PhenylketonuriasNCT04251494
A Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD).
An observational study of Phenylketonurias and Cardiovascular Diseases, run by Guy's and St Thomas' NHS Foundation Trust at 1 site in United Kingdom.
Trial summary
This study will assess the cardiovascular disease (CVD) risk in Phenylketonuria (PKU) patients on a low-phenylalanine diet (LPD). Ultrasound tests, diet information and routine blood samples will be collected once per patient at their next outpatient appointment. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Phenylketonurias, Cardiovascular Diseases
- What is being tested?
- Carotid intima media thickness measurement (CIMT), Pulse Wave Velocity measurement and Ankle-brachial pressure index (ABPI)
- How many people
- 32 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 23 Feb 2019to 22 Apr 2021
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Study population: Phenylketonuria patients at Guys and St Thomas' Centre for Inherited Metabolic Diseases, who have a diagnosis of Phenylketonuria at birth (following the introduction of the newborn screening programme), and who are over the age of 18 will be invited to take part in the study. The population is predominantly caucasian and middle aged.
Who may be able to join
- Phenylketonuria patients at Guys and St Thomas' Centre for Inherited Metabolic Diseases, who have a diagnosis of Phenylketonuria at birth (following the introduction of the newborn screening programme), and who are over the age of 18 will be invited to take part in the study.
- The study will only include patients diagnosed at birth, which will allow a better assessment of the effects of the diet, which are not influenced by a late diagnosis and a prior diet which is not low-Phenylalanine.
Who cannot join
- Phenylketonuria patients at Guys and St Thomas' Centre for Inherited Metabolic Diseases, who have a diagnosis of Phenylketonuria after birth (not diagnosed during newborn screening), will be excluded from taking part in the study.
- If patients are pregnant, or plan to be pregnant during the study they will also be excluded.
- Patients with a previous history of cardiovascular disease, and/or a history of cardiovascular disease in their immediate family will also be excluded.
- Patients that are unable to understand and consent to the study (i.e. due to language issues or lacking capacity) will also be excluded.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
Guy's and St Thomas' NHS Foundation Trust
London, SE1 9RT
Radha Ramachandran, 02071880848, Radha.Ramachandran@gstt.nhs.uk
About this study
Official title: An Observational, Cross-sectional Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD).
This study will assess the cardiovascular disease (CVD) risk in Phenylketonuria (PKU) patients on a low-phenylalanine diet (LPD).
Ultrasound tests, diet information and routine blood samples will be collected once per patient at their next outpatient appointment.
32 adults with PKU will be studied and compared to reference data for healthy people. The results will show if the PKU CVD risk differs from healthy people, and if CVD risk varies within people with PKU.
Read the detailed description
Cardiovascular disease (CVD) is the largest cause of deaths worldwide each year. Many factors affect CVD including diet, genetics, exercise and smoking. It is unclear if Phenylketonuria (PKU) and the low-phenylalanine diet (LPD) affect CVD risk.
CVD risk can be predicted by measuring the thickness of the artery wall in the neck which supplies blood to the head. Thickness is measured using a non-invasive, safe ultrasound test (similar to pregnancy jelly scans). A wall thicker than normal indicates an increased CVD risk. A wall thinner than normal indicates a lower CVD risk.
- Conditions
- Phenylketonurias, Cardiovascular Diseases
- Being tested
- Carotid intima media thickness measurement (CIMT), Pulse Wave Velocity measurement, Ankle-brachial pressure index (ABPI), Diet questionnaire and diet diary, Routine blood samples
- Keywords
- PKU, CVD, Cardiovascular disease risk
- Medical subject headings
- Phenylketonurias, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Carotid intima media thickness measurement (CIMT)Diagnostic test
CIMT is a measurement of the thickness of the two inner layers of the common carotid artery wall. Using ultrasound, a linear array transducer is placed on the surface of the skin at the neck. Electrocardiogram(ECG) electrodes are placed on each wrist and one on the ankle. An image of the common carotid artery (CCA) is then produced in anterior, posterior and lateral views, and saved along with the ECG recording. The wall thickness can then be measured at the same point in the cardiac cycle using semi-automated detection software (Philips QLAB), which can reduce observer variability. The CIMT will be averaged across left and right CIMTs, as the control reference values use an average of both sides.
Pulse Wave Velocity measurementDiagnostic test
The PWV measures the speed of a pulse wave travelling between two points in the vascular systems of known distance between them. PWV will be measured by the gold standard carotid-femoral PWV method which is a direct measurement of aortic stiffness. A 3-Lead ECG is connected alongside measurements of the blood velocity trace for the left CCA 2cm proximal to the bifurcation, and for the left common femoral artery origin.
