Safety of Stopping Pancreatic Enzyme Replacement Therapy in Children With Improved Pancreatic Function After Highly Effective Modulator Therapy
The Return of the Pancreas: Evaluating Impact of CFTR Modulators on Pancreatic Function
The goal of this clinical trial is to evaluate the safety of stopping pancreatic enzyme replacement therapy (PERT) in children with cystic fibrosis (CF) receiving CFTR modulator therapy (CFTRm) who have regained pancreatic sufficiency. The main questions it aims to answer are:
- Does discontinuation of PERT affect gastrointestinal symptoms, nutritional status, pancreatic function, or body composition over 6 months?
- Does stopping PERT increase gastrointestinal symptoms or affect nutritional status, pancreatic function, or body composition compared with continuing PERT?
Researchers will evaluate changes in growth, gastrointestinal symptoms, vitamin levels, pancreatic function, and body composition following PERT discontinuation.
Participants will:
- Complete study visits and assessments over 6 months
- Continue or discontinue PERT based on study assignment
- Undergo anthropometric measurements
- Complete questionnaires about gastrointestinal symptoms
- Provide blood samples to assess vitamin levels and coagulation markers
- Provide stool samples to measure fecal elastase-1 (FE-1) and evaluate pancreatic function
調査の概要
状態
詳細な説明
This prospective clinical trial will evaluate the safety and feasibility of discontinuing pancreatic enzyme replacement therapy (PERT) in children with cystic fibrosis (CF) who regain pancreatic sufficiency after treatment with cystic fibrosis transmembrane conductance regulator modulators (CFTRm).
Pancreatic insufficiency is a common complication of CF and is typically treated with lifelong PERT. Recent studies and case reports have shown that some individuals receiving CFTR modulators experience improvement or normalization of pancreatic function, as measured by fecal elastase-1 (FE-1) levels. However, there is limited evidence regarding the safety of stopping PERT after pancreatic function recovery. This study aims to address this knowledge gap by prospectively evaluating growth, gastrointestinal symptoms, nutritional status, and pancreatic function after PERT withdrawal.
Eligible participants are children and adolescents aged 18 years or younger with cystic fibrosis and a history of pancreatic insufficiency who are receiving CFTR modulator therapy. Participants will undergo fecal elastase testing at enrollment and will be assigned to study groups based on pancreatic function status. Participants with FE-1 levels ≥200 µg/g will discontinue PERT under medical supervision, while participants with FE-1 levels <200 µg/g will continue standard therapy.
Study assessments will include growth measurements, gastrointestinal symptom questionnaires, laboratory monitoring of fat-soluble vitamins and liver function, CFTR modulator trough levels, and repeat fecal elastase testing. Body composition will also be evaluated. Participants who discontinue PERT will undergo close clinical monitoring throughout the study period for signs of malabsorption, poor weight gain, recurrent pancreatic insufficiency, or pancreatitis. PERT may be restarted if clinically indicated.
The study will also explore factors associated with successful recovery of pancreatic function and sustained pancreatic sufficiency, including demographic characteristics, nutritional status, medication history, and laboratory markers. In addition, optional biospecimen storage will allow future analyses of the metabolome and microbiome related to pancreatic function and CFTR modulator response.
This study is intended to provide feasibility and safety data to support future larger multicenter trials and to improve individualized treatment strategies for children with CF who experience recovery of pancreatic function after CFTR modulator therapy.
研究の種類
入学 (実際)
段階
- フェーズ 4
連絡先と場所
研究場所
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Indiana
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Indianapolis、Indiana、アメリカ、46202
- Riley Hospital for Children
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参加基準
適格基準
就学可能な年齢
- 子
- 大人
健康ボランティアの受け入れ
説明
Inclusion Criteria:
- Diagnosis of cystic fibrosis.
- History of pancreatic insufficiency, documented by a prior fecal elastase-1 (FE-1) concentration <200 µg/g stool.
- Current pancreatic sufficiency at study entry, defined as fecal elastase-1 (FE-1) concentration ≥200 µg/g stool after treatment with a CFTR modulator.
- Age ≤18 years.
- Current use of a CFTR modulator, including ivacaftor, elexacaftor/tezacaftor/ivacaftor, or vanzacaftor/tezacaftor/deutivacaftor.
