MRN supported a Phase 2 clinical trial evaluating an investigational oral therapy designed to boost utrophin production in boys with DMD. The protocol included five site-based visits and two off-site visits focused on laboratory sample collection and processing.
To keep patient and family burden at a minimum and ensure no missed visits, MRN was able to deliver the trial for a patient at a Ronald McDonald House. These facilities provide a supportive environment for families with children undergoing complex medical treatment, allowing them to remain close to care while maintaining a sense of normalcy.
Visits for this trial included an intensive schedule of clinical assessments and pharmacokinetic (PK) sampling across two days. Procedures included:
- Vital signs monitoring
- Recording of adverse events and concomitant medications
- Blood draws for clinical chemistry and liver function markers (including glutamate, dehydrogenase, amylase, and lipase)
- Creatine Kinase (CK) and Matrix Metalloproteinase-9 (MMP-9) assessments
- Time-critical PK sampling at pre-dose, 3 hours, and 6 hours post-dose
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