame: From enrollment to the end of treatment at 12 months
measure: The Six-minute Walk Test
timeFrame: From enrollment to the end of treatment at 12 months
measure: The Expanded Disability Status Scale (EDSS)
timeFrame: From enrollment to the end of treatment at 12 months
measure: The Multiple Sclerosis Quality of Life-54 (MSQoL-54)
timeFrame: From enrollment to the end of treatment at 12 months
Other outcomes:
Locations:
city: Charlotte
country: United States
facility: Atrium Health
state: North Carolina
Study officials:
affiliation: Wake Forest University Health Sciences
name: Mahmoud Abdelrazek, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.