A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

NCT04110496 ·Status: TERMINATED ·Phase: PHASE1

Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)

NCT04085666 ·Status: COMPLETED ·Phase: PHASE1

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

NCT05270837 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)

NCT00924703 ·Status: COMPLETED ·Phase: PHASE2

Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia

NCT03167697 ·Status: COMPLETED ·Phase: NA

A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels

NCT00225615 ·Status: COMPLETED ·Phase: PHASE3

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

NCT03810690 ·Status: WITHDRAWN ·Phase: PHASE1/PHASE2

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

NCT07241234 ·Status: RECRUITING ·Phase: PHASE1

Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU

NCT00925054 ·Status: COMPLETED ·Phase: PHASE2

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT06332807 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria

NCT03516487 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response

NCT00841100 ·Status: COMPLETED ·Phase: PHASE2

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

NCT01889862 ·Status: COMPLETED ·Phase: PHASE3

Gene Therapy Clinical Study in Adult PKU

NCT03952156 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Natural History Clinical Study in Adult PKU

NCT04768348 ·Status: TERMINATED

Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria

NCT00104260 ·Status: COMPLETED ·Phase: PHASE2

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

NCT06971731 ·Status: RECRUITING ·Phase: PHASE3

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

NCT06628128 ·Status: RECRUITING ·Phase: PHASE3

Study of BH4, a New and Simple Treatment of Mild PKU

NCT00260000 ·Status: COMPLETED ·Phase: PHASE2

Phenylketonuria, Oxidative Stress, and BH4

NCT01395394 ·Status: TERMINATED ·Phase: PHASE2

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects

NCT06687733 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU

NCT03694353 ·Status: COMPLETED ·Phase: PHASE3

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

NCT05166161 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

NCT07318909 ·Status: NOT_YET_RECRUITING ·Phase: EARLY_PHASE1