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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01847573 |
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Date of registration:
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02/05/2013 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
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Scientific title:
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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 |
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Date of first enrolment:
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May 2013 |
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Target sample size:
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17 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT01847573 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Diana M Escolar, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Akashi Therapeutics |
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Key inclusion & exclusion criteria
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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
Age minimum:
6 Years
Age maximum:
20 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Drug: HT-100
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Primary Outcome(s)
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Safety and tolerability of administering single and multiple ascending doses of HT-100 in DMD boys
[Time Frame: 1 week]
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Secondary Outcome(s)
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Pharmacokinetic plasma profile of halofuginone after single and multiple dose administration of HT-100 in DMD boys
[Time Frame: 1 week]
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Early pharmacodynamic signals of HT-100 after 4 weeks of continuous dosing in DMD boys
[Time Frame: 4 weeks]
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Safety and tolerability of administering multiple ascending doses of HT-100 in DMD boys over 4 weeks
[Time Frame: 4 weeks]
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Secondary ID(s)
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HALO-DMD-01
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HALO
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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