HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
- Registration Number
- NCT02525302
- Lead Sponsor
- Akashi Therapeutics
- Brief Summary
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).
- Detailed 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- Male
- Target Recruitment
- 10
- 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
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Cohort 1: HT-100 tablet, Dose 5 HT-100 HT-100 multiple dose administration (dose 1). Cohort 1: HT-100 tablet, Dose 1 HT-100 HT-100 multiple dose administration (dose 1). Cohort 1: HT-100 tablet, Dose 2 HT-100 HT-100 multiple dose administration (dose 1). Cohort 1: HT-100 tablet, Dose 4 HT-100 HT-100 multiple dose administration (dose 1). Cohort 1: HT-100 tablet, Dose 3 HT-100 HT-100 multiple dose administration (dose 1).
- Primary Outcome Measures
Name Time Method Number of adverse events by severity and relationship Every 6 months from enrollment for up to 3 years Trial discontinuations due to upper GI or other AEs Every 6 months from enrollment for up to 3 years Dose reduction or modification due to upper GI or other adverse events Every 6 months from enrollment for up to 3 years Vital signs (Number of subjects with clinically significant changes) 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) Every 6 months from enrollment for up to 3 years Number of subjects with clinically significant changes.
Electrocardiograms Every 6 months from enrollment for up to 3 years Number of subjects with clinically significant changes in QT interval
Echocardiograms 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 Every 6 months from enrollment for up to 3 years Number of subjects with clinically significant change in diagnostic interpretation
- Secondary Outcome Measures
Name Time Method Cardiovascular Magnetic Resonance 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) Every 6 months from enrollment for up to 3 years Number of subjects with clinically significant changes.
Motor Function Measure (MFM) Every 6 months from enrollment for up to 3 years Motor function measure (MFM) scale Every 6 months from enrollment for up to 3 years Performance of upper limb (PUL) scale Every 6 months from enrollment for up to 3 years Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes) Every 6 months from enrollment for up to 3 years Number of subjects with clinically significant changes.
Quantitative muscle testing (QMT) scores Every 6 months from enrollment for up to 3 years Timed function tests (TFTs) Every 6 months from enrollment for up to 3 years 9-hole peg test Every 6 months from enrollment for up to 3 years Assessment of upper limb function and dexterity
Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL) Every 6 months from enrollment for up to 3 years Tip pinch and key pinch tests (Number of subjects with clinically significant changes) Every 6 months from enrollment for up to 3 years Number of subjects with clinically significant changes.
Electrical impedance myography (EIM) score Every 6 months from enrollment for up to 3 years
Trial Locations
- Locations (5)
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Washington University School of Medicine
🇺🇸Saint Louis, Missouri, United States
Kennedy Krieger Institute, Johns Hopkins School of Medicine
🇺🇸Baltimore, Maryland, United States
University of California, Davis Medical Center
🇺🇸Sacramento, California, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States