Ignite Creation Date:
2024-05-06 @ 6:51 PM
Last Modification Date:
2024-10-26 @ 2:56 PM
Study NCT ID:
NCT05813678
Status:
RECRUITING
Last Update Posted:
2024-05-13
First Post:
2023-04-03
Brief Title:
A Long-term Post-marketing Safety Study of Palynziq in Patients With PKU PALace
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
A Multi-Center Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq Pegvaliase in Subjects With Phenylketonuria
Status:
RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PALace
Brief Summary:
This is a 10-year multi-center global observational study to further characterize the safety profile of pegvaliase including hypersensitivity reactions long-term safety and tolerability and the effectiveness of the additional risk minimization measures aRMMs European Union EU only in subjects receiving pegvaliase for the treatment of PKU Subjects for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment incident-users or have previously started treatment with pegvaliase at the date of enrollment prevalent-users are eligible for participation in this study
Detailed Description:
This is a 10-year multi-center global observational study to further characterize the safety profile of pegvaliase including hypersensitivity reactions long-term safety and tolerability and the effectiveness of the aRMMs EU only in subjects receiving pegvaliase for the treatment of PKU Subjects for whom a clinical decision has been made that they will receive pegvaliase to treat their PKU within 30 days following the date of enrollment incident-users or have previously started treatment with pegvaliase at the date of enrollment prevalent-users are eligible for participation in this study Once the subject has been enrolled the investigator ie subjects primary physician will be asked to provide information about the subjects medical history treatments received to manage their blood Phe levels treatment with pegvaliase and concomitant medication use The investigator will also be asked to provide evaluation of the following safety concerns Acute systemic hypersensitivity reaction Anaphylaxis Angioedema Serum sickness Severe hypersensitivity reaction Severe or Persistent arthralgia Severe injection site reaction Hypophenylalaninemia and other protocol-defined safety events following the initiation of pegvaliase therapy There are no protocol-mandated visits or procedures associated with this study Enrolled subjects should be followed per routine clinical practice at the institution based upon their diagnosis with clinical outcomes assessed at regular intervals typically every month during the induction and titration phases of pegvaliase treatment and every 6 months once the maintenance phase is reached No experimental treatment or assessments are planned as part of this observational study An Independent Adjudication Committee IAC will periodically review protocol-defined safety events including Acute systemic hypersensitivity reaction and Serum sickness
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| EUPAS34992 | REGISTRY | European Network of Centres for Pharmacoepidemiology and Pharmacovigilance | None |