Colchicine for Amyotrophic Lateral Sclerosis

Colchicine for Amyotrophic Lateral Sclerosis

Quick Info:

Status:
Not Yet Recruiting
Estimated Enrollment:
54
Phase:
2
Treatment Type:
Drug: Colchicine 1 MG Oral Tablet
Trial Type:
Randomized, Parallel Assignment, double blind
Sponsor:
Azienda Ospedaliero-Universitaria di Modena
Primary Investigator:
Contact Information:

Enrollment Criteria:

Forced Ventilation

Breathing Ability

Percent lung function (FVC) or (SVC)
≥65%
Months/Onset

Months Since Onset

Number of months since first
symptoms of ALS
<18 months
BiPap Allowed

Non-Invasive Ventilation (NIV)

Can PALS use a BiPAP in the trial?
Unknown
DPS Allowed

Diaphragm Pacer (DPS)

Can PALS use a DPS in the trial?
Unknown
Edaravone Usage

Edaravone Usage

Can a PALS use edaravone (Radicut/Radicava)
while enrolled in the trial?
Unknown

Update Notes:

11/30/2018No significant updates
10/3/2018Trial added

Locations:

Centro Sla, University of Bari , Bari
Centro Sla, Istituto Auxologico Italiano, Universi, Milano
Irccs St. Raffaele Institute of Milano , Milano
Centro Sla, Ospedale Civile S. Agostino Estense, A, Modena
Università della Campania Gianluigi Vanvitelli , Napoli
Centro Sla, Universita' Di Padova , Padova
Als Centre, "C. Mondino" National Neurological Ins University of Pavia, Pavia
, Neuromuscular Omnicentre Centre, Rome Catholic University, Rome, Roma
Centro Sla, Universita' Di Torino , Torino

Other Information:

Purpose: The study evaluates the effects of two different Colchicine doses (0.01mg/kg/day or 0.005 mg/kg/day) compared to placebo in Amyotrophic Lateral Sclerosis (ALS) patients. Disease progression as defined by changes in ALSFRS-r is the primary outcome measure. Other measures of clinical progression and survival, together with safety and tolerability of Colchicine in ALS patients will be assessed.
Eligibility: between 18 and 80 years, all genders, not accepting healthy volunteers
Details: Recent evidence supports the disruption of the ubiquitin-proteasome-system and autophagy as central events in ALS. ALS is characterized by the presence of misfolded proteins prone to oligomerize into aggregates, which exert a toxic effect by affecting several intracellular functions. Heat shock protein B8 (HSPB8) recognizes and promotes the autophagy-mediated removal of misfolded mutant SOD1 and TDP-43 fragments from ALS motor neurons (MNs). Moreover, HSPB8-BAG3-HSP70 maintains the so called "granulostasis", a surveillance mechanism that avoids the conversion of dynamic stress granules (SGs) into aggregation-prone assemblies, which are a hallmark of ALS. Colchicine enhances the expression of HSPB8 and of several autophagy players while blocking TDP-43 accumulation in neurons. Moreover, given the cross-talk between infalmmation and autophagy, the well-known antinflammatory action of Cochicine may contribute to cell homeostasis. Based on these premises, this is a phase II randomized, double-blind, placebo-controlled, multicenter (9 MND Centres in Italy: 2 centres in Milan, Pavia, Turin, Modena, Padua, Rome, Naples, Bari), clinical trial to test efficacy of Colchicine in ALS.
Collaborator(s):
News Articles and Summaries:
ALS Forum:
First Published on Clinicaltrials.gov: 10/3/2018
ClinicalTrials.gov ID: NCT03693781
Trial Protocol as Published on Clinicaltrials.gov:
ClinicalTrials.gov