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Human Neuronal Progenitor/Stem Cells

This article discusses an ongoing clinical trial investigating the use of human fetal neural precursor cells (hfNPCs), a type of human neuronal progenitor/stem cells, as a potential treatment for progressive multiple sclerosis (PMS). The study aims to evaluate the safety and efficacy of intrathecal administration of these cells compared to a sham procedure in patients with PMS. This phase 2 trial represents an important step in exploring innovative cellular therapies for this challenging neurological condition.

Table of Contents

What are Human Fetal Neural Precursor Cells (hfNPCs)?

Human Fetal Neural Precursor Cells (hfNPCs), also known as human neuronal progenitor/stem cells, are a type of cell therapy being investigated for the treatment of progressive multiple sclerosis (PMS)[1]. These cells have the potential to develop into various types of cells found in the nervous system, which makes them a promising candidate for treating neurological conditions like multiple sclerosis.

Treatment for Progressive Multiple Sclerosis

Progressive Multiple Sclerosis (PMS) is a form of multiple sclerosis characterized by a steady worsening of neurological function[1]. The use of hfNPCs is being studied as a potential treatment for PMS patients who have not responded well to other approved therapies or are ineligible for them[1].

How are hfNPCs Administered?

In the clinical trial, hfNPCs are administered through intrathecal (IT) injection[1]. This means the cells are injected directly into the fluid surrounding the brain and spinal cord. The treatment involves two separate administrations, each consisting of approximately 200 million cells, given six months apart[1].

The STEMS2 Clinical Trial

The STEMS2 trial is a Phase 2 clinical study investigating the use of hfNPCs for treating progressive multiple sclerosis[1]. Key features of the trial include:

  • It is a randomized, patient and assessor blind, multicentre study[1].
  • Participants are randomly assigned to receive either hfNPC transplantation or a sham procedure[1].
  • The study includes a 96-week follow-up period to assess the long-term effects of the treatment[1].

Who is Eligible for the Trial?

The trial has specific inclusion and exclusion criteria. Some key eligibility factors include:

  • Participants must be between 18 and 65 years old[1].
  • Diagnosed with Progressive Multiple Sclerosis[1].
  • Have an Expanded Disability Status Scale (EDSS) score between 3.0 and 8.0[1]. This scale measures the degree of disability in MS patients.
  • Have failed, been intolerant to, or ineligible for approved therapies[1].

There are also several exclusion criteria, such as certain medical conditions or recent use of specific medications, that may prevent participation[1].

Trial Objectives and Endpoints

The main goal of the STEMS2 trial is to evaluate how well hfNPCs work in treating PMS compared to a sham procedure[1]. The study will measure several outcomes, including:

  • Changes in brain volume over 96 weeks[1].
  • The proportion of patients without disease progression[1].
  • Changes in cognitive function[1].

Safety Considerations

As with any clinical trial, safety is a primary concern. The study will closely monitor participants for any adverse events related to the treatment[1]. Additionally, participants will receive tacrolimus, an immunosuppressive medication, for 24 weeks after each hfNPC administration to help prevent rejection of the transplanted cells[1].

It’s important to note that while this treatment shows promise, it is still experimental and its long-term effects and efficacy are not yet fully known. Patients considering participation in this or any clinical trial should discuss the potential risks and benefits thoroughly with their healthcare providers.

Aspect Details
Study Type Phase 2, randomized, patient and assessor blind, multicentre study
Condition Progressive Multiple Sclerosis (PMS)
Intervention Human fetal neural precursor cells (hfNPCs) transplantation
Dosage Two intrathecal administrations of 200 x 10^6±10% cells, six months apart
Control Sham procedure
Primary Outcome Change in whole brain volume (MRI) over 96 weeks
Key Secondary Outcomes Safety, brain grey/white matter volume changes, disability progression, cognitive function
Eligibility Adults 18-65 with PMS, EDSS 3.0-8.0, failed/intolerant to approved therapies
Follow-up Duration 96 weeks post-treatment

FAQ

  • What is the main goal of this clinical trial? The main goal is to evaluate the therapeutic efficacy of human fetal neural precursor cells (hfNPCs) compared to a sham procedure in patients with progressive multiple sclerosis (PMS). The trial will assess the effects of two intrathecal administrations of 200 million cells, given six months apart.
  • Who is eligible to participate in this trial? Eligible participants are adults aged 18-65 with diagnosed progressive multiple sclerosis, an Expanded Disability Status Scale (EDSS) score between 3.0 and 8.0, and who have failed, are intolerant to, or ineligible for approved therapies. Participants must also be willing to adhere to the study procedures and use effective contraception.
  • How long does the trial last? The trial consists of a screening period, a treatment phase with two administrations six months apart, and a 96-week (about 2 years) follow-up period. The total duration of participation is approximately 2.5 years.
  • What are the main outcomes being measured in this trial? The primary outcome is the change in whole brain volume evaluated through MRI over 96 weeks. Secondary outcomes include safety assessments, changes in brain grey and white matter volume, disability progression, cognitive function, and the proportion of patients with no active disease.
  • Is this treatment already approved for use? No, this is a phase 2 trial of a drug not currently authorized for use in the population involved in the planned clinical trial. The study aims to gather more information about the safety and efficacy of this potential treatment for progressive multiple sclerosis.

Ongoing Clinical Trials on Human Neuronal Progenitor/Stem Cells

  • Study on Human Fetal Neural Precursor Cells and Tacrolimus for Patients with Progressive Multiple Sclerosis

    Recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy

Glossary

  • Progressive Multiple Sclerosis (PMS): A form of multiple sclerosis characterized by a steady worsening of neurologic functioning, with or without relapses or new MRI activity.
  • Human fetal neural precursor cells (hfNPCs): A type of stem cell derived from fetal neural tissue that has the potential to develop into various types of cells in the nervous system.
  • Intrathecal (IT) administration: A method of delivering medication directly into the fluid surrounding the brain and spinal cord.
  • Expanded Disability Status Scale (EDSS): A method of quantifying disability in multiple sclerosis, ranging from 0 to 10 in 0.5 unit increments.
  • Sham procedure: A fake or placebo procedure that mimics the real treatment but does not involve active intervention, used as a control in clinical trials.
  • Magnetic Resonance Imaging (MRI): A medical imaging technique that uses magnetic fields and radio waves to create detailed images of organs and tissues in the body.
  • Tacrolimus: An immunosuppressive drug used to prevent rejection in organ transplantation and to treat certain autoimmune disorders.
  • Symbol Digit Modalities Test (SDMT): A cognitive test that assesses processing speed and working memory, often used in multiple sclerosis research.
  • Timed 25-foot walk test (T25FWT): A quantitative measure of lower extremity function used to assess walking speed in patients with neurological disorders.
  • Nine-hole peg test (9HPT): A standardized measure of finger dexterity used to assess upper extremity function in neurological disorders.

References

  1. http://clinicaltrials.eu/trial/study-on-human-fetal-neural-precursor-cells-and-tacrolimus-for-patients-with-progressive-multiple-sclerosis/