Status:
COMPLETED
Rapamycin Treatment for ALS
Lead Sponsor:
Azienda Ospedaliero-Universitaria di Modena
Collaborating Sponsors:
University of Modena and Reggio Emilia
Azienda Ospedaliero Universitaria Maggiore della Carita
Conditions:
Amyotrophic Lateral Sclerosis
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
In the last years research has pointed out potential mechanisms of pathogenesis in ALS including lack of degradation of abnormally accumulated proteins inside motor neurons, and an unbalanced function...
Detailed Description
This is a phase II randomized, double-blind, placebo-controlled, multicenter clinical trial for people with ALS. The aim is to study the biological and clinical effect of Rapamycin (in two different ...
Eligibility Criteria
Inclusion
- 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/mm³, platelets count\<100,000/mm³, 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.
Exclusion
Key Trial Info
Start Date :
September 19 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 15 2022
Estimated Enrollment :
63 Patients enrolled
Trial Details
Trial ID
NCT03359538
Start Date
September 19 2017
End Date
February 15 2022
Last Update
September 19 2024
Active Locations (7)
Enter a location and click search to find clinical trials sorted by distance.
1
Centro Sla, Irccs A.O.U. S.Martino Ist, Genova
Genova, Italy
2
Centro Clinico Nemo, Fondazione Serena Onlus, Milano
Milan, Italy
3
Centro Sla, Irccs Istituto Carlo Besta, Milano
Milan, Italy
4
Centro Sla, Ospedale Civile S. Agostino Estense, A.O.U. Modena
Modena, Italy, 41126