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A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

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ClinicalTrials.gov Identifier: NCT03625466
Recruitment Status : Recruiting
First Posted : August 10, 2018
Last Update Posted : September 12, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F)

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: LUM Drug: IVA Drug: Matched Placebos Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double blinded in Part one, and Open label in Part 2
Primary Purpose: Treatment
Official Title: An Exploratory Phase 2, 2-part, Randomized, Double-blind, Placebo-controlled Study With a Long-term, Open-label Period to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del
Actual Study Start Date : August 10, 2018
Estimated Primary Completion Date : November 2020
Estimated Study Completion Date : October 2021

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: Part 1: Double Blind Period
Subjects will receive LUM/IVA as FDC granules dependent upon weight or matched placebo at Day 1.
Drug: LUM
Fixed-dose combination (FDC) granule (LUM/IVA)
Other Names:
  • Lumacaftor
  • VX-809

Drug: IVA
FDC tablet or granule (LUM/IVA)
Other Names:
  • Ivacaftor
  • VX-770

Drug: Matched Placebos
Placebo will be used as a comparator.

Experimental: Part 2: Open Label Period
Subjects will receive LUM/IVA as FDC tablets or granules dependent upon weight at Day 1.
Drug: LUM
Fixed-dose combination (FDC) granule (LUM/IVA)
Other Names:
  • Lumacaftor
  • VX-809

Drug: IVA
FDC tablet or granule (LUM/IVA)
Other Names:
  • Ivacaftor
  • VX-770




Primary Outcome Measures :
  1. Absolute change in magnetic resonance imaging (MRI) global chest score [ Time Frame: from baseline at Week 48 ]

Secondary Outcome Measures :
  1. Absolute change in lung clearance index (LCI)2.5 [ Time Frame: from baseline through Week 48 ]
  2. Absolute change in weight-for-age z-score [ Time Frame: from baseline at Week 48 ]
  3. Absolute change in stature-for-age z-score [ Time Frame: from baseline at Week 48 ]
  4. Absolute change in body mass index (BMI)-for-age z-score [ Time Frame: from baseline at Week 48 ]


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Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Subjects with confirmed diagnosis of CF.
  • Homozygous for F508del (F/F).
  • Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit.

Key Exclusion Criteria:

  • Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
  • Solid organ or hematological transplantation.
  • History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study.

Other protocol defined Inclusion/Exclusion criteria may apply.


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): NCT03625466


Contacts
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Contact: Medical Information 6173416777 medicalinfo@vrtx.com

Locations
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Germany
Charite Paediatric Pulmonology Department Recruiting
Berlin, Germany
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin Recruiting
Giessen, Germany
Hannover Medical School Recruiting
Hannover, Germany
Heidelberg Cystic Fibrosis Center Recruiting
Heidelberg, Germany
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin Recruiting
Lubeck, Germany
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03625466     History of Changes
Other Study ID Numbers: VX16-809-121
2017-003761-99 ( EudraCT Number )
First Posted: August 10, 2018    Key Record Dates
Last Update Posted: September 12, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Disease Progression
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Disease Attributes
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action