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

Sponsor

Target PharmaSolutions, Inc. (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05842837

Collaborator

Recordati Rare Diseases (Industry)

Enrollment

Location

117.5

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Hyperammonemia Methylmalonic Acidemia Propionic Acidemia	Drug: carglumic acid

Study Design

Study Type:

Observational

Anticipated Enrollment :

20 participants

Observational Model:

Cohort

Time Perspective:

Other

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

Actual Study Start Date :

Sep 15, 2022

Anticipated Primary Completion Date :

Jun 30, 2032

Anticipated Study Completion Date :

Jun 30, 2032

Arms and Interventions

Arm	Intervention/Treatment
Patients with PA/MMA treated with Carbaglu	Drug: carglumic acid Current or previous treatment with carglumic acid

Arm

Intervention/Treatment

Patients with PA/MMA treated with Carbaglu

Drug: carglumic acid

Current or previous treatment with carglumic acid

Outcome Measures

Primary Outcome Measures

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. [10 years]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Provide a signed and dated informed consent/assent form
Receive treatment with Carbaglu
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)