pegvaliase
- Generic Name
- pegvaliase
- Brand Names
- Palynziq
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1585984-95-7
- Unique Ingredient Identifier
- N6UAH27EUV
- Background
Pegvaliase is a recombinant phenylalanine ammonia lyase (PAL) enzyme derived from Anabaena variabilis that converts phenylalanine to ammonia and trans-cinnamic acid . Both the U.S. Food and Drug Administration and European Medicines Agency approved pegvaliase-pqpz in May 2018 for the treatment of adult patients with phenylketonuria (PKU). Phenylketonuria is ...
- Indication
用于尽管接受先前可用的治疗方案但仍无法充分控制血液苯丙氨酸（Phe）水平（血液Phe水平＞600微摩尔/升）的16岁及以上苯丙酮尿症（罕见病：PKU）患者，降低其血液Phe水平 。
- Associated Conditions
- Uncontrolled phenylketonuria
- Associated Therapies
- -
A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
- Conditions
- First Posted Date
- 2024-03-12
- Last Posted Date
- 2024-08-26
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 200
- Registration Number
- NCT06305234
- Locations
- 🇺🇸
UT Southwestern Medical Center, Dallas, Texas, United States
🇺🇸Northwestern University, Chicago, Illinois, United States
🇺🇸University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
- Conditions
- First Posted Date
- 2023-04-14
- Last Posted Date
- 2024-05-13
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 450
- Registration Number
- NCT05813678
- Locations
- 🇺🇸
University of Texas Southwestern Medical Center, Dallas, Texas, United States
🇺🇸Medical College of Wisconsin, Milwaukee, Wisconsin, United States
🇮🇹Policlinico Sant'orsola Malpighi, Bologna, Italy
A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
- First Posted Date
- 2022-10-14
- Last Posted Date
- 2024-05-03
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 50
- Registration Number
- NCT05579548
- Locations
- 🇩🇪
Universitaetsklinikum Hamburg Eppendorf, Hamburg, Germany
🇮🇹Ospedale San Paolo, Milano, Italy
🇺🇸Syneos Health, Wilmington, North Carolina, United States
Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
- Conditions
- Interventions
- First Posted Date
- 2022-05-02
- Last Posted Date
- 2024-07-26
- Lead Sponsor
- University of Missouri-Columbia
- Target Recruit Count
- 13
- Registration Number
- NCT05356377
- Locations
- 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇺🇸University of Missouri, Columbia, Missouri, United States
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
- Conditions
- Interventions
- First Posted Date
- 2022-03-08
- Last Posted Date
- 2024-06-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 55
- Registration Number
- NCT05270837
- Locations
- 🇺🇸
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
🇺🇸University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania, United States
🇺🇸Phoenix Children's Hospital, Phoenix, Arizona, United States
Nutrition Status of Adults Treated With Pegvaliase
- Conditions
- First Posted Date
- 2019-01-03
- Last Posted Date
- 2021-03-04
- Lead Sponsor
- Boston Children's Hospital
- Target Recruit Count
- 18
- Registration Number
- NCT03792451
- Locations
- 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
- First Posted Date
- 2018-10-03
- Last Posted Date
- 2022-02-17
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 37
- Registration Number
- NCT03694353
- Locations
- 🇺🇸
University of Missouri Health Care, Columbia, Missouri, United States
🇺🇸Washington University School of Medicine, Saint Louis, Missouri, United States
🇺🇸University of Florida, Gainesville, Florida, United States
Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
- First Posted Date
- 2010-10-01
- Last Posted Date
- 2019-02-26
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 16
- Registration Number
- NCT01212744
- Locations
- 🇺🇸
University of Missouri, Columbia, Missouri, United States
🇺🇸Nationwide Children's Hospital, Columbus, Ohio, United States
🇺🇸The Children's Hospital, Aurora, Colorado, United States
Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
- First Posted Date
- 2009-06-19
- Last Posted Date
- 2021-10-12
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 68
- Registration Number
- NCT00924703
- Locations
- 🇺🇸
University of Florida, Gainesville, Florida, United States
🇺🇸Ann and Robert H Lurie Children's Hospital, Chicago, Illinois, United States
🇺🇸The Children's Hospital, Aurora, Colorado, United States
Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
- First Posted Date
- 2008-03-13
- Last Posted Date
- 2017-02-01
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 25
- Registration Number
- NCT00634660
- Locations
- 🇺🇸
Mount Sinai Medical Center, New York, New York, United States
🇺🇸University of Wisconsin, Madison, Wisconsin, United States
🇺🇸Children's Memorial Hospital, Chicago, Illinois, United States
Drug Development Updates
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