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
Tutkimuksen yleiskatsaus
Tila
Ehdot
Interventio / Hoito
Yksityiskohtainen kuvaus
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.
Opintotyyppi
Ilmoittautuminen (Todellinen)
Vaihe
- Vaihe 4
Yhteystiedot ja paikat
Opiskelupaikat
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Indiana
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Indianapolis, Indiana, Yhdysvallat, 46202
- Riley Hospital for Children
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Osallistumiskriteerit
Kelpoisuusvaatimukset
Opintokelpoiset iät
- Lapsi
- Aikuinen
Hyväksyy terveitä vapaaehtoisia
Kuvaus
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
Opintosuunnitelma
Miten tutkimus on suunniteltu?
Suunnittelun yksityiskohdat
- Ensisijainen käyttötarkoitus: Hoito
- Jako: Ei käytössä
- Inventiomalli: Yksittäinen ryhmätehtävä
- Naamiointi: Ei mitään (avoin tarra)
Aseet ja interventiot
Osallistujaryhmä / Arm |
Interventio / Hoito |
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Kokeellinen: 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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Mitä tutkimuksessa mitataan?
Ensisijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
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Body Mass Index (BMI)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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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Toissijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
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Gastrointestinal Symptom Severity Total Score (PAGI-SYM Total Score)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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)
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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)
Aikaikkuna: 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
Aikaikkuna: 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
Aikaikkuna: 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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Yhteistyökumppanit ja tutkijat
Sponsori
Yhteistyökumppanit
Julkaisuja ja hyödyllisiä linkkejä
Yleiset julkaisut
- Eypasch E, Williams JI, Wood-Dauphinee S, Ure BM, Schmulling C, Neugebauer E, Troidl H. Gastrointestinal Quality of Life Index: development, validation and application of a new instrument. Br J Surg. 1995 Feb;82(2):216-22. doi: 10.1002/bjs.1800820229.
- 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.
- Terlizzi V, Amato F, Castellani C, Ferrari B, Galietta LJV, Castaldo G, Taccetti G. Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype. Mol Genet Genomic Med. 2021 Apr;9(4):e1656. doi: 10.1002/mgg3.1656. Epub 2021 Mar 13.
- Stephenson KG, Lingle AJ, Baumberger KA, Dellon EP, Esther CR Jr, Meier EM, Oermann CM, Shenoy VK, Smith NR, Wimmer NS, Duehlmeyer SR, Kam CW, McKinzie CJ, Poisson MO, Elson EC. Changes in fecal elastase-1 following initiation of CFTR modulator therapy in pediatric patients with cystic fibrosis. J Cyst Fibros. 2023 Nov;22(6):996-1001. doi: 10.1016/j.jcf.2023.09.005. Epub 2023 Sep 26.
- Smith H, Rayment JH. Sustained recovery of exocrine pancreatic function in a teenager with cystic fibrosis treated with ivacaftor. Pediatr Pulmonol. 2020 Oct;55(10):2493-2494. doi: 10.1002/ppul.24952. Epub 2020 Jul 17. No abstract available.
- Schwarzenberg SJ, Vu PT, Skalland M, Hoffman LR, Pope C, Gelfond D, Narkewicz MR, Nichols DP, Heltshe SL, Donaldson SH, Frederick CA, Kelly A, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Solomon GM, Stalvey MS, Clancy JP, Rowe SM, Freedman SD; Promise Study Group. Elexacaftor/tezacaftor/ivacaftor and gastrointestinal outcomes in cystic fibrosis: Report of promise-GI. J Cyst Fibros. 2023 Mar;22(2):282-289. doi: 10.1016/j.jcf.2022.10.003. Epub 2022 Oct 21.
- Rosenfeld M, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Chilvers M, Higgins M, Tian S, Cooke J, Davies JC; KLIMB study group. An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB). J Cyst Fibros. 2019 Nov;18(6):838-843. doi: 10.1016/j.jcf.2019.03.009. Epub 2019 Apr 30.
