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

Study Overview
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Brief Summary
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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).

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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* Drug: HT-100


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 20 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1: HT-100 tablet, Dose 1<br>• Multiple dose administration: Dose 1 | Drug: HT-100<br>* May be administered in either fed or fasted state<br>* Other names: halofuginone hydrobromide delayed-release tablet;|
| Experimental: Cohort 2: HT-100 tablet, Dose 2<br>• Multiple dose administration: Dose 2 | Drug: HT-100<br>* May be administered in either fed or fasted state<br>* Other names: halofuginone hydrobromide delayed-release tablet;|
| Experimental: Cohort 3: HT-100 tablet, Dose 3<br>• Multiple dose administration: Dose 3 | Drug: HT-100<br>* May be administered in either fed or fasted state<br>* Other names: halofuginone hydrobromide delayed-release tablet;|
| Experimental: Cohort 4: HT-100 tablet, Dose 4<br>• Multiple dose administration: Dose 4 | Drug: HT-100<br>* May be administered in either fed or fasted state<br>* Other names: halofuginone hydrobromide delayed-release tablet;|
| Experimental: Cohort 5: HT-100 tablet, Dose 5<br>• Multiple dose administration: Dose 5 | Drug: HT-100<br>* May be administered in either fed or fasted state<br>* Other names: halofuginone hydrobromide delayed-release tablet;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. | Target Safety profile by review of adverse events (AEs) Physical examination findings Clinical laboratory test results Other diagnostic testing | Months 2, 4, 6, 7 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD. | Pulmonary function Motor function Muscle composition Biochemical and imaging markers | Months 4, 6, 7 | 
| Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD. | Halofuginone plasma concentrations | Months 4, 6 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne muscular dystrophy, halofuginone hydrobromide, anti-fibrotic, anti-inflammatory, muscle regeneration, protein synthesis inhibitor