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Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients

NCT02987413 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2017-08-07

No results posted yet for this study

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.

Conditions

  • Motor Neuron Disease

Interventions

BIOLOGICAL

Autologous Mesenchymal stem cells (MSCs)

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

Sponsors & Collaborators

  • IEP São Lucas - Instituto de Ensino e Pesquisa

    collaborator OTHER

  • Clinica Jordy Sinapse

    collaborator UNKNOWN

  • TECHLIFE - Centro de Tecnologia Celular

    collaborator UNKNOWN

  • Hospital e Maternidade Dr. Christóvão da Gama

    lead OTHER

Principal Investigators

  • Leandro B Agati, PhD · Hospital e Maternidade Dr Christovao da Gama

  • Eliseo J Sekiya, MD, PhD · Instituto de Ensino e Pesquisas - IEP-São Lucas

  • Adelson A Silva, MD · Instituto de Ensino e Pesquisas - IEP-São Lucas

  • Andresa Forte, MSc · TECHLIFE - Centro de Tecnologia Celular

  • Sergio S Jordy, MD · Clínica Jordy Sinapse

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-04-28
Primary Completion
2016-03-31
Completion
2017-04-05

Countries

  • Brazil

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02987413 on ClinicalTrials.gov