Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients

August 4, 2017 updated by: Hospital e Maternidade Dr. Christóvão da Gama

Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Brazil
    • SP
      • Sao Paulo, SP, Brazil, 01236-030
        • Instituto de Ensino e Pesquisas - IEP-São Lucas
    • Sao Paulo
      • Santo Andre, Sao Paulo, Brazil, 09030010
        • Hospital e Maternidade Dr Christóvão da Gama

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hospital e Maternidade Dr. Christóvão da Gama

Collaborators

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 Christóvão 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 28, 2015

Primary Completion (Actual)

March 1, 2016

Study Completion (Actual)

April 5, 2017

Study Registration Dates

First Submitted

July 1, 2016

First Submitted That Met QC Criteria

December 8, 2016

First Posted (Estimate)

December 9, 2016

Study Record Updates

Last Update Posted (Actual)

August 7, 2017

Last Update Submitted That Met QC Criteria

August 4, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HospitalMCG
  • IEPSaoLucas (Other Identifier: IEP Sao Lucas)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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