Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne muscular dystrophy, halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor
Brief summary
This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD).
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
* Completed both the single ascending dose (SAD) and multiple ascending dose (MAD) phases of predecessor study HALO-DMD-01 * Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01 * Ability to provide written informed consent * Ambulatory or non-ambulatory
Exclusion criteria
* Recent, substantial change in use of cardiac medications or medications affecting muscle function * Clinically significant major disease, not related to DMD * Significantly compromised cardio-respiratory function * History of severe allergic or anaphylactic reactions * Prior treatment with another investigational product in past 6 months * Inability to undergo magnetic resonance imaging (MRI) * Current drug or alcohol abuse or prior treatment for abuse
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. | Months 2, 4, 6, 7 | * Target Safety profile by review of adverse events (AEs) * Physical examination findings * Clinical laboratory test results * Other diagnostic testing |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD. | Months 4, 6, 7 | * Pulmonary function * Motor function * Muscle composition * Biochemical and imaging markers |
| Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD. | Months 4, 6 | Halofuginone plasma concentrations |
Countries
United States
Outcome results
None listed