Sapropterin for Visceral Myopathy
What You Need to Know Before You Apply
What is the purpose of this trial?
There is currently no approved treatment for multisystem smooth muscle dysfunction syndrome (MSMDS). This single-patient study is the first to be conducted in a child with MSMDS in Canada and was designed to provide the child with access to sapropterin treatment. The molecule we will be using, sapropterin (Kuvan), is already approved and available for other indications. This disease is caused by a genetic variant in the ACTA2 gene. This variant prevents the small units of actin fibers, which are the molecular motors of the smooth muscle cell, from assembling correctly. The goal is to gather data so that the drug can be approved for this indication and thus treat the patient.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participant receives KUVAN® (sapropterin dihydrochloride) 100 mg Powder Packets once daily
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Sapropterin
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participant will receive KUVAN® (sapropterin dihydrochloride) 100 mg Powder Packets once daily.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Gregor Andelfinger
Lead Sponsor
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