Cystic Fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: SOC Classification code 10021881 Term: Infections and infestations System Organ Class: 10021881 - Infections and infestations
Conditions
Interventions
Product Name: Zadaxin
Product Code: [Ta1]
Pharmaceutical Form: Lyophilisate for solution for injection
CAS Number: 62304-98-7
Current Sponsor code: Ta1
Other descriptive name: Thymosin alpha 1
Concent
Sponsors
SciClone Pharmaceuticals Limited Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Male or female, from 18 years of age (inclusive) at the time of screening. • Signed Informed Consent. • Confirmed diagnosis of CF, based on the following: two sweat chloride tests with a result = 60 mEq/L or two CFTR mutations on genetic test. • CF diagnosis independently of genetic mutations. • Clinical stability with no change in symptoms and/or medication within 4 weeks prior to screening. • Body mass index (BMI) = 15.0 (kg/m2). • Non-tobacco/nicotine-containing product user for a minimum of 6 months prior to screening. • Percent Predicted Forced Expiratory Volume (ppFEV1) > 40%, predicted at screening. • Female with pregnancy test negative and using an acceptable contraception method, except if postmenopausal for more than 2 years or sterilized for more than 3 months. • Blood pressure: DBP values between 60 and 89 mmHg, and SBP values between 90 and 139 mmHg. ECG normal or wave changes not considered clinically significant. • Pulse betweem 50 and 120 bpm unless deemed clinically. insignificant by the PI. 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 24 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: • Clinical/laboratory/radiological/spirometric signs of unstable or unexpectedly deteriorating respiratory disease (30 days prior to the screening). • Any malignancy or chronic organ failure or disease that depart from the patient's usual baseline level of health as a patient with CF. • Patients with “F508del homozygous mutation” treated successfully with corrector potentiators, according to physicians’ judgment. • Intravenous antibiotic use in the last 4 weeks before screening. • Treatment with oxygen. • History of organ or hematological transplantation. • Kidney (creatinine 2-fold of the normal upper limit) or hepatic alterations (Child Pugh score equal to B or C). • History or presence of alcoholism or drug abuse within 2 years prior to the screening. • Personal or family history of prolonged QT syndrome; or a QTc interval >430 msec (males) or > 450 msec (females) using Bazett's formula (QTcB) or deemed clinically significant by the PI. • In the judgment of the PI, clinically significant hemoptysis (>30 ml per episode) within the last 180 days. • History of allergy, hypersensitivity, intolerance to Thymosin alpha1 and to its excipients (Mannitol, monobasic sodium phosphate monohydrate, dibasic sodium phosphate heptahydrate) • Ongoing or prior participation in an investigational drug study within 30 days of screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The evaluation of the activity of Thymosin alpha 1 in patients with cystic fibrosis (CF) by means of variation of inflammatory cytokines (IL-1ß, IL-8, IL-17A, IL-6 and TNF-alpha) in serum and induced sputum.;Secondary Objective: - Evaluate the safety and tolerability of Thymosin alpha 1 by means of type, incidence, severity, timing, seriousness and relatedness of reported AEs, physical examinations, ECG and laboratory tests. - Evaluate the variation of neutrophil elastase in induced sputum, of c-reactive protein in blood and of sweat chloride concentration. - Evaluate and compare the first efficacy results about Thymosin alpha1 as assessed by changes before and after the treatment in lung functionality and quality of life.;Primary end point(s): The activity of Thymosin alpha 1 will be evaluated by means of variation of inflammatory cytokines (IL-1ß, IL-8, IL-17A, IL-6 and TNF-alpha) in serum and induced sputum,;Timepoint(s) of evaluation of this end point: It will be considered the difference between the values at the end of the first treatment period (week 8) and those at the baseline. | — |
Countries
Italy
Contacts
Public ContactCRO
Medi Service S.r.l
Outcome results
None listed