welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
study id #: NCT02667483
condition: Duchenne Muscular Dystrophy
status: active, not recruitingpurpose:
This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: February 15, 2019
start date: October 2015
estimated completion: December 2019
phase of development: Phase 1/Phase 2
size / enrollment: 7
- Number of participants with treatment-emergent adverse events (TEAEs) by the end of the trial [Time Frame: 48 Weeks of Part 2-Extension]
TEAEs are adverse events (including clinically significant laboratory values) temporally associated with use of DS-5141b, whether or not attributable to the product.
- Maximum concentration (Cmax) of DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Area under the curve (AUC) for DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Time to maximum concentration (Tmax) of DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Half-life (T1/2) of DS-5141b [Time Frame: Week 48 of Part 2-Extension]
- Dystrophin protein expression in muscle tissue [Time Frame: Week 48 of Part 2-Extension]
- Production of exon 45-skipped dystrophin mRNA in muscle tissue [Time Frame: Week 48 of Part 2-Extension]
• Confirmation of out-of-frame deletion(s) that could be corrected by dystrophin gene exon 45 skipping.
• Intact muscles of adequate quality for biopsy to allow evaluation of the efficacy of the study drug.
• Boys aged from 5 years to <11 years.
• Patients able to walk at least 325 meters in the 6-minutes walk test.
• Glucocorticoid-naive patients, or patients who have used glucocorticoids for at least 6 months prior to enrollment in this study with no dose changes for at least 3 months prior to enrollment
• A genetic mutation that cannot be expected the expression of dystrophin protein by dystrophin gene exon 45 skipping.
• A concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
• Current or history of severe disorder.
• Left ventricular ejection fraction (LEVF) <55%.
• Corrected QT interval (QTc) >0.45 sec.
Study of Ataluren in >=2 to <5 Year-Old Males With Duchenne Muscular DystrophyThis is a Phase 2, multiple-dose, open-l...
PTC Therapeutics Announces Positive Data from its Translarna™ Phase II Clinical Trial in Children as Young as Two ...PTC Therapeutics, Inc. today announced t...
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Musc...This is a multi-center, randomized, doub...
Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, ran...Background: Duchenne muscular dystrophy...
PhaseOut DMD: a Phase 2, proof of concept, clinical study of utrophin modulation with ezutromidThis study investigates the hypothesis t...
First Patient Dosed in Phase II Clinical Trial in Duchenne Muscular DystrophyAntisense Therapeutics today announced d...
Catabasis Pharmaceuticals Announces Publication Of Phase 1 Clinical Results Of Edasalonexent (CAT-1004) In Duchenne ...Catabasis Pharmaceuticals, Inc., a clini...