- Ronan NJ, Einarsson GG, Deane J, Fouhy F, Rea M, Hill C, Shanahan F, Elborn JS, Ross RP, McCarthy M, Murphy DM, Eustace JA, Mm T, Stanton C, Plant BJ. Modulation, microbiota and inflammation in the adult CF gut: A prospective study. J Cyst Fibros. 2022 Sep;21(5):837-843. doi: 10.1016/j.jcf.2022.06.002. Epub 2022 Jun 25.
- Ratjen F, Hug C, Marigowda G, Tian S, Huang X, Stanojevic S, Milla CE, Robinson PD, Waltz D, Davies JC; VX14-809-109 investigator group. Efficacy and safety of lumacaftor and ivacaftor in patients aged 6-11 years with cystic fibrosis homozygous for F508del-CFTR: a randomised, placebo-controlled phase 3 trial. Lancet Respir Med. 2017 Jul;5(7):557-567. doi: 10.1016/S2213-2600(17)30215-1. Epub 2017 Jun 9.
- Ramsey ML, Li SS, Lara LF, Gokun Y, Akshintala VS, Conwell DL, Heintz J, Kirkby SE, McCoy KS, Papachristou GI, Patel A, Singh VK, Hart PA. Cystic fibrosis transmembrane conductance regulator modulators and the exocrine pancreas: A scoping review. J Cyst Fibros. 2023 Mar;22(2):193-200. doi: 10.1016/j.jcf.2022.08.008. Epub 2022 Aug 23.
- Pope CE, Vo AT, Hayden HS, Weiss EJ, Durfey S, McNamara S, Ratjen A, Grogan B, Carter S, Nay L, Parsek MR, Singh PK, McKone EF, Aitken ML, Rosenfeld MR, Hoffman LR. Changes in fecal microbiota with CFTR modulator therapy: A pilot study. J Cyst Fibros. 2021 Sep;20(5):742-746. doi: 10.1016/j.jcf.2020.12.002. Epub 2020 Dec 31.
- Nichols AL, Davies JC, Jones D, Carr SB. Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor. Paediatr Respir Rev. 2020 Sep;35:99-102. doi: 10.1016/j.prrv.2020.04.003. Epub 2020 Apr 14.
- Moshiree B, Freeman AJ, Vu PT, Khan U, Ufret-Vincenty C, Heltshe SL, Goss CH, Schwarzenberg SJ, Freedman SD, Borowitz D, Sathe M; GALAXY Study Group. Multicenter prospective study showing a high gastrointestinal symptom burden in cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):266-274. doi: 10.1016/j.jcf.2022.10.006. Epub 2022 Oct 29.
- Megalaa R, Gopalareddy V, Champion E, Goralski JL. Time for a gut check: Pancreatic sufficiency resulting from CFTR modulator use. Pediatr Pulmonol. 2019 Aug;54(8):E16-E18. doi: 10.1002/ppul.24353. Epub 2019 May 7.
- McNamara JJ, McColley SA, Marigowda G, Liu F, Tian S, Owen CA, Stiles D, Li C, Waltz D, Wang LT, Sawicki GS. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. Lancet Respir Med. 2019 Apr;7(4):325-335. doi: 10.1016/S2213-2600(18)30460-0. Epub 2019 Jan 24.
- Kounis I, Levy P, Rebours V. Ivacaftor CFTR Potentiator Therapy is Efficient for Pancreatic Manifestations in Cystic Fibrosis. Am J Gastroenterol. 2018 Jul;113(7):1058-1059. doi: 10.1038/s41395-018-0123-7. Epub 2018 Jun 11. No abstract available.
- Goralski JL, Hoppe JE, Mall MA, McColley SA, McKone E, Ramsey B, Rayment JH, Robinson P, Stehling F, Taylor-Cousar JL, Tullis E, Ahluwalia N, Chin A, Chu C, Lu M, Niu T, Weinstock T, Ratjen F, Rosenfeld M. Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2023 Jul 1;208(1):59-67. doi: 10.1164/rccm.202301-0084OC.