Ankle-brachial pressure index (ABPI)Diagnostic test
A blood pressure cuff is placed around the ankle and upper arm of the participant. A continuous wave Doppler probe is used over the surface of the skin to locate the artery distal to the cuff (such as the brachial artery in the arm, or the anterior tibial and posterior tibial arteries in the ankle). The cuff is then inflated, and the systolic pressure identified by listening cessation of the sounds produced by the probe corresponding to blood flow in the artery. The ABPI is then the ratio of the highest systolic ankle pressure divided by the highest systolic brachial pressure for the left and right sides of the body.
Diet questionnaire and diet diaryOther
Dietary information will be collected in two formats. One will be a 14-item questionnaire (Martínez-González et al., 2012), which is a simplified version of a comprehensive food frequency questionnaire. This provides a quick measure of compliance to a Mediterranean dietary pattern and a rough measure of protective and adverse dietary factors that could affect individual CVD risk. The second will be a 3-day diet diary (3DDD). This will consist of participants recording the foods that they consume and the quantities, which will then be analysed for macro- and micronutrient composition once completed.
Routine blood samplesOther
Blood samples will be taken from participants during their outpatient appointment. Patients routinely have blood samples taken during this clinic. The study will use the results from the routine blood tests, and also extra blood samples to collect data on other biomarkers. The laboratory facilities at Guy's and St Thomas Hospitals will be used to conduct analysis, no other laboratories will be used. Data will be collected on Phenylalanine levels, lipids, vitamin B12 and related biomarkers.
| Study group | Role | What they receive |
|---|---|---|
| Phenylketonuria (PKU) participants | During their outpatient clinic appointment, participants will:
* Undergo routine height and weight measurements and blood tests. A full patient history will be taken, including a record of cardiovascular disease within the family. Blood samples will be collected, including phenylalanine, lipids, vitamin B12 and related biomarkers.
* Complete a 14-item diet history questionnaire, and fill in a 3 day diet diary before they arrive at their outpatient clinic appointment, which will be collected after the participant signs the informed written consent form.
* Undergo assessment of carotid Intima-media thickness (CIMT), pulse wave velocity (PWV), ankle brachial pressure index (ABPI) and systolic and diastolic blood pressure. Receives: Diagnostic Test: Carotid intima media thickness measurement (CIMT); Diagnostic Test: Pulse Wave Velocity measurement; Diagnostic Test: Ankle-brachial pressure index (ABPI); Other: Diet questionnaire and diet diary; Other: Routine blood samples | |
| Age and gender matched reference controls | Only Phenylketonuria (PKU) patients will be studied. Controls are generated from the literature. Reference CIMT values exist for a healthy population based on age and gender (Engelen et al., 2013), eliminating the need to assess age- and gender-matched controls. The study would otherwise require performing blood tests and vascular assessments on healthy individuals, generating a risk of harm and a possible incidental finding. It would be inconvenient for controls because they would need to travel to hospital and undergo invasive venepuncture. |
What is being measured
The primary outcome is the main question the study is designed to answer.
A single carotid-intima media thickness (CIMT) measurement in Phenylketonuria (PKU) participants and the corresponding age- and gender-matched controls.
A single CIMT measurement in PKU participants at their most recent outpatient appointment, and the corresponding age- and gender-matched CIMT, generated from the male (1) or female (2) equation for a healthy population using each participants' age and gender (Engelen et al., 2013).
Measured: 10 minutes
Secondary outcomes (6)
Pulse Wave Velocity (PWV)
will be compared to PKU patient biomarkers (median annual Phenylalanine levels, biomarkers and dietary factors) to assess if there is any correlation between compliance to diet and variation in cardiovascular risk within PKU patients.
Measured: 10 minutes
Ankle brachial pressure index (ABPI)
will be compared to PKU patient biomarkers (median annual Phe levels, biomarkers and dietary factors) to assess if there is any correlation between compliance to diet and variation in cardiovascular risk within PKU patients.
Measured: 10 minutes
Vitamin B12 levels from blood sample
will be compared to PKU patient measures (PWV, ABPI and CIMT) to assess if there is any correlation between compliance to diet and variation in cardiovascular risk within PKU patients.
Measured: 5 minutes
Blood Phenylalanine levels
will be compared to PKU patient measures (PWV, ABPI and CIMT) to assess if there is any correlation between compliance to diet and variation in cardiovascular risk within PKU patients.
