BridgeMD
Development and delivery of personalized 3D-printed drugs to improve treatment adherence in children with rare diseases.
EU CTIS ID: 2024-519378-37-00
What this study is testing
The aim is to compare the tolerability of the commercial biotin drug prescribed as usual treatment in pediatric patients at the Hospital Clínico Universitario de Santiago de Compostela with respect to the biotin master formula (chewable formulation) prepared in the Pharmacy Service of the same center, in order to improve the care and adherence of pediatric patients diagnosed with the rare hereditary metabolic disease
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Outpatients of both sexes, between 3-18 years of age (both ages included within the range). Diagnosis of one of the two rare metabolic diseases under study: methylcrotonylglycinuria treated with Medebiotin tablets or injectable solution, biotinidase deficiency treated with Medebiotin tablets or injectable solution. The patient has no swallowing problems at the time of signing the informed consent document by the parent(s) or guardian(s). Good compliance with the treatment prescribed by the specialist for each patient during the last year (Medebiotin tablets or injectable solution). This implies that the patient has not presented any metabolic decompensation in the last year.
You likely can't join if
- Known hypersensitivity to any of the excipients of the biotin master formulation. Any disorder or situation (severe metabolic decompensation) which, in the opinion of the investigating physician, would pose a risk for the subject not to comply with the prescribed treatment.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.