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NCT05040178 · ClinicalTrials.gov registry record
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
A clinical trial of Methylmalonic Acidemia and Propionic Acidemia, sponsored by Recordati Group.
- Recruiting
- Registry status
- 20
- Enrollment target
- 5
- Study locations
NCT05040178: Recruiting study of Methylmalonic Acidemia and Propionic Acidemia, sponsored by Recordati Group.
NCT05040178 is a study of Methylmalonic Acidemia and Propionic Acidemia that is actively recruiting participants, run by Recordati Group. The registered enrollment target is 20 participants, below the 424-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (95% lower). The trial reports 5 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05040178, a study of Methylmalonic Acidemia and Propionic Acidemia, is actively recruiting participants, sponsored by Recordati Group.
- RECRUITING
- Registry status
- 20 participants
- Enrollment target
- 5
- Study locations
Study Summary
To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
Primary Outcome
Multiple plasma ammonia levels will be collected only during treatment with Carbaglu® according to prescribing information and routine medical practice in terms of visit frequency.
Conditions Studied
Interventions
- DRUG Carglumic Acid
Study Locations (5)
District of Columbia
- Children's National Hospital - Washington D.C.
Florida
- University of South Florida - Tampa
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Indiana
- Riley Children's Hospital - Indianapolis
New York
- Icahn School of Medicine at Mt. Sinai - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2022-06-30 |
| Est. Completion | 2032-06-30 |
What NCT05040178 shows while recruiting
NCT05040178 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 20 participants, a relatively small participant target, below the 424-participant average among 6 other Methylmalonic Acidemia trials with a reported enrollment target (95% lower).
The record links to 3 conditions, with Methylmalonic Acidemia appearing as the primary indexed condition, and to 1 intervention - of which Carglumic Acid is the first listed.
NCT05040178 lists 5 locations in 5 states (District of Columbia, Florida, Illinois).
Frequently Asked Questions
What is clinical trial NCT05040178 about?
NCT05040178 is a clinical study titled "An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics". To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.
What is the current status of trial NCT05040178?
This trial is currently recruiting. The enrollment target is 20 participants. The study started on 2022-06-30. Estimated completion is 2032-06-30.
What conditions does trial NCT05040178 study?
This clinical trial studies the following conditions: Methylmalonic Acidemia, Propionic Acidemia, Hyperammonemia.
What interventions are being tested in trial NCT05040178?
The interventions under investigation include: Carglumic Acid (DRUG).
Who is sponsoring clinical trial NCT05040178?
This trial is sponsored by Recordati Group, which has 11 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05040178 being conducted?
This trial has 5 study locations across District of Columbia, Florida, Illinois, Indiana, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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