Amyotrophic Lateral Sclerosis MedDRA version: 14.0 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: EPREX*1SIR 40000UI/ML 1ML
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ERYTHROPOIETIN
CAS Number: 11096-26-7
Concentration unit: U/ml unit(s)/millilitre
Concentration t
Sponsors
ISTITUTO NEUROLOGICO "CARLO BESTA"
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: STUDY POPULATION: 60 patients with diagnosis of laboratory-supported probable, probable, or definite ALS according to El Escorial revised criteria. PATIENTE ELIGIBILITY: 1. age 18-75 years 2. diagnosis of sporadic or familial ALS 3. onset of weakness =65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: EXCLUSION CRITERIA: 1. hematocrit >49% in men and >47% in women, hemoglobin value >15 g/dl in men and >13 g/dl in women 2. tracheotomy 3. clinical diagnosis of frontotemporal dementia, Parkinson or Alzheimer disease 5. history and/or instrumental evidence of previous myocardial infarction or thrombotic vascular events (such as stroke, transient ischemic attack, pulmonary emboli, retinal thrombosis, deep vein or arterial thrombosis including asymptomatic carotid and/or vertebral stenosis >30%) 6. clinically evident cardiac disease (ischemic heart disease, congestive heart failure, arrhythmia) 7. uncontrolled hypertension (systolic blood pressure >160 mmHg and diastolic blood pressure >95 mmHg irrespective of anti-hypertensive treatments at two consecutive evaluations) 8. active malignancy, polycythemia, myeloproliferative disorders 9. hypercoagulable disorders (ref. Deitcher S, in Current Clinical Medicine 2009, Cleveland Clinic ed., Elsevier) 10. porphyria 11. known hypersensitivity to human albumin 12. female subjects pregnant or lactating 13. use of post-menopausal drugs (estrogen, progestinic, or their combination) 14. use of hormonal contraception drugs 15. use of experimental drug or participation in a clinical trial within 3 months prior to screening 16. previous treatment with hematopoietic stem cells
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The project aims to assess: 1) safety and tolerability of rhEPO given at different doses and by different routes; 2) modulation of potential biomarkers involved in the neuroprotective activity of EPO; 3) percentage of rhEPO crossing the BBB in ALS patients. Since ALS patients are at risk for some events included in the primary outcome (e.g. venous thrombosis), a placebo arm will be included.;Secondary Objective: To assess safety and tolerability of rhEPO administered iv for 4 months or sc open in 60 patients with ALS. Patients will be treated fortnightly with 120,000, 80,000, or 40,000 IU / ml. Safety will be analyzed in relation to adverse events associated with the erythropoietic activity of rhEPO and compared to the placebo arm. Biomarkers will be investigated including EPCs, VEGF and rhEPO modulated by NO. It will assess the percentage of rhEPO through the BBB.;Primary end point(s): Discontinuation of treatment because of hematocrit or haemoglobin values, adverse events, or death;Timepoint(s) of evaluation of this end point: 4 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Changes in biomarkers analyzed between treatment groups;Timepoint(s) of evaluation of this end point: 4 months | — |
Countries
Italy
Contacts
Public ContactServizio Ricerca e Sviluppo Clinico
Fondazione IRCCS Istituto Neurologico Carlo Besta
Outcome results
None listed