Amyotrophic lateral sclerosis MedDRA version: 17.0 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: Suspension of human autologous MSC 3P in 1,5 ml
Product Code: AMSC
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: Human autologous mesenchymal stem cells
Current Spon
Sponsors
Bioinova, s.r.o.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To participate in this trial, patients will have to meet following criteria: 1. established diagnosis of definite ALS according to El Escorial kriteria and data available from detailed neurologic observation (including ALSFRS, Norris scale, spirometry, brain and spinal cord MRI) for at least 6 months prior to the study entrance, 2. riluzole naive or at stable dose for at least 2 months, 3. patients between 18–65 years, both sexes, life expectancy more than 2 years, 4. patients able to provide written informed consent. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients meeting any of the following criteria will be excluded from the participation in this study: 1. FVC less than 70% , 2. in case of primary bulbar paralysis less than 15 points on Norris bulbar scale, 3. less than 15 points on Norris spinal scale, 4. pregnancy, breastfeeding 5. coagulopathy, 6. skin infection at the site of bone marrow aspiration or application of the cell product, 7. gastrostomy, 8. any significant medical condition that would compromise the safety of the patient (e.g. recent myocardial infarction, congestive heart failure, renal failure, liver failure, cancer, systemic infection, reccurent thromboembolic disease .....), 9. alcohol or drug abuse, 10. cancer, 11. women of childbearing potential not using effective contraception (established oral contraception, intrauterine device, ligation of the uterine tube) including proven contraceptive measures taken by their sexual partners, 12. fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Safety: To assess an absence of complications at the site of intrathecal infusion and no new neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributed to the natural progression of the disease. ;Secondary Objective: Efficacy: To assess progress of the disease measured according to ALS functional rating scale (ALSFRS) and Norris scale and spirometry (FVC) after the AMSC treatment.;Primary end point(s): 1) Complication at the site of intrathecal infusion 2) New neurologic deficit (i. e. meningism, paraplegia, urinary incontinence) not attributable to the natural progression of the disease.;Timepoint(s) of evaluation of this end point: ad 1: at Visits IV, V, and VI (1 day to 3 months after treatment administration) ad 2: at Visits IV, V, VI and IX (1 day to 12 months after treatment administration) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): ALS disease progression (assessment of functional deterioration milestones);Timepoint(s) of evaluation of this end point: At 3, 6, 9, 12 and 18 months following treatment administration. | — |
Countries
Czech Republic
Contacts
Public ContactHeadquarters
Bioinova, s.r.o.
Outcome results
None listed