A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
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| ClinicalTrials.gov Identifier: NCT02725567 |
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Recruitment Status :
Completed
First Posted : April 1, 2016
Results First Posted : September 7, 2023
Last Update Posted : September 7, 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: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment |
| Condition |
Cystic Fibrosis |
| Intervention |
Drug: IVA |
| Enrollment | 57 |
Participant Flow
| Recruitment Details | This study was conducted in participants with cystic fibrosis (CF) who were less than (<) 24 months of age at Day 1 and have an ivacaftor-responsive CF transmembrane conductance regulator (CFTR) mutation. |
| Pre-assignment Details |
| Arm/Group Title | Part A: 3 to < 24 Months | Part B + A/B: 1 to < 24 Months |
|---|---|---|
 Arm/Group Description |
Participants weighing 5 to less than (<) 7 kilogram (kg) received 25 milligram (mg) IVA (ivacaftor), 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4. |
Participants 4 to <6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to <7 kg received 25 mg IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to <4 months weighing 3 kg to <5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age. |
| Period Title: Part A (Up to 5 Days) | ||
| Started | 19 | 0 |
| Completed | 19 | 0 |
| Not Completed | 0 | 0 |
| Period Title: Part B + A/B ( Up to 24 Weeks) | ||
| Started | 0 | 43 [1] |
| Completed | 0 | 40 |
| Not Completed | 0 | 3 |
| Reason Not Completed | ||
| Lost to Follow-up | 0 | 1 |
| Physician Decision | 0 | 1 |
| Withdrawal of Consent (not due to adverse event) | 0 | 1 |
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[1]
Out of the 19 participants in the reporting arm Part A, 5 also participated in the reporting arm "Part B + Part A/B.
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Baseline Characteristics
| Arm/Group Title | Ivacaftor (IVA) | |
|---|---|---|
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Arm/Group Description |
Part A: Participants weighing 5 to <7 kg received 25 mg IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA administered q12h on Days 1 through 3 and 1 morning dose on Day 4. Participants 4 to <6 months of age and ≥5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to <7 kg received 25 mg IVA, 7 to <14 kg received 50 mg IVA, and those weighing 14 to <25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to <4 months weighing 3 kg to <5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age. |
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| Overall Number of Baseline Participants | 57 | |
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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.
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Age, Customized
[1] Measure Type: Count of Participants Unit of measure: Participants |
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| Part A | Number Analyzed | 19 participants |
| 12 to <24 months |
7 36.8%
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| 6 to <12 months |
6 31.6%
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| 4 to <6 months |
4 21.1%
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| 1 to <4 months |
2 10.5%
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| Part B+ A/B | Number Analyzed | 43 participants |
| 12 to <24 months |
19 44.2%
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| 6 to <12 months |
11 25.6%
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| 4 to <6 months |
6 14.0%
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| 1 to <4 months |
7 16.3%
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[1]
Measure Analysis Population Description: The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.
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Sex: Female, Male
[1] Measure Type: Count of Participants Unit of measure: Participants |
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| Part A | Number Analyzed | 19 participants |
| Female |
10 52.6%
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| Male |
9 47.4%
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| Part B + A/B | Number Analyzed | 43 participants |
| Female |
22 51.2%
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| Male |
21 48.8%
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[1]
Measure Analysis Population Description: The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.
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Race/Ethnicity, Customized
[1] Measure Type: Count of Participants Unit of measure: Participants |
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| Part A | Number Analyzed | 19 participants |
| Hispanic or Latino |
0 0.0%
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| Not Hispanic or Latino |
19 100.0%
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| Not collected per local regulations |
0 0.0%
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| Part B + A/B | Number Analyzed | 43 participants |
| Hispanic or Latino |
1 2.3%
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| Not Hispanic or Latino |
42 97.7%
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| Not collected per local regulations |
0 0.0%
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[1]
Measure Analysis Population Description: The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.
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Race/Ethnicity, Customized
[1] Measure Type: Count of Participants Unit of measure: Participants |
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| Part A | Number Analyzed | 19 participants |
| White |
19 100.0%
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| Black or African American |
0 0.0%
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| Asian |
0 0.0%
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| American Indian or Alaska Native |
0 0.0%
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| Native Hawaiian or Other Pacific Islander |
0 0.0%
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| Not collected per local Regulations |
0 0.0%
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| Part B + A/B | Number Analyzed | 43 participants |
| White |
43 100.0%
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| Black or African American |
0 0.0%
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| Asian |
0 0.0%
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| American Indian or Alaska Native |
0 0.0%
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| Native Hawaiian or Other Pacific Islander |
0 0.0%
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| Not collected per local Regulations |
0 0.0%
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[1]
Measure Analysis Population Description: The Baseline data were planned to be presented separately for Part A and for the combined Part B+ A/B. Here, "Number Analyzed" signifies participants who were evaluable for specified part of the study.
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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: | NCT02725567 |
| Other Study ID Numbers: |
VX15-770-124 2015-001997-16 ( EudraCT Number ) |
| First Submitted: | March 14, 2016 |
| First Posted: | April 1, 2016 |
| Results First Submitted: | June 27, 2023 |
| Results First Posted: | September 7, 2023 |
| Last Update Posted: | September 7, 2023 |