Pegvaliase

DRUG7 trials

Sponsors

BioMarin Pharmaceutical, Boston Children's Hospital

Conditions

PhenylketonuriaPhenylketonuria (PKU)Phenylketonuria, MaternalPhenylketonurias

Phase 3

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

CompletedNCT03694353

BioMarin PharmaceuticalPhenylketonuria (PKU)

Start: 2018-09-13End: 2021-01-13Updated: 2022-02-17

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

Active, not recruitingNCT05270837

BioMarin PharmaceuticalPhenylketonuria (PKU)

Start: 2022-06-17End: 2027-10-31Target: 55Updated: 2025-09-12

Phase 4

Immune Modulation During Palynziq® Treatment in Adults (IMPALA)

Not yet recruitingNCT07477691

BioMarin PharmaceuticalPhenylketonuria

Start: 2026-03-31End: 2027-09-30Target: 12Updated: 2026-03-17

Unknown Phase

Nutrition Status of Adults Treated With Pegvaliase

CompletedNCT03792451

Boston Children's HospitalPhenylketonurias

Start: 2019-01-17End: 2020-12-31Updated: 2021-03-04

A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

RecruitingNCT05579548

BioMarin PharmaceuticalPhenylketonuria, Maternal

Start: 2022-11-22End: 2032-10-04Target: 50Updated: 2026-03-24

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

RecruitingNCT05813678

BioMarin PharmaceuticalPhenylketonuria (PKU)

Start: 2022-06-06End: 2033-11-01Target: 450Updated: 2026-03-25

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

RecruitingNCT06305234

BioMarin PharmaceuticalPhenylketonuria (PKU)

Start: 2024-01-20End: 2033-11-18Target: 200Updated: 2026-03-24

Related Papers

Long-term management strategies for pegvaliase use in phenylketonuria: Lessons learned from the phase-3 PRISM open-label extension study.

2025-05-20

Pegvaliase for the treatment of phenylketonuria: Final results of a long-term phase 3 clinical trial program.

2024-04-239 citations

Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Analysis of phase 3 clinical trial data.

2023-12-2412 citations