Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 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
Results First Posted : January 6, 2023
Last Update Posted : January 6, 2023
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Sponsor:
Vertex Pharmaceuticals Incorporated
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
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| Study Type | Interventional |
|---|---|
| Study Design | Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
| Condition |
Cystic Fibrosis |
| Interventions |
Drug: LUM Drug: IVA |
| Enrollment | 61 |
Participant Flow
| Recruitment Details | |
| Pre-assignment Details | This study was conducted in participants with cystic fibrosis (CF) aged 1 through less than 2 years of age who are homozygous for F508del. A total of 61 participants were enrolled in Parts A and B of the study. One participant in Part B was enrolled but not dosed in this study. Therefore, data for 60 participants are reported in the participant flow and baseline characteristics sections. |
| Arm/Group Title | Part A: LUM/IVA | Part B: LUM/IVA |
|---|---|---|
 Arm/Group Description |
Participants weighing 7 to less than( <)10 kilograms (kg) at screening received lumacaftor (LUM) 75 milligrams (mg)/ ivacaftor (IVA) 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days. | Participants weighing 7 to <9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight. |
| Period Title: Overall Study | ||
| Started | 14 | 46 |
| Completed | 13 | 43 |
| Not Completed | 1 | 3 |
| Reason Not Completed | ||
| Withdrawal of Consent (not due to adverse event) | 0 | 1 |
| Adverse Event | 1 | 1 |
| Other | 0 | 1 |
Baseline Characteristics
| Arm/Group Title | Part A: LUM/IVA | Part B: LUM/IVA | Total | |
|---|---|---|---|---|
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Arm/Group Description |
Participants weighing 7 to less than(<)10 kilograms (kg) at screening received lumacaftor (LUM) 75 milligrams (mg)/ ivacaftor (IVA) 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days. | Participants weighing 7 to <9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight. | Total of all reporting groups | |
| Overall Number of Baseline Participants | 14 | 46 | 60 | |
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Baseline Analysis Population Description |
All participants who received at least one dose of the study drug during the treatment period were included in the baseline analysis. The baseline analysis was collected for the overall treatment arm, irrespective of a weight-based dose regimen. Therefore, the analysis is reported in a single arm for each study part (Part A: LUM/IVA and Part B: LUM/IVA).
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 14 participants | 46 participants | 60 participants | |
| <=18 years |
14 100.0%
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46 100.0%
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60 100.0%
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| Between 18 and 65 years |
0 0.0%
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0 0.0%
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0 0.0%
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| >=65 years |
0 0.0%
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0 0.0%
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0 0.0%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 14 participants | 46 participants | 60 participants | |
| Female |
7 50.0%
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24 52.2%
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31 51.7%
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| Male |
7 50.0%
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22 47.8%
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29 48.3%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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| Number Analyzed | 14 participants | 46 participants | 60 participants | |
| Hispanic or Latino |
1 7.1%
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2 4.3%
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3 5.0%
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| Not Hispanic or Latino |
13 92.9%
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37 80.4%
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50 83.3%
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| Unknown or Not Reported |
0 0.0%
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7 15.2%
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7 11.7%
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Race/Ethnicity, Customized
Measure Type: Count of Participants Unit of measure: Participants |
Number Analyzed | 14 participants | 46 participants | 60 participants |
| White |
14 100.0%
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34 73.9%
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48 80.0%
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| Black or African American |
0 0.0%
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1 2.2%
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1 1.7%
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| Asian |
0 0.0%
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0 0.0%
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0 0.0%
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| American Indian or Alaska Native |
0 0.0%
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1 2.2%
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1 1.7%
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| Native Hawaiian or Other Pacific Islander |
0 0.0%
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0 0.0%
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0 0.0%
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| Not collected per local regulations |
0 0.0%
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7 15.2%
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7 11.7%
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| Other |
0 0.0%
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3 6.5%
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3 5.0%
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Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
| Name/Title: | Medical Monitor |
| Organization: | Vertex Pharmaceuticals Incorporated |
| Phone: | 617-341-6777 |
| EMail: | medicalinfo@vrtx.com |
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 Submitted: | July 18, 2018 |
| First Posted: | July 26, 2018 |
| Results First Submitted: | October 28, 2022 |
| Results First Posted: | January 6, 2023 |
| Last Update Posted: | January 6, 2023 |