Clinical trial NCT01847573: Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: halofuginone hydrobromide
anti-fibrotic
anti-inflammatory
muscle regeneration
protein synthesis inhibitor
Lead sponsor: Processa Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2013-05-07
Enrollment: 17
Sex: MALE
Minimum age (years): 6
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
timeFrame: 1 week
Secondary outcomes:
measure: Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
timeFrame: 1 week
measure: Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
timeFrame: 4 weeks
measure: Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
timeFrame: 4 weeks
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California, Davis Medical Center
state: California
city: Baltimore
country: United States
facility: Kennedy Krieger Institute, Johns Hopkins School of Medicine
state: Maryland
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Akashi Therapeutics
name: Diana M Escolar, MD
role: STUDY_DIRECTOR
References: