Your donation will make a difference:
Cystic Fibrosis Trust
Forum

Please Log in

Log in

Not a registered user? Register with us

This trial has been added to your watchlist.

Do you want to receive email notifications when the trial status changes?

No Yes

Unfollowing a trial will remove it from the watchlist and stop status change email notifications

Are you sure you want to unfollow this trial?

Yes, remove from watchlist

HIT-CF Organoid Study

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

Participating Centres
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

Not Applicable

Full title

HIT-CF Project: Stratifying Cystic Fibrosis Patients Based on Intestinal Organoid Response To Different CFTR-modulators

New innovative and effective ‘mutation-specific’ drugs are available or in development for up to 90% of people with CF (ie. those with the most common 20-30 mutations). These are called ‘CFTR modulators.’ However, this leaves an important minority (~10%) of people with CF with no modulator in development as their mutations are very rare and only a small number of individuals have these specific mutations, making studying these patients in traditional trials very challenging. Therefore, the Human Individualized Treatment for CF (HIT-CF) project has been developed to address this unmet need, ie. to develop personalised treatments for this important group. HIT-CF is a Europe-wide, multi-centre project, which comprises two parts: This IRAS submission relates to the first part - the organoid study - in which patients with rare CFTR mutations will be recruited and intestinal organoids produced from rectal biopsies from each patient will be generated. Intestinal organoids, sometimes called ‘mini-intestines,’ are cell cultures made from stem cells so they contain the same mutations as the person from whom the biopsies are derived. Using this laboratory model, candidate modulator drugs can then be tested to assess for responsiveness (thus potentially predicting response in real life). In the second part of HIT-CF - the clinical trial - these modulators will then be tested in patients identified from the organoid study as likely responders, thus increasing the likelihood of showing efficacy. We plan to test drugs on organoids derived from 500 patients across Europe.

Trial Reference Number

119685

Intervention
the name of the treatment or therapy being researched

Rectal biopsy

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

500

Last edited date

04 September 2019

CF sponsor

University Medical Centre Utrecht

CF sponsor type

Academic

Who can take part?

Top inclusion criteria
  • Male or female with confirmed diagnosis of CF
  • Adult age on the date of informed consent for biopsy taking
Top exclusion criteria
  • Subject has at least one of the following CFTR-mutations: F508del, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, A455E, 3849+10kbC>T
  • Subject has a combination of any two of the following mutations: G542X, 1717-1G>A, 621+1G>T, 3120+1G>A, 1898+1G->A, CFTRdele2,3 and 2183AA->G
  • If you have just one of the mutations in the above bullet point along with another mutation not listed at all, then you may be eligible
  • If in doubt about your eligibility status, please speak to your CF team

CF centres running this trial

Recruiting

King's College Hospital (Denmark Hill)

Address

Denmark Hill London SE5 9RS

Recruitment ends

March 2020

Contact

Waller , Michael

Get in touch

Recruiting

Royal Brompton and Harefield NHS Foundation Trust

Address

Royal Brompton Hospital Sydney Street London Greater London SW3 6NP

Recruitment ends

December 2019

Contact

,

Get in touch

Recruiting

Southampton General Hospital

Address

Tremona Road Southampton Hampshire SO16 6YD

Recruitment starts

September 2019

Recruitment ends

July 2020

Contact

Carroll, Mary

Get in touch

Recruiting

ST JAMES'S UNIVERSITY HOSPITAL

Address

BECKETT STREET, LEEDS, WEST YORKSHIRE LS9 7TF

Recruitment ends

December 2020

Contact

Peckham, Daniel

Get in touch
Show all participating centres