Phase 3
Completed N=60
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Cystic Fibrosis
Source: ClinicalTrials.gov NCT03601637 ↗
Enrolled (actual)
60
Serious AEs
8.3%
Results posted
Jan 2023
Primary outcomePrimary: Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA — 14600; 12600; 16600; 13900 nanograms per milliliter (ng/mL)
◆ Published Evidence
Established
71citations · ~12 / year
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Summary
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
Linked Publications (3)
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
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A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for <i>F508del-CFTR</i>.
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Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA |
14600; 12600; 16600; 13900; 1620; 1320 | — |
| PRIMARY Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA |
12000; 12800; 8380; 10500; 169; 185 | — |
| PRIMARY Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
44; 5 | — |
| SECONDARY Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
12; 0 | — |
| SECONDARY Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA) |
1410; 1470; 1460; 1370; 606; 842 | — |
| SECONDARY Part B: Absolute Change in Sweat Chloride |
-29.1 | — |
| SECONDARY Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA) |
13500; 9030; 887; 21900; 9370; 1000 | — |
Eligibility Criteria
Key Inclusion Criteria
- Participants 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 participants
- Solid organ or hematological transplantation
Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03601637) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.