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

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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

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).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: HT-100 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 17 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
Actual Study Start Date : October 2013
Actual Primary Completion Date : April 30, 2016
Actual Study Completion Date : April 30, 2016


Arm Intervention/treatment
Experimental: Cohort 1: HT-100 tablet, Dose 1
• Multiple dose administration: Dose 1
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet

Experimental: Cohort 2: HT-100 tablet, Dose 2
• Multiple dose administration: Dose 2
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet

Experimental: Cohort 3: HT-100 tablet, Dose 3
• Multiple dose administration: Dose 3
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet

Experimental: Cohort 4: HT-100 tablet, Dose 4
• Multiple dose administration: Dose 4
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet

Experimental: Cohort 5: HT-100 tablet, Dose 5
• Multiple dose administration: Dose 5
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet




Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. 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

  2. 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



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Ages Eligible for Study:   6 Years to 20 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01978366


Locations
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United States, California
University of California, Davis Medical Center
Sacramento, California, United States, 95817
United States, Maryland
Kennedy Krieger Institute, Johns Hopkins School of Medicine
Baltimore, Maryland, United States, 21205
United States, Missouri
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Akashi Therapeutics
Investigators
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Study Director: Diana M Escolar, MD AkashiTherapeutics

Additional Information:
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Responsible Party: Akashi Therapeutics
ClinicalTrials.gov Identifier: NCT01978366     History of Changes
Other Study ID Numbers: HALO-DMD-02
HALO ( Other Identifier: Akashi Therapeutics )
First Posted: November 7, 2013    Key Record Dates
Last Update Posted: March 12, 2019
Last Verified: March 2019

Keywords provided by Akashi Therapeutics:
Duchenne muscular dystrophy
halofuginone hydrobromide
anti-fibrotic
anti-inflammatory
muscle regeneration
protein synthesis inhibitor

Additional relevant MeSH terms:
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Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Halofuginone
Antineoplastic Agents
Coccidiostats
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Protein Synthesis Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors