Duchenne Muscular Dystrophy
Conditions
Keywords
halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor
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.
Interventions
DRUGHT-100
May be administered in either fed or fasted state
Sponsors
Processa Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
6 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
Main Inclusion Criteria: * 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
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Outcome results
None listed