Observing the effects of Carbaglu® in treating MMA and PA in patients
A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations
This study is testing how safe Carbaglu® is for treating high ammonia levels in people with Methylmalonic Acidemia and Propionic Acidemia, both kids and adults.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 20 (estimated) |
| Sex | All |
| Sponsor | Recordati Group Industry-sponsored |
| Locations | 5 sites (Washington D.C., District of Columbia and 4 other locations) |
| Trial ID | NCT05040178 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather clinical safety information regarding the use of Carbaglu® in treating hyperammonemia caused by Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in both pediatric and adult patients. Participants will receive treatment as per standard medical practice, and data will be collected on various outcomes, including treatment details, plasma ammonia levels, and any adverse effects. The study will focus on both short-term and long-term safety profiles of Carbaglu® in these patient populations.
Who should consider this trial
Good fit: Ideal candidates include individuals diagnosed with Methylmalonic Acidemia or Propionic Acidemia who are being treated with Carbaglu®.
Not a fit: Patients who are not diagnosed with MMA or PA or those not receiving Carbaglu® treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety and efficacy of Carbaglu® for patients suffering from MMA and PA.
How similar studies have performed: While this study is observational, similar studies have shown promising results in understanding the safety profiles of treatments for metabolic disorders.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Provision of signed and dated informed consent/assent form 2. Prescribed and treated with Carbaglu® 3. Have an established diagnosis of PA or MMA defined as follows: * Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR * Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness). AND/OR * Confirmation by molecular genetic testing Exclusion Criteria: * None
Where this trial is running
Washington D.C., District of Columbia and 4 other locations
- Children's National Hospital — Washington D.C., District of Columbia, United States (Recruiting)
- University of South Florida — Tampa, Florida, United States (Recruiting)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- Riley Children's Hospital — Indianapolis, Indiana, United States (Recruiting)
- Icahn School of Medicine at Mt. Sinai — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Nicholas Ah Mew, MD — Children's National Research Institute
- Study coordinator: Anne Marie Cesario
- Email: cesario.a@recordati.com
- Phone: 908-849-4907
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.