Actively Recruiting
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Led by RECORDATI GROUP · Updated on 2025-04-15
20
Participants Needed
5
Research Sites
521 weeks
Total Duration
On this page
Sponsors
R
RECORDATI GROUP
Lead Sponsor
T
Target PharmaSolutions, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
CONDITIONS
Official 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 provided
- Prescribed and treated with Carbaglu4
- Established diagnosis of Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA) confirmed by urine organic acid analysis or molecular genetic testing
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
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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