- ICH GCP
- USA klinikai vizsgálatok nyilvántartása
- Klinikai vizsgálat NCT02477319
A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT) (PROSPECT)
A tanulmány áttekintése
Részletes leírás
Tanulmány típusa
Beiratkozás (Tényleges)
Kapcsolatok és helyek
Tanulmányi helyek
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Alabama
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Birmingham, Alabama, Egyesült Államok, 35233
- University of Alabama at Birmingham
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California
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Los Angeles, California, Egyesült Államok, 90027
- Childrens Hospital Los Angeles
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Palo Alto, California, Egyesült Államok, 94394
- Lucile S. Packard Children's Hospital
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Colorado
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Aurora, Colorado, Egyesült Államok, 80045
- The Children's Hospital Colarado
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Denver, Colorado, Egyesült Államok, 80206
- National Jewish Health
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Illinois
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Chicago, Illinois, Egyesült Államok, 60611-2605
- Ann & Robert H. Lurie Children's Hospital of Chicago
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Indiana
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Indianapolis, Indiana, Egyesült Államok
- Indianapolis University Hospital; James Whitcomb Riley Hospital for Children
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Kansas
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Kansas City, Kansas, Egyesült Államok, 66160
- The University of Kansas Hospital
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Maryland
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Baltimore, Maryland, Egyesült Államok, 21287
- John Hopkins University
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Massachusetts
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Boston, Massachusetts, Egyesült Államok, 02114
- Massachusetts General Hospital
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Boston, Massachusetts, Egyesült Államok, 02115
- Children's Hospital Boston
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Michigan
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Detroit, Michigan, Egyesült Államok, 48201
- Children's Hospital of Michigan
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Grand Rapids, Michigan, Egyesült Államok, 49503
- Devon Children's Hospital at Spectrum Health
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Minnesota
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Minneapolis, Minnesota, Egyesült Államok, 55455
- University of Minnesota
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Missouri
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Kansas City, Missouri, Egyesült Államok, 64108
- Children's Mercy Hospital
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Saint Louis, Missouri, Egyesült Államok, 63104
- Cardinal Glennon Children's Medical Center
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Saint Louis, Missouri, Egyesült Államok
- St. Louis Children's Hospital
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New Hampshire
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Lebanon, New Hampshire, Egyesült Államok
- Dartmouth Hitchcock Medical Center
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New York
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Buffalo, New York, Egyesült Államok, 14222
- Women and Children's Hospital of Buffalo
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New York, New York, Egyesült Államok, 10032
- Columbia University Medical Center
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Valhalla, New York, Egyesült Államok, 10595
- Maria Fareri Children's Hospital; Westchester Medical Center
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North Carolina
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Chapel Hill, North Carolina, Egyesült Államok, 27599
- University of North Carolina At Chapel Hill
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Ohio
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Akron, Ohio, Egyesült Államok, 44308
- Akron Children's Hospital
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Cincinnati, Ohio, Egyesült Államok, 45229
- Cincinnati Children's Hospital Medical Center
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Cleveland, Ohio, Egyesült Államok, 44106
- University Hospital of Cleveland
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Columbus, Ohio, Egyesült Államok
- Nation Wide Childrens Hospital
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Oregon
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Portland, Oregon, Egyesült Államok, 97239
- Oregon Health & Sciences University
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Pennsylvania
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Hershey, Pennsylvania, Egyesült Államok, 17033
- Hershey Medical Center; Penn State Children's Hospital
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Philadelphia, Pennsylvania, Egyesült Államok
- Children's Hospital of Philadelphia
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Pittsburgh, Pennsylvania, Egyesült Államok, 15213
- Children's Hospital of Pittsburgh of UPMC
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South Carolina
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Charleston, South Carolina, Egyesült Államok, 29403
- Medical University of South Carolina
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Tennessee
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Nashville, Tennessee, Egyesült Államok, 37232-9500
- The Children's Hospital at Vanderbilt
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Texas
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Houston, Texas, Egyesült Államok, 77030
- Baylor College of Medicine/Texas Children's Hospital
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Utah
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Salt Lake City, Utah, Egyesült Államok, 84132
- Primary Children's Hospital
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Washington
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Seattle, Washington, Egyesült Államok, 98195
- University of Washington Medical Center
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Seattle, Washington, Egyesült Államok, 98145-9807
- Seattle Children's Hospital
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Wisconsin
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Milwaukee, Wisconsin, Egyesült Államok, 53226
- Froedtert Hospital
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Milwaukee, Wisconsin, Egyesült Államok, 55455
- Children's Hospital of Wisconsin
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Részvételi kritériumok
Jogosultsági kritériumok
Tanulmányozható életkorok
Egészséges önkénteseket fogad
Tanulmányozható nemek
Mintavételi módszer
Tanulmányi populáció
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.
Leírás
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.
Tanulási terv
Hogyan készül a tanulmány?
Tervezési részletek
Kohorszok és beavatkozások
Csoport / Kohorsz |
Beavatkozás / kezelés |
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Part A
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Part B
CF patients who are homozygous for the F508del
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Mit mér a tanulmány?
Elsődleges eredményintézkedések
Eredménymérő |
Intézkedés leírása |
Időkeret |
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Sweat Chloride by Cohort (Part A Only)
Időkeret: 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)
Időkeret: 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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Együttműködők és nyomozók
Szponzor
Együttműködők
Publikációk és hasznos linkek
Tanulmányi rekorddátumok
Tanulmány főbb dátumok
Tanulmány kezdete
Elsődleges befejezés (Tényleges)
A tanulmány befejezése (Tényleges)
Tanulmányi regisztráció dátumai
Először benyújtva
Először nyújtották be, amely megfelel a minőségbiztosítási kritériumoknak
Első közzététel (Becslés)
Tanulmányi rekordok frissítései
Utolsó frissítés közzétéve (Tényleges)
Az utolsó frissítés elküldve, amely megfelel a minőségbiztosítási kritériumoknak
Utolsó ellenőrzés
Több információ
A tanulmányhoz kapcsolódó kifejezések
További vonatkozó MeSH feltételek
Egyéb vizsgálati azonosító számok
- PROSPECT
Ezt az információt közvetlenül a clinicaltrials.gov webhelyről szereztük be, változtatás nélkül. Ha bármilyen kérése van vizsgálati adatainak módosítására, eltávolítására vagy frissítésére, kérjük, írjon a következő címre: register@clinicaltrials.gov. Amint a változás bevezetésre kerül a clinicaltrials.gov oldalon, ez a webhelyünkön is automatikusan frissül. .
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