A Phase 3 study of Viltolarsen in boys with DMD
Research type
Research Study
Full title
A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)
IRAS ID
269244
Contact name
Minal Kara
Contact email
Sponsor organisation
NS Pharma Inc
Eudract number
2019-002076-13
Duration of Study in the UK
2 years, 5 months, 0 days
Research summary
Summary of Research
Duchenne muscular dystrophy (DMD) is a disorder of progressive weakness leading to severe disability and ultimately death caused by a deficiency of the dystrophin protein. The symptoms of DMD are often first noted at about 3 to 5 years of age. Current treatment of DMD is with glucocorticoid however these have significant side effects.The study aims to measure how well the study drug (viltolarsen) works by performing strength and function assessments throughout the study. The study will also measure the safety of viltolarsen and how boys tolerate it.
Boys aged 4 to 7 will receive intravenous (IV) (into their vein) infusions of viltolarsen injection or placebo administered once weekly over a 48-week period. Participants will be dosed at 80 mg/kg/ week.
The placebo will look like viltolarsen, but it will not contain active ingredients.This is a randomised, placebo-controlled, double−blind study meaning neither the participant, their parent/ guardian nor the study doctor will know which medication is being given.
NS Pharma Inc are sponsoring this multicentre study and it is anticipated that approximately 74 participants will be randomised to viltolarsen or placebo in a 1:1 ratio across Europe, Asia, and North and south America.
Summary of Results
This was a Phase 3 study to evaluate the efficacy, safety, and PK of viltolarsen in ambulant boys with Duchenne Muscular Dystrophy (DMD) with ages 4 to <8 years.
This study was a randomised, double-blind, placebo-controlled, multi-center study meaning participants were randomly assigned to receiving vitolarsen or placebo. Participants had an equal chance of receiving vitolarsen or placebo. The placebo looked the same as vitolarsen, but without the active ingredients.
Participants received 80 mg/kg viltolarsen administered intravenously (IV) weekly over a 48-week treatment period. In this study, treatment with viltolarsen was well tolerated.
The primary objective was to compare the efficacy of viltolarsen administered IV at weekly doses of 80 mg/kg over a 48-week treatment period versus placebo controls in boys aged 4 to <8 years with DMD using the Time to Stand Test (TTSTAND) as a measure of strength and function.
The primary efficacy conclusion was that there was no statistically significant difference in the change from baseline in the primary endpoint, TTSTAND (velocity), between the 2 treatment groups.
The inability to observe statistically significant improvements compared to placebo (ie, standard of care) in the functional assessments used in this Phase 3 study suggested the difficulty of conducting studies in DMD patients, who are highly heterogeneous.https://gbr01.safelinks.protection.outlook.com/?url=https%3A%2F%2Fclick.pstmrk.it%2F3ts%2Fclinicaltrials.gov%252Fstudy%252FNCT04060199%253Fa%253D31%2FNBTI%2Fya65AQ%2FAQ%2F1585723c-9022-4f02-8a9f-00d0e604db30%2F1%2FjZCsOoC-r3&data=05%7C02%7Csheffield.rec%40hra.nhs.uk%7C12bb864e78cb401c464808dd1067e8e7%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638684760651744778%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=%2BhJO2bSyBkGLq5iNrZ%2B3vxwL1yBGCdg6p8X%2BfE2I60A%3D&reserved=0
REC name
Yorkshire & The Humber - Sheffield Research Ethics Committee
REC reference
19/YH/0372
Date of REC Opinion
2 Mar 2020
REC opinion
Further Information Favourable Opinion