Exclusion Criteria:
- CF-related diabetes requiring current insulin use
- Advanced CF liver disease as defined by nodular liver, advanced fibrosis (F4), multi-lobular cirrhosis with or without portal hypertension, non-cirrhotic portal hypertension
- Short gut syndrome as defined by need for surgical bowel resection and subsequent need for parenteral nutrition for > 60 days or bowel length less than 25%
- Moderate to severe malnutrition, defined as a BMI-for-age z score ≤ -2 for participants aged ≥2 years or a weight-for-length z score ≤ -2 for participants aged <2 years
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：処理
- 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 |
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実験的：Intervention: Discontinuation of Pancreatic Enzyme Replacement Therapy
Participants with cystic fibrosis who demonstrate pancreatic sufficiency, defined as fecal elastase-1 (FE-1) ≥200 µg/g after treatment with CFTR modulator therapy, will discontinue pancreatic enzyme replacement therapy (PERT) under medical supervision.
Participants will undergo follow-up assessments over 6 months, including monitoring of growth, gastrointestinal symptoms, nutritional laboratory markers, and repeat fecal elastase testing to evaluate the safety and sustainability of pancreatic function recovery after PERT discontinuation.
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Participants with cystic fibrosis who demonstrate pancreatic sufficiency, defined as fecal elastase-1 (FE-1) ≥200 µg/g after treatment with CFTR modulator therapy, will discontinue pancreatic enzyme replacement therapy (PERT) under medical supervision.
Participants will undergo follow-up assessments over 6 months, including monitoring of growth, gastrointestinal symptoms, nutritional laboratory markers, and repeat fecal elastase testing to evaluate the safety and sustainability of pancreatic function recovery after PERT discontinuation.
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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 |
|---|---|---|
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Body Mass Index (BMI)
時間枠：6 months
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Description: BMI calculated as body weight in kilograms divided by height in meters squared (kg/m²), using weight and height measurements obtained at each study visit. Time Frame: Baseline (Day 0 ± 28 days), Day 90 ± 28 days, Day 180 ± 28 days Type: Continuous |
6 months
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Weight (kg)
時間枠：6 months
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Description: Participant body weight measured using a calibrated digital clinical scale. Weight will be recorded in kilograms (kg) to the nearest 0.1 kg. Time Frame: Baseline (Day 0 ± 28 days), Day 90 ± 28 days, Day 180 ± 28 days Type: Continuous |
6 months
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Height (cm)
時間枠：6 months
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Description: Participant standing height measured using a wall-mounted stadiometer. Height will be recorded in centimeters (cm) to the nearest 0.1 cm. Time Frame: Baseline (Day 0 ± 28 days), Day 90 ± 28 days, Day 180 ± 28 days Type: Continuous |
6 months
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二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
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Gastrointestinal Symptom Severity Total Score (PAGI-SYM Total Score)
時間枠：6 months
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Description: Participants complete the validated Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The PAGI-SYM total score is calculated as the mean of all completed items and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse gastrointestinal symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Heartburn/Regurgitation Symptom Severity (Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index [PAGI-SYM] Heartburn/Regurgitation Domain Score)
時間枠：6 months
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Description: Heartburn/regurgitation domain score from the Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The domain score is calculated as the mean of the completed items within the heartburn/regurgitation domain and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Nausea/Vomiting Symptom Severity (Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index [PAGI-SYM] Nausea/Vomiting Domain Score)
時間枠：6 months
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Description: Nausea/vomiting domain score from the Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The domain score is calculated as the mean of the completed items within the nausea/vomiting domain and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Postprandial Fullness/Early Satiety Symptom Severity (Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index [PAGI-SYM] Postprandial Fullness/Early Satiety Domain Score)
時間枠：6 months
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Description: Postprandial fullness/early satiety domain score from the Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The domain score is calculated as the mean of the completed items within the postprandial fullness/early satiety domain and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Bloating Symptom Severity (Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index [PAGI-SYM] Bloating Domain Score)
時間枠：6 months
