welcome to DuchenneXchange- a positively charged Duchenne muscular dystrophy community.
- join today!
- log in
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
study id #: NCT03218995
condition: Duchenne Muscular Dystrophy
status: active, not recruitingpurpose:
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: April 08, 2020
start date: August 16, 2017
estimated completion: March 15, 2021
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]
• Eligible Sexes: male
• 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
Long-Term Treatment With Eteplirsen in Nonambulatory Patients With Duchenne Muscular DystrophyThis analysis aims to describe the outco...
ICER Releases Draft Evidence Report on Treatments for Duchenne Muscular DystrophyThe Institute for Clinical and Economic ...
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular DystrophyThis is a multicenter, double blind, pla...
Phase 1/2 Study in Boys With Duchenne Muscular DystrophyThe MoveDMD study is a 3-part, Phase 1/2...
MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dys...NF-κB is activated from infancy in DMD,...
Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)Duchenne/Becker muscular dystrophy (DMD/...
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular DystrophyThis study is single arm, single center ...
Capricor Therapeutics Announces Positive Results from its Interim Analysis in the HOPE-2 Trial to Treat Patients wit...Capricor Therapeutics, Inc. (NASDAQ: CAP...
PhaseOut DMD: a Phase 2, proof of concept, clinical study of utrophin modulation with ezutromidThis study investigates the hypothesis t...