Last updated on February 2018

Rapamycin Treatment for ALS


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Amyotrophic Lateral Sclerosis
  • Age: Between 18 - 75 Years
  • Gender: Male or Female

Inclusion criteria:

  • Patient diagnosed with a laboratory supported , clinically "probable" or "definite" amyotrophic lateral sclerosis according to the Revised El Escorial criteria (Brooks, 2000)
  • Familial or sporadic ALS
  • Female or male patients aged between 18 and 75 years old
  • Disease duration from symptoms onset no longer than 18 months at the screening visit
  • Patient 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
  • Patient with a FVC 70 % predicted normal value for gender, height, and age at the screening visit
  • Patient able and willing to comply with study procedures as per protocol
  • Patient able to understand, and capable of providing informed consent at screening visit prior to any protocol-specific procedures
  • Use of effective contraception both for males and females

Exclusion Criteria:

  • Prior use of Sirolimus
  • Prior allergy/sensitivity to Sirolimus or macrolides
  • Any medical disorder that would make immunosuppression contraindicated, including but not limited to, acute infections requiring antibiotics, patients with known diagnosis of HIV, tuberculosis, hepatitis B or C infection or history of malignancy
  • Severe comorbidities (heart, renal, liver failure), autoimmune diseases or any type of interstitial lung disease
  • White blood cells<4,000/mm, platelets count<100,000/mm, hematocrit<30%
  • Patient 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 superoxide dismutase 1 (SOD1) mutation or with familial ALS and a family member carrying SOD1 mutation.

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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