HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02 | DuchenneXchange

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terminated

HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

key information

study id #: NCT02525302

condition: Duchenne Muscular Dystrophy

status: terminated

purpose:

This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies. HALO-DMD-03 will provide safety and strength and function data on continuous long-term dosing. Data from this study will be used to inform the safety, tolerability, and dose selection for a future trial of HT-100 in boys with 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/NCT02525302

last updated: April 01, 2019

study details

start date: May 2015

estimated completion: December 30, 2016

phase of development: Phase 2

size / enrollment: 10

study description: As a follow-on study to the initial clinical studies of HT-100 in DMD (Protocols HALO-DMD-01 and HALO-DMD-02), this open-label study is designed to provide data on continuous long-term dosing. Subjects will be entered into the study without cessation of dosing, in a staggered fashion, into the same cohort assignment they had in the predecessor studies.
Up to 30 subjects who have completed dosing in HALO-DMD-02 will be offered the opportunity to continue on the same dose regimen until market approval of HT-100 or termination of the study by the Sponsor. Reasons for termination could include, among others, safety concerns or lack of efficacy, based on analysis of combined data from all HT-100 studies. Safety data from subjects approaching the end the HALO-DMD-02 participation will be individually reviewed by the Medical Monitor and the subject's physician (Principal Investigator [PI]).
If the Medical Monitor and the PI agree there are no clinically significant safety signals (absence of clinically significant laboratory or clinical abnormalities to date), the subject will be considered eligible and offered continuation of dosing. To avoid an interruption in dosing, subjects will immediately be screened for participation and enrolled upon completing the predecessor trial, HALO-DMD-02. Participation is in this study HALO-DMD-03 is optional.
Safety and pharmacodynamics (PD) monitoring will continue throughout the subject's study participation. Dose reduction/modification might occur or individual subjects' participation in the trial may be discontinued if any Adverse Events (AEs) suggest that HT-100 is not sufficiently well tolerated.

primary outcomes:

  • Number of adverse events by severity and relationship [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Dose reduction or modification due to upper GI or other adverse events [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Trial discontinuations due to upper GI or other AEs [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Vital signs (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant changes
  • Laboratory values (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant changes.
  • Electrocardiograms [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant changes in QT interval
  • Echocardiograms [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant changes in left ventricular ejection fraction, end systolic and diastolic interventricular septal thickness, left ventricular posterior wall thickness
  • Cardiovascular Magnetic Resonance [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant change in diagnostic interpretation.

secondary outcomes:

  • Cardiovascular Magnetic Resonance [Time Frame: Every 6 months from enrollment for up to 3 years]
    Circumferential strain and myocardial fibrotic areas
  • Pulmonary function testing (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years ]
    Number of subjects with clinically significant changes.
  • Motor function measure (MFM) scale [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Performance of upper limb (PUL) scale [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant changes.
  • Quantitative muscle testing (QMT) scores [ Time Frame: Every 6 months from enrollment for up to 3 years ]
  • Timed function tests (TFTs) [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Motor Function Measure (MFM) [Time Frame: Every 6 months from enrollment for up to 3 years]
  • Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL) [Time Frame: Every 6 months from enrollment for up to 3 years]
  • 9-hole peg test [Time Frame: Every 6 months from enrollment for up to 3 years]
    Assessment of upper limb function and dexterity
  • Tip pinch and key pinch tests (Number of subjects with clinically significant changes) [Time Frame: Every 6 months from enrollment for up to 3 years]
    Number of subjects with clinically significant changes.
  • Electrical impedance myography (EIM) score [Time Frame: Every 6 months from enrollment for up to 3 years]

inclusion criteria:
• Completed both previous studies HALO-DMD-01 and HALO-DMD-02
• Ability to provide written informed consent
• Ability to understand and follow site and protocol instruction for the entire duration of the study

exclusion criteria:
Answering yes to any of the following make the subject NOT eligible to participate in the study.
• Clinically significant major disease not related to DMD that would make it not safe to be in the study or affect ability to follow the protocol
• History of severe allergic or anaphylactic reactions
• Recent report of drug/alcohol abuse

study contacts

sponsor: Akashi Therapeutics

investigators: Diana M Escolar, MD

locations: United States