Study of the use of the drug Thymosin alpha 1 in adults patients with Cystic Fibrosis.

• Register: EUCTR
• Last refreshed on: 16 December 2024
• Main ID: EUCTR2019-001441-40-IT
• Date of registration: 24/05/2021
• Prospective Registration: No
• Primary sponsor: SciClone Pharmaceuticals Limited Ltd
• Public title: Study of the use of the drug Thymosin alpha 1 in adults patients with Cystic Fibrosis.
• Scientific title: A phase II trial to assess the activity and tolerability of Thymosin alpha 1 in Cystic Fibrosis Patients. - Thymosin alpha 1 in Cystic Fibrosis patients
• Date of first enrolment: 17/12/2019
• Target sample size: 24
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-001441-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Italy

Contacts

• Name: CRO
• Affiliation: Medi Service S.r.l

• Name: CRO
• Affiliation: Medi Service S.r.l

Key inclusion & exclusion 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:
• 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.


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

Health Condition(s) or Problem(s) studied

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

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Intervention(s)

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
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)

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,

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.

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.

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.

Secondary ID(s)

SC-Ta1-CF-001

Ethics review

Status: Approved
Approval date: 17/12/2019
Contact: