An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics

RECORDATI GROUP20 enrolled

Overview

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice. Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Hyperammonemia Methylmalonic Acidemia Propionic Acidemia

Interventions

Carglumic AcidDRUG

Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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 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). AND/OR * Confirmation by molecular genetic testing Exclusion Criteria: * None

Locations (5)

Children's National Hospital

Washington D.C., District of Columbia, United States

RECRUITING

University of South Florida

Tampa, Florida, United States

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Outcomes

Primary Outcomes

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.

Adverse Event frequency and severity

Any Carbaglu® related adverse events will be be collected and reported

Secondary Outcomes

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.

Central Contacts

Anne Marie Cesario

CONTACT

908-849-4907 cesario.a@recordati.com

Data from ClinicalTrials.gov

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