240. フェニルケトン尿症 Phenylketonuria Clinical trials / Disease details
臨床試験数 : 143 / 薬物数 : 90 - (DrugBank : 10) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 5
| 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 | NCT04480567 (ClinicalTrials.gov) | September 24, 2020 | 8/7/2020 | AAV Gene Therapy Study for Subjects With PKU | A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With Phenylketonuria | Phenylketonuria (PKU) | Drug: BMN 307 | BioMarin Pharmaceutical | NULL | Active, not recruiting | 15 Years | N/A | All | 100 | Phase 1/Phase 2 | United States;United Kingdom |
| 2 | EUCTR2019-001878-28-GB (EUCTR) | 17/12/2019 | 21/11/2019 | A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuria | A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 µmol/L - A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 | Phenylketonuria MedDRA version: 20.0;Level: PT;Classification code 10034872;Term: Phenylketonuria;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Code: BMN 307 | BioMarin Pharmaceutical Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 1;Phase 2 | United States;Taiwan;Spain;Turkey;Australia;Germany;Italy;United Kingdom |