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Clinical Trial Summary

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


Clinical Trial Description

This study is being conducted to obtain short-term and long-term clinical safety information from adult and pediatric patients treated for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA). This is an observational/non-interventional study. Patients will be treated per the prescribing information and routine medical practice. Only available data will be collected as part of the study including developmental outcomes, details of treatment with Carbaglu® and other treatments for hyperammonemia including dietary and protein management, plasma ammonia levels, pregnancy and maternal complications, adverse effects on the developing fetus and neonate, adverse effects on the infant through first year of life. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05040178
Study type Observational
Source Recordati Rare Diseases
Contact Anne Marie Cesario
Phone 908-849-4907
Email cesario.a@recordati.com
Status Recruiting
Phase
Start date June 30, 2022
Completion date June 30, 2032

See also
  Status Clinical Trial Phase
Enrolling by invitation NCT05842837 - A Non-Interventional Post-Authorization Study of Carbaglu for the Treatment of Hyperammonemia Due to MMA and PA
Completed NCT01599286 - Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia Phase 2
Completed NCT02426775 - Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia Phase 3
Terminated NCT04581785 - Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia Phase 1/Phase 2
Recruiting NCT05295433 - An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in Other Clinical Studies of mRNA-3705 Phase 1/Phase 2
Terminated NCT04836494 - A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia Phase 1
Completed NCT03484767 - "The MaP Study": Mapping the Patient Journey in MMA and PA
Terminated NCT04732429 - Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Phase 2
Recruiting NCT00078078 - Clinical and Laboratory Study of Methylmalonic Acidemia
Active, not recruiting NCT05506254 - Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy
Recruiting NCT04176523 - Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach
Recruiting NCT01289158 - Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study N/A
Withdrawn NCT01341379 - Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate Phase 2
Recruiting NCT04899310 - A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Phase 1/Phase 2
Terminated NCT05438485 - Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia