Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients

Phase 1

Completed

• Conditions: Motor Neuron Disease
• Interventions: Biological: Autologous Mesenchymal stem cells (MSCs)

• Registration Number: NCT02987413
• Lead Sponsor: Hospital e Maternidade Dr. Christóvão da Gama

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-04-28
• Primary Completion: 2016-03
• Study First Submitted: 2016-07-01
• Study First Submitted QC: 2016-12-08
• Study First Posted: 2016-12-09
• Study Completion: 2017-04-05
• Status Verified: 2017-08
• Last Update Submitted: 2017-08-04
• Last Update Posted: 2017-08-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous Mesenchymal stem cells	Autologous Mesenchymal stem cells (MSCs)	Two escalated intrathecal infusions of autologous mesenchymal stem cell

Primary Outcome Measures

Name	Time	Method
Serious Adverse Events related to the treatment	12 months	Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up

Secondary Outcome Measures

Name	Time	Method
Revised ALS Functional Rating Scale (ALSFRS-R)	12 months	Quality of life from patients according to functional scales will be evaluated before and after interventions with 1, 3, 6 and 12 months.

Trial Locations

Locations (2)

Hospital e Maternidade Dr Christovao da Gama; 🇧🇷 Santo Andre, Sao Paulo, Brazil

Instituto de Ensino e Pesquisas - IEP-São Lucas; 🇧🇷 Sao Paulo, SP, Brazil