Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

Phase 2TerminatedNCT01847573

Processa Pharmaceuticals17 enrolled

Overview

The main purpose of this study is to test the safety and tolerability of different, increasing doses of an experimental medication called HT-100 in boys and young men with Duchenne muscular dystrophy (DMD). The study medication, HT-100, is a medicine that may help promote healthy muscle regeneration, diminish inflammation and the resulting damage to muscle, and decrease the scar tissue that forms in the muscles of children with DMD. In this study, pharmacokinetic sampling, or measurements of the amount of HT-100 in the bloodstream will also be taken.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Duchenne Muscular Dystrophy

Interventions

HT-100

Eligibility

Sex: MALEMin age: 6 YearsMax age: 20 Years

Medical Language ↔ Plain English

Main Inclusion Criteria: * Ambulatory or non-ambulatory * Diagnosis of DMD with confirmation of minimal to no dystrophin * Corticosteroid naive or on therapy for at least 12 months (stable dose and regimen) Main Exclusion Criteria: * Recent, substantial change in use of cardiac medications or medications affecting muscle function * Inability to undergo magnetic resonance imaging (MRI) * Significantly compromised cardio-respiratory function * Prior treatment with another investigational product in past 6 months

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

Nationwide Children's Hospital

Columbus, Ohio, United States

Outcomes

Primary Outcomes

Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys

Safety profile by review of adverse events (AEs), physical examination findings, clinical laboratory test results, and other diagnostic testing

Time frame: 1 week

Secondary Outcomes

Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys

Halofuginone plasma concentrations

Time frame: 1 week

Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks

Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing

Time frame: 4 weeks

Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys

Pharmacodynamic measures relevant to DMD pathology: * Pulmonary function * Motor function * Muscle composition * Biochemical and imaging markers

Time frame: 4 weeks