Clinical trial NCT01978366: Open Label Extension Study of HT-100 in Patients With DMD
Official title: An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne muscular dystrophy
halofuginone hydrobromide
anti-fibrotic
anti-inflammatory
muscle regeneration
protein synthesis inhibitor
Lead sponsor: Processa Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2013-11-07
Enrollment: 17
Sex: MALE
Minimum age (years): 6
Maximum age (years): 20
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.
timeFrame: Months 2, 4, 6, 7
Secondary outcomes:
measure: Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.
timeFrame: Months 4, 6, 7
measure: Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.
timeFrame: Months 4, 6
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California, Davis Medical Center
state: California
city: Baltimore
country: United States
facility: Kennedy Krieger Institute, Johns Hopkins School of Medicine
state: Maryland
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: AkashiTherapeutics
name: Diana M Escolar, MD
role: STUDY_DIRECTOR
References: