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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01847573
• Other study ID #: HALO-DMD-01
• Secondary ID: HALO
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: May 2013
• Est. completion date: March 30, 2016

Study information

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

Clinical Trial Summary

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.

Recruitment information / eligibility

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

Eligibility

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

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 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	1 week
Secondary	Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys	Halofuginone plasma concentrations	1 week
Secondary	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	4 weeks
Secondary	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	4 weeks

External Links

•   Sponsor company website