Clinical Trials Logo

Clinical Trial Summary

20 participants with PKU will build-up their dietary intake of PKU sphere over 2-16 weeks, depending on their level of metabolic control whilst doing so. Participants will complete a gastrointestinal specific and PKU specific questionnaire at the Baseline clinic visit and record the amount of PKU sphere taken each day. Dried blood spots are taken twice per week. Once built up to a clinically appropriate intake of PKU sphere, or after 16 weeks, participants enter a 4-week Evaluation Period. The amount of PKU sphere taken per day continues to be recorded by participants. Gastrointestinal tolerance over the preceding seven days is recorded at the end of each week. Evaluations of PKU sphere's palatability are made at the end of weeks two and four of the Evaluation Period. Dried blood spots are taken once per week. The participant attends an End of Study Visit at the clinic and the investigator decides whether they should continue taking PKU sphere.


Clinical Trial Description

20 participants with phenylketonuria (PKU) will introduce PKU sphere into their diet. PKU sphere is a food for special medical purposes (as defined by Regulation (EU) No 609/2013 on food for specific groups), used for the dietary management of phenylketonuria from 3 years of age. The study begins with a Baseline clinic visit. Informed consent is taken by the investigator before the participant completes a PKU specific quality of life questionnaire, and a GI specific questionnaire. The participant takes a dried blood spot in order for the investigator to check their technique is correct. Build-up and Stabilisation Period. This will last between 2 and 16 weeks, depending on how long the participant takes to reach their maximum recommended intake of PKU sphere. Participants will take a dried blood spot twice per week in order for their health care professional to monitor their phenylalanine and tyrosine levels. After three consecutive dried blood spot results within the target range, the participant's HCP can advise to swap one portion of amino acid-based protein substitute for PKU sphere. This method of building up the amount of PKU sphere to be taken is informed directly by Vitaflo's 'Practical Guide to PKU sphere'. The amount of PKU sphere taken each day is recorded by the participant in their study diary. Once the maximum recommended amount of PKU sphere is being taken by the participant, or after 16 weeks, the participant moves into the four-week Evaluation Period. Participants then take a dried blood spot once per week, but continue to record their intake of PKU sphere each day. At the end of each Evaluation Period week, participants record any gastrointestinal symptoms experienced over the preceding seven days. At the end of Evaluation Period weeks two and four, participants will also record their thoughts on PKU sphere's palatability. This includes a Likert scale rating of the product's appearance, taste, aftertaste, smell, texture, packaging and ease of use. At the end of the Evaluation Period, participants return to the clinic for the End of Study visit. The PKU and GI specific questionnaires completed at Baseline are repeated and the Investigator discusses the suitability of the participant to continue to take PKU sphere as part of their regular therapeutic diet. Following the visit, the Investigator rates the utility of the Practical Guide to PKU sphere. A Follow-up Phone Call will relay the results of any outstanding dried blood spot results to participants following their End of Study Visit. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT04679467
Study type Interventional
Source Vitaflo International, Ltd
Contact
Status Completed
Phase N/A
Start date January 15, 2020
Completion date July 31, 2022

See also
  Status Clinical Trial Phase
Recruiting NCT05998109 - PheCheck Feasibility Study
Not yet recruiting NCT04433728 - Life With Phenylketonuria. Adult Neurological Outcome of PCU Screened Patients From 1971 to 2002.
Completed NCT01209819 - Bone Mineral Density in Adults With Hyperphenylalaninemia N/A
Active, not recruiting NCT05174559 - Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU) N/A
Not yet recruiting NCT04969809 - Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients N/A
Recruiting NCT04404530 - Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Recruiting NCT05827536 - Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU. N/A
Active, not recruiting NCT05971563 - Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers N/A
Completed NCT04368624 - PKU Skin Stripping
Completed NCT04452513 - A Prospective Clinical Study of Phenylketonuria (PKU)
Completed NCT05497050 - The Effect of Nursing Empowerment Program N/A
Completed NCT04943393 - Remote Neurocognitive and Psychological Assessment in PKU
Completed NCT05096988 - Evaluation of PKU Sphere Liquid N/A
Completed NCT00225615 - A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels Phase 3
Recruiting NCT05128149 - Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
Terminated NCT05229549 - Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria N/A
Enrolling by invitation NCT05356377 - Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
Completed NCT02445521 - Testing of Four Home Phenylalanine Monitoring Prototype Devices N/A
Active, not recruiting NCT04898829 - Evaluation of PKU Explore France N/A
Not yet recruiting NCT04302194 - Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.