Phenylketonurias Clinical Trial
Official title:
An Acceptability Study to Evaluate the Tolerance, Adherence and Metabolic Control of Patients With Phenylketonuria (PKU) Consuming PKU Sphereâ„¢, a Glycomacropeptide (GMP) Based Protein Substitute, When Introduced According to a Practical Guide to PKU Sphere.
Verified date | February 2024 |
Source | Vitaflo International, Ltd |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 13 |
Est. completion date | July 31, 2022 |
Est. primary completion date | July 31, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 3 Years and older |
Eligibility | Inclusion Criteria: - Patients diagnosed with PKU or hyperphenylalaninemia via new born screening and early treated, requiring >50% of protein intake from protein substitute. - Aged 3 years and above. - Female patients of childbearing potential must have a negative urine pregnancy test prior to completing the screening procedures. - Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/guardian for participants < 18 years. - Willingly given, written assent (if appropriate) for those < 18 years. Exclusion Criteria: - Pregnant, planning pregnancy or breastfeeding. - Intake of sapropterin dihydrochloride (Kuvan), PEG PAL or large neutral amino acids within 30 days prior to screening visit. - Patients currently taking their maximum recommended amount of protein substitute from a GMP based protein substitute. - Patients who are unable/unwilling to replace their current GMP protein substitute (if any) with PKU sphere. - Patients with known soya, milk or fish allergies / intolerance. - Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol. - Any co-morbidity, which in the opinion of the Investigator, would preclude participation in the study. - Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening visit. |
Country | Name | City | State |
---|---|---|---|
Italy | Sant'Orsola-Malpighi Polyclinic (adults) | Bologna | |
Italy | Sant'Orsola-Malpighi Polyclinic (paediatrics) | Bologna | |
Italy | Ospedale San Paolo | Milano | |
Italy | A.O.R.N. Santobono-Pausilipon - Ospedale Santobono | Napoli |
Lead Sponsor | Collaborator |
---|---|
Vitaflo International, Ltd |
Italy,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in adherence to recomended amounts of PKU sphere | Patient reported data on actual compared to prescribed intakes of PKU sphere | Recorded daily from start of 2-16 week Build-up and Stabilisation Period to end of 4 week Evaluation Period, assessed by HCPs periodically as per routine practice. | |
Primary | Change in gastrointestinal tolerance | Patient reported data on any gastrointestinal symptoms experienced | Data on previous 7 days collected by patients at the end of each Evaluation Period week. This data will be assessed by HCPs periodically as per routine practice. | |
Primary | Patient evaluation of PKU sphere's palatability, Evaluation Period week 2 | Patient reported Likert-scale evaluation of PKU sphere's appearance, smell, taste, aftertaste, texture, packaging/presentation and ease of use. A higher score denotes a more positive response. | End of Evaluation Period week 2. | |
Primary | Patient evaluation of PKU sphere's palatability, Evaluation Period week 4 | Patient reported Likert-scale evaluation of PKU sphere's appearance, smell, taste, aftertaste, texture, packaging/presentation and ease of use. A higher score denotes a more positive response. | End of Evaluation Period week 4. | |
Primary | Change in metabolic control (phenylalanine) over Build-up and Stabilisation Period. | Phenylalanine levels obtained from dried blood spots | Dried blood spots taken twice weekly during 2-16 week Build-up and Stabilisation Period. | |
Primary | Change in metabolic control (phenylalanine) over Evaluation Period. | Phenylalanine levels obtained from dried blood spots | Dried blood spots taken once weekly during 4-week Evaluation Period. | |
Primary | Change in metabolic control (tyrosine) over Build-up and Stabilisation Period. | Tyrosine levels obtained from dried blood spots | Dried blood spots taken twice weekly during 2-16 week Build-up and Stabilisation Period. | |
Primary | Change in metabolic control (tyrosine) over Evalaution Period. | Tyrosine levels obtained from dried blood spots | Dried blood spots taken once weekly during 4-week Evalaution Period. | |
Primary | Change in gatrointestinal quality of life | Age-specific, GI related quality of life questionnaire completed by participants. The specific version of the questionnaire to be used by each age group is listed below:
Age 2-4 years (Parental completion) = MAPI PedsQL Gastrointestinal Symptoms Scales, Toddlers (2-4) Age 5-7 years (Parental completion) = MAPI PedsQL Gastrointestinal Symptoms Scales, Young Children (5-7) Age 8-12 years (Parental completion) = MAPI PedsQL Gastrointestinal Symptoms Scales, Children (8-12) Age 13-18 years = MAPI PedsQL Gastrointestinal Symptoms Scales, Teens (13-18) Age 18 years and upwards = Birmingham IBS Symptom Questionnaire The MAPI PedsQL Gastrointestinal Symptoms Scale questionnaires and the Birmingham IBS symptom Questionnaire indicate fewer problems with higher calculated scores. |
Baseline visit (day one) and End of Study visit (post day-28 of Evaluation Period) | |
Primary | Change in PKU specific quality of life | Age-specific, PKU related quality of life questionnaire completed by participants. The specific version of the questionnaire to be used by each age group is listed below:
Age >9 years = MAPI phenylketonuria impact and treatment Quality Of Life Questionnaire (PKU-QOL), Parents Age 9-11 years = MAPI phenylketonuria impact and treatment Quality Of Life Questionnaire (PKU-QOL), Children Age 12-17 years = MAPI phenylketonuria impact and treatment Quality Of Life Questionnaire (PKU-QOL), Adolescent Age 18+ years = MAPI phenylketonuria impact and treatment Quality Of Life Questionnaire (PKU-QOL), Adult With all versions of the PKU-QOL, a higher calculated score denotes higher quality of life. |
Baseline visit (day one) and End of Study visit (post day-28 of Evaluation Period) |
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