Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients
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|ClinicalTrials.gov Identifier: NCT02987413|
Recruitment Status : Completed
First Posted : December 9, 2016
Last Update Posted : August 7, 2017
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 or disease||Intervention/treatment||Phase|
|Motor Neuron Disease||Biological: Autologous Mesenchymal stem cells (MSCs)||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial|
|Actual Study Start Date :||April 28, 2015|
|Actual Primary Completion Date :||March 2016|
|Actual Study Completion Date :||April 5, 2017|
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
- 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
- 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.
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
|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|
|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|