An investigation of the effect of CTX-4430 on lung inflammation in patients with cystic fibrosis

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 20.0Level: PTClassification code 10011763Term: Cystic fibrosis lungSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2015-002677-38-DE
• Lead Sponsor: Celtaxsys Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-11-03
• Last Update Posted: 18 June 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 195

Inclusion Criteria

Patients are eligible to participate in the study if they meet all of the following inclusion criteria:
1. 18 to 30 years of age inclusive at the time of Screening
2. Documented, confirmed diagnosis of pulmonary CF (defined as follows):
• CF signs and symptoms AND
• Either 2 CFTR mutations on genetic testing OR sweat chloride =60 mEq/L
3. Medically stable, in the Investigator’s opinion
4. At least 1 pulmonary exacerbation, based on the Investigator’s judgment, in the 12 months before Screening
5. Resolution of any pulmonary exacerbation of CF at least 14 days before Screening, in the Investigator’s opinion
6. On a stable regimen of CF treatments with no change for at least 14 days before Screening and between Screening and Baseline
7. If on ivacaftor or ivacaftor-lumacaftor combination, on a stable regimen for at least 8 weeks before Baseline
8. No clinical or radiological evidence, per the Investigator’s site procedures, of clinically significant lung abnormalities (e.g., major atelectasis, pneumothorax)
9. FEV1 =50 percent predicted at Screening
10. Resting oxygen saturation >92% on room air
11. Body mass index (BMI) =17.0 kg/m2
12. No smoking (including electronic cigarettes) for at least 6 months before Screening and agreement not to use such products for the duration of the study
13. Females of childbearing potential must have a negative pregnancy test at Screening (unless surgically sterile) and must agree to use an effective contraception method from screening throughout the duration of the study
14. Able to perform spirometry according to European Respiratory Society/American Thoracic Society guidance
15. Able to swallow investigational product (IP) whole
16. Able to comply with the study procedures, in the opinion of the Investigator
17. Has provided informed consent to participate in the study.
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 195
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients are eligible to participate in this study if they meet none of the following exclusion criteria:
1. In the opinion of the Investigator, any significant clinical/laboratory/radiological/spirometric sign of unstable or unexpectedly deteriorating respiratory disease within 14 days before Screening or between Screening and Baseline (These clinical/laboratory/radiological/ spirometric signs include, but are not limited to, features suggestive of a pulmonary exacerbation as suggested by the modified Fuchs’ criteria.)
2. A medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results, in the opinion of the Investigator
3. History of organ transplantation
4. History of either alcoholism or drug abuse in the opinion of the investigator
5. Clinically significant hemoptysis (e.g., > approximately 30 cc per episode, or clinically significant in the Investigator’s opinion) within 180 days before Screening
6. Colonization with organisms associated with a more rapid decline in respiratory function in CF patients (e.g. all Burkholderia species, Mycobacterium abscessus). Subjects with a history of a positive culture could be considered free of colonization if she/he has had 6 subsequent respiratory tract cultures negative for these bacteria within the past 24 months prior to Screening, with one of these cultures obtained within 6 months prior to Screening
7. Active allergic bronchopulmonary aspergillosis at Screening or at Baseline
8. Any clinically significant ECG abnormality, in the Investigator’s opinion
9. Positive serology for HIV-1 or HIV-2 antibody, hepatitis C virus antibody, or hepatitis B surface antigen at Screening
10. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =2x the upper limit of normal (ULN) at Screening
11. Bilirubin >1.25 x ULN at Screening. Subjects with known Gilbert’s syndrome can be included with bilirubin >1.25 x ULN
12. Any subject with cirrhosis of the liver
13. Any subject with portal hypertension
14. Neutrophil count <1.5 x 109/L at Screening
15. Platelet count <150,000/µl at Screening
16. Clinically unstable pancreatic function, in the opinion of the Investigator. Evidence of unstable pancreatic function could include:
• Clinically significant weight loss (=5% after a previously stable period)
• Evidence of uncontrolled hyperglycemia or recent hypoglycemia
• Change in pancreatic enzyme requirements in the 60 days before Screening
17. Use of systemic corticosteroids, or systemic antimicrobial therapy (other than chronic antimicrobial use, e.g. azithromycin, flucloxacillin, itraconazole) within 14 days before Screening or between Screening and Baseline
18. Regular use (>3 times per week) of a high-dose NSAID (e.g., >1.6 g ibuprofen/day) within 60 days before Screening or between Screening and Baseline
19. Participation in a clinical trial for any medical/device product within 30 days before Screening (participation in a noninterventional or observational study is permitted)
20. Pregnant or nursing women

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified