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

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ClinicalTrials.gov Identifier: NCT01847573
Recruitment Status : Terminated (Dosing stopped)
First Posted : May 7, 2013
Last Update Posted : September 3, 2020
Sponsor:
Information provided by (Responsible Party):
Processa Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE May 2, 2013
First Posted Date  ICMJE May 7, 2013
Last Update Posted Date September 3, 2020
Study Start Date  ICMJE May 2013
Actual Primary Completion Date March 30, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 6, 2013)
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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 6, 2013)
  • Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys [ Time Frame: 1 week ]
    Halofuginone plasma concentrations
  • Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks [ Time Frame: 4 weeks ]
    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 ]
    Pharmacodynamic measures relevant to DMD pathology:
    • Pulmonary function
    • Motor function
    • Muscle composition
    • Biochemical and imaging markers
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet
Study Arms  ICMJE
  • 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 4a: HT-100 tablet, Dose 4
    • Single dose administration: Dose 4
    • Multiple dose administration: Dose 4
    Intervention: Drug: HT-100
  • Experimental: Cohort 4b: HT-100 tablet, Dose 5
    * Multiple dose administration: Dose 5
    Intervention: Drug: HT-100
  • Experimental: Cohort 5: HT-100 tablet, Dose 6
    * Multiple dose administration: Dose 5
    Intervention: Drug: HT-100
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 21, 2017)
17
Original Estimated Enrollment  ICMJE
 (submitted: May 6, 2013)
30
Actual Study Completion Date  ICMJE March 30, 2016
Actual Primary Completion Date March 30, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 6 Years to 20 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01847573
Other Study ID Numbers  ICMJE HALO-DMD-01
HALO ( Other Identifier: Akashi Therapeutics )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Processa Pharmaceuticals
Study Sponsor  ICMJE Processa Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Diana M Escolar, MD Akashi Therapeutics
PRS Account Processa Pharmaceuticals
Verification Date March 2019

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