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Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study) (VOCAL)

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ClinicalTrials.gov Identifier: NCT02445053
Recruitment Status : Active, not recruiting
First Posted : May 15, 2015
Last Update Posted : August 5, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
To describe the effectiveness of Kalydeco® treatment in patients with cystic fibrosis (CF) who have 1 of 8 non G551D gating CFTR mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D).

Condition or disease Intervention/treatment
Cystic Fibrosis Drug: ivacaftor

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Study Type : Observational
Estimated Enrollment : 90 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)
Actual Study Start Date : April 2015
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor


Intervention Details:
  • Drug: ivacaftor
    Other Name: Kalydeco, VX-770


Primary Outcome Measures :
  1. Pulmonary exacerbations - Number of pulmonary exacerbations and duration of treatment for pulmonary exacerbations during Kalydeco treatment compared to the period before Kalydeco treatment [ Time Frame: 48 Months ]
  2. Respiratory Microbiology - Percentage of patients with cultures positive for Pseudomonas aeruginosa during Kalydeco treatment compared to the period before Kalydeco treatment [ Time Frame: 48 Months ]
  3. Respiratory Microbiology - Percentage of patients with cultures positive for bacteria other than Pseudomonas aeruginosa and for fungi during Kalydeco treatment compared to the period before Kalydeco treatment [ Time Frame: 48 Months ]
  4. FEV1: Absolute change in percent predicted FEV1 during Kalydeco treatment [ Time Frame: 48 Months ]
  5. Measures of nutritional status: Absolute change in weight, weight-for-age Z score, body mass index (BMI), and BMI-for-age Z-score during Kalydeco treatment [ Time Frame: 48 Months ]
  6. Comorbidities: Incidence and prevalence of comorbidities during Kalydeco treatment compared to the period before Kalydeco treatment [ Time Frame: 48 Months ]
  7. Mortality: Incidence and cause of deaths [ Time Frame: 48 Months ]
  8. Organ transplantation: Incidence and reason for organ transplantations [ Time Frame: 48 Months ]

Other Outcome Measures:
  1. To explore the effect of Kalydeco treatment on Health-Related Quality of Life (HRQoL) in patients with CF and in caregivers of pediatric patients enrolled in the study. [ Time Frame: 48 Months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Male and female patients with CF, age 6 years or older, who have 1 of the following CFTR mutations on at least 1 allele: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
Criteria

Inclusion Criteria:

  • Male or female with confirmed diagnosis of CF16
  • At least 1 allele with 1 of the following CFTR mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D
  • Six years of age or older on the date of signed (Informed Consent Form) ICF, and where appropriate, date of assent
  • Signed ICFs and, where appropriate, signed Assent Form
  • Able to understand the study requirements and comply with study data collection procedures

Exclusion Criteria:

  • Previously exposed to Kalydeco, except currently treated patients who started Kalydeco treatment within 6 months of enrollment
  • Currently enrolled in a Kalydeco interventional study or other interventional therapeutic clinical study directed at CFTR modulation
  • History of organ transplantation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02445053


Locations
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Italy
Bari, Italy
Messina, Italy
Milano, Italy
Milan, Italy
Naples, Italy
Potenza, Italy
Verona, Italy
Netherlands
Den Haag, South Holland, Netherlands
Amsterdam, Netherlands
Gronningen, Netherlands
Heidelberglaan, Netherlands
Nijmegen, Netherlands
Rotterdam, Netherlands
United Kingdom
London, Greater London, United Kingdom
Birmingham, West Midlands, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02445053     History of Changes
Other Study ID Numbers: VX14-770-116
2014-002704-24 ( EudraCT Number )
First Posted: May 15, 2015    Key Record Dates
Last Update Posted: August 5, 2019
Last Verified: August 2019
Keywords provided by Vertex Pharmaceuticals Incorporated:
Cystic Fibrosis
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action