246. Methylmalonic acidemia Clinical trials / Disease details
Clinical trials : 23 / Drugs : 31 - (DrugBank : 8) / Drug target genes : 17 - Drug target pathways : 23
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05040178 (ClinicalTrials.gov) | June 30, 2022 | 16/6/2021 | An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics | 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 | Hyperammonemia;Methylmalonic Acidemia;Propionic Acidemia | Drug: Carglumic Acid | Recordati Rare Diseases | Target PharmaSolutions, Inc. | Recruiting | N/A | N/A | All | 20 | United States | |
| 2 | NCT04284917 (ClinicalTrials.gov) | November 28, 2019 | 24/2/2020 | Long-term Efficacy of Carglumic Acid in Organic Acidemia. | Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). | Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) | Drug: Carglumic Acid (Carbaglu®) | National Taiwan University Hospital | NULL | Enrolling by invitation | 18 Years | N/A | All | 5 | N/A | Taiwan |
| 3 | NCT04176523 (ClinicalTrials.gov) | January 15, 2019 | 20/11/2019 | Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach | Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods Approach | Methylmalonic Acidemia;Propionic Acidemia | Drug: Carglumic Acid | Recordati Rare Diseases | NULL | Recruiting | 6 Months | 99 Years | All | 75 | France;Germany;Italy;Norway;Spain;Sweden;United Kingdom | |
| 4 | NCT02426775 (ClinicalTrials.gov) | November 2015 | 22/4/2015 | Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia | Randomized Multicentre Comparative Trial to Evaluate the Long Term Effectiveness of the Use of Carbaglu® in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA) | Propionic Acidemia;Methylmalonic Acidemia | Drug: Carglumic Acid | King Abdullah International Medical Research Center | NULL | Completed | N/A | 15 Years | All | 33 | Phase 3 | Saudi Arabia |
| 5 | NCT01597440 (ClinicalTrials.gov) | September 2012 | 10/5/2012 | Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia | Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia | Propionic Acidemia;Methylmalonic Acidemia | Drug: N-carbamylglutamate;Other: Standard of Care | Mendel Tuchman | Children's Research Institute;Boston Children’s Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Lucile Packard Children's Hospital;University of Colorado, Denver | Terminated | N/A | 4 Weeks | All | 1 | Phase 2 | United States |
| 6 | NCT01599286 (ClinicalTrials.gov) | September 2012 | 11/5/2012 | Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia | Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia | Propionic Acidemia, Type I and/or Type II;Methylmalonic Acidemia;Carbamoyl-Phosphate Synthase I Deficiency Disease;Ornithine Carbamoyltransferase Deficiency | Drug: Carbaglu;Drug: Placebo;Drug: Standard of Care Treatment | Mendel Tuchman | Children's National Research Institute;Boston Children's Hospital;University Hospitals Cleveland Medical Center;University of California, Los Angeles;Children's Hospital of Philadelphia;Stanford University;Icahn School of Medicine at Mount Sinai;University of Pittsburgh;Children's Hospital Colorado | Active, not recruiting | N/A | 99 Years | All | 114 | Phase 2 | United States |
| 7 | NCT01341379 (ClinicalTrials.gov) | December 2010 | 22/4/2011 | Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate | Increasing Ureagenesis in Inborn Errors of Metabolism With N-Carbamylglutamate | Urea Cycle Disorders, Inborn;Inborn Errors of Metabolism;Propionic Acidemia;Methylmalonic Acidemia;Carbamyl Phosphate Synthetase Deficiency | Drug: N-carbamylglutamate | Children's Hospital of Philadelphia | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Withdrawn | 3 Years | 70 Years | Both | 0 | Phase 2 | United States |