Trial | NCT01847573  Report issue

Title

Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
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

  • Phase

    Phase 1/Phase 2

  • Study Type

    Interventional

  • Status

    Terminated

  • Study Participants

    17
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.
Study Started
May 31
2013
Primary Completion
Mar 30
2016
Study Completion
Mar 30
2016
Last Update
Sep 03
2020

Drug HT-100

May be administered in either fed or fasted state

  • Other names: halofuginone hydrobromide delayed-release tablet

Cohort 1: HT-100 tablet, Dose 1 Experimental

Single dose administration: Dose 1 Multiple dose administration: Dose 1

Cohort 2: HT-100 tablet, Dose 2 Experimental

Single dose administration: Dose 2 Multiple dose administration: Dose 2

Cohort 3: HT-100 tablet, Dose 3 Experimental

Single dose administration: Dose 3 Multiple dose administration: Dose 3

Cohort 4a: HT-100 tablet, Dose 4 Experimental

Single dose administration: Dose 4 Multiple dose administration: Dose 4

Cohort 4b: HT-100 tablet, Dose 5 Experimental

* Multiple dose administration: Dose 5

Cohort 5: HT-100 tablet, Dose 6 Experimental

* Multiple dose administration: Dose 5

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
No Results Posted