BMN 307 ( DrugBank: - )

---

1 disease

ID	Disease name (Link within this page)	Number of trials
240	Phenylketonuria	2

240. Phenylketonuria

---

Clinical trials : 143 /  Drugs : 90 - (DrugBank : 10) / Drug target gene : 1 - Drug target pathways : 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