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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02525302
• Other study ID #: HALO-DMD-03
• Status: Terminated
• Phase: Phase 2
• Start date: May 2015
• Est. completion date: December 30, 2016

Study information

• Verified date: March 2019
• Source: Akashi Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Terminated
• Enrollment: 10
• Est. completion date: December 30, 2016
• Est. primary completion date: December 30, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 20 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• HT-100
HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.

Locations

Country	Name	City	State
United States	Kennedy Krieger Institute, Johns Hopkins School of Medicine	Baltimore	Maryland
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of California, Davis Medical Center	Sacramento	California
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Akashi Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Pharmacokinetics peak plasma concentration (Cmax)		Pre-dose and 2-4 hour post-dose
Primary	Number of adverse events by severity and relationship		Every 6 months from enrollment for up to 3 years
Primary	Dose reduction or modification due to upper GI or other adverse events		Every 6 months from enrollment for up to 3 years
Primary	Trial discontinuations due to upper GI or other AEs		Every 6 months from enrollment for up to 3 years
Primary	Vital signs (Number of subjects with clinically significant changes)	Number of subjects with clinically significant changes	Every 6 months from enrollment for up to 3 years
Primary	Laboratory values (Number of subjects with clinically significant changes)	Number of subjects with clinically significant changes.	Every 6 months from enrollment for up to 3 years
Primary	Electrocardiograms	Number of subjects with clinically significant changes in QT interval	Every 6 months from enrollment for up to 3 years
Primary	Echocardiograms	Number of subjects with clinically significant changes in left ventricular ejection fraction, end systolic and diastolic interventricular septal thickness, left ventricular posterior wall thickness	Every 6 months from enrollment for up to 3 years
Primary	Cardiovascular Magnetic Resonance	Number of subjects with clinically significant change in diagnostic interpretation	Every 6 months from enrollment for up to 3 years
Secondary	Cardiovascular Magnetic Resonance	Circumferential strain and myocardial fibrotic areas	Every 6 months from enrollment for up to 3 years
Secondary	Pulmonary function testing (Number of subjects with clinically significant changes)	Number of subjects with clinically significant changes.	Every 6 months from enrollment for up to 3 years
Secondary	Motor function measure (MFM) scale		Every 6 months from enrollment for up to 3 years
Secondary	Performance of upper limb (PUL) scale		Every 6 months from enrollment for up to 3 years
Secondary	Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes)	Number of subjects with clinically significant changes.	Every 6 months from enrollment for up to 3 years
Secondary	Quantitative muscle testing (QMT) scores		Every 6 months from enrollment for up to 3 years
Secondary	Timed function tests (TFTs)		Every 6 months from enrollment for up to 3 years
Secondary	Motor Function Measure (MFM)		Every 6 months from enrollment for up to 3 years
Secondary	Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL)		Every 6 months from enrollment for up to 3 years
Secondary	9-hole peg test	Assessment of upper limb function and dexterity	Every 6 months from enrollment for up to 3 years
Secondary	Tip pinch and key pinch tests (Number of subjects with clinically significant changes)	Number of subjects with clinically significant changes.	Every 6 months from enrollment for up to 3 years
Secondary	Electrical impedance myography (EIM) score		Every 6 months from enrollment for up to 3 years

External Links

•   Sponsor company website