Adult Phenylketonuria Non Treated PatientsGLEEPHENNCT03924180
Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients
A study testing Dietary Supplement for PKU patients in people with Adult Phenylketonuria Non Treated Patients, run by University Hospital, Tours at 7 sites in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adult Phenylketonuria Non Treated Patients
- What is being tested?
- Dietary Supplement for PKU patients
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 7 sitesFrance
- Study dates
- 19 Sep 2019to 5 Sep 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
France7 sites
CHU-ANGERS -Médecine Interne
Angers, 49933
CHU du Morvan-Département de Pédiatrie et génétique médicale,
Brest, 29609
Hôpital Femme-Mère-Enfant-Centre de Référence des Maladies Héréditaires du Métabolisme de Lyon
Bron, 69677
CHU de LILLE-Hôpital Claude HURIEZ-Service d'Endocrinologie
Lille, 59037
CHU-Service de Réanimation Pédiatrique / Néonatalogie, Consultation spécialisée en Maladies Héréditaires du Métabolisme
Nantes, 44000
CHU-RENNES-Hôpital Sud-Service de Génétique-Clinique
Rennes, 35203
CHRU-Hôpital Bretonneau - Service de Médecine Interne-Nutrition
Tours, Centre-Val de Loire, 37044
About this study
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.
Read the 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.
- Being tested
- Dietary Supplement for PKU patients
- Keywords
- Amino acid, GMP, PKU
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dietary Supplement for PKU patientsDietary 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.
| Study group | Role | What they receive |
|---|---|---|
| GMP - Dietary Supplement for PKU patients | Experimental | Glycomacropeptides -GMP Glytactin Receives: Dietary Supplement: Dietary Supplement for PKU patients |
| Control -Amino acids mixtures | Active comparator | Mixtures of conventional amino acids. Receives: Dietary Supplement: Dietary Supplement for PKU patients |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of phenylalaninemia on blotter
Rate of phenylalaninemia on blotter measured bi-monthly during the 6 months of the study.
Measured: 6 months
Secondary outcomes (7)
Therapeutic compliance
Therapeutic compliance measured after 3 months and 6 months of treatment
Measured: 6 months
Evolution of neuropsychological tests
Neuropsychological tests measured after 3 months and 6 months of treatment
Measured: 6 months
MRI brain M0, M6 evolution
MRI brain evolution between inclusion and 6 months of treatment
Measured: 6 months
Bone remodeling markers
Bone remodeling markers at inclusion and 6 months of treatment
Measured: 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
Measured: 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
Measured: 6 months
Gastrointestinal tolerance at M3 and M6
Evolution of Gastrointestinal tolerance after 3 months and 6 months of treatment
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2019
- Study start19 Sep 2019
- Primary completion14 Mar 2022
- Study completion5 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adult Phenylketonuria Non Treated PatientsStudies of the same condition, open and under way.
- More trials run by University Hospital, ToursThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Multicenter randomized controlled trial performed in open and 2 parallel groups: "GMP" versus "amino acids" group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Tours
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Adrien BIGOT, MD-PHD, Study director, University Hospital of TOURS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03924180
- Sponsor's protocol ID
- DR180127 - GLEEPHEN
- Other IDs
- 2018-A03244-51
- Acronym
- GLEEPHEN
- Status last verified
- Sep 2022
- First submitted
- 22 Apr 2019
- Last updated
- 21 Sep 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria Patients
- ClinicalTrials.gov ID
- NCT03924180
- Acronym / sponsor ID
- GLEEPHEN / DR180127 - GLEEPHEN
- Keywords
- Amino acid, GMP, PKU
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- University Hospital, ToursUniversity, hospital or other
- Study start
- 19 Sep 2019 (actual)
- Primary completion
- 14 Mar 2022 (actual)
- Study completion
- 5 Sep 2022 (actual)
- First posted
- 23 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Multicenter randomized controlled trial performed in open and 2 parallel groups: "GMP" versus "amino acids" group
| Arm | Type | Receives |
|---|---|---|
| GMP - Dietary Supplement for PKU patients | Experimental | Dietary Supplement: Dietary Supplement for PKU patientsGlycomacropeptides -GMP Glytactin |
| Control -Amino acids mixtures | Active comparator | Dietary Supplement: Dietary Supplement for PKU patientsMixtures of conventional amino acids. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Dietary Supplement for PKU patientsFor 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. | Dietary supplement | Control -Amino acids mixtures, GMP - Dietary Supplement for PKU patients |
Primary outcome
Rate of phenylalaninemia on blotter
Time frame: 6 months
Rate of phenylalaninemia on blotter measured bi-monthly during the 6 months of the study.
7 secondary outcomes
Therapeutic compliance
Time frame: 6 months
Evolution of neuropsychological tests
Time frame: 6 months
MRI brain M0, M6 evolution
Time frame: 6 months
Bone remodeling markers
Time frame: 6 months
Evolution of quality of life (PKU QoL score), mood (POMS test - Fillion 1999), at M0, M3, M6.
Time frame: 6 months
Nutritional and clinical markers evaluated at inclusion and 6 months of treatment
Time frame: 6 months
Gastrointestinal tolerance at M3 and M6
Time frame: 6 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2022.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Adrien BIGOT, MD-PHD study director
University Hospital of TOURS
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Dietary Supplement for PKU patients in people with Adult Phenylketonuria Non Treated Patients, run by University Hospital, Tours at 7 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of phenylalaninemia on blotter (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Sep 2022.