PKU Microtablets Case Studies
Evaluating the Adherence, Tolerance, Acceptability and Safety of a New Amino Acid Based, Microtablet Protein Substitute in PKU: a Case Study Series
Phenylalanine (Phe) free protein substitutes are typically presented in ready to drink liquid or powder format and are made up with water to a set volume. Despite recent advancements related to the taste, scent and texture of commercially available protein substitutes, a proportion of PKU patients choose to consume tablet-based protein substitutes. Protein substitutes in tablet format may help overcome taste, scent and texture concerns associated with powdered and liquid preparations. Indeed, previous research has shown that protein substitute tablets are an effective strategy to manage PKU in children, teenagers and adults. Nonetheless, the daily burden and discipline required often impedes compliance, especially alongside the restrictive low-protein diet. Achieving compliance with multivitamin supplements when given alongside protein substitutes therefore becomes especially challenging. This is particularly true for adolescent, adult and maternal populations.
PKU Microtablets have recently been developed. PKU Microtablets are a new phenylalanine-free tablet protein substitute combined with vitamins, minerals and trace elements. As patient acceptability is fundamental to the successful use of any protein substitute, investigation is needed to assess the compliance, tolerance and acceptability of PKU Microtablets as part of a low phenylalanine dietary regimen in PKU patients.
This series of case-studies aims to evaluate the gastrointestinal tolerance, acceptability, compliance, and safety of this protein substitute in both adults and children from 4 years. These case studies will last 29 days in total, including a 1-day baseline period followed by a 28-day intervention period. The case studies will be conducted across multiple specialist metabolic centres in the UK.
연구 개요
연구 유형
등록 (추정된)
단계
- 해당 없음
연락처 및 위치
연구 연락처
- 이름: Ellen Taylor
- 전화번호: +447900724091
- 이메일: ellen.taylor@nutricia.com
연구 장소
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Bristol, 영국
- University Hospitals Bristol and Weston NHS Foundation Trust
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참여기준
자격 기준
공부할 수 있는 나이
- 어린이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
설명
Inclusion Criteria:
- Male or female
- Over 4 years of age
- Patients diagnosed with any phenotype of PKU characterised by hyperphenylalaninaemia
- Have been compliant in taking at least one protein substitute, providing at least 20g protein equivalent, for at least 1 month prior to trial commencement
- Have a prescribed daily phenylalanine allowance for PKU
- Written or electronic informed consent from patient, and/or from parent/caregiver if applicable
- Participants who are anticipated to be able to take at least 20g protein equivalent of the case study product per day
Exclusion Criteria:
- Pregnant or lactating
- Requiring parenteral nutrition
- Major hepatic or renal dysfunction
- Participation in other studies within 1 month prior to entry of this study
- Allergy to any of the study product ingredients
- Investigator concern around willingness/ability of patient or parent/caregiver to comply with protocol requirement
- Treatment with Sapropterin dihydrochloride for less than 6 months
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 기초 과학
- 할당: 해당 없음
- 중재 모델: 단일 그룹 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
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실험적: PKU Microtabet intervention product
Dietary Supplement: PKU Microtablet intervention product
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PKU Microtablet intervention product
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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Gastro-intetsinal tolerance
기간: Measured at baseline (day 1) and end of intervention (day 29)
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GI tolerance will be recorded by the Dietitian at baseline on Day 1 and at the end of the case study period (Day 29).
GI tolerance will be recorded using a standardised questionnaire to capture perceived severity (none, mild, moderate or severe) of common gastrointestinal symptoms (diarrhoea, constipation, bloating, abdominal discomfort, vomiting and nausea).
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Measured at baseline (day 1) and end of intervention (day 29)
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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규정 준수
기간: 기준선 (1 일) 및 중재 종료 (29 일)에서 측정
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환자의 일반적인 단백질 대체물 (기준선 중) 및 연구 제품 (사례 연구 기간 동안)의 권장 섭취를 준수하는 것은 기준선 (1 일) 및 사례 연구 (29 일)의 영양사에 의해 평가됩니다.
환자 및/또는 부모/간병인은 지난 24 시간 동안 평균적으로 복용 한 단백질 대체의 양을 회상해야합니다.
이것은 영양사가 소비하는 것이 권장되는 양과 비교됩니다.
환자의 치료를 관리하는 영양사가 처방 한 일일 금액은 사례 연구가 시작될 때 기록되며 사례 연구 중에이 처방전의 변경도 주목할 것입니다.
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기준선 (1 일) 및 중재 종료 (29 일)에서 측정
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Acceptability
기간: Measured at baseline (day 1) and end of intervention (day 29)
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Acceptability (ease of use and liking) of the patients' usual protein substitute and the study product will be assessed by the Dietitian at baseline (Day 1) and the end of the case study period (Day 29) by a series of questions posed to the patient and/or parent/caregiver.
Acceptability questions will assess the patient's sensory experience, ease of use, tolerance, and overall self-rated acceptability on a 5-point Likert scale from Terrible (worst) to Great (best).
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Measured at baseline (day 1) and end of intervention (day 29)
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Blood Phe and Tyrosine
기간: Day 2 (baseline) and day 29 (end of intervention)
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Participants will be required to provide a total of two fasting fingerprick blood samples as dried blood spots; one on the first day of the intervention period (Day 2) before consuming any of the case study product, and one at the end of the intervention period (Day 29).
These tests will be conducted in addition to any usual blood tests the participants conduct as part of their usual disease management, which vary in frequency with the age of the participant and the local policy.
Dietitians will also be asked to describe the patient's metabolic control during the case study period.
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Day 2 (baseline) and day 29 (end of intervention)
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Anthropometry - Height
기간: Day 1 (baseline) and day 29 (end of intervention)
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Height will be measured, using standard methods, recorded to the nearest 0.1cm (at baseline only) and used to calculate body mass index (BMI, kg/m2).
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Day 1 (baseline) and day 29 (end of intervention)
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Anthropometry - Weight
기간: Day 1 (baseline) and day 29 (end of intervention)
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For safety purposes, at baseline (Day 1) and at the end of the case study (Day 29), body weight (kg) will be measured where possible using standard methods, recorded to the nearest 0.1kg using a weighing scale without heavy clothing.
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Day 1 (baseline) and day 29 (end of intervention)
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Patient History
기간: Day 1 (baseline) and day 29 (end of intervention)
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A detailed patient history will be recorded at baseline to assess previous compliance to protein substitutes, relevant medical and clinical issues, medication(s) prescribed, and any other relevant information related to the patient's condition or dietary management.
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Day 1 (baseline) and day 29 (end of intervention)
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Safety/ Adverse events
기간: Throughout the case study series (day 1 to day 29).
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All adverse events will be recorded, throughout the case study.
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Throughout the case study series (day 1 to day 29).
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공동 작업자 및 조사자
스폰서
수사관
- 연구 책임자: Gary Hubbard, Dr, Nutricia, Inc.
연구 기록 날짜
연구 주요 날짜
연구 시작 (추정된)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- PKUM2025
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
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