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

NCT ID: NCT02917681

Last Updated: 2016-09-28

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 28 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-09-30
• Study Completion Date: 2019-08-31

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.

Conditions

AMYOTROPHIC LATERAL SCLEROSIS

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Two intrathecal MSC injections

Intervention Type: OTHER

Interventions

Two intrathecal MSC injections

Intervention Type: OTHER

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pontifícia Universidade Católica do Paraná

OTHER

Sponsor Role: collaborator

University of Sao Paulo General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Gerson Chadi

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Gerson Chadi, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Full Professor

Locations

University of Sao Paulo School of Medicine Clinics Hospital

São Paulo, São Paulo, Brazil

Site Status: RECRUITING

Countries

Brazil

Central Contacts

Fabrício Castro Borba, MD

Role: CONTACT

contato@projetoelabrasil.com.br

551130617460

Jéssica Ruivo Maximino, PhD

Role: CONTACT

contato@projetoelabrasil.com.br

551130617460

Other Identifiers

401922/2014-6

Identifier Type: -

Identifier Source: org_study_id