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
  Purpose

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.


Condition Intervention Phase
Duchenne Muscular Dystrophy
Drug: HT-100
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Halo Therapeutics, LLC:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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


Estimated Enrollment: 30
Study Start Date: May 2013
Estimated Study Completion Date: February 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1: HT-100 tablet, Dose 1
  • Single dose administration: 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
  • Single dose administration: 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
  • Single dose administration: 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
  • Single dose administration: 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
  • Single dose administration: 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

  Eligibility

Ages Eligible for Study:   6 Years to 20 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01847573

Locations
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
St 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
Halo Therapeutics, LLC
Investigators
Study Director: Diana M. Escolar, MD Halo Therapeutics
  More Information

Additional Information:
No publications provided

Responsible Party: Halo Therapeutics, LLC
ClinicalTrials.gov Identifier: NCT01847573     History of Changes
Other Study ID Numbers: HALO-DMD-01, HALO
Study First Received: May 2, 2013
Last Updated: June 12, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Halo Therapeutics, LLC:
halofuginone hydrobromide
anti-fibrotic
anti-inflammatory
muscle regeneration
protein synthesis inhibitor

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Halofuginone
Antineoplastic Agents
Therapeutic Uses
Pharmacologic Actions
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

ClinicalTrials.gov processed this record on October 16, 2014