Amyotrophic lateral sclerosis (ALS) is a severe progressive neurological disorder characterized by a selective degeneration of spinal, bulbar, and cortical motor neurons.
Conditions
Interventions
Trade Name: TEVAGRASTIM
Product Name: filgrastim
Pharmaceutical Form: Injection
Pharmaceutical form of the placebo: Injection
Route of administration of the placebo: Intravenous use
Trade Name: MANNI
Sponsors
Università degli Studi di Torino e Azienda Ospedaliera Città della Salute e della Scienza di Torino
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: a. patients with definite, probable, probable laboratory-supported or possible ALS b. age 20-75 years; c. disease duration =65 years) yes F.1.3.1 Number of subjects for this age range 90
Exclusion criteria
Exclusion criteria: a. previous polio infection ; b. motor neuron diseases other than ALS (progressive bulbar palsy , progressive muscular atrophy , primary lateral sclerosis ); c. clinical involvement of other neurological systems (sensory, extrapyramidal , oculomotor, cerebellar); d. serious clinical conditions such as cardiovascular disease, uncontrolled hypertension, renal or hepatic impairment , dysthyroidism , Respiratory Distress Syndrome (ARDS) , sickle-cell anaemia; e. subjects who have participated in any drug trial within 12 weeks prior to recruitment; f . concomitant malignancy, present or past, with the exception of different melanoma skin cancers and carcinoma in situ of the cervix; g . patients with severe congenital neutropaenia syndrome with abnormal cytogenetics; h . patients with rare hereditary problems of fructose intolerance; i . present or past malignant myeloproliferative diseases, secondary polycythemia, splenomegaly with a diameter> 14 cm, thrombophilia was found; j . presence of percutaneous gastrostomy at the time of recruitment ; k . presence of NIV at the time of recruitment; l. subjects who have participated in any drug trial within 12 weeks prior to recruitment; n . drug addiction , alcoholism, or psychiatric disorders; o . women who are pregnant or breastfeeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: assessment of the safety and efficacy of granulocyte-colony stimulating factor compared with placebo in ALS patients, evaluated with a placebo-controlled, double blind, parallel groups design.;Secondary Objective: evaluation of cytokine and chemokine levels in the cerebrospinal fluid and serum of patients before and after the treatment with granulocyte-colony stimulating factor and placebo;Primary end point(s): a. modification of progression rates of total disability score (ALS-FRS-R) during the 72 weeks of the study; b. modification of manual muscle testing scores (MRC scale), FVC and QoL during the 72 weeks of the study; c. time to death, tracheostomy or use of Non-Invasive Ventilation =18 h/day; d. evaluation of treatment safety and tolerability ;Timepoint(s) of evaluation of this end point: all the end points will be assessed for the entire duration of the study at week 0, 4, 12, 24, 36, 48 and including time point of follow-up at weeks 60 and 72. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): a. modification of progression rates of total disability score (ALS-FRS-R) during the 72 weeks of the study; b. modification of manual muscle testing scores (MRC scale), FVC and QoL during the 72 weeks of the study; c. time to death, tracheostomy or use of Non-Invasive Ventilation =18 h/day; d. evaluation of treatment safety and tolerability ;Timepoint(s) of evaluation of this end point: all the end points will be assessed for the entire duration of the study at week 0, 4, 12, 24, 36, 48 and including time point of follow-up at weeks 60 and 72. | — |
Countries
Italy
Contacts
Public ContactCRESLA, SCDU Neurologia 2
Università degli Studi di Torino e Azienda Ospedaliera Città della Salute e della Scienza di Torino
Outcome results
None listed