Study Assessing PTI-428 Safety, Tolerability, and Pharmacokinetics in Subjects With Cystic Fibrosis on KALYDECO® as Background Therapy

Details

Therapeutic approach
Restore CFTR Function
Trial status
Closed with results Participating Centres
Trials Tracker ID
TT001826
Last updated
16/03/2018

Full title

A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.

Study details

The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.


Phase
Phase 1
Length of participation
21 weeks
Recruitment target
16
CF sponsor
Proteostasis Therapeutics, Inc.
CF sponsor type
Commercial

Who can take part?

Age range
18+
Including people

Stable on Kalydeco dosing for minimum of 3 months prior to study
Non-smoker and non-tobacco user for a minimum of 30 days prior to screening

Excluding people

Participation in another clinical trial or treatment with an investigational agent within 30 days.
History of organ transplantation.

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CF centres running this trial

Closed

Belfast - Celerion Research Institute

CTAP centre
NHS Trust
Belfast Health & Social Care Trust
Address
22-24 Lisburn Road
BT9 6AD
Local site investigator
Damian Downey
Trial Coordinators
Catherine McNeill
Closed

Manchester - Medicines Evaluation Unit

CTAP centre
NHS Trust
Manchester University NHS Foundation Trust
Address
The Langley Building
Southmoor Road, Wythenshawe
M23 9QZ
Local site investigator
Professor Alex Horsley
Trial Coordinators
Natalie Hill
Sarah Sampson