245. Propionic acidemia
9 clinical trials,   15 drugs   (DrugBank: 3 drugs),   1 drug target gene,   6 drug target pathways
Searched query = "Propionic acidemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
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 |
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1 | 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 |