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

Phase 2TerminatedNCT01978366

Processa Pharmaceuticals17 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

HT-100DRUG

May be administered in either fed or fasted state

Eligibility

Sex: MALEMin age: 6 YearsMax age: 20 Years

Medical Language ↔ Plain English

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

Locations (5)

University of California, Davis Medical Center

Sacramento, California, United States

Kennedy Krieger Institute, Johns Hopkins School of Medicine

Baltimore, Maryland, United States

Washington University School of Medicine

St Louis, Missouri, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Outcomes

Primary Outcomes

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

Time frame: Months 2, 4, 6, 7

Secondary Outcomes

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

Time frame: Months 4, 6, 7

Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.

Halofuginone plasma concentrations

Time frame: Months 4, 6

Data from ClinicalTrials.gov

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