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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Sciclone Pharmaceuticals International (SG) Pte. Ltd.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

7 Dec 2021 → 7 Mar 2025

Decision date (initial)

2024-11-04

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-518102-41-00

EudraCT number

2019-001441-40

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Pharmacodynamic

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 sputum.

Secondary objectives 3

1. 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.
2. Evaluate the variation of a combined score of inflamamtory cytokines, neutrophil elastase in induced sputum, of c-reactive protein in blood and of sweat chloride concentration.
3. 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.

Conditions and MedDRA coding

Cystic Fibrosis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 11

1. Male or female, from 18 years of age (inclusive) at the time of screening.
2. Signed Informed Consent.
3. 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.
4. CF diagnosis independently of genetic mutations.
5. Clinical stability with no change in symptoms and/or medication within 4 weeks prior to screening.
6. Body mass index (BMI) = 15.0 (kg/m2).
7. Non-tobacco/nicotine-containing product user for a minimum of 6 months prior to screening.
8. Percent Predicted Forced Expiratory Volume (ppFEV1) > 40%, predicted at screening.
9. 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.
10. 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.
11. Pulse betweem 50 and 120 bpm unless deemed clinically. insignificant by the PI.

Exclusion criteria 12

1. Clinical/laboratory/radiological/spirometric signs of unstable or unexpectedly deteriorating respiratory disease (30 days prior to the screening).
2. Any malignancy or chronic organ failure or disease that depart from the patient's usual baseline level of health as a patient with CF.
3. Patients with “F508del homozygous mutation” treated successfully with corrector potentiators, according to physicians’ judgment.
4. Intravenous antibiotic use in the last 4 weeks before screening.
5. Treatment with oxygen.
6. History of organ or hematological transplantation.
7. Kidney (creatinine 2-fold of the normal upper limit) or hepatic alterations (Child Pugh score equal to B or C).
8. History or presence of alcoholism or drug abuse within 2 years prior to the screening.
9. 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.
10. In the judgment of the PI, clinically significant hemoptysis (>30 ml per episode) within the last 180 days.
11. History of allergy, hypersensitivity, intolerance to Thymosin alpha1 and to its excipients (Mannitol, monobasic sodium phosphate monohydrate, dibasic sodium phosphate heptahydrate)
12. Ongoing or prior participation in an investigational drug study within 30 days of screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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 sputum.

Secondary endpoints 3

1. Safety and tolerability will be evaluated during the course of the study by means of: - Type, incidence, severity, timing, seriousness and relatedness of reported AEs. - Physical examinations. - Vital signs. - ECGs. - Clinical laboratory results (local lab).
2. Activity will be evaluated during the course of the study by monitoring: - variation of a combined score of inflammatory cytokines; - variation of neutrophil elastase in sputum; - variation of blood C-reactive protein; - variation of the sweat chloride concentration tests.
3. Efficacy will be evaluated by monitoring during the course of the study of: - change in predicted forced expiratory volume in 1 second (ppFEV1). - change in quality of life measured by CFQ-R questionnaire

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Sciclone Pharmaceuticals International (SG) Pte. Ltd.

Sponsor organisation

Sciclone Pharmaceuticals International (SG) Pte. Ltd.

Address

1 George Street, #23-03 #23-03

City

Singapore

Postcode

049145

Country

Singapore

Scientific contact point

Organisation

Sciclone Pharmaceuticals International (SG) Pte. Ltd.

Contact name

Cevi Wang

Public contact point

Organisation

Sciclone Pharmaceuticals International (SG) Pte. Ltd.

Contact name

Cevi Wang

Third parties 3

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications