Phenylketonurias Clinical Trial
— express plusOfficial title:
A Study To Evaluate The Acceptability Of the Express Plus Range, Foods For Special Medical Purposes (FSMP), In Children And Adults With Inborn Errors of Metabolism (IEM)
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.
Status | Recruiting |
Enrollment | 40 |
Est. completion date | April 30, 2025 |
Est. primary completion date | October 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 3 Years and older |
Eligibility | Inclusion Criteria: - Diagnosis of PKU or associated disorder for example, Tetrahydrobiopterin (BH4) deficiency, requiring a low protein diet and Phe-free L-amino acid supplements (protein substitute). - Established on PKU express or an equivalent protein substitute - In the opinion of the study investigator, the participant is able to take the study product and meets at least one of the following criteria: 1. taking an age inappropriate protein substitute 2. struggling with adherence to current protein substitute 3. delayed in transitioning - Aged 3 years and above - Willingly given, written, informed consent from patient or parent/guardian. - Willingly given, written assent (if appropriate). Exclusion Criteria: - Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein substitute. - Patients who are tube-fed. - Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol. - Any co-morbidity/condition 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. - Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file). - Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period. (N.B.: Women who become pregnant unexpectedly during this study may, in consultation with their doctor, continue on the study's dietary product if they wish but will not have any investigations that would not normally be carried out during pregnancy.) - Those with allergies to fish, milk or soya. |
Country | Name | City | State |
---|---|---|---|
United Kingdom | Royal Belfast Hospital for Sick Children | Belfast | |
United Kingdom | Birmingham Women's and Children's Hospital | Birmingham | |
United Kingdom | St. Luke's Hospital | Bradford | |
United Kingdom | Bristol Royal Hospital for Children | Bristol | |
United Kingdom | Greater Glasgow and Clyde NHS Foundation Trust | Glasgow | |
United Kingdom | Great Ormond Street Hospital for Children | London | |
United Kingdom | Nottingham Children's Hospital | Nottingham |
Lead Sponsor | Collaborator |
---|---|
Vitaflo International, Ltd |
United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Questionnaire of self-reported adherence to the prescribed amount of study product | Participants will record the amount of PKU express taken each day compared to the amount recommended by their dietitian | Days 1-28 | |
Primary | Transition time | Time taken for participants to transition from their current diet to the inclusion of one sachet of PKU express plus per day | Transition period Weeks 1-6 (maximum) | |
Primary | Change in gastrointestinal tolerance from week 1 to week 4 | Participants will self-report any gastrointestinal symptoms experience over the course of the study in daily study diaries | Days 1-7 and days 21-28 | |
Primary | Product acceptability rated on a Likert scale by the patient after 28-day intake | Participants will answer questions relating to PKU express plus' palatability and ease of use following the end of study | Visit 2 (end of 28 day evaluation period) | |
Primary | Change in general neophobia | Participants to complete general neophobia questionnaire at baseline and end of study to compare results | Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period) | |
Primary | Change in food neophobia | Participants to complete food neophobia questionnaire at baseline and end of study to compare results | Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period) | |
Primary | Nutritional suitability: change Phenylalanine levels | PKU express plus' nutritional suitability will be assessed by evaluating Phenylalanine levels recorded as part of routine care | Visit 1 until visit 2 (day 28) | |
Primary | Nutritional suitability: change Tyrosine levels | PKU express plus' nutritional suitability will be assessed by evaluating Tyrosine levels recorded as part of routine care | Visit 1 until visit 2 (day 28) |
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