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Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis

This study is currently recruiting participants.
Verified September 2016 by Gerson Chadi, University of Sao Paulo General Hospital
Sponsor:
ClinicalTrials.gov Identifier:
NCT02917681
First Posted: September 28, 2016
Last Update Posted: September 28, 2016
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Pontifícia Universidade Católica do Paraná
Information provided by (Responsible Party):
Gerson Chadi, University of Sao Paulo General Hospital
  Purpose
The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated

Condition Intervention Phase
AMYOTROPHIC LATERAL SCLEROSIS Other: Two intrathecal MSC injections Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Gerson Chadi, University of Sao Paulo General Hospital:

Primary Outcome Measures:
  • Safety [ Time Frame: 10 months ]
    Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery


Secondary Outcome Measures:
  • Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) [ Time Frame: 10 months ]
    Specific scale for assessing ALS disease progression

  • Change in Handheld dynamometry [ Time Frame: 10 months ]
  • Change in Electrical Impedance Myography [ Time Frame: 10 months ]
  • Change in Forced Vital Capacity (FVC) [ Time Frame: 10 months ]
  • Change in Isometric Strength [ Time Frame: 10 months ]
  • Changes in morphometric parameters of MRI of encephalon and spinal cord [ Time Frame: 10 months ]

Estimated Enrollment: 28
Study Start Date: September 2016
Estimated Study Completion Date: August 2019
Estimated Primary Completion Date: February 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: MSC injection
Patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.
Other: Two intrathecal MSC injections

Detailed Description:
The study is an open-label, unmasked, uncontrolled phase I and II trial to evaluate safety and efficacy of two injections of autologous mesenchymal stem cells (MSC) in Amyotrophic Lateral Sclerosis (ALS) patients. Patients will be recruited trough a web-based registration system, open for all ALS Brazilian patients. The patients will be enrolled after in-person screening at the University of Sao Paulo Medical School Clinics Hospital and inclusion criteria fulfilled. There will be 9 national calls for registration, two months apart each. The enrolled patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.
  Eligibility

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age between 18 and 70
  2. Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)
  3. ALSFRS-r ≥30 at enrollment
  4. Forced Vital Capacity ≥65% of the height and weight standard
  5. No-pregnancy agreement
  6. Regional accessibility to the study site
  7. Capability to give away agreed consensus
  8. Patients will be followed at Academic Institutions at their hometown

Exclusion Criteria:

  1. Previous cellular therapy
  2. Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections
  3. Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures
  4. Any other disease that may interfere with the study
  5. Any other neurological diseases
  6. Aspartate or alanine aminotransferases elevated >3x normality upper limit
  7. Serum creatinine >2x normality upper limit
  8. Hepatitis B and C, HIV, HTLV I and II and syphilis
  9. Immunosuppressant drug use within 6 weeks from the study's screening
  10. Pregnancy or breast-feeding
  11. Acquired or inherited Immunodeficiency
  12. Participation in other clinical trials
  13. Non-invasive ventilation, tracheostomy or diaphragm pacing
  14. Substance abuse within one year and other unstable mental health diseases according to researcher's judgement
  15. Gastrostomy or any alternative feeding means
  16. Inappropriate in-vitro MSC expansion
  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): NCT02917681


Contacts
Contact: Fabrício Castro Borba, MD 551130617460 contato@projetoelabrasil.com.br
Contact: Jéssica Ruivo Maximino, PhD 551130617460 contato@projetoelabrasil.com.br

Locations
Brazil
University of Sao Paulo School of Medicine Clinics Hospital Recruiting
Sao Paulo, SP, Brazil, 05403000
Sponsors and Collaborators
University of Sao Paulo General Hospital
Pontifícia Universidade Católica do Paraná
Investigators
Principal Investigator: Gerson Chadi, MD, PhD Full Professor
  More Information

Responsible Party: Gerson Chadi, Professor, University of Sao Paulo General Hospital
ClinicalTrials.gov Identifier: NCT02917681     History of Changes
Other Study ID Numbers: 401922/2014-6
First Submitted: September 26, 2016
First Posted: September 28, 2016
Last Update Posted: September 28, 2016
Last Verified: September 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Gerson Chadi, University of Sao Paulo General Hospital:
ALS
mesenchymal stem cells
intrathecal

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