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- Klinische proef NCT02477319
A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT) (PROSPECT)
Studie Overzicht
Gedetailleerde beschrijving
Studietype
Inschrijving (Werkelijk)
Contacten en locaties
Studie Locaties
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Alabama
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Birmingham, Alabama, Verenigde Staten, 35233
- University of Alabama at Birmingham
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California
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Los Angeles, California, Verenigde Staten, 90027
- Childrens Hospital Los Angeles
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Palo Alto, California, Verenigde Staten, 94394
- Lucile S. Packard Children's Hospital
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Colorado
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Aurora, Colorado, Verenigde Staten, 80045
- The Children's Hospital Colarado
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Denver, Colorado, Verenigde Staten, 80206
- National Jewish Health
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Illinois
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Chicago, Illinois, Verenigde Staten, 60611-2605
- Ann & Robert H. Lurie Children's Hospital of Chicago
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Indiana
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Indianapolis, Indiana, Verenigde Staten
- Indianapolis University Hospital; James Whitcomb Riley Hospital for Children
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Kansas
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Kansas City, Kansas, Verenigde Staten, 66160
- The University of Kansas Hospital
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Maryland
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Baltimore, Maryland, Verenigde Staten, 21287
- John Hopkins University
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Massachusetts
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Boston, Massachusetts, Verenigde Staten, 02114
- Massachusetts General Hospital
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Boston, Massachusetts, Verenigde Staten, 02115
- Children's Hospital Boston
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Michigan
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Detroit, Michigan, Verenigde Staten, 48201
- Children's Hospital of Michigan
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Grand Rapids, Michigan, Verenigde Staten, 49503
- Devon Children's Hospital at Spectrum Health
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Minnesota
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Minneapolis, Minnesota, Verenigde Staten, 55455
- University of Minnesota
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Missouri
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Kansas City, Missouri, Verenigde Staten, 64108
- Children's Mercy Hospital
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Saint Louis, Missouri, Verenigde Staten, 63104
- Cardinal Glennon Children's Medical Center
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Saint Louis, Missouri, Verenigde Staten
- St. Louis Children's Hospital
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New Hampshire
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Lebanon, New Hampshire, Verenigde Staten
- Dartmouth Hitchcock Medical Center
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New York
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Buffalo, New York, Verenigde Staten, 14222
- Women and Children's Hospital of Buffalo
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New York, New York, Verenigde Staten, 10032
- Columbia University Medical Center
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Valhalla, New York, Verenigde Staten, 10595
- Maria Fareri Children's Hospital; Westchester Medical Center
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North Carolina
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Chapel Hill, North Carolina, Verenigde Staten, 27599
- University of North Carolina at Chapel Hill
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Ohio
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Akron, Ohio, Verenigde Staten, 44308
- Akron Children's Hospital
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Cincinnati, Ohio, Verenigde Staten, 45229
- Cincinnati Children's Hospital Medical Center
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Cleveland, Ohio, Verenigde Staten, 44106
- University Hospital of Cleveland
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Columbus, Ohio, Verenigde Staten
- Nation Wide Childrens Hospital
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Oregon
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Portland, Oregon, Verenigde Staten, 97239
- Oregon Health & Sciences University
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Pennsylvania
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Hershey, Pennsylvania, Verenigde Staten, 17033
- Hershey Medical Center; Penn State Children's Hospital
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Philadelphia, Pennsylvania, Verenigde Staten
- Children's Hospital of Philadelphia
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Pittsburgh, Pennsylvania, Verenigde Staten, 15213
- Children's Hospital of Pittsburgh of UPMC
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South Carolina
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Charleston, South Carolina, Verenigde Staten, 29403
- Medical University of South Carolina
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Tennessee
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Nashville, Tennessee, Verenigde Staten, 37232-9500
- The Children's Hospital at Vanderbilt
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Texas
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Houston, Texas, Verenigde Staten, 77030
- Baylor College of Medicine/Texas Children's Hospital
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Utah
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Salt Lake City, Utah, Verenigde Staten, 84132
- Primary Children's Hospital
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Washington
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Seattle, Washington, Verenigde Staten, 98195
- University of Washington Medical Center
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Seattle, Washington, Verenigde Staten, 98145-9807
- Seattle Children's Hospital
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Wisconsin
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Milwaukee, Wisconsin, Verenigde Staten, 53226
- Froedtert Hospital
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Milwaukee, Wisconsin, Verenigde Staten, 55455
- Children's Hospital of Wisconsin
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Deelname Criteria
Geschiktheidscriteria
Leeftijden die in aanmerking komen voor studie
Accepteert gezonde vrijwilligers
Geslachten die in aanmerking komen voor studie
Bemonsteringsmethode
Studie Bevolking
Part A N = 260 (210 CF, 50 non-CF controls)
- Cohort 1: 50 non-CF control subjects ≥ 12 years of age, with at least 15 subjects 12 - 21 yrs of age
- Cohort 2: 50 Partial CFTR Function CF subjects with at least one class IV/V CFTR mutation, ≥ 12 years of age
- Cohort 3 160 Absent CFTR Function CF subjects with two class I/II mutations ≥ 12 years of age
Part B
Up to 250 CF subjects who are homozygous for F508del mutation and who are prescribed ivacaftor/lumacafor for clinical care will be allowed to enroll. This will include :
- Cohort 3 subjects homozygous for F508del mutation from Part A who are prescribed ivacaftor/lumacaftor will be invited to participate in Part B (up to 100 potential subjects).
- Up to 150 additional CF subjects homozygous for F508del mutation ≥ 12 years of age who did not participate in Part A but are otherwise eligible for participation in Part B.
Beschrijving
Inclusion Criteria Part A COHORT 1:
1. Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
2. Be willing and able to adhere to the study visit schedule and other protocol requirements 3. Male or female ≥ 12 years of age at Visit 1. 4. Have a body mass index (BMI) of:
- For subjects ≥ 18 years of age: ≤ 30 kg/m2
For subjects 12 - 17 years of age: ≤ 95th percentile 5. Be a non-smoker for ≥ 1 year at screening and have ≤ 10 pack-year history of smoking.
6. To participate in the optional DNA banking component of this study, subject must have signed the informed consent indicating willingness to participate in the genomic component of the study. Refusal to give consent for this component does not exclude a subject from participation in the study.
Inclusion Cohorts 2-3
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
- Male or female ≥ 12 years of age at Visit 1.
Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and the following criteria: Cohort 2: (Partial Function CFTR CF)
Two mutations in the CFTR gene:
- At least one allele must be a Class IV or V mutation
- The second allele can be within any CFTR mutation class.
- Pancreatic sufficient (based on the absence of daily PERT use)
- At least one historic sweat chloride ≥60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) OR sweat chloride results ≥ 40, but < 60mEQ/L upon permission of the PROSPECT Investigator-Sponsors.
Cohort 3: (Absent Function CF)
• Two class I or II CFTR mutations
- Enrolled in the Cystic Fibrosis Foundation Patient Registry. Patients may enroll in the Registry at Visit 1 if not previously enrolled.
- Clinically stable with no significant changes in health status within 2 weeks prior to Visit 1.
- Be a non-smoker for ≥ 1 year at screening and have ≤ 10 pack-year history of smoking.
- To participate in the optional DNA banking component of this study, subject must have signed the informed consent indicating willingness to participate in the genomic component of the study. Refusal to give consent for this component does not exclude a subject from participation in the study
Part B Inclusion
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative.
- Physician decision to treat with ivacaftor/lumacaftor.
- Completion of at least Visit 1 and Visit 2 of Part A
Exclusion Criteria PART A COHORT 1
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- A history of any clinically significant medical illness or medical disorder that requires ongoing systemic medical therapy, including (but not limited to) cardiovascular disease, neuromuscular disease, hematological disease including bleeding disorders, chronic respiratory disease (including persistent asthma), hepatic or gastrointestinal (GI) disease, neurological disease, neoplastic disease, renal diseases, or endocrine disorders including diabetes.
- Acute illness requiring any new prescription or over-the-counter treatment within 14 days prior to Visit 1.
- Major or traumatic surgery within 12 weeks prior to Visit 1.
- For females of child-bearing potential: a positive pregnancy test at Visit 1.
- Initiation of any new chronic therapy within 28 days prior to Visit 1.
- Use of an investigational agent within 28 days prior to Visit 1.
Exclusion Part A COHORTS 2-3
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- Initiation of newly prescribed antibiotics [oral, intravenous (IV), and/or inhaled] for acute respiratory symptoms within 2 weeks of Visit 1.
- Major or traumatic surgery within 12 weeks prior to Visit 1.
- For females of child-bearing potential: a positive pregnancy test at Visit 1.
- Initiation of any new chronic therapy (e.g., ibuprofen Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 1.
- Use of an investigational agent within 28 days prior to Visit 1.
- Use of oral corticosteroids in doses exceeding 10 mg prednisone/day or 20 mg prednisone/every other day (subjects on oral steroids will be on stable doses for > 12 weeks prior to visit 1).
- Active treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled).
- Use of CFTR modulator therapy such as ivacaftor (Kalydeco®) within 28 days prior to Visit 1.
- History of lung or liver transplantation, or listing for organ transplantation.
Exclusion PART B
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- Initiation of newly prescribed antibiotics [oral, intravenous (IV), and/or inhaled] for acute respiratory symptoms within 2 weeks of Visit 4.
- Initiation of any new chronic therapy (e.g., ibuprofen, Pulmozyme®, hypertonic saline, azithromycin, TOBI®, Cayston®) within 4 weeks prior to Visit 4.
- Use of an investigational agent within 28 days prior to Visit 4.
- Use of oral corticosteroids in doses exceeding 10 mg prednisone/day or 20 mg prednisone/every other day (subjects on oral steroids will be on stable doses for > 12 weeks prior to Visit 4).
- Active treatment for nontuberculous mycobacterial (NTM) infection, consisting of ≥ two antibiotics (oral, IV, and/or inhaled).
- Use of CFTR modulator therapy such as ivacaftor (Kalydeco®) within 28 days prior to Visit 4.
Studie plan
Hoe is de studie opgezet?
Ontwerpdetails
Cohorten en interventies
Groep / Cohort |
Interventie / Behandeling |
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Part A
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Part B
CF patients who are homozygous for the F508del
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Wat meet het onderzoek?
Primaire uitkomstmaten
Uitkomstmaat |
Maatregel Beschrijving |
Tijdsspanne |
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Sweat Chloride by Cohort (Part A Only)
Tijdsspanne: For cohort 1, sweat chloride at Day 0 is time frame. For cohorts 2-3, sweat chloride averaged across all 3 visits at days 0, 14 and 90 is time frame.
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This is the primary endpoint for Part A per the PROSPECT protocol. Mean sweat chloride was not reported for Part B, as it is not a relevant statistic. For cohort 1, sweat chloride is from day 0 only. For cohorts 2-3, sweat chloride was averaged from days 0, 14, 90 via a random intercept longitudinal model. |
For cohort 1, sweat chloride at Day 0 is time frame. For cohorts 2-3, sweat chloride averaged across all 3 visits at days 0, 14 and 90 is time frame.
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6 Month Change in FEV1 Percent Predicted (Part B Only)
Tijdsspanne: Baseline and 6 months
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This is the primary endpoint for Part B per the PROSPECT protocol.
Change in FEV1 Percent Predicted is only relevant for Part B as it captures changes in lung function post-initiation of Ivacaftor/Lumacaftor.
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Baseline and 6 months
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Medewerkers en onderzoekers
Medewerkers
Publicaties en nuttige links
Studie record data
Bestudeer belangrijke data
Studie start
Primaire voltooiing (Werkelijk)
Studie voltooiing (Werkelijk)
Studieregistratiedata
Eerst ingediend
Eerst ingediend dat voldeed aan de QC-criteria
Eerst geplaatst (Schatting)
Updates van studierecords
Laatste update geplaatst (Werkelijk)
Laatste update ingediend die voldeed aan QC-criteria
Laatst geverifieerd
Meer informatie
Termen gerelateerd aan deze studie
Aanvullende relevante MeSH-voorwaarden
Andere studie-ID-nummers
- PROSPECT
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Synspira, Inc.BeëindigdLongziekten | Taaislijmziekte | Longziekte | Antibioticaresistente infectie | Aandoening van de luchtwegen | Cystic Fibrosis Longexacerbatie | Longontsteking | Burkholderia-infecties | Long infectie | Multi-antibioticaresistentie | Longontsteking | Longinfectie Pseudomonaal | Cystic Fibrosis Long | Cystic Fibrosis... en andere voorwaardenVerenigd Koninkrijk
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Dartmouth-Hitchcock Medical CenterTrustees of Dartmouth CollegeIngetrokkenAan cystic fibrosis gerelateerde diabetes | Cystic Fibrosis Leverziekte | CF - Cystische fibroseVerenigde Staten
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University Hospital TuebingenVoltooidGecombineerde droog poeder tobramycine en vernevelde colistine-inhalatie bij CF-patiënten (CotoCFII)Cystic Fibrosis Met Pulmonale Manifestaties