NCT05100823Terminated
Validation of Therapeutic Efficacy Targeting the Splicing Variants in Cystic Fibrosis and CFTR Pathologies
Source: ClinicalTrials.gov · Last updated 18 Sept 2025
Reason given: Enrolment period completed and sufficient number of enrolments to achieve study objectives
Brief summary (registry, in English)
Cystic Fibrosis, an inherited autosomal recessive disease, arises from mutations in the CFTR gene. For intronic mutations affecting splicing events, oligonucleotides therapy has the potential to restore the production of the full length CFTR protein. Recent scientific research has demonstrated the potential of this approach to restore full length mRNA CFTR in in vitro human airway cells. The study aims to validate the therapeutic efficacy of oligonucleotide blockers (ONB) that target splicing defects associated to splicing variants in epithelia obtained from patients with Cystic Fibrosis and CFTR-related disorders.
- Status (registry)
- Terminated
- Study type
- Interventional
- Enrollment
- 16 (actual)
- First submitted
- 6 Sept 2021
- Start date
- 30 Mar 2022
- Primary completion date
- 17 Feb 2025
- Lead sponsor
- University Hospital, Montpellier
- Diseases
- Cystic fibrosis
- NCT number
- NCT05100823
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- The subject is in a period of exclusion determined by a previous study.
Other illness
- The subject has porphyria, or has hepatic insufficiency, or suffers from epilepsy, or suffers from conduction disorders, or suffers from severe heart failure, has a cons-indication to the use of a local anesthetic spray.
- Specific non-inclusion criteria for rectal sampling:
- the subject has a bleeding disorder
Other
- The subject is under judicial protection, under guardianship or under curatorship
- The subject does not accept to sign consent
- It turns out to be impossible to give informed information to the subject
- The subject does not read the French language fluently
- The subject is a pregnant or breastfeeding woman
- the subject has thrombocytopenia
- The patient has severe inflammation of the rectum.
Who the study is looking for
- The subject must have given their free and informed consent and signed the consent
- The subject must be affiliated or beneficiary of a health insurance plan Women and men are included
- The patient is at least 12 years old.
- The patient has cystic fibrosis or a CFTR pathology and therefore carries two mutations (with at least one mutation affecting splicing) in the CFTR gene.
- Patients who volunteer for rectal biopsy collection (only from Montpellier University Hospital) must be at least 18 years old.
Age in the registry: 12 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Montpellier University Hospital | Montpellier | France | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.