Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients
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| ClinicalTrials.gov Identifier: NCT02987413 |
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Recruitment Status :
Completed
First Posted : December 9, 2016
Last Update Posted : August 7, 2017
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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 |
| Allocation: | N/A |
| 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 |
| Actual Study Start Date : | April 28, 2015 |
| Actual Primary Completion Date : | March 2016 |
| Actual Study Completion Date : | April 5, 2017 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Autologous Mesenchymal stem cells
Two escalated intrathecal infusions of autologous mesenchymal stem cell
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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.
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
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
| 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 | |
| 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 |
| Responsible Party: | Hospital e Maternidade Dr. Christóvão da Gama |
| ClinicalTrials.gov Identifier: | NCT02987413 |
| Other Study ID Numbers: |
HospitalMCG IEPSaoLucas ( Other Identifier: IEP Sao Lucas ) |
| First Posted: | December 9, 2016 Key Record Dates |
| Last Update Posted: | August 7, 2017 |
| Last Verified: | August 2017 |
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Amyotrophic Lateral Sclerosis |
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Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |