Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
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| ClinicalTrials.gov Identifier: NCT03601637 |
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Recruitment Status :
Completed
First Posted : July 26, 2018
Last Update Posted : November 29, 2021
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Sponsor:
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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Brief Summary:
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: LUM Drug: IVA | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 60 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del |
| Actual Study Start Date : | August 23, 2018 |
| Actual Primary Completion Date : | October 29, 2021 |
| Actual Study Completion Date : | October 29, 2021 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
Drug Information available for:
Ivacaftor
| Arm | Intervention/treatment |
|---|---|
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Experimental: Part A Cohort 1 [aged 18 to <24 months]
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
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Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
Drug: IVA FDC granule (LUM/IVA)
Other Names:
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Experimental: Part A Cohort 2 [12 to <18months]
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
|
Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
Drug: IVA FDC granule (LUM/IVA)
Other Names:
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Experimental: Part B
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
|
Drug: LUM
Fixed Dose Combination (FDC) granule (LUM/IVA)
Other Names:
Drug: IVA FDC granule (LUM/IVA)
Other Names:
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Primary Outcome Measures :
- Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of LUM and IVA [ Time Frame: from baseline through Day 15 ]
- Part B: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 2 weeks after the last dose) ]
Secondary Outcome Measures :
- Part A: Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 10 days after the last dose) ]
- Part A: Average observed pre-dose concentrations (Ctrough) of LUM and IVA metabolites [ Time Frame: from baseline through Day 15 ]
- Part B: Absolute change in sweat chloride [ Time Frame: from baseline at Week 24 ]
- Part B: Average observed pre-dose concentration (Ctrough) of LUM, IVA, and their respective metabolites [ Time Frame: from baseline through Week 24 ]
Information from the National Library of Medicine
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| Ages Eligible for Study: | 12 Months to 23 Months (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Key Inclusion Criteria:
- Subjects will be 1 to less than 2 years of age on Day 1 of the relevant part of the study.
- Homozygous for F508del (F/F).
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.
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): NCT03601637
Locations
Show 27 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Vertex Pharmaceuticals Incorporated |
| ClinicalTrials.gov Identifier: | NCT03601637 |
| Other Study ID Numbers: |
VX16-809-122 2017-004794-13 ( EudraCT Number ) |
| First Posted: | July 26, 2018 Key Record Dates |
| Last Update Posted: | November 29, 2021 |
| Last Verified: | November 2021 |
| 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 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 |