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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-001317-28-NL

Enrollment

Unknown

Registered

2015-05-19

Start date

2015-07-30

Completion date

Unknown

Last updated

2017-05-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic fibrosis

Interventions

Trade Name: Salbutamol Pharmaceutical Form: Tablet

Eligibility

Sex/Gender

All

Inclusion criteria

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) Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Main Objective: to evaluate the clinical effect of a long term treatment (8 weeks) with oral B2-agonists in CF patients with residual CFTR function;Secondary Objective: 1. Evaluate the correlations between individual B2-agonist-induced CFTR function in vitro (organoid-based measurements) and the long term clinical treatment effect (eg. lung function and airway resistance). 2. Assess the effect of the salbutamol concentration in the blood on CFTR function in the background of patient specific parameters. We will do this by examining the CFTR-stimulating potential of the patients’ blood in vitro (in the organoid model). ;Primary end point(s): Pulmonary function before and after treatment with salbutamol ;Timepoint(s) of evaluation of this end point: Before and after treatment with salbutamol	—

Measure

Time frame

Main Objective: to evaluate the clinical effect of a long term treatment (8 weeks) with oral B2-agonists in CF patients with residual CFTR function;Secondary Objective: 1. Evaluate the correlations between individual B2-agonist-induced CFTR function in vitro (organoid-based measurements) and the long term clinical treatment effect (eg. lung function and airway resistance). 2. Assess the effect of the salbutamol concentration in the blood on CFTR function in the background of patient specific parameters. We will do this by examining the CFTR-stimulating potential of the patients’ blood in vitro (in the organoid model). ;Primary end point(s): Pulmonary function before and after treatment with salbutamol ;Timepoint(s) of evaluation of this end point: Before and after treatment with salbutamol

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Secondary

Measure	Time frame
Secondary end point(s): • Fraction exhaled Nitric Oxide (FeNO) and Nasal Nitric Oxide (nNO); • BMI (=weight (in Kg)/Length2 (in cm)); • Quality of life (measured with Cystic Fibrosis Questionnaire (CFQ)); • Bile salt measurements in plasma and the feces; • Elastase measurements in the feces; • SCC measurements; • Upper and lower airway microbial profiles (microbiome) (conventional culturing, high throughout pyrosequencing (16S rRNA) for bacterial diversity and relative abundance). • Correlation between individual salbutamol induced CFTR function in vitro (organoid-based measurements) and the in vivo treatment effect; • The CFTR stimulating ability of the concentration of salbutamol in the patient’s blood samples, examined by in vitro testing (in the organoid model);;Timepoint(s) of evaluation of this end point: Before and after treatment with salbutamol	—

Measure

Time frame

Secondary end point(s): • Fraction exhaled Nitric Oxide (FeNO) and Nasal Nitric Oxide (nNO); • BMI (=weight (in Kg)/Length2 (in cm)); • Quality of life (measured with Cystic Fibrosis Questionnaire (CFQ)); • Bile salt measurements in plasma and the feces; • Elastase measurements in the feces; • SCC measurements; • Upper and lower airway microbial profiles (microbiome) (conventional culturing, high throughout pyrosequencing (16S rRNA) for bacterial diversity and relative abundance). • Correlation between individual salbutamol induced CFTR function in vitro (organoid-based measurements) and the in vivo treatment effect; • The CFTR stimulating ability of the concentration of salbutamol in the patient’s blood samples, examined by in vitro testing (in the organoid model);;Timepoint(s) of evaluation of this end point: Before and after treatment with salbutamol

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Countries

Netherlands

Contacts

Public ContactGitte Berkers

UMC Utrecht

g.berkers-3@umcutrecht.nl0031887553741

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026