Bronchodilcation as a CFTR activator in CF

• Register: EUCTR
• Last refreshed on: 8 August 2016
• Main ID: EUCTR2014-000057-37-NL
• Date of registration: 12/02/2014
• Prospective Registration: Yes
• Primary sponsor: University Medical Centre Utrecht
• Public title: Bronchodilcation as a CFTR activator in CF
• Scientific title: A B2-agonist as a CFTR activator in CF - ABBA study
• Date of first enrolment: 30/04/2014
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-000057-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: yes Open: yes Single blind: no Double blind: no Parallel group: no Cross over: Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Netherlands

Contacts

• Name: Sabine Michel
• Affiliation: University Medical Center Utrecht

• Name: Sabine Michel
• Affiliation: University Medical Center Utrecht

Key inclusion & exclusion criteria

Inclusion criteria:
- CFTR genotype compound/A455E or compound/R117H
- CFTR measurement available in intestinal biopsies
- 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 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Severe acute exacerbation or pulmonary infection (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
- HBA1C > 45 mmol/mol
- Use of 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 or within 1 month prior to the start
- Inability to follow instructions of the investigator



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Cystic Fibrosis

Intervention(s)

Product Name: Salbutamol
Product Code: R03AC02
Pharmaceutical Form: Inhalation vapour
INN or Proposed INN: SALBUTAMOL
CAS Number: 18559-94-9
Concentration unit: µg microgram(s)
Concentration type: up to
Concentration number: 400-

Product Name: Salbutamol
Product Code: R03CC02
Pharmaceutical Form: Tablet
INN or Proposed INN: SALBUTAMOL
CAS Number: 18559-94-9
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 4-

Primary Outcome(s)

Main Objective: To investigate the clinical response on treatment with a B2-agonist in a CF-patient with residual CFTR function by examining change in nasal potential difference (NPD) and sweat chloride concentration (SCC).

Secondary Objective: 1. To compare the efficacy of B2-agonist treatment per inhalation with oral B2-agonist by measuring the NPD and SCC.
2. To investigate the correlations between individual B2-agonist-induced CFTR function in vitro (using organoid-based measurements) and treatment effect in vivo (by measuring the NPD and SCC)
3. To assess whether the dosage of B2-agonist used in the clinical study is sufficient to stimulate CFTR function, by testing the CFTR stimulating effect of patients’ blood samples in vitro, on autologous organoid cultures.

Primary end point(s): NAsal Potential Difference measurement
Sweat Chloride Concentration

Timepoint(s) of evaluation of this end point: At 4 studyvisits endpoints will be measured.

Secondary Outcome(s)

Secondary end point(s): NPD
SCC
Blood sample
CFTR function in organoids

Timepoint(s) of evaluation of this end point: Also measured at all 4 studyvisits

Secondary ID(s)

ABBA-2014

Source(s) of Monetary Support

NCFS