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
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
University of Sao Paulo School of Medicine Clinics Hospital
São Paulo, São Paulo, Brazil
RECRUITINGSafety
Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery
Time frame: 10 months
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
Specific scale for assessing ALS disease progression
Time frame: 10 months
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
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