Measured: 5 minutes
Blood low-density lipoprotein (LDL) cholesterol levels
will be compared to PKU patient measures (PWV, ABPI and CIMT) to assess if there is any correlation between compliance to diet and variation in cardiovascular risk within PKU patients.
Measured: 5 minutes
Diet diary and diet questionnaire (Mediterranean Adherence Screener, 0-14 scale, 14 indicating full adherence, 0 indicating no adherence)
will be compared to PKU patient measures (PWV, ABPI and CIMT) to assess if there is any correlation between compliance to diet and variation in cardiovascular risk within PKU patients.
Measured: 3 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2020
- Study start23 Feb 2019
- Primary completion22 Apr 2021 (estimated)
- Study completion22 Apr 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- More trials run by Guy's and St Thomas' NHS Foundation TrustThe 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
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guy's and St Thomas' NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Radha Ramachandran, Principal investigator, GSTT
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04251494
- Sponsor's protocol ID
- 254922
- Status last verified
- Jan 2020
- First submitted
- 25 Sep 2019
- Last updated
- 23 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Krikke M, Arends JE, Van Lelyveld S, Hoepelman A, Visseren F. Greater carotid intima media thickness at a younger age in HIV-infected patients compared with reference values for an uninfected cohort. HIV Med. 2017 Apr;18(4):275-283. doi: 10.1111/hiv.12428. Epub 2016 Aug 1.
PubMed 27477496Engelen L, Ferreira I, Stehouwer CD, Boutouyrie P, Laurent S; Reference Values for Arterial Measurements Collaboration. Reference intervals for common carotid intima-media thickness measured with echotracking: relation with risk factors. Eur Heart J. 2013 Aug;34(30):2368-80. doi: 10.1093/eurheartj/ehs380. Epub 2012 Nov 27.
PubMed 23186808Bots ML, Evans GW, Tegeler CH, Meijer R. Carotid Intima-media Thickness Measurements: Relations with Atherosclerosis, Risk of Cardiovascular Disease and Application in Randomized Controlled Trials. Chin Med J (Engl). 2016 Jan 20;129(2):215-26. doi: 10.4103/0366-6999.173500.
PubMed 26830994Hermida-Ameijeiras A, Crujeiras V, Roca I, Calvo C, Leis R, Couce ML. Arterial stiffness assessment in patients with phenylketonuria. Medicine (Baltimore). 2017 Dec;96(51):e9322. doi: 10.1097/MD.0000000000009322.
PubMed 29390507Htun P, Nee J, Ploeckinger U, Eder K, Geisler T, Gawaz M, Bocksch W, Fateh-Moghadam S. Fish-Free Diet in Patients with Phenylketonuria Is Not Associated with Early Atherosclerotic Changes and Enhanced Platelet Activation. PLoS One. 2015 Aug 20;10(8):e0135930. doi: 10.1371/journal.pone.0135930. eCollection 2015.
PubMed 26291823
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Observational, Cross-sectional Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD).
- ClinicalTrials.gov ID
- NCT04251494
- Acronym / sponsor ID
- 254922
- Conditions
- Phenylketonurias, Cardiovascular Diseases
- Keywords
- PKU, CVD, Cardiovascular disease risk
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Guy's and St Thomas' NHS Foundation TrustUniversity, hospital or other
- Study start
- 23 Feb 2019 (actual)
- Primary completion
- 22 Apr 2021 (estimated)
- Study completion
- 22 Apr 2021 (estimated)
- First posted
- 5 Feb 2020 (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.
This study will assess the cardiovascular disease (CVD) risk in Phenylketonuria (PKU) patients on a low-phenylalanine diet (LPD).
Ultrasound tests, diet information and routine blood samples will be collected once per patient at their next outpatient appointment.
32 adults with PKU will be studied and compared to reference data for healthy people. The results will show if the PKU CVD risk differs from healthy people, and if CVD risk varies within people with PKU.
Detailed description
Cardiovascular disease (CVD) is the largest cause of deaths worldwide each year. Many factors affect CVD including diet, genetics, exercise and smoking. It is unclear if Phenylketonuria (PKU) and the low-phenylalanine diet (LPD) affect CVD risk.
