Cystic Fibrosis Mucoviscidosis
Conditions
Interventions
Patients will be randomized to receive genistein or placebo during a period of
8 weeks (treatment period 1). After an 4-week washout, patients will be crossed
over to receive the opposite treatment
Sponsors
Universitair Medisch Centrum Utrecht
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: *CFTR genotype associated with residual CFTR function; *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
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 tamoxifen therapy 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 outcomes
Primary
| Measure | Time frame |
|---|---|
| The main study parameter is pulmonary function (Forced Expiratory volume in 1 second; FEV1) measured before and after the use of genistein and before and after the use of placebo. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints to evaluate in vivo effect are: * Sweat chloride concentration (SCC) * Airway resistance (Rint and bodybox) * Body Mass Index (BMI) * Quality of life (measured with the Cystic Fibrosis Questionnnaire; (CFQ)) * Elastase measurements in the feces * The CFTR stimulating ability of the concentration of genistein in the patient*s blood samples. We will also determine the plasma levels of genistein; Assessment of ß-adrenergic sweat secretion by evaporimetry is included as an exploratory endpoint. | — |
Countries
Netherlands
Outcome results
None listed