welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
study id #: NCT03218995
condition: Duchenne Muscular Dystrophy
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, PK, and efficacy of once-weekly IV infusions of eteplirsen in approximately 12 male patients, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.
mechanism of action: Exon-skipping to promote dystrophin production
last updated: December 28, 2018
start date: August 16, 2017
estimated completion: August 31, 2020
phase of development: Phase 2
size / enrollment: 12
- Incidence of adverse events [Time Frame: Up to 96 Weeks]
- Abnormal changes from baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis) [Time Frame: Change from Baseline]
- Abnormal changes from baseline or worsening of vital signs [Time Frame: Change from Baseline]
- Abnormal changes from baseline or worsening of physical examination findings [Time Frame: Change from Baseline]
- Abnormal changes from baseline or clinically significant worsening of electrocardiogram (ECG) and echocardiogram (ECHO) [Time Frame: Change from Baseline]
- Maximum plasma concentration [Time Frame: 24 Weeks]
- Time of Cmax (Tmax) [Time Frame: 24 Weeks]
- Area under the concentration-time curve (AUC) [Time Frame: 24 Weeks]
- Apparent volume of distribution at steady state (Vss) [Time Frame: 24 Weeks]
- Clearance (CL) [Time Frame: 24 Weeks]
- Elimination half-life (t½) [Time Frame: 24 Weeks]
- Amount of drug eliminated in urine (Ae%) [Time Frame: 24 Weeks]
• Male between 6 months to 48 months of age (inclusive)
• Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping
• Parent(s) or legal guardian(s) who is willing to provide written informed consent
• Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing
• Received previous or current treatment with any experimental treatment
• Clinically significant illness other than DMD
• Clinically significant laboratory abnormality
• Any other condition that could interfere with the patient's participation
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)This is a Phase 2, open-label, single ar...
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular DystrophiesThe study will include 120 participants ...
ICER Releases Draft Evidence Report on Treatments for Duchenne Muscular DystrophyThe Institute for Clinical and Economic ...
Sarepta Receives Negative CHMP Re-examination Opinion for EteplirsenSarepta Therapeutics, Inc., a commercial...
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)Duchenne muscular dystrophy (DMD) is a g...
Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dyst...Duchenne/Becker muscular dystrophy (DMD/...
A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged 2 to 5 years with nonsense mutation ...Nonsense mutation Duchenne muscular dyst...
Wave Life Sciences Duchenne Muscular Dystrophy Clinical Trial Selected for FDA Complex Innovative Trial Designs Pilo...Wave Life Sciences Ltd., a biotechnology...
Local boy with Duchenne muscular dystrophy begins promising gene-targeted therapySeven-year-old Wyatt Hubbard was a bit t...