Abstract | PURPOSE: METHODS: This multicenter, Phase 1 study enrolled 12 patients, divided equally into three groups (A-C). Group A were given 50 mg/kg on Days 1 and 11, and 50 mg/kg bid on Days 2 to 10. Group B and C received 100 mg/kg on Days 1 and 11; Group B and Group C were given 100 mg/kg bid and 100 mg/kg tid, respectively, on Days 2 to 10. A safety review was performed on all patients following the single dose and there was at least 2 weeks between each dose escalation, for safety and PK review. Adverse events (AEs) were monitored throughout the study. RESULTS: CONCLUSIONS:
SMT C1100 was well tolerated in pediatric DMD patients. TRIAL REGISTRATION: ClinicalTrials.gov NCT02383511.
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Authors | Valeria Ricotti, Stefan Spinty, Helen Roper, Imelda Hughes, Bina Tejura, Neil Robinson, Gary Layton, Kay Davies, Francesco Muntoni, Jonathon Tinsley |
Journal | PloS one
(PLoS One)
Vol. 11
Issue 4
Pg. e0152840
( 2016)
ISSN: 1932-6203 [Electronic] United States |
PMID | 27055247
(Publication Type: Clinical Trial, Phase I, Journal Article, Multicenter Study, Observational Study, Research Support, Non-U.S. Gov't)
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Chemical References |
- Benzoxazoles
- SMT C1100
- UTRN protein, human
- Utrophin
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Topics |
- Benzoxazoles
(administration & dosage, adverse effects, pharmacokinetics)
- Child
- Child, Preschool
- Humans
- Male
- Muscular Dystrophy, Duchenne
(blood, drug therapy, pathology)
- Utrophin
(metabolism)
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