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terminated

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

key information

study id #: NCT01978366

condition: Duchenne Muscular Dystrophy

status: terminated

purpose:

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).

intervention: HT-100

mechanism of action: Small molecule designed to reduce fibrosis and inflammation

results: https://clinicaltrials.gov/ct2/show/results/NCT01978366

last updated: August 01, 2019

study details

start date: October 2013

estimated completion: April 30, 2016

phase of development: Phase 2

size / enrollment: 17

primary outcomes:

  • 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

secondary outcomes:

  • 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

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

study contacts

sponsor: Akashi Therapeutics

investigators: Diana M Escolar, MD

locations: United States

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