An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorRECORDATI GROUP
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®.
Eligibility criteria
Qualifiers
Provision of signed and dated informed consent/assent form
Prescribed and treated with Carbaglu®
Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR
Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).
Disqualifiers
None
Trial design
Treatments tested in this trial
- Carglumic Acid
Treatment groups
20 Participants
are divided into 1 treatment groupSponsors and collaborators
RECORDATI GROUP
Lead sponsor
Target PharmaSolutions, Inc.
Collaborator
Source ClinicalTrials.gov