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

Details

Therapeutic category
The type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise

Restore CFTR Function

Trial status

Open to recruitment

Phase
The different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use

Phase I

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.

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.

Trial type

Medication

Length of participation
The length of time a participant will take part in a trial, from the first to the last appointment

21 weeks

Intervention
The name of the treatment or therapy being researched

PTI-428,Placebo

Recruitment target
The number of participants who need to be recruited for the trial in the UK

16

Last edited date

03 November 2017

Sponsor

Proteostasis Therapeutics, Inc.

Sponsor type

Commercial

Who can take part?

Age

18+

Top inclusion criteria
  • Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization.
  • Non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.
  • Confirmed diagnosis of CF.
Top exclusion criteria
  • Participation in another clinical trial or treatment with an investigational agent within 30 days.
  • History of organ transplantation.
  • Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.

CF centres running this trial

Recruiting

Celerion Research Institute

Address

22-24 Lisburn Rd Belfast BT9 6AD

Recruitment starts

July 2017

Recruitment ends

February 2018

Contact

Downey, Damian

Get in touch

Recruiting

Medicines Evaluation Unit

Address

The Langley Building Southmoor Road Wythenshawe Manchester M23 9QZ

Recruitment ends

February 2018

Contact

Downey, Damian

Get in touch