Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients

NCT02987413 | Completed Phase 1 DMC

• 2 other identifiers: HospitalMCGIEPSaoLucas
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 2

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.

Conditions

Motor Neuron Disease

Keywords

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Serious Adverse Events related to the treatment

  Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up

  12 months

Secondary Outcomes (1)

• Revised ALS Functional Rating Scale (ALSFRS-R)

  12 months

Study Arms (1)

Autologous Mesenchymal stem cells

EXPERIMENTAL

Two escalated intrathecal infusions of autologous mesenchymal stem cell

Biological: Autologous Mesenchymal stem cells (MSCs)

Interventions

Autologous Mesenchymal stem cells (MSCs) BIOLOGICAL

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

Autologous Mesenchymal stem cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Hospital e Maternidade Dr. Christóvão da Gama: lead
• IEP São Lucas - Instituto de Ensino e Pesquisa: collaborator
• Clinica Jordy Sinapse: collaborator
• TECHLIFE - Centro de Tecnologia Celular: collaborator

Study Sites (2)

Hospital e Maternidade Dr Christovao da Gama

Santo André, São Paulo, 09030010, Brazil

Location

Instituto de Ensino e Pesquisas - IEP-São Lucas

São Paulo, São Paulo, 01236-030, Brazil

Location

Related Links

• Institutional website

MeSH Terms

Conditions

Motor Neuron Disease; Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Leandro B Agati, PhD

  Hospital e Maternidade Dr Christovao da Gama

  STUDY DIRECTOR

• 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

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 1, 2016
• First Posted: December 9, 2016
• Study Start: April 28, 2015
• Primary Completion: March 1, 2016
• Study Completion: April 5, 2017
• Last Updated: August 7, 2017

Record last verified: 2017-08

Locations

BR (2)