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Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
study id #: NCT01847573
condition: Duchenne Muscular Dystrophy
The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.
mechanism of action: Small molecule designed to reduce fibrosis and inflammation
last updated: August 01, 2019
start date: May 2013
estimated completion: March 30, 2016
phase of development: Phase 1/Phase 2
size / enrollment: 17
- Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys [Time Frame: 1 week]
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
- Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys [Time Frame: 1 week]
Halofuginone plasma concentrations
- Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks [Time Frame: 4 weeks]
Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing
- Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys [Time Frame: 4 weeks]
Pharmacodynamic measures relevant to DMD pathology:
• Pulmonary function
• Motor function
• Muscle composition
• Biochemical and imaging markers
• Ambulatory or non-ambulatory
• Diagnosis of DMD with confirmation of minimal to no dystrophin
• Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen)
• Recent, substantial change in use of cardiac medications or medications affecting muscle function
• Inability to undergo magnetic resonance imaging (MRI)
• Significantly compromised cardio-respiratory function
• Prior treatment with another investigational product in past 6 months
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