Phase 2CompletedAge-related condition
Rapamycin Treatment for ALS (RAP-ALS)
Rapamycin (Sirolimus) Treatment for Amyotrophic Lateral Sclerosis
- Sponsor
- Azienda Ospedaliero-Universitaria di Modena (Academic or other)
- Enrollment
- 63 enrolled
- Conditions
- Amyotrophic Lateral Sclerosis
- Interventions
- Rapamycin; Placebo Oral Tablet
- Ages
- adults, older adults
- Registry
- NCT03359538
Full record on ClinicalTrials.gov
From the registry
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 of the immune system leading to the prevalence of a neurotoxic function over neuroprotection. These two mechanisms contribute to ALS progression hence representing important therapeutic targets to modify disease expression. With a phase II clinical trial the investigators aim to study the biological response in ALS treated with Rapamycin, to obtain predictive information for a larger study. Eight Italian Centres will enroll 63 patients; treatment will be double blinded to patients and physicians, and will last 18 weeks.Follow up will be carried out for 36 months (total duration: 54 weeks).
Primary outcomes
- T-reg number
History
Last registry update
Study completion
Primary completion
Registered
Started
Papers citing this trial
- Metabolic effects of Rapamycin in Amyotrophic Lateral Sclerosis patients: secondary analysis of an RCT
Frontiers in pharmacology
- Co-deposition of SOD1, TDP-43 and p62 proteinopathies in ALS: evidence for multifaceted pathways underlying neurodegeneration
Acta neuropathologica communications