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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Halo Therapeutics, LLC
ClinicalTrials.gov Identifier:
NCT01847573
First received: May 2, 2013
Last updated: June 12, 2014
Last verified: June 2014

May 2, 2013
June 12, 2014
May 2013
December 2014   (final data collection date for primary outcome measure)
Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing
Same as current
Complete list of historical versions of study NCT01847573 on ClinicalTrials.gov Archive Site
  • Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys [ Time Frame: 1 week ] [ Designated as safety issue: Yes ]
    Halofuginone plasma concentrations
  • Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]

    Pharmacodynamic measures relevant to DMD pathology:

    • Pulmonary function
    • Motor function
    • Muscle composition
    • Biochemical and imaging markers
Same as current
Not Provided
Not Provided
 
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

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.

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
  • Experimental: Cohort 1: HT-100 tablet, Dose 1
    • Single dose administration: Dose 1
    • Multiple dose administration: Dose 1
    Intervention: Drug: HT-100
  • Experimental: Cohort 2: HT-100 tablet, Dose 2
    • Single dose administration: Dose 2
    • Multiple dose administration: Dose 2
    Intervention: Drug: HT-100
  • Experimental: Cohort 3: HT-100 tablet, Dose 3
    • Single dose administration: Dose 3
    • Multiple dose administration: Dose 3
    Intervention: Drug: HT-100
  • Experimental: Cohort 4: HT-100 tablet, Dose 4
    • Single dose administration: Dose 4
    • Multiple dose administration: Dose 4
    Intervention: Drug: HT-100
  • Experimental: Cohort 5: HT-100 tablet, Dose 5
    • Single dose administration: Dose 5
    • Multiple dose administration: Dose 5
    Intervention: Drug: HT-100
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
30
February 2015
December 2014   (final data collection date for primary outcome measure)

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
Male
6 Years to 20 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01847573
HALO-DMD-01, HALO
Yes
Halo Therapeutics, LLC
Halo Therapeutics, LLC
Not Provided
Study Director: Diana M. Escolar, MD Halo Therapeutics
Halo Therapeutics, LLC
June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP