Clinical trial NCT02525302: HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
Official title: HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: DMD
neuromuscular
Duchenne
Lead sponsor: Akashi Therapeutics
Sponsor class: INDUSTRY
First posted: 2015-08-17
Enrollment: 10
Sex: MALE
Minimum age (years): 6
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

1. Completed both previous studies HALO-DMD-01 and HALO-DMD-02
2. Ability to provide written informed consent
3. 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.

1. 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
2. History of severe allergic or anaphylactic reactions
3. Recent report of drug/alcohol abuse

Primary outcomes:
measure: Number of adverse events by severity and relationship
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Dose reduction or modification due to upper GI or other adverse events
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Trial discontinuations due to upper GI or other AEs
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Vital signs (Number of subjects with clinically significant changes)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Laboratory values (Number of subjects with clinically significant changes)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Electrocardiograms
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Echocardiograms
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Cardiovascular Magnetic Resonance
timeFrame: Every 6 months from enrollment for up to 3 years
Secondary outcomes:
measure: Cardiovascular Magnetic Resonance
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Pulmonary function testing (Number of subjects with clinically significant changes)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Motor function measure (MFM) scale
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Performance of upper limb (PUL) scale
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Quantitative muscle testing (QMT) scores
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Timed function tests (TFTs)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Motor Function Measure (MFM)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: 9-hole peg test
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Tip pinch and key pinch tests (Number of subjects with clinically significant changes)
timeFrame: Every 6 months from enrollment for up to 3 years
measure: Electrical impedance myography (EIM) score
timeFrame: Every 6 months from enrollment for up to 3 years
Other outcomes:
measure: Pharmacokinetics peak plasma concentration (Cmax)
timeFrame: Pre-dose and 2-4 hour post-dose
Locations:
city: Sacramento
country: United States
facility: University of California, Davis Medical Center
state: California
city: Baltimore
country: United States
facility: Kennedy Krieger Institute, Johns Hopkins School of Medicine
state: Maryland
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Askashi Therapeutics
name: Diana M Escolar, MD
role: STUDY_DIRECTOR
References: