<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03783286</url>
  </required_header>
  <id_info>
    <org_study_id>18-015299</org_study_id>
    <nct_id>NCT03783286</nct_id>
  </id_info>
  <brief_title>Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects</brief_title>
  <official_title>Nutritional Impact of Ivacaftor Treatment in 4 Month to 2 Year Old Children With CF Gating Mutations</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Children's Hospital of Philadelphia</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Vertex Pharmaceuticals Incorporated</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Children's Hospital of Philadelphia</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this research study is to determine the effects of clinically prescribed&#xD;
      ivacaftor treatment on 4-24 month old children with CF and gating mutations on sleeping&#xD;
      energy expenditure, growth status and gut health and function.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Ivacaftor is a novel FDA approved therapy for patients with CF and gating mutations who are 4&#xD;
      months and older. This investigator-Initiated study is designed to evaluate the nutritional,&#xD;
      growth and GI impact of ivacaftor treatment for the youngest (4-24 months) patient cohort and&#xD;
      for whom FDA approval has recently been granted. This proposal directly extends the previous&#xD;
      highly informative nutrition and weight gain investigation of ivacaftor treatment in the&#xD;
      older patient cohort (1). The primary aims of the study are to evaluate the impact of 12&#xD;
      weeks of ivacaftor treatment in 4-24 month old subjects with CF and gating mutations on&#xD;
      sleeping energy expenditure, growth status and gut health and function in 18 children with&#xD;
      protocol evaluations at baseline (pre-treatment) and 6 and 12 weeks after clinically&#xD;
      prescribed ivacaftor treatment has begun. Other outcomes of significant clinical interest in&#xD;
      young subjects with CF will be explored. All subjects will be evaluated at the Children's&#xD;
      Hospital of Philadelphia (CHOP) and will be recruited both regionally and nationally to&#xD;
      ensure timely enrollment.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">February 6, 2019</start_date>
  <completion_date type="Actual">December 30, 2021</completion_date>
  <primary_completion_date type="Actual">December 30, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Sleeping Energy Expenditure</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's SEE. Using indirect calorimetry, SEE will be assessed using a computerized metabolic cart Vmax ENCORE at each protocol visit while the child is asleep. SEE will be assessed in the morning if possible and careful note of previous feeding of the child, including the time of day, amount of food, and feeding interval prior to test</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Anthropometric Assessment</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's BMI. Investigators will compare the results to BMI Z scores over 12 weeks compared to baseline.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Fecal Elastase I/Pancreatic Function</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's pancreatic function. Pancreatic function will be assessed at two visits by obtaining spot stool samples with fecal elastase 1. The concentration of fecal elastase I is indicative of pancreatic function.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Fecal Calprotectin/Gut Inflammation</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's gut health and function. Spot stool samples will be obtained to determine fecal calprotectin, a marker for gut inflammation.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Plasma Total Fatty Acids:</measure>
    <time_frame>4 to 6 months</time_frame>
    <description>Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's dietary fat absorption. A total plasma fatty acid panel will be assessed to measure the change in status of 22 fatty acids, the concentration of plasma fatty acids is indicative of dietary fat absorption.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Dietary Intake</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Three day weighed food record will be obtained and to determine changes in dietary caloric intake and micro and macronutrient intake over the course of 12 weeks on ivacaftor treatment. The weighed food in grams will be used to determine the calories consumed, as well as calories from fat. The caloric intake will be used to determine micro and macro nutrient intake.</description>
  </other_outcome>
  <other_outcome>
    <measure>Serum fat soluble vitamins A, D, E and K, bile acids, and serum calprotectin</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will examine the changes in serum vitamin A, E, D and K concentrations after 12 weeks of Ivacaftor treatment. Additionally, investigators will examine the changes in total serum bile acids concentration and 14 bile acid species. Additionally, serum calprotectin will be obtained as a marker of lung and gut inflammation.</description>
  </other_outcome>
  <other_outcome>
    <measure>Muscle/Fat Stores</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will measure body composition to determine muscle and fat store changes over the course of 12 weeks on ivacaftor treatment compared to baseline.</description>
  </other_outcome>
  <other_outcome>
    <measure>Growth Status/Growth Velocity</measure>
    <time_frame>12 Weeks</time_frame>
    <description>Investigators will observe the changes in growth status/growth velocity. This will be assessed amongst three different measurements- length (cm), weight (kg) and head circumference (cm). Each value will be used to calculate the growth velocity percentile of the subjects over the course of 12 weeks on ivacaftor treatment compared to baseline.</description>
  </other_outcome>
  <enrollment type="Actual">15</enrollment>
  <condition>Cystic Fibrosis</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ivacaftor</intervention_name>
    <description>Ivacaftor (Kalydeco®, Vertex Pharmaceuticals Inc.) was the first of a new class of drugs that improved CFTR gating dysfunction (2, 3, 4). For such mutations (G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D) that permit CFTR expression at the cell membrane but compromise its activity.</description>
    <other_name>Kalydeco</other_name>
  </intervention>
  <biospec_retention>Samples Without DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Blood and stool collections&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        4-24 month old subjects with a diagnosis of cystic fibrosis with at least one CFTR gating&#xD;
        mutation of these ten (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1439D,&#xD;
        or R117H) approved for treatment in a usual state of good health with a clinical decision&#xD;
        to start ivacaftor treatment.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Cystic fibrosis with at least one CFTR gating mutation (E56K, G178R, S549R, S977F,&#xD;
             F1074L, 2789+5G→A,P67L, E193K, G551D, F1052V, D1152H, 3272-26A→G, R74W, L206W, G551S,&#xD;
             K1060T, G1244E, 3849+10kbC→T, D110E, R347H, D579G, A1067T, S1251N, D110H, R352Q,&#xD;
             711+3A→G, G1069R, S1255P, R117C, A455E, E831X, R1070Q, D1270N, R117H, S549N, S945L,&#xD;
             R1070W, G1349D) approved for treatment&#xD;
&#xD;
          -  Age: 4-24 months of age&#xD;
&#xD;
          -  In their usual state of good health&#xD;
&#xD;
          -  A clinical decision has been made for subject to begin ivacaftor treatment&#xD;
&#xD;
          -  Family committed to the 4 to 6 month study protocol with visits to CHOP that will last&#xD;
             2 or 3 days for the baseline visit (Visit 1) prior to ivacaftor and the 12 week visit&#xD;
             (Visit 3) after clinically prescribed ivacaftor treatment has begun, and will last 2&#xD;
             days for the 6 week visit (Visit 2) after ivacaftor treatment has begun.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  On parenteral nutrition&#xD;
&#xD;
          -  Use of any medications which are as inhibitors or inducers of cytochrome P450 (CYP) 3A&#xD;
&#xD;
          -  Liver function tests elevated above 3x the reference range for age and sex&#xD;
&#xD;
          -  Other illness affecting growth or nutritional status&#xD;
&#xD;
          -  Other contraindications described for ivacaftor therapy&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>4 Months</minimum_age>
    <maximum_age>2 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Virginia Stallings, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital of Philadelphia</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19146</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <reference>
    <citation>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.</citation>
    <PMID>30029855</PMID>
  </reference>
  <reference>
    <citation>Van Goor F, Hadida S, Grootenhuis PD, Burton B, Cao D, Neuberger T, Turnbull A, Singh A, Joubran J, Hazlewood A, Zhou J, McCartney J, Arumugam V, Decker C, Yang J, Young C, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu P. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18825-30. doi: 10.1073/pnas.0904709106. Epub 2009 Oct 21.</citation>
    <PMID>19846789</PMID>
  </reference>
  <reference>
    <citation>Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordonez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.</citation>
    <PMID>21083385</PMID>
  </reference>
  <reference>
    <citation>Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.</citation>
    <PMID>22047557</PMID>
  </reference>
  <reference>
    <citation>Energy and protein requirements. Report of a joint FAO/WHO/UNU Expert Consultation. World Health Organ Tech Rep Ser. 1985;724:1-206. No abstract available.</citation>
    <PMID>3937340</PMID>
  </reference>
  <reference>
    <citation>Schofield WN. Predicting basal metabolic rate, new standards and review of previous work. Hum Nutr Clin Nutr. 1985;39 Suppl 1:5-41.</citation>
    <PMID>4044297</PMID>
  </reference>
  <reference>
    <citation>7. World Health Organization. WHO Child Growth Standards: Length/height-for-age, weight-for-age, weight-for-length, weight-for-height, and body mass index-for-age. Geneva, Switzerland: WHO Press, World Health Organization; 2006.</citation>
  </reference>
  <reference>
    <citation>Borowitz D, Baker SS, Duffy L, Baker RD, Fitzpatrick L, Gyamfi J, Jarembek K. Use of fecal elastase-1 to classify pancreatic status in patients with cystic fibrosis. J Pediatr. 2004 Sep;145(3):322-6. doi: 10.1016/j.jpeds.2004.04.049.</citation>
    <PMID>15343184</PMID>
  </reference>
  <reference>
    <citation>Borowitz D, Lin R, Baker SS. Comparison of monoclonal and polyclonal ELISAs for fecal elastase in patients with cystic fibrosis and pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2007 Feb;44(2):219-23. doi: 10.1097/MPG.0b013e31802c41de.</citation>
    <PMID>17255835</PMID>
  </reference>
  <reference>
    <citation>O'Connor MG, Seegmiller A. The effects of ivacaftor on CF fatty acid metabolism: An analysis from the GOAL study. J Cyst Fibros. 2017 Jan;16(1):132-138. doi: 10.1016/j.jcf.2016.07.006. Epub 2016 Jul 26.</citation>
    <PMID>27473897</PMID>
  </reference>
  <reference>
    <citation>11. World Health Organization. WHO Child Growth Standards: Head circumference-for-age, arm circumference-for-age, triceps skinfold-for-age, and subscapular skinfold-for-age. Geneva, Switzerland: WHO Press, World Health Organization; 2007.</citation>
  </reference>
  <reference>
    <citation>12. WHO Multicenter Growth Reference Study Group. WHO Child Growth Standards: Growth Velocity Based on Weight, Length and Head Circumference: Methods and Development. Geneva, Switzerland: WHO Press: World Health Organization; 2009.</citation>
  </reference>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>December 18, 2018</study_first_submitted>
  <study_first_submitted_qc>December 18, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">December 21, 2018</study_first_posted>
  <last_update_submitted>March 10, 2022</last_update_submitted>
  <last_update_submitted_qc>March 10, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 11, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Children's Hospital of Philadelphia</investigator_affiliation>
    <investigator_full_name>Virginia Stallings</investigator_full_name>
    <investigator_title>Director of Nutrition Center Children's Hospital of Philadelphia, Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania</investigator_title>
  </responsible_party>
  <keyword>CF transmembrane conductance regulator</keyword>
  <keyword>CFTR</keyword>
  <keyword>Gating Mutations</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cystic Fibrosis</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ivacaftor</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>December 4, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/86/NCT03783286/Prot_SAP_003.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Informed Consent Form</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>Yes</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>December 4, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/86/NCT03783286/ICF_002.pdf</document_url>
    </provided_document>
  </provided_document_section>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03783286
org study id: 18-015299
nct id: NCT03783286
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
is us export: No
The purpose of this research study is to determine the effects of clinically prescribed
      ivacaftor treatment on 4-24 month old children with CF and gating mutations on sleeping
      energy expenditure, growth status and gut health and function.
Ivacaftor is a novel FDA approved therapy for patients with CF and gating mutations who are 4
      months and older. This investigator-Initiated study is designed to evaluate the nutritional,
      growth and GI impact of ivacaftor treatment for the youngest (4-24 months) patient cohort and
      for whom FDA approval has recently been granted. This proposal directly extends the previous
      highly informative nutrition and weight gain investigation of ivacaftor treatment in the
      older patient cohort (1). The primary aims of the study are to evaluate the impact of 12
      weeks of ivacaftor treatment in 4-24 month old subjects with CF and gating mutations on
      sleeping energy expenditure, growth status and gut health and function in 18 children with
      protocol evaluations at baseline (pre-treatment) and 6 and 12 weeks after clinically
      prescribed ivacaftor treatment has begun. Other outcomes of significant clinical interest in
      young subjects with CF will be explored. All subjects will be evaluated at the Children's
      Hospital of Philadelphia (CHOP) and will be recruited both regionally and nationally to
      ensure timely enrollment.
observational model: Cohort
time perspective: Prospective
measure: Sleeping Energy Expenditure
time frame: 12 Weeks
description: Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's SEE. Using indirect calorimetry, SEE will be assessed using a computerized metabolic cart Vmax ENCORE at each protocol visit while the child is asleep. SEE will be assessed in the morning if possible and careful note of previous feeding of the child, including the time of day, amount of food, and feeding interval prior to test
measure: Anthropometric Assessment
time frame: 12 Weeks
description: Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's BMI. Investigators will compare the results to BMI Z scores over 12 weeks compared to baseline.
measure: Fecal Elastase I/Pancreatic Function
time frame: 12 Weeks
description: Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's pancreatic function. Pancreatic function will be assessed at two visits by obtaining spot stool samples with fecal elastase 1. The concentration of fecal elastase I is indicative of pancreatic function.
measure: Fecal Calprotectin/Gut Inflammation
time frame: 12 Weeks
description: Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's gut health and function. Spot stool samples will be obtained to determine fecal calprotectin, a marker for gut inflammation.
measure: Plasma Total Fatty Acids:
time frame: 4 to 6 months
description: Investigators will examine the effects of 12 weeks of Ivacaftor treatment on subject's dietary fat absorption. A total plasma fatty acid panel will be assessed to measure the change in status of 22 fatty acids, the concentration of plasma fatty acids is indicative of dietary fat absorption.
measure: Dietary Intake
time frame: 12 Weeks
description: Three day weighed food record will be obtained and to determine changes in dietary caloric intake and micro and macronutrient intake over the course of 12 weeks on ivacaftor treatment. The weighed food in grams will be used to determine the calories consumed, as well as calories from fat. The caloric intake will be used to determine micro and macro nutrient intake.