CVD risk can be predicted by measuring the thickness of the artery wall in the neck which supplies blood to the head. Thickness is measured using a non-invasive, safe ultrasound test (similar to pregnancy jelly scans). A wall thicker than normal indicates an increased CVD risk. A wall thinner than normal indicates a lower CVD risk.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phenylketonuria (PKU) participants | – | Diagnostic Test: Carotid intima media thickness measurement (CIMT), Diagnostic Test: Pulse Wave Velocity measurement, Diagnostic Test: Ankle-brachial pressure index (ABPI), Other: Diet questionnaire and diet diary, Other: Routine blood samplesDuring their outpatient clinic appointment, participants will: * Undergo routine height and weight measurements and blood tests. A full patient history will be taken, including a record of cardiovascular disease within the family. Blood samples will be collected, including phenylalanine, lipids, vitamin B12 and related biomarkers. * Complete a 14-item diet history questionnaire, and fill in a 3 day diet diary before they arrive at their outpatient clinic appointment, which will be collected after the participant signs the informed written consent form. * Undergo assessment of carotid Intima-media thickness (CIMT), pulse wave velocity (PWV), ankle brachial pressure index (ABPI) and systolic and diastolic blood pressure. |
| Age and gender matched reference controls | – | –Only Phenylketonuria (PKU) patients will be studied. Controls are generated from the literature. Reference CIMT values exist for a healthy population based on age and gender (Engelen et al., 2013), eliminating the need to assess age- and gender-matched controls. The study would otherwise require performing blood tests and vascular assessments on healthy individuals, generating a risk of harm and a possible incidental finding. It would be inconvenient for controls because they would need to travel to hospital and undergo invasive venepuncture. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Phenylketonuria patients at Guys and St Thomas' Centre for Inherited Metabolic Diseases, who have a diagnosis of Phenylketonuria at birth (following the introduction of the newborn screening programme), and who are over the age of 18 will be invited to take part in the study. The population is predominantly caucasian and middle aged.
Key inclusion criteria (2 listed)
- Phenylketonuria patients at Guys and St Thomas' Centre for Inherited Metabolic Diseases, who have a diagnosis of Phenylketonuria at birth (following the introduction of the newborn screening programme), and who are over the age of 18 will be invited to take part in the study.
- The study will only include patients diagnosed at birth, which will allow a better assessment of the effects of the diet, which are not influenced by a late diagnosis and a prior diet which is not low-Phenylalanine.
Key exclusion criteria (4 listed)
- Phenylketonuria patients at Guys and St Thomas' Centre for Inherited Metabolic Diseases, who have a diagnosis of Phenylketonuria after birth (not diagnosed during newborn screening), will be excluded from taking part in the study.
- If patients are pregnant, or plan to be pregnant during the study they will also be excluded.
- Patients with a previous history of cardiovascular disease, and/or a history of cardiovascular disease in their immediate family will also be excluded.
- Patients that are unable to understand and consent to the study (i.e. due to language issues or lacking capacity) will also be excluded.
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 |
|---|---|---|
| Carotid intima media thickness measurement (CIMT)CIMT is a measurement of the thickness of the two inner layers of the common carotid artery wall. Using ultrasound, a linear array transducer is placed on the surface of the skin at the neck. Electrocardiogram(ECG) electrodes are placed on each wrist and one on the ankle. An image of the common carotid artery (CCA) is then produced in anterior, posterior and lateral views, and saved along with the ECG recording. The wall thickness can then be measured at the same point in the cardiac cycle using semi-automated detection software (Philips QLAB), which can reduce observer variability. The CIMT will be averaged across left and right CIMTs, as the control reference values use an average of both sides. | Diagnostic test | Phenylketonuria (PKU) participants |
| Pulse Wave Velocity measurementThe PWV measures the speed of a pulse wave travelling between two points in the vascular systems of known distance between them. PWV will be measured by the gold standard carotid-femoral PWV method which is a direct measurement of aortic stiffness. A 3-Lead ECG is connected alongside measurements of the blood velocity trace for the left CCA 2cm proximal to the bifurcation, and for the left common femoral artery origin. | Diagnostic test | Phenylketonuria (PKU) participants |
| Ankle-brachial pressure index (ABPI)A blood pressure cuff is placed around the ankle and upper arm of the participant. A continuous wave Doppler probe is used over the surface of the skin to locate the artery distal to the cuff (such as the brachial artery in the arm, or the anterior tibial and posterior tibial arteries in the ankle). The cuff is then inflated, and the systolic pressure identified by listening cessation of the sounds produced by the probe corresponding to blood flow in the artery. The ABPI is then the ratio of the highest systolic ankle pressure divided by the highest systolic brachial pressure for the left and right sides of the body. | Diagnostic test | Phenylketonuria (PKU) participants |
| Diet questionnaire and diet diaryDietary information will be collected in two formats. One will be a 14-item questionnaire (Martínez-González et al., 2012), which is a simplified version of a comprehensive food frequency questionnaire. This provides a quick measure of compliance to a Mediterranean dietary pattern and a rough measure of protective and adverse dietary factors that could affect individual CVD risk. The second will be a 3-day diet diary (3DDD). This will consist of participants recording the foods that they consume and the quantities, which will then be analysed for macro- and micronutrient composition once completed. | Other | Phenylketonuria (PKU) participants |
| Routine blood samplesBlood samples will be taken from participants during their outpatient appointment. Patients routinely have blood samples taken during this clinic. The study will use the results from the routine blood tests, and also extra blood samples to collect data on other biomarkers. The laboratory facilities at Guy's and St Thomas Hospitals will be used to conduct analysis, no other laboratories will be used. Data will be collected on Phenylalanine levels, lipids, vitamin B12 and related biomarkers. | Other | Phenylketonuria (PKU) participants |
Primary outcome
A single carotid-intima media thickness (CIMT) measurement in Phenylketonuria (PKU) participants and the corresponding age- and gender-matched controls.
