245. Propionic acidemia Clinical trials / Disease details
Clinical trials : 13 / Drugs : 17 - (DrugBank : 3) / Drug target gene : 1 - Drug target pathways : 6
| 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) | March 2022 | 16/6/2021 | A Surveillance Protocol of Carbaglu® for the Treatment of Hyperammonemia | A Surveillance Protocol of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations | Hyperammonemia | Drug: Carglumic Acid | Recordati Rare Diseases | NULL | Not yet recruiting | N/A | N/A | All | 10 | United States | |
| 2 | NCT05130437 (ClinicalTrials.gov) | November 9, 2021 | 3/11/2021 | A Long-Term Extension Study to Evaluate the Safety and Clinical Activity of mRNA-3927 | A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study | Propionic Acidemia | Biological: mRNA-3927 | ModernaTX, Inc. | NULL | Recruiting | 1 Year | N/A | All | 36 | Phase 1/Phase 2 | United States;Canada;United Kingdom |
| 3 | NCT04159103 (ClinicalTrials.gov) | April 15, 2021 | 7/11/2019 | Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia | A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants With Propionic Acidemia | Propionic Acidemia | Biological: mRNA-3927 | ModernaTX, Inc. | NULL | Recruiting | 1 Year | N/A | All | 36 | Phase 1/Phase 2 | United States;Canada;United Kingdom |
| 4 | NCT04836494 (ClinicalTrials.gov) | March 25, 2021 | 29/3/2021 | 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 | A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia | Healthy Volunteers;Propionic Acidemia;Methylmalonic Acidemia;Organic Acidemia | Drug: BBP-671;Drug: Placebo | CoA Therapeutics, Inc., a BridgeBio company | NULL | Recruiting | 18 Years | 55 Years | All | 128 | Phase 1 | United States |
| 5 | NCT04732429 (ClinicalTrials.gov) | March 15, 2021 | 21/1/2021 | Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia | A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study | Methylmalonic Acidemia;Propionic Acidemia | Drug: HST5040;Drug: Placebo | HemoShear Therapeutics | NULL | Recruiting | 2 Years | N/A | All | 12 | Phase 2 | United States |
| 6 | EUCTR2019-003529-36-GB (EUCTR) | 18/12/2020 | 03/03/2020 | A clinical study to evaluate the medicinal product in Patients with Propionic Acidemia | A Global, Phase 1/2, Open-label, Dose Optimization Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia | Propionic Acidemia- Propionic acidemia is an autosomal recessive disorder caused by loss-of-function mutations in PCCA or PCCB (and in rare instances, mutations in both PCCA and PCCB), resulting in the lossof enzymatic activity of the mitochondrial complex PCC MedDRA version: 21.0;Level: LLT;Classification code 10080615;Term: Propionic acidemia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03] | Product Name: mRNA-3927 INN or Proposed INN: N/A Other descriptive name: CX-016492 INN or Proposed INN: NA Other descriptive name: CX-017950 | ModernaTX, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 36 | Phase 1;Phase 2 | United States;Canada;United Kingdom | ||
| 7 | 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 |
| 8 | 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;Italy;Norway;Spain;United Kingdom | |
| 9 | 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 |
| 10 | 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 |
| 11 | 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 |
| 12 | 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 |
| 13 | NCT00645879 (ClinicalTrials.gov) | July 2008 | 25/3/2008 | Anaplerotic Therapy in Propionic Acidemia | Safety & Efficacy of Investigational Products: Ornithine Alpha-ketoglutarate, Glutamine, or Disodium Citrate on Hyperammonemia in Propionic Acidemia. | Propionic Acidemia | Drug: ornithine alpha ketoglutarate;Drug: glutamine;Drug: disodium citrate | Nicola Longo | National Institutes of Health (NIH);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Completed | 5 Years | 12 Years | Both | 3 | Phase 1 | United States |