10 completed, 5 recruiting, 1 active not recruiting. 4 Phase 2, 4 Phase 3, 1 Phase 1, 1 Phase 4.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 4 | Completed 4 |
| Phase 3 | 4 | Completed 3, Active not recruiting 1 |
| Phase 1 | 1 | Completed 1 |
| Phase 4 | 1 | Recruiting 1 |
Follow pegvaliase: new trials by email Literature Review of pegvaliase trials Research Report on pegvaliase
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Immune Modulation During Palynziq® Treatment in Adults (IMPALA) Pegvaliase, Methotrexate - Phenylketonuria | Phase 4 | Recruiting | BioMarin Pharmaceutical | 12 | 2026-05-29 | 2026-06-17 |
| A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade) Pegvaliase - Phenylketonuria (PKU) | Recruiting | BioMarin Pharmaceutical | 200 | 2024-01-20 | 2026-03-24 | |
| A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding Pegvaliase - Phenylketonuria, Maternal | Recruiting | BioMarin Pharmaceutical | 50 | 2022-11-22 | 2026-03-24 | |
| Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria Pegvaliase, Diet Only - Phenylketonuria (PKU) | Phase 3 | Active not recruiting | BioMarin Pharmaceutical | 55 | 2022-06-17 | 2026-07-21 |
| A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace) Pegvaliase - Phenylketonuria (PKU) | Recruiting | BioMarin Pharmaceutical | 450 | 2022-06-06 | 2026-03-25 | |
| Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU Pegvaliase-Pqpz - Phenylketonurias | Completed | University of Missouri-Columbia | 6 | 2022-05-25 | 2025-12-29 | |
| Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) Palynziq - Phenylketonurias | Recruiting | Emory University | 45 | 2019-10-08 | 2026-01-14 | |
| Nutrition Status of Adults Treated With Pegvaliase Pegvaliase - Phenylketonurias | Completed | Boston Children's Hospital | 18 | 2019-01-17 | 2021-03-04 | |
| Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU Pegvaliase - Phenylketonuria (PKU) | Phase 3 | Completed | BioMarin Pharmaceutical | 37 | 2018-09-13 | 2022-02-17 |
| Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU BMN165 20mg/day, BMN165 40mg/day, Placebo - Phenylketonuria (PKU) | Phase 3 | Completed | BioMarin Pharmaceutical | 215 | 2013-07-29 | 2021-05-21 |
| An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 BMN 165 - Phenylketonuria | Phase 3 | Completed | BioMarin Pharmaceutical | 261 | 2013-05-01 | 2019-02-26 |
| A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks BMN 165 (rAvPAL-PEG) - Phenylketonuria | Phase 2 | Completed | BioMarin Pharmaceutical | 24 | 2012-05-01 | 2019-08-06 |
| Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) rAvPAL-PEG - Phenylketonuria | Phase 2 | Completed | BioMarin Pharmaceutical | 16 | 2011-03-01 | 2019-02-26 |
| Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) rAvPAL-PEG - Phenylketonuria | Phase 2 | Completed | BioMarin Pharmaceutical | 68 | 2010-01-13 | 2021-10-12 |
| Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU rAvPAL-PEG 0.001 mg/kg, rAvPAL-PEG 0.003 mg/kg, rAvPAL-PEG 0.01 mg/kg - Phenylketonuria | Phase 2 | Completed | BioMarin Pharmaceutical | 40 | 2009-09-01 | 2019-02-26 |
| Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria rAvPAL-PEG - Phenylketonuria | Phase 1 | Completed | BioMarin Pharmaceutical | 25 | 2008-05-01 | 2017-02-01 |
Phenylketonurias (15) · Phenylketonuria, Maternal (1)
BioMarin Pharmaceutical (13) · Boston Children's Hospital (1) · Emory University (1) · University of Missouri-Columbia (1)
United States (16) · Germany (3) · Italy (2) · Canada (1)
Ari Gershman, MD (2) · Celeste Decker, MD (1) · Debra Lounsbury (1) · Krista Viau, PhD, RD (1) · Markus Merilainen, MD (1) · Rani Singh, PhD, RDN, LD (1) · Shawn Christ, PhD (1)
PMID 32593547, PMID 40411344, PMID 34826353, PMID 24743000, PMID 29973227