BioMarin Pharmaceutical: Phenylketonurias trials

The registry lists 33 Phenylketonurias studies with BioMarin Pharmaceutical as lead sponsor. 3 are open to participants now and 17 have results posted.

Phase 1 3Phase 2 8Phase 3 11Phase 4 3No phase 9(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• Immune Modulation During Palynziq® Treatment in Adults (IMPALA)RecruitingNCT07477691Phase 4
• A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)RecruitingNCT06305234
• A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)RecruitingNCT05813678
• Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With PhenylketonuriaActive, not recruitingNCT05270837Phase 3Results posted
• Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKUCompletedNCT03694353Phase 3Results posted
• A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302CompletedNCT02468570Results posted
• Sapropterin on Cognitive Abilities in Young Adults With PhenylketonuriaTerminatedNCT01977820Phase 2Results posted
• Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKUCompletedNCT01889862Phase 3Results posted
• An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165CompletedNCT01819727Phase 3Results posted
• A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 WeeksCompletedNCT01560286Phase 2Results posted
• Kuvan® in Phenylketonuria Patients Less Than 4 Years OldCompletedNCT01376908Phase 3Results posted
• Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)CompletedNCT01212744Phase 2Results posted
• Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) PatientsCompletedNCT01114737Phase 3Results posted
• Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKUCompletedNCT00925054Phase 2Results posted
• Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)CompletedNCT00924703Phase 2Results posted
• Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKUCompletedNCT00838435Phase 3Results posted
• Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 DeficiencyCompletedNCT00355264Phase 2Results posted
• Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006CompletedNCT00332189Phase 3Results posted
• Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted DietCompletedNCT00272792Phase 3Results posted
• Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine LevelsCompletedNCT00104247Phase 3Results posted
• Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to PalynziqCompletedNCT06780332Phase 4
• AAV Gene Therapy Study for Subjects with PKUActive, not recruitingNCT04480567Phase 1/2
• A Prospective Clinical Study of Phenylketonuria (PKU)CompletedNCT04452513
• Retrospective Observational Safety Effectiveness With Kuvan in hpACompletedNCT03864029
• A Non-Interventional Interview Study of Phenylketonuria (PKU) AdultsCompletedNCT03505125
• Kuvan®'s Effect on the Cognition of Children With PhenylketonuriaCompletedNCT01965912Phase 4
• Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 DeficiencyCompletedNCT01016392
• A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult SubjectsCompletedNCT00789568Phase 1
• PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryCompletedNCT00778206
• Safety and Tolerability Study of rAvPAL-PEG to Treat PhenylketonuriaCompletedNCT00634660Phase 1
• Sapropterin Expanded Access ProgramApproved for marketingNCT00484991
• A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine LevelsCompletedNCT00225615Phase 3
• Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With PhenylketonuriaCompletedNCT00104260Phase 2