An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Recruiting
Observational Study
Hyperammonemia
Methylmalonic Acidemia
Propionic Acidemia
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 20 (estimated)
- Sponsor
- RECORDATI GROUP · Industry
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About This Trial
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
1. Provision of signed and dated informed consent/assent form
2. Prescribed and treated with Carbaglu®
3. Have an established diagnosis of PA or MMA defined as follows:
* Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated meth…
Contacts