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
This trial does not accept healthy volunteersQualifiers
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 population
Patients with PA or MMA of any age and gender, including pregnant women, are eligible for enrollment in the study as long as they meet the eligibility criteria.
Trial design
Design model
Cohort
Time perspective
Other
Treatments tested in this trial
Carglumic Acid
DrugCurrent or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: Male and Female Adult and Pediatric Participants1 intervention
Trial outcomes
Primary outcomes
1
Effects of Carbaglu® on plasma ammonia levels
Multiple plasma ammonia levels will be collected only during treatment with Carbaglu® according to prescribing information and routine medical practice in terms of visit frequency.
Time frame
Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment.2
Adverse Event frequency and severity
Any Carbaglu® related adverse events will be be collected and reported
Time frame
Patients treated with Carbaglu® will be managed chronically (out-patient) or acutely (hospital in-patient). In both cases, data will be collected for approximately 1 year following discontinuation of Carbaglu treatment.Secondary outcomes
1
Fetal Outcomes and Pregnancy Outcomes
Pregnancy risks including maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant (through the first year of life).
Time frame
Collection of pregnancy information for patients who becomes pregnant while participating in the trial or at time of enrollment. Pregnancy reports and reports involving neonates and infants up to 1 year of age must be reported to RRD Pharmacovigilance.Sponsors and contacts
Click on the lead sponsor to view all of their trials.
RECORDATI GROUP
Lead sponsor
Target PharmaSolutions, Inc.
Collaborator
Source ClinicalTrials.gov