AMYOTROPHIC LATERAL SCLEROSIS
Conditions
Keywords
ALS, mesenchymal stem cells, intrathecal
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.
Interventions
OTHERTwo intrathecal MSC injections
Sponsors
Pontifícia Universidade Católica do Paraná
University of Sao Paulo General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety | 10 months | Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Handheld dynamometry | 10 months | — |
| Change in Electrical Impedance Myography | 10 months | — |
| Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) | 10 months | Specific scale for assessing ALS disease progression |
| Change in Isometric Strength | 10 months | — |
| Changes in morphometric parameters of MRI of encephalon and spinal cord | 10 months | — |
| Change in Forced Vital Capacity (FVC) | 10 months | — |
Countries
Brazil
Contacts
Primary ContactFabrício Castro Borba, MD
Backup ContactJéssica Ruivo Maximino, PhD
Outcome results
None listed