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Trial record 1 of 1 for:    809-116 | Cystic Fibrosis
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A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

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ClinicalTrials.gov Identifier: NCT03125395
Recruitment Status : Active, not recruiting
First Posted : April 24, 2017
Last Update Posted : December 20, 2017
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Lumacaftor Drug: Ivacaftor Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 57 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Rollover Study to Evaluate the Safety of Long-term Treatment With Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Actual Study Start Date : May 12, 2017
Estimated Primary Completion Date : July 26, 2019
Estimated Study Completion Date : July 26, 2019

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: Treatment Cohort
Subjects <6 years of age and <14 kg at enrollment: LUM 100 mg/IVA 125 mg q12h. Subjects <6 years of age and ≥14 kg at enrollment: LUM 150 mg/IVA 188 mg q12h. Subjects ≥6 years of age at enrollment, regardless of weight: LUM 200 mg/IVA 250 mg q12h.
Drug: Lumacaftor
LUM/IVA will be administered q12h
Other Name: LUM

Drug: Ivacaftor
LUM/IVA will be administered q12h
Other Name: IVA

No Intervention: Observational Cohort



Primary Outcome Measures :
  1. Safety and tolerability assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From baseline through safety follow-up (up to 98 weeks). ]
    Number of subjects with AEs and SAEs will be reported.


Secondary Outcome Measures :
  1. Absolute change from baseline in sweat chloride [ Time Frame: From baseline through 96 weeks. ]
    Sweat samples will be collected using an approved collection device. Absolute change in sweat chloride will be reported.

  2. Absolute change from baseline in body mass index (BMI) [ Time Frame: From baseline through 96 weeks. ]
    BMI is defined as weight in kilogram (kg) divided by height*height in square meter (m^2). Absolute change in BMI will be reported.

  3. Absolute change from baseline in BMI-for-age Z-score [ Time Frame: From baseline through 96 weeks. ]
    BMI is defined as weight in kg divided by height*height in m^2. Z-score is a statistical measure to evaluate how a single data point compares to a standard. Absolute change in BMI-for-age Z-score will be reported.

  4. Absolute change from baseline in weight [ Time Frame: From baseline through 96 weeks. ]
    Weight will be measured in kg and absolute change will be reported.

  5. Absolute change from baseline in weight-for-age Z-score [ Time Frame: From baseline through 96 weeks. ]
    Weight will be measured in kg. Z-score is a statistical measure to evaluate how a single data point compares to a standard. Absolute change in weight-for-age Z-score will be reported.

  6. Absolute change from baseline in stature [ Time Frame: From baseline through 96 weeks. ]
    Stature (height) will be measured in centimeter (cm) and absolute change will be reported.

  7. Absolute change from baseline in stature-for-age Z-score [ Time Frame: From baseline through 96 weeks. ]
    Stature (height) will be measured in cm. Z-score is a statistical measure to evaluate how a single data point compares to a standard. Absolute change in stature-for-age Z-score will be reported.

  8. Time-to-first pulmonary exacerbation [ Time Frame: From baseline through 96 weeks. ]
    Pulmonary exacerbation refers to the intermittent episodes of acute decline of lung function and worsening of symptoms. Time to first pulmonary exacerbation will be reported.

  9. Number of pulmonary exacerbations [ Time Frame: From baseline through 96 weeks. ]
    Pulmonary exacerbation refers to the intermittent episodes of acute decline of lung function and worsening of symptoms. Number of pulmonary exacerbations during the study will be reported.

  10. Number of Cystic Fibrosis (CF)-related hospitalizations [ Time Frame: From baseline through 96 weeks. ]
    Number of hospitalizations due to CF during the study will be reported.

  11. Absolute change from baseline in fecal elastase-1 (FE-1) levels [ Time Frame: From baseline through 96 weeks. ]
    Absolute change in FE-1 levels will be reported.

  12. Absolute change from baseline in serum levels of immunoreactive trypsinogen (IRT) [ Time Frame: From baseline through 96 weeks. ]
    Absolute change in IRT serum levels will be reported.

  13. Change from baseline in sputum microbiology cultures [ Time Frame: From baseline through 96 weeks. ]
    Change in sputum microbiology cultures will be reported.

  14. Absolute change from baseline in lung clearance index (LCI)2.5 [ Time Frame: From baseline through 96 weeks. ]
    LCI is a measure of ventilation inhomogeneity that is based on tidal breathing techniques. LCI 2.5 will be defined as the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. Absolute change in LCI2.5 will be reported.

  15. Absolute change from baseline in LCI5.0 [ Time Frame: From baseline through 96 weeks. ]
    LCI is a measure of ventilation inhomogeneity that is based on tidal breathing techniques. LCI 5.0 will be defined as the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value. Absolute change in LCI5.0 will be reported.



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects entering the Treatment Cohort must meet the following criteria:

  • Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study VX15-809-115 Part B (Study 115B, NCT02797132)
  • Willing to remain on a stable CF medication regimen through the Safety Follow-up Visit

Subjects entering the Observational Cohort must meet 1 of the following criteria:

  • Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study 115B, but do not want to enroll in the Treatment Cohort.
  • Received at least 4 weeks of LUM/IVA treatment and completed visits up to Week 24 and the Safety Follow-up Visit, if required, of Study 115B but are not taking LUM/IVA at the end of the Study 115B Treatment Period (i.e., Week 24) because of a drug interruption and either did not receive Vertex approval to enroll in the Treatment Cohort or do not want to enroll in the Treatment Cohort.
  • Permanently discontinued LUM/IVA in Study 115B after receiving at least 4 weeks of treatment and remained in the study from the time of treatment discontinuation through the Week 24 Visit and Safety Follow-up Visit, if required.

Exclusion Criteria (Treatment Cohort Only):

  • Prematurely discontinued LUM/IVA treatment in Study 115B.
  • History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering LUM/IVA to the subject
  • History of drug intolerance or other serious reactions to LUM/IVA in Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor.
  • Subjects with a history of allergy or hypersensitivity to LUM/IVA.
  • Liver function test (LFT) abnormality meeting criteria for LUM/IVA treatment interruption at the completion of Study 115B, for which no convincing alternative etiology is identified.
  • QTc value at the completion of Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor
  • History of poor compliance with LUM/IVA and/or procedures in Study 115B as deemed by the investigator.
  • Participation in an investigational drug trial (including studies investigating LUM and/or IVA) other than Study 115B.

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): NCT03125395


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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03125395     History of Changes
Other Study ID Numbers: VX16-809-116
First Posted: April 24, 2017    Key Record Dates
Last Update Posted: December 20, 2017
Last Verified: December 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Fibrosis
Cystic 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