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

Description

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

Conditions

Hyperammonemia, Methylmalonic Acidemia, Propionic Acidemia

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Study Overview

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Study Details

Study overview

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

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

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

Condition
Hyperammonemia
Intervention / Treatment

-

Contacts and Locations

Washington

Children's National Hospital, Washington, District of Columbia, United States, 20010

Tampa

University of South Florida, Tampa, Florida, United States, 33606

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Indianapolis

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

New York

Icahn School of Medicine at Mt. Sinai, New York, New York, United States, 10029

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.

For general information about clinical research, read Learn About Studies.

Eligibility 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).
  • * Confirmation by molecular genetic testing
  • * None

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Recordati Rare Diseases,

William Ludlum, MD, STUDY_DIRECTOR, Recordati Rare Diseases Inc.

Nicholas Ah Mew, MD, PRINCIPAL_INVESTIGATOR, Children's National Research Institute

Study Record Dates

2032-06-30