RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2022-06-30
Study Completion:2032-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05040178

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Provision of signed and dated informed consent/assent form
  2. 2. Prescribed and treated with Carbaglu®
  3. 3. Have an established diagnosis of PA or MMA defined as follows:
  4. * 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
  5. * 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).
  6. * Confirmation by molecular genetic testing
  1. * None

Contacts and Locations

Study Contact

Anne Marie Cesario
CONTACT
908-849-4907
cesario.a@recordati.com

Principal Investigator

William Ludlum, MD
STUDY_DIRECTOR
Recordati Rare Diseases Inc.
Nicholas Ah Mew, MD
PRINCIPAL_INVESTIGATOR
Children's National Research Institute

Study Locations (Sites)

Children's National Hospital
Washington, District of Columbia, 20010
United States
University of South Florida
Tampa, Florida, 33606
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
Riley Children's Hospital
Indianapolis, Indiana, 46202
United States
Icahn School of Medicine at Mt. Sinai
New York, New York, 10029
United States

Collaborators and Investigators

Sponsor: 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

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 Registration Dates

Study Start Date2022-06-30
Study Completion Date2032-06-30

Study Record Updates

Study Start Date2022-06-30
Study Completion Date2032-06-30

Terms related to this study

Keywords Provided by Researchers

  • PA & MMA

Additional Relevant MeSH Terms

  • Hyperammonemia
  • Methylmalonic Acidemia
  • Propionic Acidemia