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.
調査の概要
研究の種類
入学 (推定)
段階
- 適用できない
連絡先と場所
研究連絡先
- 名前：Ellen Taylor
- 電話番号：+447900724091
- メール：ellen.taylor@nutricia.com
研究場所
-
-
-
Bristol、イギリス
- University Hospitals Bristol and Weston NHS Foundation Trust
-
-
参加基準
適格基準
就学可能な年齢
- 子
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
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
研究計画
研究はどのように設計されていますか？
デザインの詳細
- 主な目的：基礎科学
- 割り当て：なし
- 介入モデル：単一グループの割り当て
- マスキング：なし（オープンラベル）
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
|
実験的：PKU Microtabet intervention product
Dietary Supplement: PKU Microtablet intervention product
|
PKU Microtablet intervention product
|
この研究は何を測定していますか？
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Gastro-intetsinal tolerance
時間枠：Measured at baseline (day 1) and end of intervention (day 29)
|
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).
|
Measured at baseline (day 1) and end of intervention (day 29)
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
コンプライアンス
時間枠：ベースライン（1日目）および介入終了（29日目）で測定
|
患者の通常のタンパク質代替物（ベースライン中）および研究産物（ケーススタディ期間中）の推奨摂取の順守は、ベースライン（1日目）およびケーススタディの終わり（29日目）の栄養士によって評価されます。
患者および/または両親/介護者は、過去24時間以内に採取したタンパク質代替量の量を平均して思い出すように求められます。
これは、栄養士が消費することが推奨される金額と比較されます。
患者のケアを管理する栄養士によって規定された1日額は、ケーススタディの開始時に記録され、ケーススタディ中のこの処方箋の変更も注目されます。
|
ベースライン（1日目）および介入終了（29日目）で測定
|
|
Acceptability
時間枠：Measured at baseline (day 1) and end of intervention (day 29)
|
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).
|
Measured at baseline (day 1) and end of intervention (day 29)
|
|
Blood Phe and Tyrosine
時間枠：Day 2 (baseline) and day 29 (end of intervention)
|
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.
|
Day 2 (baseline) and day 29 (end of intervention)
|
|
Anthropometry - Height
時間枠：Day 1 (baseline) and day 29 (end of intervention)
|
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).
|
Day 1 (baseline) and day 29 (end of intervention)
|
|
Anthropometry - Weight
時間枠：Day 1 (baseline) and day 29 (end of intervention)
|
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.
|
Day 1 (baseline) and day 29 (end of intervention)
|
|
Patient History
時間枠：Day 1 (baseline) and day 29 (end of intervention)
|
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.
|
Day 1 (baseline) and day 29 (end of intervention)
|
|
Safety/ Adverse events
時間枠：Throughout the case study series (day 1 to day 29).
|
All adverse events will be recorded, throughout the case study.
|
Throughout the case study series (day 1 to day 29).
|
協力者と研究者
スポンサー
捜査官
- スタディディレクター：Gary Hubbard, Dr、Nutricia, Inc.
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
追加の関連 MeSH 用語
その他の研究ID番号
- PKUM2025
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。