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| ID | Type | Description | Link |
|---|---|---|---|
| HALO | Other Identifier | Akashi Therapeutics |
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Dosing stopped
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1: HT-100 tablet, Dose 1 | Experimental | • Multiple dose administration: Dose 1 |
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| Cohort 2: HT-100 tablet, Dose 2 | Experimental | • Multiple dose administration: Dose 2 |
|
| Cohort 3: HT-100 tablet, Dose 3 | Experimental | • Multiple dose administration: Dose 3 |
|
| Cohort 4: HT-100 tablet, Dose 4 | Experimental | • Multiple dose administration: Dose 4 |
|
| Cohort 5: HT-100 tablet, Dose 5 | Experimental | • Multiple dose administration: Dose 5 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| HT-100 | Drug | May be administered in either fed or fasted state |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD. |
| Months 2, 4, 6, 7 |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD. |
| Months 4, 6, 7 |
| Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Diana M Escolar, MD | AkashiTherapeutics | 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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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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|
Halofuginone plasma concentrations |
| Months 4, 6 |
| 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 |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |