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

Primary Purpose

AMYOTROPHIC LATERAL SCLEROSIS

Status
Unknown status
Phase
Phase 1
Locations
Brazil
Study Type
Interventional
Intervention
Two intrathecal MSC injections
Sponsored by
University of Sao Paulo General Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for AMYOTROPHIC LATERAL SCLEROSIS focused on measuring ALS, mesenchymal stem cells, intrathecal

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Sao Paulo School of Medicine Clinics HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

MSC injection

Arm Description

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.

Outcomes

Primary Outcome Measures

Safety
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)
Specific scale for assessing ALS disease progression
Change in Handheld dynamometry
Change in Electrical Impedance Myography
Change in Forced Vital Capacity (FVC)
Change in Isometric Strength
Changes in morphometric parameters of MRI of encephalon and spinal cord

Full Information

First Posted
September 26, 2016
Last Updated
September 27, 2016
Sponsor
University of Sao Paulo General Hospital
Collaborators
Pontifícia Universidade Católica do Paraná
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1. Study Identification

Unique Protocol Identification Number
NCT02917681
Brief Title
Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
September 2016
Overall Recruitment Status
Unknown status
Study Start Date
September 2016 (undefined)
Primary Completion Date
February 2019 (Anticipated)
Study Completion Date
August 2019 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Sao Paulo General Hospital
Collaborators
Pontifícia Universidade Católica do Paraná

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
AMYOTROPHIC LATERAL SCLEROSIS
Keywords
ALS, mesenchymal stem cells, intrathecal

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
28 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
MSC injection
Arm Type
Experimental
Arm Description
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.
Intervention Type
Other
Intervention Name(s)
Two intrathecal MSC injections
Primary Outcome Measure Information:
Title
Safety
Description
Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery
Time Frame
10 months
Secondary Outcome Measure Information:
Title
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
Description
Specific scale for assessing ALS disease progression
Time Frame
10 months
Title
Change in Handheld dynamometry
Time Frame
10 months
Title
Change in Electrical Impedance Myography
Time Frame
10 months
Title
Change in Forced Vital Capacity (FVC)
Time Frame
10 months
Title
Change in Isometric Strength
Time Frame
10 months
Title
Changes in morphometric parameters of MRI of encephalon and spinal cord
Time Frame
10 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age between 18 and 70 Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000) ALSFRS-r ≥30 at enrollment Forced Vital Capacity ≥65% of the height and weight standard No-pregnancy agreement Regional accessibility to the study site Capability to give away agreed consensus Patients will be followed at Academic Institutions at their hometown Exclusion Criteria: Previous cellular therapy Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections 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 Any other disease that may interfere with the study Any other neurological diseases Aspartate or alanine aminotransferases elevated >3x normality upper limit Serum creatinine >2x normality upper limit Hepatitis B and C, HIV, HTLV I and II and syphilis Immunosuppressant drug use within 6 weeks from the study's screening Pregnancy or breast-feeding Acquired or inherited Immunodeficiency Participation in other clinical trials Non-invasive ventilation, tracheostomy or diaphragm pacing Substance abuse within one year and other unstable mental health diseases according to researcher's judgement Gastrostomy or any alternative feeding means Inappropriate in-vitro MSC expansion
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Fabrício Castro Borba, MD
Phone
551130617460
Email
contato@projetoelabrasil.com.br
First Name & Middle Initial & Last Name or Official Title & Degree
Jéssica Ruivo Maximino, PhD
Phone
551130617460
Email
contato@projetoelabrasil.com.br
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gerson Chadi, MD, PhD
Organizational Affiliation
Full Professor
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Sao Paulo School of Medicine Clinics Hospital
City
Sao Paulo
State/Province
SP
ZIP/Postal Code
05403000
Country
Brazil
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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