Safety and Tolerability of RTX-134 in Adults With Phenylketonuria

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

NCT07241234 ·Status: RECRUITING ·Phase: PHASE1

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

NCT01889862 ·Status: COMPLETED ·Phase: PHASE3

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

NCT03694353 ·Status: COMPLETED ·Phase: PHASE3

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

NCT00925054 ·Status: COMPLETED ·Phase: PHASE2

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

NCT05166161 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects

NCT06687733 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

A Study of RO4917523 in Pediatric Patients With Fragile X Syndrome

NCT01750957 ·Status: COMPLETED ·Phase: PHASE2

Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)

NCT02750618 ·Status: COMPLETED ·Phase: PHASE2

A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

NCT06302348 ·Status: RECRUITING ·Phase: PHASE3

Phenylketonuria, Oxidative Stress, and BH4

NCT01395394 ·Status: TERMINATED ·Phase: PHASE2

Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients

NCT04256655 ·Status: WITHDRAWN ·Phase: PHASE1

Natural History Clinical Study in Adult PKU

NCT04768348 ·Status: TERMINATED

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

NCT06628128 ·Status: RECRUITING ·Phase: PHASE3

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

NCT04188964 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

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

NCT05270837 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006

NCT00332189 ·Status: COMPLETED ·Phase: PHASE3

An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT05367960 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE3

A Study of RO4917523 in Patients With Fragile X Syndrome

NCT01517698 ·Status: COMPLETED ·Phase: PHASE2

Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety

NCT00432822 ·Status: TERMINATED ·Phase: PHASE2/PHASE3

Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria

NCT04534842 ·Status: COMPLETED ·Phase: PHASE2

Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein Substitute

NCT04309331 ·Status: COMPLETED ·Phase: NA

Long-Term Follow-up of Subjects Who Were Treated With ST-920

NCT05039866 ·Status: ENROLLING_BY_INVITATION

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

NCT06971731 ·Status: RECRUITING ·Phase: PHASE3

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

NCT01433354 ·Status: TERMINATED ·Phase: PHASE2/PHASE3

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

NCT04085666 ·Status: COMPLETED ·Phase: PHASE1