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Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02987413
First Posted: December 9, 2016
Last Update Posted: August 7, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
IEP São Lucas - Instituto de Ensino e Pesquisa
Clinica Jordy Sinapse
TECHLIFE - Centro de Tecnologia Celular
Information provided by (Responsible Party):
Hospital e Maternidade Dr. Christóvão da Gama
  Purpose

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.


Condition Intervention Phase
Motor Neuron Disease Biological: Autologous Mesenchymal stem cells (MSCs) Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial

Resource links provided by NLM:


Further study details as provided by Hospital e Maternidade Dr. Christóvão da Gama:

Primary Outcome Measures:
  • Serious Adverse Events related to the treatment [ Time Frame: 12 months ]
    Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up


Secondary Outcome Measures:
  • Revised ALS Functional Rating Scale (ALSFRS-R) [ Time Frame: 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.


Enrollment: 3
Actual Study Start Date: April 28, 2015
Study Completion Date: April 5, 2017
Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Autologous Mesenchymal stem cells
Two escalated intrathecal infusions of autologous mesenchymal stem cell
Biological: Autologous Mesenchymal stem cells (MSCs)
2 intrathecal autologous MSCs infusions (1x10^8 cells) will be performed, escalated from 30 days apart

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02987413


Locations
Brazil
Hospital e Maternidade Dr Christovao da Gama
Santo Andre, Sao Paulo, Brazil, 09030010
Instituto de Ensino e Pesquisas - IEP-São Lucas
Sao Paulo, SP, Brazil, 01236-030
Sponsors and Collaborators
Hospital e Maternidade Dr. Christóvão da Gama
IEP São Lucas - Instituto de Ensino e Pesquisa
Clinica Jordy Sinapse
TECHLIFE - Centro de Tecnologia Celular
Investigators
Study Director: Leandro B Agati, PhD Hospital e Maternidade Dr Christovao da Gama
Study Chair: Eliseo J Sekiya, MD, PhD Instituto de Ensino e Pesquisas - IEP-São Lucas
Study Chair: Adelson A Silva, MD Instituto de Ensino e Pesquisas - IEP-São Lucas
Study Chair: Andresa Forte, MSc TECHLIFE - Centro de Tecnologia Celular
Study Chair: Sergio S Jordy, MD Clínica Jordy Sinapse
  More Information

Additional Information:
Responsible Party: Hospital e Maternidade Dr. Christóvão da Gama
ClinicalTrials.gov Identifier: NCT02987413     History of Changes
Other Study ID Numbers: HospitalMCG
IEPSaoLucas ( Other Identifier: IEP Sao Lucas )
First Submitted: July 1, 2016
First Posted: December 9, 2016
Last Update Posted: August 7, 2017
Last Verified: August 2017

Keywords provided by Hospital e Maternidade Dr. Christóvão da Gama:
Amyotrophic Lateral Sclerosis

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases