Ignite Creation Date:
2025-12-25 @ 1:43 AM
Ignite Modification Date:
2025-12-26 @ 1:41 AM
Study NCT ID:
NCT01395394
Status:
TERMINATED
Last Update Posted:
2014-08-28
First Post:
2011-06-23
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phenylketonuria, Oxidative Stress, and BH4
Sponsor:
Emory University
Organization:
Study Overview
Official Title:
The Ability of Kuvan® (Sapropterin Dihydrochloride) to Prevent Meal-induced Lipid Peroxidation and Endothelial Dysfunction in Patients With Phenylketonuria: a Pilot Study
Status:
TERMINATED
Status Verified Date:
2014-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Difficulty with recruitment and agreed with Sponsor to report completed data as case reports
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to see how tetrahydrobiopterin therapy (BH4; also known as sapropterin dihydrochloride or Kuvan) affects measures of oxidative stress and endothelial function in patients with Phenylketonuria (PKU).
Detailed Description:
Oxidative stress will be induced by having participants eat a meal in polyunsaturated fats. The investigators will see how participants respond to the meal challenge over the course of 6 hours.
* PKU participants who are currently on BH4 therapy will be evaluated while taking BH4 with the meal at a single study visit.
* PKU participants who did not responded to BH4 therapy will be evaluated twice. At the first study visit the investigators will see how you react to the meal challenge when you are not given BH4. You will be given BH4 for two weeks, then return for a second meal challenge study visit while taking BH4.
* Healthy controls will be evaluated after consuming the meal only (no BH4) at a single study visit.
* PKU participants who are currently on BH4 therapy will be evaluated while taking BH4 with the meal at a single study visit.
* PKU participants who did not responded to BH4 therapy will be evaluated twice. At the first study visit the investigators will see how you react to the meal challenge when you are not given BH4. You will be given BH4 for two weeks, then return for a second meal challenge study visit while taking BH4.
* Healthy controls will be evaluated after consuming the meal only (no BH4) at a single study visit.
Study Oversight
Has Oversight DMC:
True
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?: