Ignite Creation Date:
2024-05-06 @ 8:19 PM
Last Modification Date:
2024-10-26 @ 3:25 PM
Study NCT ID:
NCT06337864
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-03-29
First Post:
2024-03-15
Brief Title:
Effect of Large Neutral Amino Acids in Adults with Classical Phenylketonuria
Sponsor:
Rigshospitalet Denmark
Organization:
Rigshospitalet Denmark
Study Overview
Official Title:
Safety and Efficacy of Treatment with Large Neutral Amino Acids in Patients with Classical Phenylketonuria
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
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:
None
Brief Summary:
The overall aim of this study is to evaluate LNAA treatment as a potential alternative to conventional dietary treatment for PKU This study investigates the effects of LNAA treatment compared to the classic dietary treatment on cerebral dopamine synthesis in patients with classic PKU We will assess LNAAs effectiveness on neurotransmitter synthesis cognitive function mental health and safety compared to the standard diet
Detailed Description:
Standard treatment for Phenylketonuria PKU involves a lifelong phenylalanine-restricted diet Strict adherence to the diet is crucial but often challenging Large neutral amino acid LNAA supplementation is a potential alternative therapeutic approach for PKU management The proposed mechanism involves competitive inhibition of phenylalanine Phe transport across the blood-brain barrier by high-dose LNAA leading to reduced brain Phe levels However further investigation is needed to validate its efficacy and safety for PKU management
A randomized open-label crossover trial will be conducted to assess the safety and efficacy of LNAA supplementation in PKU patients After completion of the crossover study participants will have the option to participate in an open-label extension study aimed at evaluating the long-term safety and efficacy of LNAA
A healthy control group will be recruited to obtain baseline outcome measures This project is expected to provide much-needed insights into the potential of LNAA in PKU management The study also aims to gain a deeper understanding of the underlying pathophysiology of the disease Finally this work could lead to more personalized management strategies for PKU patients
A randomized open-label crossover trial will be conducted to assess the safety and efficacy of LNAA supplementation in PKU patients After completion of the crossover study participants will have the option to participate in an open-label extension study aimed at evaluating the long-term safety and efficacy of LNAA
A healthy control group will be recruited to obtain baseline outcome measures This project is expected to provide much-needed insights into the potential of LNAA in PKU management The study also aims to gain a deeper understanding of the underlying pathophysiology of the disease Finally this work could lead to more personalized management strategies for PKU patients
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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