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Organoid Study R334W

NCT04254705 · Status: WITHDRAWN · Phase: NA · Type: INTERVENTIONAL

Last updated 2023-04-18

No results posted yet for this study

Summary

In contrary to what is seen in FRT cells, rectal organoids of patients with a R334W mutation do respond to CFTR modulators ivacaftor and lumacaftor. The present study will investigate the response to modulators in organoids of 30 patients with CF and a R334W mutation, to allow further stratificaton for a future clinical trial assessing the clinical effect of ivacaftor/tezacaftor in patients with CF and a R334W mutation.

Conditions

Interventions

PROCEDURE

Rectal Biopsy

Suction biopsy or forceps biopsy

Sponsors & Collaborators

  • Vertex Pharmaceuticals Incorporated

    collaborator INDUSTRY

  • KU Leuven

    collaborator OTHER

  • University of Lisbon

    collaborator OTHER

  • Universitaire Ziekenhuizen KU Leuven

    lead OTHER

Principal Investigators

  • François Vermeulen, MD PhD · Universitaire Ziekenhuizen KU Leuven

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
99 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-03-01
Primary Completion
2020-09-01
Completion
2021-03-01

Countries

  • Belgium
  • France
  • Italy
  • Portugal
  • Spain

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04254705 on ClinicalTrials.gov