Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-04-28

Study Completion Date

2017-04-05

Brief Summary

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Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord, leading to bulbar, respiratory, and limb weakness. There is no effective treatment, and the disease usually progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates for cellular therapy in ALS.

Design-A phase 1 open-safety clinical trial. 4 patients will be selected according to a restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal autologous stem cells infusions escalated in two intrathecal administrations in patients with ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response, laboratorial and magnetic resonance imaging of patients submitted to cellular escalating doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale and functional scales.

Conclusion: This study is a primary step before a large randomized double-blind clinical trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS patients, initial data of efficacy in addition to improved quality of life.

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Detailed Description

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Conditions

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Motor Neuron Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Autologous Mesenchymal stem cells

Two escalated intrathecal infusions of autologous mesenchymal stem cell

Group Type EXPERIMENTAL

Autologous Mesenchymal stem cells (MSCs)

Intervention Type BIOLOGICAL

2 intrathecal autologous MSCs infusions (1x10\^8 cells) will be performed, escalated from 30 days apart

Interventions

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Autologous Mesenchymal stem cells (MSCs)

2 intrathecal autologous MSCs infusions (1x10\^8 cells) will be performed, escalated from 30 days apart

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* Women and males over 18-year-old.
* Diagnosis of ALS in agreement with the criteria of "EL SCORE"
* Less than 24 months of evolution of the disease (from the beginning of the symptoms).
* Good understanding of the protocol and aptitude to grant the informed consent
* Infertile women (post-menopause or hysterectomized)
* Brazilian citizen and permanent resident.

Exclusion Criteria

* Any significant medical condition (congestive heart failure, angina, respiratory failure, and others)
* Any auto-immune disease
* Any malignant diseases
* Systemic infection
* Mental illness
* Depressive state

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No