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

University of Sao Paulo General Hospital

Status and phase: Unknown; Phase 2; Phase 1

Conditions: AMYOTROPHIC LATERAL SCLEROSIS

Treatments: Other: Two intrathecal MSC injections

Study type: Interventional

Funder types: Other

Identifiers

NCT02917681

401922/2014-6

Details and patient eligibility

About

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

Full 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.

Enrollment: 28 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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28 participants in 1 patient group

MSC injection

Experimental group

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.

Treatment:

Other: Two intrathecal MSC injections