Actively Recruiting
A Non-Interventional Post-Authorization Safety Study of Carbaglu4 for Hyperammonemia Due to Methylmalonic Acidemia and Propionic Acidemia in Adults and Children
Led by RECORDATI GROUP · Updated on 2025-04-15
20
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
Sponsors
R
RECORDATI GROUP
Lead Sponsor
T
Target PharmaSolutions, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are collecting both short-term and long-term safety information from adults and children treated for hyperammonemia caused by Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This observational study focuses on patients receiving Carbaglu4 as part of their usual medical care, aiming to understand the effects of this treatment in real-world settings. Participants will be treated according to standard medical practices, receiving Carbaglu4 as prescribed by their doctors. The study gathers data on patients treated either as outpatients or inpatients, including details about Carbaglu4 dosing, other treatments for hyperammonemia like diet and protein management, and pregnancy-related outcomes. Data collection continues for about one year after stopping Carbaglu4. During the study, researchers will review plasma ammonia levels and record any adverse events, including their frequency and severity. They will also collect information on pregnancy outcomes and effects on infants up to one year old. No additional treatments or interventions are administered beyond routine care. This allows for monitoring safety and developmental outcomes over time in this patient group.
CONDITIONS
Brief Title
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed and dated informed consent or assent form
- Currently prescribed and treated with Carbaglu4
- Have an established diagnosis of Propionic Acidemia (PA) confirmed by urine organic acid analysis showing elevated methylcitric acid and normal methylmalonic acid without biotin-related disorders, or
- Have an established diagnosis of Methylmalonic Acidemia (MMA) confirmed by urine organic acid analysis showing elevated methylmalonic acid without vitamin B12 dependent disorder, or
- Confirmation of PA or MMA by molecular genetic testing
You will not qualify if you...
- None reported for this study, all patients meeting inclusion can participate
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 year following discontinuation of treatment
Participants who undergo routine care with Carbaglu® are observed to collect clinical safety information including plasma ammonia levels, pregnancy and fetal outcomes, and adverse effects up to one year following discontinuation of treatment.
Visits as per routine medical practice
Trial Site Locations
Total: 5 locations
1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
2
University of South Florida
Tampa, Florida, United States, 33606
Actively Recruiting
3
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
4
Riley Children's Hospital
Indianapolis, Indiana, United States, 46202
Actively Recruiting
5
Icahn School of Medicine at Mt. Sinai
New York, New York, United States, 10029
Actively Recruiting
Research Team
A
Anne Marie Cesario
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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