PKUNCT06629987
Determining the Most Efficient Screening Cut Off Levels for Phenylketonuria (PKU)
An observational study of PKU, run by Central Hospital, Nancy, France at 1 site in France.
Trial summary
studying the screening of PKU in france with the new tandemMS Read more about this study
- Who is it for?
- Ages 3 days and older
- Conditions the study is for
- PKU
- How many people
- 360 participants
- Where
- 1 siteFrance
- Study dates
- 1 Jan 2021to 31 Dec 2022
- How long will I take part?
- 1 Year
- Phase
- Observational
- 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 3 days and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: all french population born who had a screening
Who may be able to join
- having screening in france
Who cannot join
- refusing neonatal screening
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Determining the Most Efficient Screening Cut Off Levels for Phenylketonuria (PKU) by Assessing Two Years of Screening with Tandem Mass Spectrometry (MS) : a French National Cohort Study
studying the screening of PKU in france with the new tandemMS
Read the detailed description
Nationwide cohort study of the new pku screening for patient beetwen 180 and 360 µmol/L.
- Conditions
- PKU
- Keywords
- screening
What is being measured
The primary outcome is the main question the study is designed to answer.
developping an PKU: id phenylanine over 360µmol/L
based on level of phenylalanine during the year of follow up
Measured: 1 year
Secondary outcomes (1)
screening value
determining the bets screening value
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2024
- Study start1 Jan 2021
- Primary completion1 Jan 2021
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PKUStudies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 360 participants (actual)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Central Hospital, Nancy, France
- Sponsor type
- University, hospital or other
- Collaborators
- University Hospital, Lille, Hospices Civils de Lyon, University Hospital, Strasbourg, France, CHU de Reims, Centre Hospitalier Universitaire, Amiens, Centre Hospitalier Universitaire Dijon, Nantes University Hospital, University Hospital, Tours, Clinical Research Center of Reunion island, University Hospital, Caen, Rennes University Hospital, University Hospital, Bordeaux, University Hospital, Toulouse, University Hospital, Montpellier
- Responsible party
- Principal investigator, HESS Valentin, Central Hospital, Nancy, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06629987
- Sponsor's protocol ID
- 2022PI186
- Status last verified
- Oct 2024
- First submitted
- 4 Oct 2024
- Last updated
- 9 Oct 2024
- 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 9 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Determining the Most Efficient Screening Cut Off Levels for Phenylketonuria (PKU) by Assessing Two Years of Screening with Tandem Mass Spectrometry (MS) : a French National Cohort Study
- ClinicalTrials.gov ID
- NCT06629987
- Acronym / sponsor ID
- 2022PI186
- Conditions
- PKU
- Keywords
- screening
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 360 participants (actual)
- Lead sponsor
- Central Hospital, Nancy, FranceUniversity, hospital or other
- Collaborators
- University Hospital, Lille, Hospices Civils de Lyon, University Hospital, Strasbourg, France, CHU de Reims, Centre Hospitalier Universitaire, Amiens, Centre Hospitalier Universitaire Dijon, Nantes University Hospital, University Hospital, Tours, Clinical Research Center of Reunion island, University Hospital, Caen, Rennes University Hospital, University Hospital, Bordeaux, University Hospital, Toulouse, University Hospital, Montpellier
- Study start
- 1 Jan 2021 (actual)
- Primary completion
- 1 Jan 2021 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 8 Oct 2024 (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.
studying the screening of PKU in france with the new tandemMS
Detailed description
Nationwide cohort study of the new pku screening for patient beetwen 180 and 360 µmol/L.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 1 Year
Who may qualify
- Ages
- 3 days and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- all french population born who had a screening
Key inclusion criteria (1 listed)
- having screening in france
Key exclusion criteria (1 listed)
- refusing neonatal screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
developping an PKU: id phenylanine over 360µmol/L
Time frame: 1 year
based on level of phenylalanine during the year of follow up
1 secondary outcome
screening value
Time frame: 2 years
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 9 Oct 2024.
Related trials for PKU
RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of PKU, run by Central Hospital, Nancy, France at 1 site 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 1 inclusion and 1 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 9 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
developping an PKU: id phenylanine over 360µmol/L (time frame: 1 year). 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 9 Oct 2024.