Time frame: 10 minutes
A single CIMT measurement in PKU participants at their most recent outpatient appointment, and the corresponding age- and gender-matched CIMT, generated from the male (1) or female (2) equation for a healthy population using each participants' age and gender (Engelen et al., 2013).
6 secondary outcomes
Pulse Wave Velocity (PWV)
Time frame: 10 minutes
Ankle brachial pressure index (ABPI)
Time frame: 10 minutes
Vitamin B12 levels from blood sample
Time frame: 5 minutes
Blood Phenylalanine levels
Time frame: 5 minutes
Blood low-density lipoprotein (LDL) cholesterol levels
Time frame: 5 minutes
Diet diary and diet questionnaire (Mediterranean Adherence Screener, 0-14 scale, 14 indicating full adherence, 0 indicating no adherence)
Time frame: 3 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jul 2020.
- Background: PubMed 27477496Krikke M, Arends JE, Van Lelyveld S, Hoepelman A, Visseren F. Greater carotid intima media thickness at a younger age in HIV-infected patients compared with reference values for an uninfected cohort. HIV Med. 2017 Apr;18(4):275-283. doi: 10.1111/hiv.12428. Epub 2016 Aug 1.
- Background: PubMed 23186808Engelen L, Ferreira I, Stehouwer CD, Boutouyrie P, Laurent S; Reference Values for Arterial Measurements Collaboration. Reference intervals for common carotid intima-media thickness measured with echotracking: relation with risk factors. Eur Heart J. 2013 Aug;34(30):2368-80. doi: 10.1093/eurheartj/ehs380. Epub 2012 Nov 27.
- Background: PubMed 26830994Bots ML, Evans GW, Tegeler CH, Meijer R. Carotid Intima-media Thickness Measurements: Relations with Atherosclerosis, Risk of Cardiovascular Disease and Application in Randomized Controlled Trials. Chin Med J (Engl). 2016 Jan 20;129(2):215-26. doi: 10.4103/0366-6999.173500.
- Background: PubMed 29390507Hermida-Ameijeiras A, Crujeiras V, Roca I, Calvo C, Leis R, Couce ML. Arterial stiffness assessment in patients with phenylketonuria. Medicine (Baltimore). 2017 Dec;96(51):e9322. doi: 10.1097/MD.0000000000009322.
- Background: PubMed 26291823Htun P, Nee J, Ploeckinger U, Eder K, Geisler T, Gawaz M, Bocksch W, Fateh-Moghadam S. Fish-Free Diet in Patients with Phenylketonuria Is Not Associated with Early Atherosclerotic Changes and Enhanced Platelet Activation. PLoS One. 2015 Aug 20;10(8):e0135930. doi: 10.1371/journal.pone.0135930. eCollection 2015.
- Individual participant dataSharing planned: No
Related trials for Phenylketonurias
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.
Radha Ramachandran principal investigator
GSTT
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 Phenylketonurias and Cardiovascular Diseases, run by Guy's and St Thomas' NHS Foundation Trust at 1 site in United Kingdom.
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 2 inclusion and 4 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: status not confirmed, as of the sponsor's last update on 23 Jul 2020. 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 United Kingdom. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Radha Ramachandran (02071880848, Radha.Ramachandran@gstt.nhs.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
A single carotid-intima media thickness (CIMT) measurement in Phenylketonuria (PKU) participants and the corresponding age- and gender-matched controls. (time frame: 10 minutes). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04251494, which also lists 5 linked publications.
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 23 Jul 2020.