Clinical trial • Not applicable • Rare Disease|Respiratory

tezacaftor, elexacaftor, ivacaftor for Cystic fibrosis

Not applicable trial of tezacaftor, elexacaftor, ivacaftor for Cystic fibrosis. 50 participants.

Overview

Not applicable trial across 4 sites in Italy.

Recruits 50 paediatric patients.

Pregnancy Exclusion: Pregnancy and/or breastfeeding.
Vulnerable Population: Vulnerable population selected (minors). Informed consent is required; age-specific subject information and informed consent forms are provided (documents listed: L1_SIS and ICF 6-11 yr; L1_SIS and ICF 12-17 yr; L1_SIS and ICF adults). Parental/legal guardian consent and privacy forms are provided (documents: L1_SIS and ICF parents; L1_SIS and ICF legal guardian; parents_privacy/adult_privacy). Assent/consent processes are handled with these age-specific ICFs and parent/guardian forms (languages not specified).

{"criterion_text":"- CF patients with at least one F508del mutation treated with the combination ETI for a time sufficient to reach steady-state (8 days).\n- Males and females aged ≥6 years.\n- Informed consent to participate in the study and to process the patient's personal data obtained prior to the collection of any study data."}

{"criterion_text":"- Evidence of inadequate compliance to treatment.\n- Pregnancy and/or breastfeeding.\n- Any conditions that may affect the ability to complete informed consent.\n- Any other severe systemic disorders that may compromise the PK parameters.\n- Use of any drug capable of moderately/strongly inhibiting or strongly inducing hepatic biotransformation.\n- Denial of the informed consent."}

{"endpoint_text":"- Plasma concentrations of ELX, TEZ and IVA at steady-state (Css).","definition_or_measurement_approach":"Measurement of plasma concentrations of elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) at steady-state (Css)."}

{"endpoint_text":"- Population pharmacokinetics (popPK) analysis of ELX, TEZ and IVA at steady state.","definition_or_measurement_approach":"Population pharmacokinetic (popPK) analysis of ELX, TEZ and IVA at steady state."}
{"endpoint_text":"- Harvest of every AE/Adverse Drug Reaction (ADR).","definition_or_measurement_approach":"Collection and recording of all adverse events/adverse drug reactions reported during ETI treatment."}
{"endpoint_text":"- Correlation between PK of ELX, TEZ and IVA and number of AEs","definition_or_measurement_approach":"Statistical correlation analysis between pharmacokinetic parameters of ELX/TEZ/IVA and the number of adverse events."}
{"endpoint_text":"- Correlation between PK and ETI efficacy (ppFEV1 and Cl- values at T0) in the 3 groups classifications (responders, non responders and unresolved responders)","definition_or_measurement_approach":"Correlation analysis between PK parameters and efficacy measures (percent predicted FEV1 and chloride values at T0) across classifications of responders, non-responders, and unresolved responders."}

Planned Sample Size: 50
Recruitment Window Months: 24
Consent Approach: Informed consent is required prior to any study data collection. Age-specific ICFs exist for 6-11 years, 12-17 years, and adults; separate parent/legal guardian consent and privacy forms and biobank consent forms for minors and adults are provided. Specific languages available are not stated.

Site Name: Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo
Department Name: Pneumologia e Fibrosi Cistica
Contact Person Name: Massimo Maschio
Contact Person Email: massimo.maschio@burlo.trieste.it

Investigational Product Name: Kaftrio 37.5 mg/25 mg/50 mg film-coated tablets
Active Substance: tezacaftor, elexacaftor, ivacaftor
Modality: Small molecule
Routes Of Administration: ORAL USE
Route: Oral
Authorisation Status: Authorised
Orphan Designation: Yes
Dose Levels: 37.5 mg/25 mg/50 mg
Maximum Dose: 225 mg

Investigational Product Name: Kaftrio 75 mg/50 mg/100 mg film-coated tablets
Active Substance: tezacaftor, elexacaftor, ivacaftor
Modality: Small molecule
Routes Of Administration: ORAL USE
Route: Oral
Authorisation Status: Authorised
Orphan Designation: Yes
Dose Levels: 75 mg/50 mg/100 mg
Maximum Dose: 450 mg

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