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Clinical Research Studies

HALO-DMD

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

Prinicipal Investigator:

Jay Han, M.D.

 Contact:

Erica Goude, MS, CCRP
pmr.research@ucdmc.ucdavis.edu
916-734-0968

Study Details:

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.

Intervention(s)
halofuginone hydrobromide delayed-release tablet (HT-100) at multiple doses.

For more details please visit:

ClinicalTrials.gov

Eligibility

Inclusion Criteria:

  • Males
  • 6-20 years of age at time of dosing day 0
  • Ambulatory (able to independently walk 10 meters) or non-ambulatory
  • Diagnosis of DMD confirmed by at least 1 of the following:
    •  Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical presentation consistent with typical DMD (participants with scant presence of dystrophin, e.g., due to revertant muscle fibers, may be enrolled in the study with approval of the Medical Monitor). 
      OR
    • Positive gene deletion test (missing 1 or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as “out-of-frame,” and clinical presentation consistent with typical DMD (participants with deletions of exons 1-24, not definitely associated with DMD, but predicted as “out-of-frame” may be enrolled in the study with approval of the Medical Monitor).
      OR
    • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) definitively associated with DMD, and clinical presentation consistent with typical DMD (participants with mutations not definitively associated with DMD, but predicted as “out-of-frame,” may be enrolled in the study with approval of the Medical Monitor).
  • Corticosteroid therapy that meets both of the following:
    • Receiving therapy for at least 12 calendar months prior to dosing day 0.
    • Receiving a stable absolute dose and schedule for at least 6 months prior to dosing day 0 (no adjustments for increased weight are allowed for at least 6 months prior to day 0).
      OR
    • Corticosteroid naïve, defined as never having been treated with corticosteroids or having discontinued corticosteroid treatment for 12 months or more before Screening visit.

  • Participant and parent/guardian must provide written informed consent and assent for participating in the study.
  • Participant must possess the ability, per the Principal Investigator (PI), to understand and comply with protocol instruction for the entire duration of the study.

Exclusion Criteria: 

  • Currently enrolled in another treatment clinical trial.
  • History of significant concomitant illness or significant impairment of renal or hepatic function.
  • Use of regular daily aspirin or other medication with antiplatelet effects within 3 weeks of first dose of study medication.
  • Regular participation in vigorous exercise.
  • Symptomatic heart failure with cardiac ejection fraction <25% 

Recruitment Status

Currently Recruiting