Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Open Label Extension Study of HT-100 in Patients With DMD

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01978366
Recruitment Status : Terminated (Dosing stopped)
First Posted : November 7, 2013
Last Update Posted : March 12, 2019
Sponsor:
Information provided by (Responsible Party):
Akashi Therapeutics

Tracking Information
First Submitted Date  ICMJE October 31, 2013
First Posted Date  ICMJE November 7, 2013
Last Update Posted Date March 12, 2019
Actual Study Start Date  ICMJE October 2013
Actual Primary Completion Date April 30, 2016   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 31, 2013)
Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. [ Time Frame: Months 2, 4, 6, 7 ]
  • Target Safety profile by review of adverse events (AEs)
  • Physical examination findings
  • Clinical laboratory test results
  • Other diagnostic testing
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT01978366 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 31, 2013)
  • Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD. [ Time Frame: Months 4, 6, 7 ]
    • Pulmonary function
    • Motor function
    • Muscle composition
    • Biochemical and imaging markers
  • Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD. [ Time Frame: Months 4, 6 ]
    Halofuginone plasma concentrations
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 Open Label Extension Study of HT-100 in Patients With DMD
Official Title  ICMJE An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
Brief Summary This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE 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
    • Multiple dose administration: Dose 1
    Intervention: Drug: HT-100
  • Experimental: Cohort 2: HT-100 tablet, Dose 2
    • Multiple dose administration: Dose 2
    Intervention: Drug: HT-100
  • Experimental: Cohort 3: HT-100 tablet, Dose 3
    • Multiple dose administration: Dose 3
    Intervention: Drug: HT-100
  • Experimental: Cohort 4: HT-100 tablet, Dose 4
    • Multiple dose administration: Dose 4
    Intervention: Drug: HT-100
  • Experimental: Cohort 5: HT-100 tablet, Dose 5
    • 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: July 5, 2017)
17
Original Estimated Enrollment  ICMJE
 (submitted: October 31, 2013)
30
Actual Study Completion Date  ICMJE April 30, 2016
Actual Primary Completion Date April 30, 2016   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Completed both the single ascending dose (SAD) and multiple ascending dose (MAD) phases of predecessor study HALO-DMD-01
  • Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01
  • Ability to provide written informed consent
  • Ambulatory or non-ambulatory

Exclusion Criteria:

  • Recent, substantial change in use of cardiac medications or medications affecting muscle function
  • Clinically significant major disease, not related to DMD
  • Significantly compromised cardio-respiratory function
  • History of severe allergic or anaphylactic reactions
  • Prior treatment with another investigational product in past 6 months
  • Inability to undergo magnetic resonance imaging (MRI)
  • Current drug or alcohol abuse or prior treatment for abuse
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 NCT01978366
Other Study ID Numbers  ICMJE HALO-DMD-02
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 Akashi Therapeutics
Study Sponsor  ICMJE Akashi Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Diana M Escolar, MD AkashiTherapeutics
PRS Account Akashi Therapeutics
Verification Date March 2019

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