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Long-term management of organic acidemia with CARBAGLU®

Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach

Observational Recordati Group · NCT04176523

This study looks at how well carglumic acid helps people with propionic acidemia and methylmalonic acidemia over a long time, while also talking to patients and caregivers about their experiences.

Quick facts

Study typeObservational
Enrollment95 (estimated)
Ages6 Months to 99 Years
SexAll
SponsorRecordati Group Industry-sponsored
Locations32 sites (Bordeaux and 31 other locations)
Trial IDNCT04176523 on ClinicalTrials.gov

What this trial studies

This observational study focuses on the long-term management of patients with propionic acidemia (PA) and methylmalonic acidemia (MMA) who have been treated with carglumic acid for at least six months. It involves collecting treatment characteristics, clinical outcomes, and healthcare utilization data at multiple time points over a period of 54 months. Additionally, qualitative interviews with patients and caregivers will be conducted to understand the disease and treatment burden. The study aims to gather comprehensive data from up to 85 patients across six European countries.

Who should consider this trial

Good fit: Ideal candidates include patients diagnosed with methylmalonic acidemia or propionic acidemia who have been on carglumic acid treatment for at least six months.

Not a fit: Patients who have not been treated with carglumic acid for the required duration or those with other unrelated conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance understanding of the long-term effects of carglumic acid treatment, potentially improving management strategies for patients with organic acidemias.

How similar studies have performed: While this study employs a mixed methods approach, similar studies focusing on long-term management of organic acidemias have shown promise in understanding treatment impacts.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

1. Patient has confirmed diagnosis of an organic acidemia (e.g., MMA or PA)
2. Patient initiated treatment with carglumic acid for long-term management of MMA or PA
3. Patient has been treated with carglumic acid for a minimum of 6 months
4. Patient (or caregiver) is able to comply with all prospective study procedures
5. Patient (or caregiver) is able to provide informed consent

Exclusion Criteria:

* None

Where this trial is running

Bordeaux and 31 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Methylmalonic AcidemiaPropionic Acidemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.