Adult Phenylketonuria Non Treated Patients Clinical Trial
— GLEEPHENOfficial title:
Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients
| Verified date | September 2022 |
| Source | University Hospital, Tours |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
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.
| Status | Completed |
| Enrollment | 13 |
| Est. completion date | September 5, 2022 |
| Est. primary completion date | March 14, 2022 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility | Inclusion Criteria: - Patient age =18 years on an empty stomach - Phenylketonuric patient Patient with Phenylalaninemia = 900µmol / L on a blotter performed during the screening period (or average of blotter results =900 µmol / L if several blotters performed during the screening period) - Untreated or insufficiently treated patient: not taking or insufficiently Dietary foods for special medical purposes for his PKU, regardless of diet, at the discretion of the investigator - Patient having signed a free, informed and express consent - Patient requiring a diet restricted in natural proteins Exclusion Criteria: - Protected patient: court bail - Patient with concomitant diseases / conditions that may compromise the study, at the discretion of the investigator - Participated in a clinical trial or trial to evaluate PKU foods or treatments in the last 7 days prior to inclusion or planned during the next 6 months - Participation in an interventional study with health products during the next 6 months - Pregnancy project within 6 months, pre-conception diet, pregnancy or breastfeeding - Refusal to consume only validated complements for the protocol - Phenylketonuria undergoing treatment with BH4 - Allergy to the product under study |
| Country | Name | City | State |
|---|---|---|---|
| France | CHU-ANGERS -Médecine Interne | Angers | |
| France | CHU du Morvan-Département de Pédiatrie et génétique médicale, | Brest | |
| France | Hôpital Femme-Mère-Enfant-Centre de Référence des Maladies Héréditaires du Métabolisme de Lyon | Bron | |
| France | CHU de LILLE-Hôpital Claude HURIEZ-Service d'Endocrinologie | Lille | |
| France | CHU-Service de Réanimation Pédiatrique / Néonatalogie, Consultation spécialisée en Maladies Héréditaires du Métabolisme | Nantes | |
| France | CHU-RENNES-Hôpital Sud-Service de Génétique-Clinique | Rennes | |
| France | CHRU-Hôpital Bretonneau - Service de Médecine Interne-Nutrition | Tours | Centre |
| Lead Sponsor | Collaborator |
|---|---|
| University Hospital, Tours |
France,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Rate of phenylalaninemia on blotter | Rate of phenylalaninemia on blotter measured bi-monthly during the 6 months of the study. | 6 months | |
| Secondary | Therapeutic compliance | Therapeutic compliance measured after 3 months and 6 months of treatment | 6 months | |
| Secondary | Evolution of neuropsychological tests | Neuropsychological tests measured after 3 months and 6 months of treatment | 6 months | |
| Secondary | MRI brain M0, M6 evolution | MRI brain evolution between inclusion and 6 months of treatment | 6 months | |
| Secondary | Bone remodeling markers | Bone remodeling markers at inclusion and 6 months of treatment | 6 months | |
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | Gastrointestinal tolerance at M3 and M6 | Evolution of Gastrointestinal tolerance after 3 months and 6 months of treatment | 6 months |