A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy
Overview
- Phase
- Phase 1
- Intervention
- HT-100
- Conditions
- Duchenne Muscular Dystrophy
- Sponsor
- Processa Pharmaceuticals
- Enrollment
- 17
- Locations
- 5
- Primary Endpoint
- Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
- Status
- Terminated
- Last Updated
- 5 years ago
Overview
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.
Investigators
Eligibility Criteria
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)
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
Arms & Interventions
Cohort 1: HT-100 tablet, Dose 1
* Single dose administration: Dose 1 * Multiple dose administration: Dose 1
Intervention: HT-100
Cohort 2: HT-100 tablet, Dose 2
* Single dose administration: Dose 2 * Multiple dose administration: Dose 2
Intervention: HT-100
Cohort 3: HT-100 tablet, Dose 3
* Single dose administration: Dose 3 * Multiple dose administration: Dose 3
Intervention: HT-100
Cohort 4a: HT-100 tablet, Dose 4
* Single dose administration: Dose 4 * Multiple dose administration: Dose 4
Intervention: HT-100
Cohort 4b: HT-100 tablet, Dose 5
\* Multiple dose administration: Dose 5
Intervention: HT-100
Cohort 5: HT-100 tablet, Dose 6
\* Multiple dose administration: Dose 5
Intervention: HT-100
Outcomes
Primary Outcomes
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
Secondary Outcomes
- Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys(1 week)
- Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks(4 weeks)
- Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys(4 weeks)