Study evaluating the treatment with colchicine for the amyotrophic lateral sclerosis

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 54

Inclusion Criteria

- Patients diagnosed with a laboratory supported , clinically probable” or definite” amyotrophic lateral sclerosis according to the Revised El Escorial criteria (Brooks, 2000)
- Sporadic ALS
- ALS phenotypes: classic or bulbar
- Female or male patients aged between 18 and 80 years old
- Disease duration from symptoms onset no longer than 18 months at the screening visit
- Patients treated with a stable dose of Riluzole (100 mg/day) for at least 30 days prior to screening
- Patients with a weight > 50 kg and a BMI =18
- Patients with a FVC (Forced Vital Capacity) equal or more than 65 % predicted normal value for gender, height, and age at the screening visit
- Patients able and willing to comply with study procedures as per protocol
- Patients able to understand, and capable of providing informed consent at screening visit prior to any protocol-specific procedures
- Use of highly effective contraception both for males and females
Are the trial subjects under 18? no
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 29
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 25

Exclusion Criteria

- Prior use of colchicine
- Prior allergy/sensitivity to colchicine
- Receiving colchicine or other anti-inflammatory drugs (such as corticosteroids, methotrexate, anti-neoplastic, Interleukin 1-1b antagonist, Tumor necrosis factor-alpha inhibitor)
- Receiving food or co-medications such as strong-moderate cytochrome P450 3A4 inhibitors that will result in elevated plasma level of colchicine
- Inflammatory disorders (SLE, Rheumatoid arthritis, connective tissue disorder) or chronic infections (HIV, hepatitis B or C infection) or significant history of malignancy
- Severe renal (eGFR< 30ml/min/1.73m2), or liver failure or liver aminotransferase (ALT/AST > 2x Upper limit of normal)
- Existing blood dyscrasia (e.g., myelodysplasia)
- White blood cells<4,000/mm³, platelets count<100,000/mm³, hematocrit<30%
- Severe comorbidities (heart, renal, liver failure), autoimmune diseases or any type of interstitial lung disease
- Patients who underwent non invasive ventilation, tracheotomy and /or gastrostomy
- Women who are pregnant or breastfeeding
- Participation in pharmacological studies within the last 30 days before screening
- Patients with known pathogenic mutations (SOD1, TARDBP, FUS, C9ORF72)
- Patients with familial ALS defined as presence of at least one first degree family member (parents/son/daughter/brother/sister) affected by ALS
- Patients with the following ALS phenotypes: flail arm, flail leg, UMN-p, respiratory, PLS, progressive muscular atrophy