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

Propionic Acidemia Clinical Trial

Official title:

A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05040178
• Other study ID #: CARBAGLU-RRDUS-PASS-0573
• Status: Recruiting
• Start date: June 30, 2022
• Est. completion date: June 30, 2032

Study information

• Verified date: January 2024
• Source: Recordati Rare Diseases
• Contact: Anne Marie Cesario
• Phone: 908-849-4907
• Email: cesario.a[@]recordati.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Description:

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.

Other known NCT identifiers

• NCT05842837

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: June 30, 2032
• Est. primary completion date: June 30, 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Acidosis
• Amino Acid Metabolism, Inborn Errors
• Hyperammonemia
• Methylmalonic Acidemia
• Propionic Acidemia

Intervention

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

Locations

Country	Name	City	State
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Riley Children's Hospital	Indianapolis	Indiana
United States	Icahn School of Medicine at Mt. Sinai	New York	New York
United States	University of South Florida	Tampa	Florida
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (2)

Lead Sponsor	Collaborator
Recordati Rare Diseases	Target PharmaSolutions, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	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.
Primary	Adverse Event frequency and severity	Any Carbaglu® related adverse events will be be collected and reported	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	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).	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.