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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-A03244-51 | Registry Identifier | IRCB |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| GMP - Dietary Supplement for PKU patients | Experimental | Glycomacropeptides -GMP Glytactin |
|
| Control -Amino acids mixtures | Active Comparator | Mixtures of conventional amino acids. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dietary Supplement for PKU patients | Dietary Supplement | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Rate of phenylalaninemia on blotter | Rate of phenylalaninemia on blotter measured bi-monthly during the 6 months of the study. | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Therapeutic compliance | Therapeutic compliance measured after 3 months and 6 months of treatment | 6 months |
| Evolution of neuropsychological tests | Neuropsychological tests measured after 3 months and 6 months of treatment |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Adrien BIGOT, MD-PHD | University Hospital of TOURS | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHRU-Hôpital Bretonneau - Service de Médecine Interne-Nutrition | Tours | Centre-Val de Loire | 37044 | France | ||
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| ID | Term |
|---|---|
| D019587 | Dietary Supplements |
| ID | Term |
|---|---|
| D005502 | Food |
| D000066888 | Diet, Food, and Nutrition |
| D010829 | Physiological Phenomena |
| D019602 | Food and Beverages |
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Multicenter randomized controlled trial performed in open and 2 parallel groups: "GMP" versus "amino acids" group
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| 6 months |
| MRI brain M0, M6 evolution | MRI brain evolution between inclusion and 6 months of treatment | 6 months |
| Bone remodeling markers | Bone remodeling markers at inclusion and 6 months of treatment | 6 months |
| Evolution of quality of life (PKU QoL score), mood (POMS test - Fillion 1999), at M0, M3, M6. | Evolution of quality of life scores at inclusion, 3 months and 6 months of treatment | 6 months |
| Nutritional and clinical markers evaluated at inclusion and 6 months of treatment | Evolution of nutritional and clinical markers at inclusion and 6 months of treatment | 6 months |
| Gastrointestinal tolerance at M3 and M6 | Evolution of Gastrointestinal tolerance after 3 months and 6 months of treatment | 6 months |
| CHU-ANGERS -Médecine Interne |
| Angers |
| 49933 |
| France |
| CHU du Morvan-Département de Pédiatrie et génétique médicale, | Brest | 29609 | France |
| Hôpital Femme-Mère-Enfant-Centre de Référence des Maladies Héréditaires du Métabolisme de Lyon | Bron | 69677 | France |
| CHU de LILLE-Hôpital Claude HURIEZ-Service d'Endocrinologie | Lille | 59037 | France |
| CHU-Service de Réanimation Pédiatrique / Néonatalogie, Consultation spécialisée en Maladies Héréditaires du Métabolisme | Nantes | 44000 | France |
| CHU-RENNES-Hôpital Sud-Service de Génétique-Clinique | Rennes | 35203 | France |