NCT05040178View on ClinicalTrials.gov

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

RecruitingOBSERVATIONAL
Enrollment

20

Participants

Timeline

Start Date

June 30, 2022

Primary Completion Date

June 30, 2032

Study Completion Date

June 30, 2032

Conditions
HyperammonemiaMethylmalonic AcidemiaPropionic Acidemia
Interventions
DRUG

Carglumic Acid

Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.

Trial Locations (5)

10029

RECRUITING

Icahn School of Medicine at Mt. Sinai, New York

20010

RECRUITING

Children's National Hospital, Washington D.C.

33606

RECRUITING

University of South Florida, Tampa

46202

RECRUITING

Riley Children's Hospital, Indianapolis

60611

RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

All Listed Sponsors
collaborator

Target PharmaSolutions, Inc.

INDUSTRY

lead

Recordati Rare Diseases

INDUSTRY