Amyotrophic Lateral Sclerosis MedDRA version: 21.1 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: FamC-1
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: WHARTON’S JELLY-DERIVED MESENCHYMAL STEM CELLS
Other descriptive name: WHARTON’S JELLY-DERIVED MESENCHYMAL STEM
Sponsors
Polski Bank Komórek Macierzystych JSC (PBKM)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Adult patients (at least 18 years old) 2. The minimum patient's weight is not less then 40 kg. 3. Diagnosis of sporadic ALS, definite or probable, as defined by El Escorial World Federation of Neurology criteria 4. History of ALS symptoms less than 2 years duration from the first symptoms of the disease 5. More than 6 months from diagnosis of the disease 6. Disease progression at 6 past months at least 3 points during this period of time 7. ALSFRS-R scale of at least 30 at screening appointment 8. Forced vital capacity >70% of predicted value for age, gender and height 9. Treatment with stable dose of riluzole before baseline visit (for at least 1 month) 10. Capable of providing written informed consent 11. Able to comply with study requirements and willing to follow all study procedures and follow-up visits 12. Women of child-bearing age and men with partners of child-bearing potential must agree to use two forms of contraceptive therapy throughout the course of the trial 13. Women of child–bearing age must undergo pregnancy test 14. Polish-language native speakers or patients who are proficient in the Polish language 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 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1. Pregnancy or breastfeeding 2. Tracheostomy 3. Ventilator dependence 4. Renal disease with creatinine >2mg/dl 5. Liver disease with ALT, AST or GGTP 2-fold higher than upper normal limit 6. Positive test for HBV, HCV, HIV with NAT method 7. Positive tests for syphilis 8. Any other clinically significant abnormalities on laboratory evaluation 9. Any condition that would compromise ability of undergoing lumbar puncture 10. Active systemic disease 11. Autoimmune disease (Hashimoto disease under control is allowed) 12. Uncontrolled diabetes 13. Pulmonary disease that could affect interpretation of spirometry 14. Neurological concomitant disease 15. Unstable psychiatric concomitant disease 16. High risk of suicide 17. History of substance abuse within past year 18. History of malignancy, within the previous 5 years, including melanoma with exception of localized skin cancers 19. Any other clinically significant medical condition that can compromise patient’s safety in the opinion of the investigator 20. Treatment with immunomodulatory drugs (for example immunoglobulins, corticosteroids or other immunosuppressant) in last 6 months 21. Participation in another clinical trial in last 6 months 22. Previous cellular therapy of any kind 23. Hypersensitivity to any component used in the cell culture 24. Nuchal rigidity and other signs of meningitis 25. Patients on chronic anticoagulation treatment (heparin/ warfarin/acenocumarol/(N)OAC)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to assess the number of (S)AESI rate in the whole group of patients during 123 months follow up. (S)AESI are defined as: 1. Meningitis and encephalitis. 2. Toxic encephalopathy. 3. High fever >39°C. 4. Epileptic seizures that are not connected to conditions above (meningitis, encephalitis, toxic encephalopathy, high fever). ;Secondary Objective: Secondary objectives of the ALSTEM study is to assess the safety and efficacy of investigated medication more deep and wide than in primary one. All methods of efficacy evaluations which are planned to use in a course of ALSTEM study, as ALSFRS-R scale, spirometry, muscle strength examination, memory examination, quality of life assessment, laboratory tests of blood plasma and cerebrospinal fluid or other measurements are defined particularly by secondary endpoints. ;Primary end point(s): The number of (S)AESI during treatment and 3 months follow-up period of time. (S)AESI are defined as: 1. Meningitis and encephalitis. 2. Toxic encephalopathy. 3. High fever >39°C. 4. Epileptic seizures that are not connected to conditions above (meningitis, encephalitis, toxic encephalopathy, high fever). ;Timepoint(s) of evaluation of this end point: 3 months after administration of the first IMP dose. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Disease progression assessed in ALSFRS-R scale during screening, run-in period (-60d and -30d), at baseline and at 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 and 18 month FU. 2. Pulmonary function decline assessed in spirometry during screening, run-in period (-60d and -30d), at baseline and at 1, 2, 3, 6, 9 and 12 month FU. 3. Muscle strength decline assessed in physical examination during screening, run-in period (-60d and -30d), at baseline and at 1, 2, 3, 6, 9 and 12 month FU. 4. Upper motor neuron function assessed in UMNS during screening, run-in period (-60d and -30d), at baseline and at 1, 2, 3, 6, 9 and 12 month FU. 5. Cognitive function assessed in ECAS at screening and 12 month FU. 6. Quality of life changes, assessed by EQ-5D questionnaire during screening, run-in period (-60d and -30d), at baseline and at 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 and 18 month FU. 7. The change of defined cytokines, chemokines, growth factors, cystatin C and pNFH level assessed in the samples of CSF obtained during run-in visit (-60d), at baseline and at 1, 2 and 6 month FU (12 month FU optional) 8. The change of defined cytokines, chemokines and cystatin C level assessed in the samples of blood serum obtained during screening visit, run-in period (-60 d and -30 d), at baseline and at 1, 2, 3, 6, 9 and 12 month FU. 9. The change of creatinine and p75ECD level assessed in the samples of urine obtained during screening visit, run-in period (-60 d and -30 d), at baseline and at 1, 2, 3, 6, 9 and 12 month FU. 10. Muscle function changes, assessed based on EMG examination during screening visit, at baseline and at 1, 2, 6 and 12 month FU. 11. The change of the brain visualization in MRI (T1, T2 and DTI) on the run-in visit (-60 d), 6 month FU and 12 month FU. 12. SAE/AE and (S)AESI during 18 month FU. 13. Survival period to disease progression during 18 month FU. 14. Mortality rate during 18 M follow-up. ;Timepoint(s) of evaluation of this end point: bas | — |
Countries
Poland
Contacts
Public ContactPunkt Informacyjny Badania Kliniczn
Natalia Zmijewska
Outcome results
None listed