A study to investigate the safety of Kaftrio® in children with CF aged 12 to less than 24 months (VX22-445-122)

Details

CTAP badge
Therapeutic approach
Restore CFTR Function
Trial status
In setup Participating Centres
Trials Tracker ID
TT013111
Last updated
08/12/2023

Full title

A Phase 3, open-label study evaluating the pharmacokinetics, safety, and tolerability of Elexacaftor/Tezacaftor/Ivacaftor in children with cystic fibrosis aged 12 to less than 24 months

Study details

This study is aiming to learn more about the safety and tolerability of the combination modulator treatment made up of Elexacaftor (ELX), Tezacaftor (TEZ) and Ivacaftor (IVA) (also known by its brand name Kaftrio) in children aged 12 to less than 24 months.

This treatment is already approved for use in the UK for certain groups of people with CF, but has not yet been approved for this age group.

There will be two parts to the study: Part A and Part B.

Children taking part in Part A will receive ELX/TEZ/IVA in the morning and IVA in the evening. Participation will last for around 10 weeks.

Children taking part in Part B will receive ELX/TEZ/IVA in the morning and IVA in the evening, with the dose(s) to be based on the outcome of Part A. Participation will last for around 32 weeks.


Study updates

Part A of this study is now closed to recruitment. Part B will open around May 2024. 


Phase
Phase 3
Length of participation
10 to 32 weeks
Recruitment target
26
CF sponsor
Vertex
CF sponsor type
Commercial

Who can take part?

Age range
Aged 12 to less than 24 months
Including people

Aged 12 to less than 24 months
Two copies of the F508del mutation or one copy of F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)
At least one F508del mutation in the CFTR gene or another ELX/TEZ/IVA-responsive CFTR mutation

Excluding people

Clinically significant cirrhosis with or without portal hypertension
Lung infection with organisms associated with a more rapid decline in pulmonary status
Solid organ or hematological transplantation

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CF centres running this trial

Closed

Great Ormond Street Hospital for Children (London)

CTAP centre
NHS Trust
Great Ormond Street Hospital for Children NHS Foundation Trust
Address
Great Ormond Street
WC1N 3JH
Trial Coordinators
Lucy Holt
Closed

Leeds Children's Hospital

CTAP centre
NHS Trust
The Leeds Teaching Hospitals NHS Trust
Address
Clarendon Wing
Belmont Grove
LS2 9NS
Trial Coordinators
Yasmin Khanagha
Closed

Liverpool - Alder Hey Children's Hospital

CTAP centre
NHS Trust
Alder Hey Children's NHS Foundation Trust
Address
Eaton Road
West Derby
L12 2AP
Trial Coordinators
Victoria King
Closed

Royal Brompton Hospital (London) - Paediatrics

CTAP centre
NHS Trust
Guy's and St Thomas' NHS Foundation Trust
Address
Royal Brompton Hospital
Sydney Street
SW3 6NP
Trial Coordinators
[email protected]
Closed

Southampton General Hospital (Paediatrics)

CTAP centre
NHS Trust
University Hospital Southampton NHS Foundation Trust
Address
Tremona Road
SO16 6YD
Trial Coordinators
Matthew Harvey