Open Label Extension Study of HT-100 in Patients With DMD

Status:	Terminated
Conditions:	Neurology, Orthopedic
Therapuetic Areas:	Neurology, Orthopedics / Podiatry
Healthy:	No
Age Range:	6 - 20
Updated:	3/14/2019
Start Date:	October 2013
End Date:	April 30, 2016

An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01

This study is designed to provide 6-months continuous dosing with the study medication,
called HT-100, on participants who successfully completed the predecessor study
(HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability,
pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics
(measuring how much drug is in the bloodstream) in participants with a broad spectrum of
Duchenne muscular dystrophy (DMD).

Inclusion Criteria:

- Completed both the single ascending dose (SAD) and multiple ascending dose (MAD)
phases of predecessor study HALO-DMD-01

- Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01

- Ability to provide written informed consent

- Ambulatory or non-ambulatory

Exclusion Criteria:

- Recent, substantial change in use of cardiac medications or medications affecting
muscle function

- Clinically significant major disease, not related to DMD

- Significantly compromised cardio-respiratory function

- History of severe allergic or anaphylactic reactions

- Prior treatment with another investigational product in past 6 months

- Inability to undergo magnetic resonance imaging (MRI)

- Current drug or alcohol abuse or prior treatment for abuse

We found this trial at

sites

Cincinnati Children's Hospital Medical Center

3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229

1-513-636-4200