Rapamycin Treatment for ALS

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 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).

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

DRUG

Rapamycin

tablets containing Rapamycin/placebo will be administered based on body surface area and adjusted taking into consideration plasma rapamycin dosage

DRUG

Placebo Oral Tablet

tablets containing Rapamycin/placebo will be administered based on body surface area and adjusted taking into consideration plasma rapamycin dosage

Sponsors & Collaborators

• University of Modena and Reggio Emilia

  collaborator OTHER

• Azienda Ospedaliero Universitaria Maggiore della Carita

  collaborator OTHER

• IRCCS Azienda Ospedaliera Universitaria San Martino - IST Istituto Nazionale per la Ricerca sul Cancro, Genoa, Italy

  collaborator OTHER

• University of Turin, Italy

  collaborator OTHER

• Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

  collaborator OTHER

• Azienda Ospedaliera Niguarda Cà Granda

  collaborator OTHER

• Fondazione Salvatore Maugeri

  collaborator OTHER

• University of Padova

  collaborator OTHER

• Azienda Ospedaliero-Universitaria di Modena

  lead OTHER

Principal Investigators

• Jessica Mandrioli, MD · Azienda Ospedaliero-Universitaria di Modena

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-09-19

Primary Completion

2020-12-15

Completion

2022-02-15

Countries

• Italy

Study Locations