245. Propionic acidemia Clinical trials / Disease details
Clinical trials : 13 / Drugs : 16 - (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 | 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 |
| 2 | 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 | 42 | Phase 1/Phase 2 | United States;Canada;United Kingdom |
| 3 | 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 |