Safety and Efficacy of Treatment With Large Neutral Amino Acids in Patients With Classical Phenylketonuria
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Other
Study Type: Interventional
Study Phase: Not Applicable
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 50
Healthy Volunteers: t
View:
• Treatment initiation within the first month of life
• Intelligence quotient over 84, based upon the baseline neuropsychological evaluation
• Conventional dietary treatment up to minimum 15 years of age
• Signed informed consent
• Willing and able to comply with the protocol and study procedures
Locations
Other Locations
Denmark
Center for Inherited Metabolic Diseases
RECRUITING
Copenhagen
Copenhagen University Hospital, Rigshospitalet
NOT_YET_RECRUITING
Copenhagen
Contact Information
Primary
Allan Lund, Professor, MD, DMSc
allan.lund@regionh.dk
+4535451303
Backup
Olivia Fjellbirkeland, MD
olivia.welle.fjellbirkeland@regionh.dk
Time Frame
Start Date: 2024-11-01
Estimated Completion Date: 2026-09-30
Participants
Target number of participants: 30
Treatments
Experimental: LNAA supplementation, Then Low-Phe diet
Phase 1: LNAA Treatment Period (8 weeks) Each participant will be prescribed a specific dosage of LNAA tablets to be taken three times daily.~Phase 2: Washout (2 weeks) Participants will follow a liberalized diet and refrain from taking any LNAA or amino acid supplement.~Phase 3: Low-Phe diet (8 weeks) Each participant will follow a low-phenylalanine diet. LNAA therapy will not be administered during this phase.
Experimental: Low-Phe diet, Then LNAA supplementation
Phase 1: Low-Phe diet (8 weeks) Each participant will follow a low-phenylalanine diet. LNAA therapy will not be administered during this phase.~Phase 2: Washout (2 weeks) Participants will follow a liberalized diet and refrain from taking any LNAA or amino acid supplement.~Phase 3: LNAA Treatment Period (8 weeks) Each participant will be prescribed a specific dosage of LNAA tablets to be taken three times daily.
Related Therapeutic Areas
Sponsors
Collaborators: Prekulab Ltd ApS
Leads: Rigshospitalet, Denmark