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

Phase 2

Terminated

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Drug: HT-100

• Registration Number: NCT01978366
• Lead Sponsor: Processa Pharmaceuticals

Brief Summary

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).

Detailed Description

Not available

Study Timeline

• Study Start: 2013-10
• Study First Submitted: 2013-10-31
• Study First Submitted QC: 2013-10-31
• Study First Posted: 2013-11-07
• Primary Completion: 2016-04-30
• Study Completion: 2016-04-30
• Status Verified: 2019-07
• Disposition First Submitted: 2019-07-23
• Disposition First Submitted QC: 2019-07-23
• Disposition First Posted: 2019-07-30
• Last Update Submitted: 2020-08-31
• Last Update Posted: 2020-09-03

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 17

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 4: HT-100 tablet, Dose 4	HT-100	• Multiple dose administration: Dose 4
Cohort 1: HT-100 tablet, Dose 1	HT-100	• Multiple dose administration: Dose 1
Cohort 2: HT-100 tablet, Dose 2	HT-100	• Multiple dose administration: Dose 2
Cohort 3: HT-100 tablet, Dose 3	HT-100	• Multiple dose administration: Dose 3
Cohort 5: HT-100 tablet, Dose 5	HT-100	• Multiple dose administration: Dose 5

Primary Outcome Measures

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

Secondary Outcome Measures

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

Trial Locations

Locations (5)

Kennedy Krieger Institute, Johns Hopkins School of Medicine; 🇺🇸 Baltimore, Maryland, United States

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

University of California, Davis Medical Center; 🇺🇸 Sacramento, California, United States