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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01978366
• Other study ID #: HALO-DMD-02
• Secondary ID: HALO
• Status: Terminated
• Phase: Phase 2
• Start date: October 2013
• Est. completion date: April 30, 2016

Study information

• Verified date: July 2019
• Source: Processa Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 17
• Est. completion date: April 30, 2016
• Est. primary completion date: April 30, 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Years to 20 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• HT-100
May be administered in either fed or fasted state

Locations

Country	Name	City	State
United States	Kennedy Krieger Institute, Johns Hopkins School of Medicine	Baltimore	Maryland
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	University of California, Davis Medical Center	Sacramento	California
United States	Washington University School of Medicine	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Processa Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.	Halofuginone plasma concentrations	Months 4, 6

External Links

•   Sponsor company website