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).

Trial Health

Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_2

Timeline

Completed

Started May 2015

Geographic Reach

1 country

5 active sites

Status

terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

1.7 years study duration

Study Start

First participant enrolled

May 1, 2015

Completed

3 months until next milestone

First Submitted

Initial submission to the registry

July 18, 2015

Completed

1 month until next milestone

First Posted

Study publicly available on registry

August 17, 2015

Completed

1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 30, 2016

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2016

Completed

Last Updated

March 12, 2019

Status Verified

March 1, 2019

Enrollment Period

1.7 years

First QC Date

July 18, 2015

Last Update Submit

March 8, 2019

Conditions

Duchenne Muscular Dystrophy

Keywords

DMDneuromuscularDuchenne

Outcome Measures

Primary Outcomes (8)

Number of adverse events by severity and relationship
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
Trial discontinuations due to upper GI or other AEs
Every 6 months from enrollment for up to 3 years
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
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
Electrocardiograms
Number of subjects with clinically significant changes in QT interval
Every 6 months from enrollment for up to 3 years
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
Cardiovascular Magnetic Resonance
Number of subjects with clinically significant change in diagnostic interpretation
Every 6 months from enrollment for up to 3 years

Secondary Outcomes (12)

Cardiovascular Magnetic Resonance
Every 6 months from enrollment for up to 3 years
Pulmonary function testing (Number of subjects with clinically significant changes)
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
+7 more secondary outcomes

Other Outcomes (1)

Pharmacokinetics peak plasma concentration (Cmax)
Pre-dose and 2-4 hour post-dose