Overview of drisapersen (GSK2402968/PRO051) clinical trials

Development has been terminated
Phase I local treatment (NTR712)
• van Deutekom JC, Janson AA, Ginjaar IB, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. The New England journal of medicine 2007;357:2677-86.
van Deutekom_2007_N Engl J Med. Dec 27;357(26);2677-86

Phase II open-label trial (NTR1241) and extension study (NCT01910649)
• Goemans NM, Tulinius M, van den Akker JT, et al. Systemic administration of PRO051 in Duchenne’s muscular dystrophy. The New England journal of medicine 2011;364:1513-22.
Goemans_2011_N Engl J Med. Apr 21;364(16);1513-22
• Goemans NM, Tulinius M, van den Hauwe M, et al. Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study. PloS one 2016;11:e0161955.
Goemans_2016_PLoS One. Sep 2;11(9);e0161955

• Goemans N, Tulinius M, Kroksmark AK, Wilson R, van den Hauwe M, Campion G. Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids. Neuromuscular disorders : NMD 2017;27:203-213
Goemans_2017_Neuromuscul Disord. Mar;27(3);203-213

• Hilhorst N, Spanoudi-Kitrimi I, Goemans N, Morren MA. Injection site reactions after long-term subcutaneous delivery of drisapersen: a retrospective study. European journal of pediatrics 2019;178:253-258.

Hilhorst_2019_Eur J Pediatr. Feb;178(2);253-258

Phase I placebo-controlled trial (NCT01128855; DEMAND I)
• Flanigan KM, Voit T, Rosales XQ, et al. Pharmacokinetics and safety of single doses of drisapersen in non-ambulant subjects with Duchenne muscular dystrophy: results of a double-blind randomized clinical trial. Neuromuscular disorders : NMD 2014;24:16-24.
Flanigan_2014_Neuromuscul Disord. Jan;24(1);16-24

Phase II placebo-controlled trial (NCT01153932; DEMAND II) and open-label extension (NCT01480245)
• Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. The Lancet. Neurology 2014;13:987-96
Voit_2014_Lancet Neurol. Oct;13(10);987-96

Phase II placebo-controlled trial (NCT01462292) and open-label extension (NCT01803412/ NCT01890798)
• McDonald CM, Wong B, Flanigan KM, et al. Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy. Annals of clinical and translational neurology 2018;5:913-926.
McDonald_2018_Muscle Nerve. Nov;58(5);614-617

Phase III placebo-controlled trial (NCT01254019) and open-label extension (NCT01803412/ NCT01480245/ NCT01890798)
• Goemans N, Mercuri E, Belousova E, et al. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. Neuromuscular Disorders 2018;28:4-15
Goemans_2018_Neuromuscul Disord. Jan;28(1);4-15

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