Clinical trial NCT02917681: Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: AMYOTROPHIC LATERAL SCLEROSIS
Keywords: ALS
mesenchymal stem cells
intrathecal
Lead sponsor: University of Sao Paulo General Hospital
Sponsor class: OTHER
Collaborators: name: Pontifícia Universidade Católica do Paraná
First posted: 2016-09-28
Enrollment: 28
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Safety
timeFrame: 10 months
Secondary outcomes:
measure: Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
timeFrame: 10 months
measure: Change in Handheld dynamometry
timeFrame: 10 months
measure: Change in Electrical Impedance Myography
timeFrame: 10 months
measure: Change in Forced Vital Capacity (FVC)
timeFrame: 10 months
measure: Change in Isometric Strength
timeFrame: 10 months
measure: Changes in morphometric parameters of MRI of encephalon and spinal cord
timeFrame: 10 months
Other outcomes:
Locations:
city: Sao Paulo
country: Brazil
facility: University of Sao Paulo School of Medicine Clinics Hospital
state: SP
Study officials:
affiliation: Full Professor
name: Gerson Chadi, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: