Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Metho… (NCT04176523) | Clinical Trial Compass
RecruitingNot Applicable
Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
France, Germany, Italy95 participantsStarted 2019-01-15
Plain-language summary
This is a prospective mixed-design study focused on the long-term management of propionic aciduria (PA) and methylmalonic aciduria (MMA) with N-carbamylglutamate (NCG) maintenance therapy. Treatment characteristics, clinical outcomes, and healthcare utilization data of patients diagnosed PA or MMA treated \>6 months therapy with NCG are collected at baseline, 12 months, 18 months, 36 months and 54 months. Qualitative interviews with adult patients and caregivers are conducted \>6 months after study enrollment to gain a better understanding of the disease burden and the treatment burden of patients and their families.
Who can participate
Age range
6 Months – 99 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA)
. Patient initiated treatment with carglumic acid for long-term management of MMA or PA
. Patient has been treated with carglumic acid for a minimum of 6 months
. Patient (or caregiver) is able to comply with all prospective study procedures
. Patient (or caregiver) is able to provide informed consent
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this study is tracking decompensation events over time in patients already using CARBAGLU, can you help me understand what a decompensation event actually looks like for someone with methylmalonic or propionic acidemia, and how participating in this study might change how we monitor for them?
2This trial is listed as Phase NA, which suggests it's an observational or registry-style study rather than a treatment experiment — does that mean there's no change to my current treatment plan if I join, and what exactly would be expected of me as a participant?
3The study is measuring changes in the number and duration of decompensation events — how would my data be collected for that, and would participating give you or me any earlier insight into how well CARBAGLU is working for my specific condition?
4Since this study is still actively recruiting, is CARBAGLU already an option for me right now as part of standard care, or is joining this study the primary way I'd be able to access it?
5Given that both methylmalonic acidemia and propionic acidemia are included in this trial, does my specific diagnosis affect whether this study would be a good fit to discuss, and are there any long-term commitments or follow-up visits that might be hard to manage given our situation?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Number and Duration of Decompensation Events