Clinical Trial: ALS Association and ALS Finding a Cure® Supported Phase II RNS60 Trial is Now Enrolling

The clinical trial to test RNS60, a new compound for the treatment of ALS, which was supported in part from a $1.0 million grant through the ALS ACT initiative funded by The ALS Association and ALS Finding A Cure®, is now actively enrolling. This randomized placebo-controlled phase II study is being run by the IRCCS Mario Negri Institute for Pharmacological Research in Milan and the ALS Center of the Maggiore University Hospital in Novara Italy. We are happy to report that the first patient was dosed in mid-July 2017.

The study is carried out in collaboration with the University Hospital AOU Maggiore della Carità, Novara, Italy and involves 20 Italian ALS centers along with the Massachusetts General Hospital in Boston. See below for the trial site list. The trial is led by neurologists Dr. Ettore Beghi (LEFT), at IRCCS Mario Negri Institute for Pharmacological Research in Milan; and Dr. Letizia Mazzini (RIGHT), at the University Hospital of Novara in Novara, Italy; and Dr. Sabrina Paganoni, at Massachusetts General Hospital in Boston.

The phase II trial will test the impact of RNS60 on selected pharmacodynamic and clinical markers in 142 ALS patients concurrently treated with riluzole and followed for 12 months. RNS60 and placebo will be given intravenously on a weekly basis and by inhalation in the remaining days for a period of 6 months. Patients will be followed for 6 additional months to measure long-term clinical outcomes. The trial is designed to assess the effects of the drug on selected disease markers and to correlate the changes in these biological markers to changes in disease progression.

RNS60 is a novel compound that is active on the immune system and inflammation, two mechanisms implicated in ALS disease processes. The drug protects the integrity of neural cells by specifically modifying inflammatory pathways.

RNS60 exerts a protective effect on motor neurons in ALS cell and animal models. For example, when treated with RNS60, the well characterized SOD1G93A mouse model demonstrated a delay in the progression of symptoms and prolonged survival.

“In a pilot trial of RNS60 in ALS patients done at the Massachusetts General Hospital, the drug was safe and well-tolerated” – Dr. Sabrina Paganoni, co-investigator for the study and physician at the Massachusetts General Hospital and Spaulding Rehabilitation Hospital in Boston, MA.

paganoni
Dr. Sabrina Paganoni

The RNS60 trial is also supported by the Get OUT Onlus Association and the Fondazione Banco Popolare, Novara. Revalesio Corporation in Tacoma, Wash. provides the active RNS60 treatment and placebo.

For further information, please contact rns60@marionegri.it

For more information, including the contact information for the Massachusetts General Hospital trial site will be made available on clinicaltrials.gov and on the Northeast ALS (NEALS) Consortium clinical trials page.

Trial sites:

Massachusetts General Hospital, Boston (USA)

Azienda Ospedaliera Universitaria “Maggiore della Carità”, Novara, Italy

Azienda Ospedaliero-Universitaria Pisana, Pisa, Italy

IRCCS Azienda Ospedaliera Universitaria “San Martino” IST, Genova, Italy

IRCCS Istituto Neurologico Nazionale “C. Mondino”, Pavia, Italy

Azienda Ospedaliera Universitaria Città della Salute e della Scienza di Torino, Italy

Azienda Ospedaliera “Spedali Civili” ed Università degli Studi di Brescia, Italy

Azienda Ospedaliera Universitaria Senese, Siena, Italy

Policlinico Umberto I – Università di Roma “La Sapienza”, Roma, Italy

Azienda Ospedaliera Universitaria Policlinico “Paolo Giaccone”, Palermo, Italy

Azienda Ospedaliera Pia Fondazione “Card. G. Panico”, Tricase (Lecce) , Italy

Centro Clinico NEMO-Fondazione Serena Onlus, Milano, Italy

Presidio Ospedaliero Provinciale – Nuovo Ospedale Civile “S. Agostino Estense” di Modena, Italy

Azienda Ospedaliera Universitaria della Seconda Università degli Studi di Napoli, Italy

ASST Valle Olona-Presidio Ospedaliero di Gallarate (Varese), Italy

IRCCS Ospedale “San Raffaele”, Milano, Italy

Azienda Ospedaliera Universitaria Consorziale Policlinico, Università degli studi Bari, Italy

Azienda Ospedaliera di Padova-Università degli studi di Padova, Italy

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