A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA

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: Enrolling by invitation

Administrative data

• NCT number: NCT05842837
• Other study ID #: CARBAGLU-RRDUS-PASS-0573
• Status: Enrolling by invitation
• Start date: September 15, 2022
• Est. completion date: June 30, 2032

Study information

• Verified date: April 2023
• Source: Target PharmaSolutions, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The objective of this study is to obtain short- and long-term clinical safety data in pediatric and adult patients with PA and MMA treated with Carbaglu, including pregnancy and fetal outcomes. 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• 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. Provide a signed and dated informed consent/assent form

2. Receive treatment with Carbaglu

3. Have an established diagnosis of PA or MMA defined as at least 1 of the following:

• Confirmation by molecular genetic testing of gene mutations responsible for PA or MMA
• 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
• 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)

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 carglumic acid

Locations

Country	Name	City	State
United States	Riley Children's Hospital	Indianapolis	Indiana

Sponsors (2)

Lead Sponsor	Collaborator
Target PharmaSolutions, Inc.	Recordati Rare Diseases

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To obtain short- and long-term clinical safety data in pediatric and adult patients with PA and MMA treated with Carbaglu, including pregnancy and fetal outcomes.	Carbaglu was approved by the US Food and Drug Administration (FDA) in January 2021 for use as an adjunctive therapy to standard of care for the treatment of acute hyperammonemia due to PA or MMA. This observational/non-interventional study is being conducted in order to obtain clinical safety data (eg, biochemical effects, clinical outcomes, and significant safety events) associated with short- and long-term use of Carbaglu in adult and pediatric patients with PA and MMA.	10 years