Ignite Creation Date:
2024-05-05 @ 5:35 PM
Last Modification Date:
2024-10-26 @ 9:33 AM
Study NCT ID:
NCT00484991
Status:
APPROVED_FOR_MARKETING
Last Update Posted:
2008-04-15
First Post:
2007-06-08
Brief Title:
Sapropterin Expanded Access Program
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
Sapropterin Expanded Access Program
Status:
APPROVED_FOR_MARKETING
Status Verified Date:
2008-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The Purpose of this study is to provide patients with hyperphenylalaninemia HPA due to Phenylketonuria PKU access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded access program EAP until commercial product is available
Detailed Description:
The sapropterin EAP SEAP is an open-label multi-center program designed to provide access to sapropterin dihydrochloride for patients diagnosed with hyperphenylalaninemia due to PKU All patients with a confirmed diagnosis of hyperphenylalaninemia due to PKU who are not currently enrolled in a clinical study with sapropterin dihydrochloride and meet the requisite inclusion criteria and do not meet any of the exclusion criteria may be eligible to participate in this program Eligible patients may receive sapropterin dihydrochloride and participate in the program until commercial drug is available however the program will not continue beyond 2 months after the product receives marketing approval from the FDA During the program physicians will use their clinical judgment to assess whether a patient is a responder to sapropterin dihydrochloride Participating physicians will measure blood Phe levels at baseline prior to treatment and then at least once between Day 7 and Day 30 following initiation of treatment to determine if the patient is a responder A responder is a patient that has a clinically significant reduction in either the absolute or percent decrease in blood phenylalanine level compared to pre-treatment levels The recommended starting dose of sapropterin dihydrochloride is 20 mgkgday Physicians treating their patients with sapropterin dihydrochloride will be able to adjust the dose within a range of 5 mgkgday to 20 mgkgday if warranted in their clinical judgment Adverse event information will be collected in an ongoing fashion through patient reporting AEs to their physician Physicians will ask patients for information regarding adverse events that have occurred since the last visit An adverse event report form will be completed in these cases and sent to BioMarin
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None