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Description: Bloating domain score from the Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The domain score is calculated as the mean of the completed items within the bloating domain and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Upper Abdominal Pain Symptom Severity (Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index [PAGI-SYM] Upper Abdominal Pain Domain Score)
時間枠：6 months
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Description: Upper abdominal pain domain score from the Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The domain score is calculated as the mean of the completed items within the upper abdominal pain domain and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Lower Abdominal Pain Symptom Severity (Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index [PAGI-SYM] Lower Abdominal Pain Domain Score)
時間枠：6 months
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Description: Lower abdominal pain domain score from the Patient Assessment of Upper Gastrointestinal Disorders Symptom Severity Index (PAGI-SYM). The domain score is calculated as the mean of the completed items within the lower abdominal pain domain and ranges from 0 (none) to 5 (very severe). Higher scores indicate worse symptom severity. Time Frame: Day 0 ± 28 days (pre-intervention), Day 90 ± 28 days, Day 180 ± 28 days. |
6 months
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Serum Vitamin A (Retinol) Concentration
時間枠：6 months
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How measured: Serum vitamin A (retinol) concentration measured in blood. Blood collected by venipuncture and analyzed by IU Health Pathology Laboratory and/or ARUP Laboratories. Results will be reported in milligrams per liter (mg/L). Time points: Day 180 ± 28 days. Unit of Measure: mg/L |
6 months
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Serum 25-Hydroxyvitamin D [25(OH)D] Concentration
時間枠：6 months
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How measured: Serum 25-Hydroxyvitamin D [25(OH)D] concentration measured in blood. Blood collected by venipuncture and analyzed by IU Health Pathology Laboratory and/or ARUP Laboratories. Results will be reported in nanograms per milliliter (ng/mL). Time points: Day 180 ± 28 days. Unit of Measure: ng/mL |
6 months
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Serum Vitamin E (Alpha-Tocopherol) Concentration
時間枠：6 months
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How measured: Serum Vitamin E (Alpha-Tocopherol) concentration measured in blood. Blood collected by venipuncture and analyzed by IU Health Pathology Laboratory and/or ARUP Laboratories. Results will be reported in milligrams per liter (mg/L). Time points: Day 180 ± 28 days. Unit of Measure: mg/L |
6 months
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International Normalized Ratio (INR)
時間枠：6 months
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What is measured: International Normalized Ratio (INR), a standardized measure of blood coagulation. How measured: Blood collected by venipuncture and analyzed by IU Health Pathology Laboratory and/or ARUP Laboratories. Time points: Day 180 ± 28 days. Unit of Measure: Unitless ratio (INR). |
6 months
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Fecal Elastase-1 (FE-1) Concentration
時間枠：6 months
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Description: Fecal elastase-1 (FE-1) concentration measured in stool as a marker of exocrine pancreatic function. Stool samples will be collected and analyzed by a certified clinical laboratory. Results will be reported in micrograms of elastase per gram of stool (µg/g stool). Higher values indicate better exocrine pancreatic function. Time Frame: Day 0 ± 28 days (pre-intervention) and Day 180 ± 28 days. Unit of Measure: µg/g stool |
6 months
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Sustainability of Pancreatic Sufficiency
時間枠：6 months
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Description: Maintenance of pancreatic sufficiency from baseline through Day 180, defined as fecal elastase-1 (FE-1) concentration greater than 200 µg/g stool at both baseline and Day 180. The outcome will be reported as the proportion of participants who remain pancreatic sufficient throughout the study period. Time Frame: Day 0 ± 28 days (pre-intervention), Day 180 ± 28 days Unit of Measure: Percent of participants maintaining pancreatic sufficiency |
6 months
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協力者と研究者
出版物と役立つリンク
一般刊行物
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- Stallings VA, Sainath N, Oberle M, Bertolaso C, Schall JI. Energy Balance and Mechanisms of Weight Gain with Ivacaftor Treatment of Cystic Fibrosis Gating Mutations. J Pediatr. 2018 Oct;201:229-237.e4. doi: 10.1016/j.jpeds.2018.05.018. Epub 2018 Jul 18.
- Hager ER, Quigg AM, Black MM, Coleman SM, Heeren T, Rose-Jacobs R, Cook JT, Ettinger de Cuba SA, Casey PH, Chilton M, Cutts DB, Meyers AF, Frank DA. Development and validity of a 2-item screen to identify families at risk for food insecurity. Pediatrics. 2010 Jul;126(1):e26-32. doi: 10.1542/peds.2009-3146.
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研究記録日
主要日程の研究
研究開始 (実際)
一次修了 (実際)
研究の完了 (実際)
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
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
- 24152
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?
IPD プランの説明
IPD 共有時間枠
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- STUDY_PROTOCOL
- SAP
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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
米国で製造され、米国から輸出された製品。
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