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

April 24, 2023 updated by: Target PharmaSolutions, Inc.

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

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

20

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study 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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Time Frame: 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.
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Target PharmaSolutions, Inc.

Collaborators

Recordati Rare Diseases

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 15, 2022

Primary Completion (Anticipated)

June 30, 2032

Study Completion (Anticipated)

June 30, 2032

Study Registration Dates

First Submitted

February 23, 2023

First Submitted That Met QC Criteria

April 24, 2023

First Posted (Estimate)

May 4, 2023

Study Record Updates

Last Update Posted (Estimate)

May 4, 2023

Last Update Submitted That Met QC Criteria

April 24, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CARBAGLU-RRDUS-PASS-0573

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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