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



Status:Terminated
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:6 - 20
Updated:3/14/2019
Start Date:May 2015
End Date:December 30, 2016

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

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).

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.

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
We found this trial at
5
sites
2315 Stockton Blvd.
Sacramento, California 95817
(916) 734-2011
University of California, Davis Medical Center UC Davis Medical Center serves a 65,000-square-mile area that...
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Sacramento, CA
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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Baltimore, MD
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660 S Euclid Ave
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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Saint Louis, MO
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