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An Expanded Access Protocol of RNS60 in Amyotrophic Lateral Sclerosis
Author(s) -
Addy Grace,
Scirocco Erica,
Gelevski Dario,
Rohrer Margot,
Roderick Aimee,
McCormack Mary,
Weiss Sadan Anat,
Scalia Jennifer,
Parikh Neil,
Giacomelli Elisa,
Locatelli Matteo,
Neel Dylan V.,
D'Agostino Derek,
Leite Alex,
Yu Hong,
Sherman Alexander V.,
Mock Jarrad,
Kalmes Andreas,
Luppino Sarah,
Babu Suma,
Berry James,
Cudkowicz Merit,
Pagai Sabrina
Publication year - 2025
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.28398
Subject(s) - medicine , amyotrophic lateral sclerosis , tolerability , adverse effect , clinical trial , placebo , randomized controlled trial , expanded access , physical therapy , disease , pathology , alternative medicine
ABSTRACT Aims RNS60 is an investigational product in clinical development for amyotrophic lateral sclerosis (ALS). RNS60 slowed disease progression in the ALS SOD1 G93A mouse model and was safe and well tolerated both in an open‐label pilot study and a randomized, placebo‐controlled, multicenter phase 2 trial in people living with ALS. The objective of this ongoing expanded access protocol (EAP) was to provide RNS60 to people living with ALS who are ineligible for controlled clinical trials and to collect data on the safety and tolerability of dosing RNS60 via twice‐daily nebulization rather than the previously studied daily nebulization with weekly intravenous administration. Methods Eligible participants (≥ 18 years old, diagnosed with ALS per investigator assessment, and ineligible for an ALS clinical trial testing RNS60) were treated with twice‐daily nebulization of RNS60 at home. Safety was evaluated by the assessment of adverse events and routine safety labs. Results A total of 84 participants have been treated with RNS60 via nebulization twice daily for up to 48 months so far. The most common treatment‐related adverse event was increased secretions [ N = 27 (32%)]. Serious adverse events (SAEs) [69 occurrences; N = 38 (45%) with at least one SAE] and deaths [ N = 24 (28%)] were deemed not related to RNS60. Discussion This EAP supports the benign side effect profile of RNS60 when administered via twice‐daily nebulization and demonstrates the feasibility of long‐term EAPs as a complementary approach to controlled trials in people with advanced ALS.