Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
- Registration Number
- NCT01847573
- Lead Sponsor
- Processa Pharmaceuticals
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 17
- 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)
Main
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Cohort 5: HT-100 tablet, Dose 6 HT-100 \* Multiple dose administration: Dose 5 Cohort 2: HT-100 tablet, Dose 2 HT-100 * Single dose administration: Dose 2 * Multiple dose administration: Dose 2 Cohort 3: HT-100 tablet, Dose 3 HT-100 * Single dose administration: Dose 3 * Multiple dose administration: Dose 3 Cohort 1: HT-100 tablet, Dose 1 HT-100 * Single dose administration: Dose 1 * Multiple dose administration: Dose 1 Cohort 4a: HT-100 tablet, Dose 4 HT-100 * Single dose administration: Dose 4 * Multiple dose administration: Dose 4 Cohort 4b: HT-100 tablet, Dose 5 HT-100 \* Multiple dose administration: Dose 5
- Primary Outcome Measures
Name Time Method Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys 1 week Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
- Secondary Outcome Measures
Name Time Method Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys 1 week Halofuginone plasma concentrations
Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks 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 4 weeks Pharmacodynamic measures relevant to DMD pathology:
* Pulmonary function
* Motor function
* Muscle composition
* Biochemical and imaging markers
Trial Locations
- Locations (5)
University of California, Davis Medical Center
🇺🇸Sacramento, California, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Kennedy Krieger Institute, Johns Hopkins School of Medicine
🇺🇸Baltimore, Maryland, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States