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| ID | Type | Description | Link |
|---|---|---|---|
| HALO | Other Identifier | Akashi Therapeutics |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: HT-100 tablet, Dose 1 | Experimental |
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| Cohort 2: HT-100 tablet, Dose 2 | Experimental |
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| Cohort 3: HT-100 tablet, Dose 3 | Experimental |
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| Cohort 4a: HT-100 tablet, Dose 4 | Experimental |
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| Cohort 4b: HT-100 tablet, Dose 5 | Experimental | * Multiple dose administration: Dose 5 |
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| Cohort 5: HT-100 tablet, Dose 6 | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HT-100 | Drug | May be administered in either fed or fasted state |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys | Halofuginone plasma concentrations | 1 week |
| Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks |
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Main Inclusion Criteria:
Main Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Diana M Escolar, MD | Akashi Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Davis Medical Center | Sacramento | California | 95817 | United States | ||
| Kennedy Krieger Institute, Johns Hopkins School of Medicine |
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| Label | URL |
|---|---|
| Sponsor company website | View source |
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* Multiple dose administration: Dose 5
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Safety profile by review of AEs, physical examination findings, clinical laboratory test results, and other diagnostic testing |
| 4 weeks |
| Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys | Pharmacodynamic measures relevant to DMD pathology:
| 4 weeks |
| Baltimore |
| Maryland |
| 21205 |
| United States |
| Washington University School of Medicine | St Louis | Missouri | 63110 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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