A B2-agonist as a CFTR activator in CF - Part 2

Phase 2

Completed

• Conditions: Cystic Fibrosis; Mucoviscidosis; 10083624

• Registration Number: NL-OMON43663
• Lead Sponsor: niversitair Medisch Centrum Utrecht

Brief Summary

Trial ended prematurely

Detailed Description

Not available

Study Timeline

• Study Completion: 2017-01-12
• Last Update Posted: 11 June 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 14

Inclusion Criteria

- CFTR genotype compound/A455E or compound/R117H
- Already had a rectal biopsy to produce an organoid
- Males and females, aged 18 years or older on the date of informed consent
- Signed informed consent form (ICF)

Exclusion Criteria

- Severe acute exacerbation or pulmonary infection during last four weeks (needing intravenous treatment and/or systemic corticosteroids)
- Known cardiovascular medical history like cardiac failure, arrhythmias, ischemic cardiac disease, long QT interval syndrome and hypertension
- Known hyperthyroidism, thyrotoxicosis, galactose intolerance, lactase deficiency or glucose-galactose malabsorption
- Haemoglobin A1C (HBA1C) > 45 mmol/mol
- Use of oral B2-agonist one week prior to the start of the study (V1)
- Use of: heart glycoside, high dose sympathomimetics, theophylline, thiazide diuretics or non-selective beta-blockers
- Pregnancy or breastfeeding
- Participation in another drug-investigating clinical study at the start
- Inability to follow instructions of the investigator

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Pulmonary function (spirometry and airway resistance measured with the bodybox<br /><br>and Rint)<br /><br>* Before and after treatment with salbutamol</p><br>