measure: Serum fat soluble vitamins A, D, E and K, bile acids, and serum calprotectin
time frame: 12 Weeks
description: Investigators will examine the changes in serum vitamin A, E, D and K concentrations after 12 weeks of Ivacaftor treatment. Additionally, investigators will examine the changes in total serum bile acids concentration and 14 bile acid species. Additionally, serum calprotectin will be obtained as a marker of lung and gut inflammation.
measure: Muscle/Fat Stores
time frame: 12 Weeks
description: Investigators will measure body composition to determine muscle and fat store changes over the course of 12 weeks on ivacaftor treatment compared to baseline.
measure: Growth Status/Growth Velocity
time frame: 12 Weeks
description: Investigators will observe the changes in growth status/growth velocity. This will be assessed amongst three different measurements- length (cm), weight (kg) and head circumference (cm). Each value will be used to calculate the growth velocity percentile of the subjects over the course of 12 weeks on ivacaftor treatment compared to baseline.
intervention type: Drug
intervention name: Ivacaftor
description: Ivacaftor (Kalydeco®, Vertex Pharmaceuticals Inc.) was the first of a new class of drugs that improved CFTR gating dysfunction (2, 3, 4). For such mutations (G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D) that permit CFTR expression at the cell membrane but compromise its activity.
other name: Kalydeco
Blood and stool collections
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 4 Months
maximum age: 2 Years
healthy volunteers: No
last name: Virginia Stallings, MD
role: Principal Investigator
affiliation: Children's Hospital of Philadelphia
facility: 
country: United States
citation: 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.
PMID: 30029855
citation: Van Goor F, Hadida S, Grootenhuis PD, Burton B, Cao D, Neuberger T, Turnbull A, Singh A, Joubran J, Hazlewood A, Zhou J, McCartney J, Arumugam V, Decker C, Yang J, Young C, Olson ER, Wine JJ, Frizzell RA, Ashlock M, Negulescu P. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A. 2009 Nov 3;106(44):18825-30. doi: 10.1073/pnas.0904709106. Epub 2009 Oct 21.
PMID: 19846789
citation: Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordonez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003. doi: 10.1056/NEJMoa0909825.
PMID: 21083385
citation: Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185.
PMID: 22047557
citation: Energy and protein requirements. Report of a joint FAO/WHO/UNU Expert Consultation. World Health Organ Tech Rep Ser. 1985;724:1-206. No abstract available.
PMID: 3937340
citation: Schofield WN. Predicting basal metabolic rate, new standards and review of previous work. Hum Nutr Clin Nutr. 1985;39 Suppl 1:5-41.
PMID: 4044297
citation: 7. World Health Organization. WHO Child Growth Standards: Length/height-for-age, weight-for-age, weight-for-length, weight-for-height, and body mass index-for-age. Geneva, Switzerland: WHO Press, World Health Organization; 2006.
citation: Borowitz D, Baker SS, Duffy L, Baker RD, Fitzpatrick L, Gyamfi J, Jarembek K. Use of fecal elastase-1 to classify pancreatic status in patients with cystic fibrosis. J Pediatr. 2004 Sep;145(3):322-6. doi: 10.1016/j.jpeds.2004.04.049.
PMID: 15343184
citation: Borowitz D, Lin R, Baker SS. Comparison of monoclonal and polyclonal ELISAs for fecal elastase in patients with cystic fibrosis and pancreatic insufficiency. J Pediatr Gastroenterol Nutr. 2007 Feb;44(2):219-23. doi: 10.1097/MPG.0b013e31802c41de.
PMID: 17255835
citation: O'Connor MG, Seegmiller A. The effects of ivacaftor on CF fatty acid metabolism: An analysis from the GOAL study. J Cyst Fibros. 2017 Jan;16(1):132-138. doi: 10.1016/j.jcf.2016.07.006. Epub 2016 Jul 26.
PMID: 27473897
citation: 11. World Health Organization. WHO Child Growth Standards: Head circumference-for-age, arm circumference-for-age, triceps skinfold-for-age, and subscapular skinfold-for-age. Geneva, Switzerland: WHO Press, World Health Organization; 2007.
citation: 12. WHO Multicenter Growth Reference Study Group. WHO Child Growth Standards: Growth Velocity Based on Weight, Length and Head Circumference: Methods and Development. Geneva, Switzerland: WHO Press: World Health Organization; 2009.
responsible party type: Principal Investigator
investigator affiliation: Children's Hospital of Philadelphia
investigator full name: Virginia Stallings
investigator title: Director of Nutrition Center Children's Hospital of Philadelphia, Professor of Pediatrics, Perelman School of Medicine at the University of Pennsylvania
mesh term: Cystic Fibrosis
mesh term: Ivacaftor
sharing ipd: No
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