NCT03924180

Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: University Hospital, Tours · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Phenylketonuria is the most common inherited metabolic disease in France and is screened for neonatal exposure. Management consists of a strict and restrictive hypoproteic diet and the intake of amino acid substitutes and dietary supplements free of phenylalanine.One of the major difficulties, which is the source of many treatment failures, is the inappetence of the amino acid supplements required during a strict hypoproteic diet. New formulations, Glycomacropeptides (GMP), have recently appeared and are considered more palatable than conventional amino acid mixtures.

Detailed description

Phenylketonuria is the most common inherited metabolic disease in France and is screened for neonatal exposure. Management consists of a strict and restrictive hypoproteic diet and the intake of amino acid substitutes and dietary supplements free of phenylalanine. If the benefits of treatment are indisputable in children in terms of cognitive prognosis, this benefit is discussed once brain development is complete, especially as many adult patients are no longer treated. However, cognitive, neurological and reversible white matter disorders undergoing treatment are increasingly reported in adult phenylketonurics. As a result, recent European recommendations advocate the maintenance of life-long treatment. One of the major difficulties, which is the source of many treatment failures, is the inappetence of the amino acid supplements required during a strict hypoproteic diet. New formulations, Glycomacropeptides (GMP), have recently appeared and are considered more palatable than conventional mixtures. PRIMARY OBJECTIVE: Demonstrate a better metabolic balance under GMP treatment than a conventional amino acid mixture in adult phenylketonuric patients when resuming treatment.

Conditions

• Adult Phenylketonuria Non Treated Patients

Interventions

Type	Name	Description
DIETARY_SUPPLEMENT	Dietary Supplement for PKU patients	For both treatment groups, the objective in total protein will be 1g / kg / day of ideal weight, in 3-6 doses / day, including natural proteins and supplemented by the products under study.

Timeline

• Start date: 2019-09-19
• Primary completion: 2022-03-14
• Completion: 2022-09-05
• First posted: 2019-04-23
• Last updated: 2022-09-21

Locations

7 sites across 1 country: France