Genistein as an add-on treatment for CF?

ID

NL-OMON24579

Nationaal Trial Register

Brief title

The TRIO study

Health condition

Cystic Fibrosis, Taaislijmziekte, CF

Sponsors and support

Primary sponsor :

University Medical Center Utrecht

Source(s) of monetary or material Support :

ZonMw

Intervention

Outcome measures

The main study parameter is pulmonary function (%FEV1) measured before and after the use
of genistein and before and after the use of placebo.

· Sweat chloride concentration (SCC), before and after the use of genistein and placebo;

· Airway resistance (Rint and bodybox), before and after the use of genistein and
placebo;

· BMI (=weight (in Kg)/Length2 (in cm)) before and after the use of genistein and
placebo;

· Quality of life (measured with CFQ-questionnaire) before and after the use of
genistein and placebo;

· Elastase measurements in the feces before and after the use of genistein and placebo;

· The CFTR stimulating ability of the concentration of genistein in the patient’s blood
samples, examined by in vitro testing (in the organoid model). We will also determine
the plasma levels of genistein by HPLC;

Exploratory endpoint:

· Assessment of ß-adrenergic sweat secretion by evaporimetry, before and after
the use of genistein and placebo.

Study objective

Adding genistein to Ivacaftor treatment leads to higher levels of restoration of the CFTR protein channel activity in patients with a mutation associated with residual CFTR function.

Study design

- Before placebo

- After using placebo for 8 weeks

- Before genistein

- After using genistein for 8 weeks

Genistein or Placebo

Public

Wilhelmina Kinderziekenhuis
Huispostnummer KH 01.419.0
Postbus 85090
G. Berkers
Utrecht 3508 AB
The Netherlands
+ 31 (0)88-75 537 41
g.berkers-3@umcutrecht.nl

Scientific

Wilhelmina Kinderziekenhuis
Huispostnummer KH 01.419.0
Postbus 85090
G. Berkers
Utrecht 3508 AB
The Netherlands
+ 31 (0)88-75 537 41
g.berkers-3@umcutrecht.nl

Inclusion criteria

· CFTR genotype: at least one S1251N mutation;

· Already had a rectal biopsy to produce an organoid;

· Use of Ivacaftor;

· Male and female patients, aged 6 years or older on the date of informed consent;

· Signed informed consent form (IC).

Exclusion criteria

· Use of genistein or curcumin at start or within four weeks prior to start of the study;

· Severe acute exacerbation or pulmonary infection during last four weeks (needing
intravenous treatment and/or systemic corticosteroids);

· (History of) hypothyroidism;

· Women who are trying to become pregnant, or are pregnant or breastfeeding;

· Women with estrogen receptor-positive tumors;

· Postmenopausal women on anti-oestrogen therapy (like tamoxifen and aromatase

blockers) for estrogen-responsive breast cancer;
· Participation in another drug-investigating clinical study at the start or within four
weeks prior to the start;

· Inability to follow instructions of the investigator.

Design

Study type :

Interventional

Intervention model :

Crossover

Allocation :

Randomized controlled trial

Masking :

Double blinded (masking used)

Control :

Placebo

Recruitment

NL

Recruitment status :

Recruiting

Start date (anticipated) :

02-06-2017

Enrollment :

20

Type :

Anticipated

Positive opinion

Date :

13-06-2017

Application type :