Bmn 307    (DrugBank: -)

1 disease
告示番号疾患名(ページ内リンク)臨床試験数
240フェニルケトン尿症2

240. フェニルケトン尿症 [臨床試験数:125,薬物数:95(DrugBank:11),標的遺伝子数:1,標的パスウェイ数:5
Searched query = "Phenylketonuria", "PKU", "Phenylalanine hydroxylase deficiency", "PAH deficiency", "Tetrahydrobiopterin deficiency", "BH4 deficiency", "BH4 reactive hyper pheemia"
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.
2 / 125 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT04480567
(ClinicalTrials.gov)
September 24, 20208/7/2020AAV Gene Therapy Study for Subjects With PKUA 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 PhenylketonuriaPhenylketonuria (PKU)Drug: BMN 307BioMarin PharmaceuticalNULLRecruiting15 YearsN/AAll100Phase 1;Phase 2United States;United Kingdom
2EUCTR2019-001878-28-GB
(EUCTR)
17/12/201921/11/2019A Phase 1/2 study to evaluate the safety and efficacy of BMN 307 gene therapy in patients with phenylketonuriaA 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 307BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
100Phase 1;Phase 2United States;Taiwan;Spain;Turkey;Australia;Germany;Italy;United Kingdom