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Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients (GLEEPHEN)

Primary Purpose

Adult Phenylketonuria Non Treated Patients

Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Dietary Supplement for PKU patients
Sponsored by
University Hospital, Tours
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Adult Phenylketonuria Non Treated Patients focused on measuring Amino acid, GMP, PKU

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • CHRU-Hôpital Bretonneau - Service de Médecine Interne-Nutrition
  • CHU-ANGERS -Médecine Interne
  • CHU du Morvan-Département de Pédiatrie et génétique médicale,
  • Hôpital Femme-Mère-Enfant-Centre de Référence des Maladies Héréditaires du Métabolisme de Lyon
  • CHU de LILLE-Hôpital Claude HURIEZ-Service d'Endocrinologie
  • CHU-Service de Réanimation Pédiatrique / Néonatalogie, Consultation spécialisée en Maladies Héréditaires du Métabolisme
  • CHU-RENNES-Hôpital Sud-Service de Génétique-Clinique

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

GMP - Dietary Supplement for PKU patients

Control -Amino acids mixtures

Arm Description

Glycomacropeptides -GMP Glytactin

Mixtures of conventional amino acids.

Outcomes

Primary Outcome Measures

Rate of phenylalaninemia on blotter
Rate of phenylalaninemia on blotter measured bi-monthly during the 6 months of the study.

Secondary Outcome Measures

Therapeutic compliance
Therapeutic compliance measured after 3 months and 6 months of treatment
Evolution of neuropsychological tests
Neuropsychological tests measured after 3 months and 6 months of treatment
MRI brain M0, M6 evolution
MRI brain evolution between inclusion and 6 months of treatment
Bone remodeling markers
Bone remodeling markers at inclusion and 6 months of treatment
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
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
Gastrointestinal tolerance at M3 and M6
Evolution of Gastrointestinal tolerance after 3 months and 6 months of treatment

Full Information

First Posted
April 22, 2019
Last Updated
September 20, 2022
Sponsor
University Hospital, Tours
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1. Study Identification

Unique Protocol Identification Number
NCT03924180
Brief Title
Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients
Acronym
GLEEPHEN
Official Title
Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients
Study Type
Interventional

2. Study Status

Record Verification Date
September 2022
Overall Recruitment Status
Completed
Study Start Date
September 19, 2019 (Actual)
Primary Completion Date
March 14, 2022 (Actual)
Study Completion Date
September 5, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Tours

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Adult Phenylketonuria Non Treated Patients
Keywords
Amino acid, GMP, PKU

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
Multicenter randomized controlled trial performed in open and 2 parallel groups: "GMP" versus "amino acids" group
Masking
None (Open Label)
Allocation
Randomized
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
GMP - Dietary Supplement for PKU patients
Arm Type
Experimental
Arm Description
Glycomacropeptides -GMP Glytactin
Arm Title
Control -Amino acids mixtures
Arm Type
Active Comparator
Arm Description
Mixtures of conventional amino acids.
Intervention Type
Dietary Supplement
Intervention Name(s)
Dietary Supplement for PKU patients
Intervention Description
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.
Primary Outcome Measure Information:
Title
Rate of phenylalaninemia on blotter
Description
Rate of phenylalaninemia on blotter measured bi-monthly during the 6 months of the study.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Therapeutic compliance
Description
Therapeutic compliance measured after 3 months and 6 months of treatment
Time Frame
6 months
Title
Evolution of neuropsychological tests
Description
Neuropsychological tests measured after 3 months and 6 months of treatment
Time Frame
6 months
Title
MRI brain M0, M6 evolution
Description
MRI brain evolution between inclusion and 6 months of treatment
Time Frame
6 months
Title
Bone remodeling markers
Description
Bone remodeling markers at inclusion and 6 months of treatment
Time Frame
6 months
Title
Evolution of quality of life (PKU QoL score), mood (POMS test - Fillion 1999), at M0, M3, M6.
Description
Evolution of quality of life scores at inclusion, 3 months and 6 months of treatment
Time Frame
6 months
Title
Nutritional and clinical markers evaluated at inclusion and 6 months of treatment
Description
Evolution of nutritional and clinical markers at inclusion and 6 months of treatment
Time Frame
6 months
Title
Gastrointestinal tolerance at M3 and M6
Description
Evolution of Gastrointestinal tolerance after 3 months and 6 months of treatment
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Adrien BIGOT, MD-PHD
Organizational Affiliation
University Hospital of TOURS
Official's Role
Study Director
Facility Information:
Facility Name
CHRU-Hôpital Bretonneau - Service de Médecine Interne-Nutrition
City
Tours
State/Province
Centre
ZIP/Postal Code
37044
Country
France
Facility Name
CHU-ANGERS -Médecine Interne
City
Angers
ZIP/Postal Code
49933
Country
France
Facility Name
CHU du Morvan-Département de Pédiatrie et génétique médicale,
City
Brest
ZIP/Postal Code
29609
Country
France
Facility Name
Hôpital Femme-Mère-Enfant-Centre de Référence des Maladies Héréditaires du Métabolisme de Lyon
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
CHU de LILLE-Hôpital Claude HURIEZ-Service d'Endocrinologie
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
CHU-Service de Réanimation Pédiatrique / Néonatalogie, Consultation spécialisée en Maladies Héréditaires du Métabolisme
City
Nantes
ZIP/Postal Code
44000
Country
France
Facility Name
CHU-RENNES-Hôpital Sud-Service de Génétique-Clinique
City
Rennes
ZIP/Postal Code
35203
Country
France

12. IPD Sharing Statement

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Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients

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