Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations

Phase 1 Completed 33 enrolled Ionis Pharmaceuticals, Inc. ALS Association · collab Muscular Dystrophy Association · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

This study will test the safety, tolerability and pharmacokinetics of single doses of ISIS 333611 administered into the spinal canal as 12 hour infusions.

Timeline

Start: 2010-01
Primary completion: 2011-12
Completion: 2012-01

Outcome

Met primary endpoint

paper ISIS 333611 was well tolerated when administered as an intrathecal infusion. PMID 23541756 ↗

Publications

Miller TM, Pestronk A, David W, Rothstein J, Simpson E, Appel SH, Andres PL, Mahoney K, Allred P, Alexander K, Ostrow LW, Schoenfeld D, Macklin EA, Norris DA, Manousakis G, Crisp M, Smith R, Bennett CF, Bishop KM, Cudkowicz ME. An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol. 2013 May;12(5):435-42. doi: 10.1016/S1474-4422(13)70061-9. Epub 2013 Mar 29.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ISIS 333611	Antisense oligonucleotide	0.15 mg	Intrathecal
Subject	ISIS 333611	Antisense oligonucleotide	0.5 mg	Intrathecal
Subject	ISIS 333611	Antisense oligonucleotide	1.5 mg	Intrathecal
Subject	ISIS 333611	Antisense oligonucleotide	3 mg	Intrathecal