- Gould MJ, Smith H, Rayment JH, Machida H, Gonska T, Galante GJ. CFTR modulators increase risk of acute pancreatitis in pancreatic insufficient patients with cystic fibrosis. J Cyst Fibros. 2022 Jul;21(4):600-602. doi: 10.1016/j.jcf.2021.09.010. Epub 2021 Oct 31.
- Freeman AJ, Sathe M, Aliaj E, Borowitz D, Fogarty B, Goss CH, Freedman S, Heltshe SL, Khan U, Riva D, Roman C, Romasco M, Schwarzenberg SJ, Ufret-Vincenty CA, Moshiree B. Designing the GALAXY study: Partnering with the cystic fibrosis community to optimize assessment of gastrointestinal symptoms. J Cyst Fibros. 2021 Jul;20(4):598-604. doi: 10.1016/j.jcf.2020.12.021. Epub 2021 Jan 13.
- Freeman AJ, Ng K, Wang F, Abu-El-Haija MA, Chugh A, Cress GA, Fishman DS, Gariepy CE, Giefer MJ, Goday P, Gonska TY, Grover AS, Lindblad D, Liu QY, Maqbool A, Mark JA, McFerron BA, Mehta MS, Morinville VD, Noel RA, Ooi CY, Perito ER, Schwarzenberg SJ, Sellers ZM, Wilschanski M, Zheng Y, Yuan Y, Andersen DK, Lowe ME, Uc A; Consortium for the Study of Chronic Pancreatitis, Diabetes, and Pancreatic Cancer (CPDPC). Pancreatic Enzyme Use Reduces Pancreatitis Frequency in Children With Acute Recurrent or Chronic Pancreatitis: A Report From INSPPIRE. Am J Gastroenterol. 2024 Oct 1;119(10):2094-2102. doi: 10.14309/ajg.0000000000002772. Epub 2024 Mar 22.
- Davies JC, Wainwright CE, Sawicki GS, Higgins MN, Campbell D, Harris C, Panorchan P, Haseltine E, Tian S, Rosenfeld M. Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial. Am J Respir Crit Care Med. 2021 Mar 1;203(5):585-593. doi: 10.1164/rccm.202008-3177OC.
- Davies JC, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Robertson S, Green Y, Cooke J, Rosenfeld M; KIWI Study Group. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016 Feb;4(2):107-15. doi: 10.1016/S2213-2600(15)00545-7. Epub 2016 Jan 21.
- Carrion A, Borowitz DS, Freedman SD, Siracusa CM, Goralski JL, Hadjiliadis D, Srinivasan S, Stokes DC. Reduction of Recurrence Risk of Pancreatitis in Cystic Fibrosis With Ivacaftor: Case Series. J Pediatr Gastroenterol Nutr. 2018 Mar;66(3):451-454. doi: 10.1097/MPG.0000000000001788.
Opintojen ennätyspäivät
Opi tärkeimmät päivämäärät
Opiskelun aloitus (Todellinen)
Ensisijainen valmistuminen (Todellinen)
Opintojen valmistuminen (Todellinen)
Opintoihin ilmoittautumispäivät
Ensimmäinen lähetetty
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen Lähetetty (Todellinen)
Tutkimustietojen päivitykset
Viimeisin päivitys julkaistu (Todellinen)
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeksi vahvistettu
Lisää tietoa
Tähän tutkimukseen liittyvät termit
Avainsanat
Muita asiaankuuluvia MeSH-ehtoja
Muut tutkimustunnusnumerot
- 24152
Yksittäisten osallistujien tietojen suunnitelma (IPD)
Aiotko jakaa yksittäisten osallistujien tietoja (IPD)?
IPD-suunnitelman kuvaus
IPD-jaon aikakehys
IPD-jaon käyttöoikeuskriteerit
IPD-jakamista tukeva tietotyyppi
- STUDY_PROTOCOL
- MAHLA
- ICF
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