Open Label Extension Study of HT-100 in Patients With DMD
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ClinicalTrials.gov Identifier: NCT01978366 |
Recruitment Status :
Terminated
(Dosing stopped)
First Posted : November 7, 2013
Last Update Posted : September 3, 2020
|
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: HT-100 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 17 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01 |
Actual Study Start Date : | October 2013 |
Actual Primary Completion Date : | April 30, 2016 |
Actual Study Completion Date : | April 30, 2016 |
Arm | Intervention/treatment |
---|---|
Experimental: Cohort 1: HT-100 tablet, Dose 1
• Multiple dose administration: Dose 1
|
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet |
Experimental: Cohort 2: HT-100 tablet, Dose 2
• Multiple dose administration: Dose 2
|
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet |
Experimental: Cohort 3: HT-100 tablet, Dose 3
• Multiple dose administration: Dose 3
|
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet |
Experimental: Cohort 4: HT-100 tablet, Dose 4
• Multiple dose administration: Dose 4
|
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet |
Experimental: Cohort 5: HT-100 tablet, Dose 5
• Multiple dose administration: Dose 5
|
Drug: HT-100
May be administered in either fed or fasted state
Other Name: halofuginone hydrobromide delayed-release tablet |
- Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. [ Time Frame: Months 2, 4, 6, 7 ]
- Target Safety profile by review of adverse events (AEs)
- Physical examination findings
- Clinical laboratory test results
- Other diagnostic testing
- Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD. [ Time Frame: 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. [ Time Frame: Months 4, 6 ]Halofuginone plasma concentrations
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Ages Eligible for Study: | 6 Years to 20 Years (Child, Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01978366
United States, California | |
University of California, Davis Medical Center | |
Sacramento, California, United States, 95817 | |
United States, Maryland | |
Kennedy Krieger Institute, Johns Hopkins School of Medicine | |
Baltimore, Maryland, United States, 21205 | |
United States, Missouri | |
Washington University School of Medicine | |
Saint Louis, Missouri, United States, 63110 | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 | |
Nationwide Children's Hospital | |
Columbus, Ohio, United States, 43205 |
Study Director: | Diana M Escolar, MD | AkashiTherapeutics |
Responsible Party: | Processa Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT01978366 |
Other Study ID Numbers: |
HALO-DMD-02 HALO ( Other Identifier: Akashi Therapeutics ) |
First Posted: | November 7, 2013 Key Record Dates |
Last Update Posted: | September 3, 2020 |
Last Verified: | July 2019 |
Duchenne muscular dystrophy halofuginone hydrobromide anti-fibrotic |
anti-inflammatory muscle regeneration protein synthesis inhibitor |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Halofuginone Antineoplastic Agents Coccidiostats |
Antiprotozoal Agents Antiparasitic Agents Anti-Infective Agents Protein Synthesis Inhibitors Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Angiogenesis Inhibitors Angiogenesis Modulating Agents Growth Substances Physiological Effects of Drugs Growth Inhibitors |