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Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03768089
Recruitment Status : Completed
First Posted : December 7, 2018
Last Update Posted : June 3, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
The purpose of this study is to evaluate safety and tolerability of VX-121 in healthy subjects and in subjects with cystic fibrosis (CF).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-121 Drug: TEZ/IVA Drug: IVA Drug: Matched Placebo Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 114 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
Actual Study Start Date : March 19, 2018
Actual Primary Completion Date : May 3, 2019
Actual Study Completion Date : May 3, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: Part A: VX-121 in Healthy Subjects (HS)
Single dose escalation.
Drug: VX-121
VX-121 tablet or suspension for oral administration.

Placebo Comparator: Part A: Placebo Drug: Matched Placebo
Matched placebo.

Experimental: Part B: VX-121 in HS
Multiple-dose escalation.
Drug: VX-121
VX-121 suspension for oral administration.

Placebo Comparator: Part B: Placebo Drug: Matched Placebo
Matched placebo.

Experimental: Part C: VX-121 in Triple Combination (TC) with TEZ/IVA in HS
Multiple-dose escalation of VX-121 in TC with TEZ/IVA.
Drug: VX-121
VX-121 tablet for oral administration.

Drug: TEZ/IVA
100 milligram (mg) TEZ/150-mg IVA, fixed-dose combination (FDC) tablet for oral administration.
Other Names:
  • VX-661/VX-770
  • Tezacaftor/Ivacaftor

Drug: IVA
150-mg film-coated tablet for oral administration.
Other Names:
  • VX-770
  • Ivacaftor

Placebo Comparator: Part C: Placebo Drug: Matched Placebo
Matched placebo.

Experimental: Part D: VX-121 in TC with TEZ/IVA in subjects with CF
VX-121 in TC with TEZ/IVA in subjects with CF.
Drug: VX-121
VX-121 tablet for oral administration.

Drug: TEZ/IVA
100 mg TEZ/150-mg IVA, FDC tablet for oral administration.
Other Names:
  • VX-661/VX-770
  • Tezacaftor/Ivacaftor

Drug: IVA
150-mg film-coated tablet for oral administration.
Other Names:
  • VX-770
  • Ivacaftor

Placebo Comparator: Part D: Placebo Drug: Matched Placebo
Matched placebo.




Primary Outcome Measures :
  1. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From baseline up to Day 56 ]

Secondary Outcome Measures :
  1. Maximum observed concentration (Cmax) of VX-121,TEZ and metabolites, IVA and metabolites [ Time Frame: From Day 1 up to Day 29 ]
  2. Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-121, TEZ and metabolites, IVA and metabolites [ Time Frame: From Day 1 up to Day 29 ]
  3. Observed pre-dose concentration (Ctrough) of VX-121, TEZ and metabolites, IVA and metabolites [ Time Frame: From Day 1 up to Day 29 ]
  4. Part D only: Absolute change in sweat chloride concentrations [ Time Frame: From baseline through Day 29 ]
  5. Part D only: Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: From baseline through Day 29 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Key Inclusion Criteria:

  • Part A, B, and C: Healthy Volunteers

    • Female subjects must be of non-childbearing potential
    • Between the ages of 18 and 55 years, inclusive
    • Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight >50 kg
  • Part D: Subjects with CF

    • Heterozygous for F508del and an MF mutation (F/MF)
    • FEV1 value ≥40% and ≤90% of predicted mean for age, sex, and height
    • Body weight ≥35 kg

Key Exclusion Criteria:

  • Part A, B and C: Healthy Volunteers

    • Any condition possibly affecting drug absorption
    • History of febrile illness or other acute illness within 5 days before the first study drug dose
  • Part D: Subjects with CF

    • History of clinically significant cirrhosis with or without portal hypertension
    • History of solid organ or hematological transplantation
    • Lung infection with organisms associated with a more rapid decline in pulmonary status

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): NCT03768089


Locations
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Netherlands
Academic Medical Center
Amsterdam, Netherlands
HagaZiekenhuis van den Haag
Den Haag, Netherlands
PRA Health Sciences Onderzoekscentrum UMCG
Groningen, Netherlands
UMC St. Radboud
Nijmegen, Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
United Kingdom
Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital
Birmingham, United Kingdom
The Medicines Evaluation Unit
Manchester, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03768089     History of Changes
Other Study ID Numbers: VX17-121-001
2018-000126-55 ( EudraCT Number )
First Posted: December 7, 2018    Key Record Dates
Last Update Posted: June 3, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes

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