Bronchopleural fistula (BPF) MedDRA version: 21.1 Level: PT Classification code 10053481 Term: Bronchopleural fistula System Organ Class: 10038738 - Respiratory, thoracic and mediastinal disorders
Conditions
Interventions
Product Name: Tissue engineered autologous cell seeded allogeneic airway scaffold
Pharmaceutical Form: Living tissue equivalent
Sponsors
Cell and Gene Therapy Catapult
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Male or female subjects 18 years or older. • Documented diagnoses of BPF through imaging and bronchoscopic examination. • BPF which involves the tracheobronchial junction or proximal bronchus. • Subjects who have failed primary repair. • Subjects who have no evidence of any primary or recurrent cancer (not limited to the surgical site) at the time of pre-operative screening as evidenced by CT and/or targeted biopsy (except for controlled or controllable basal cell carcinoma. • Subjects who have signed and dated written informed consent to participate in the study. • Females of childbearing potential (i.e. not surgically sterilised or post-menopausal for at least 2 years) must have a negative serum or urine pregnancy test. • Male and female subjects of childbearing potential (i.e. not surgically sterilised or post-menopausal for at least 2 years) must use forms of highly effective methods of contraception, which are defined as hormonal methods of contraception (oral, injection or implant), barrier methods (condom or occlusive cap (diaphragm or cervical/vault caps)) with spermicidal foam/gel/cream/film/suppository, or true abstinence for 1 month following surgery. • Subjects who have produced viable cells from BMA. 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 5 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Subjects who have received previous treatment with another Advanced Technology Medicinal Product (ATMP). • Subjects with ECOG performance status of 3 or 4. • Subjects deemed not suitable for surgery by the MDT. • Uncontrolled diabetes, defined as HbA1c levels above 7.0 %. • Any medical condition contraindicating the ability to tolerate general anaesthetic in the judgement of a consultant anaesthetist. • Subjects who have a severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or in the judgment of the investigator, would make the subject inappropriate for entry into this trial. • Subjects with clinically significant renal and liver impairment. • Subjects undergoing an immunosuppression regimen or suffering from a primary immunodeficiency syndrome. • Subjects with any known hypersensitivity to the culture and transport media compounds. • Subjects with current or recurrent disease that could affect the administration, the action or disposition of the investigational product, or clinical or laboratory assessments. • Subjects with clinically relevant or recent (within 2 years) history of substance abuse, including alcohol. • Subject who has participated in any other interventional clinical trial within previous 30 days of the start of this study. • Subjects with known presence of Human Immunodeficiency Virus (HIV) antibody, Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody. • Subject who, in the investigator’s judgement, is unlikely to complete all protocol required study visits or procedures, including follow-up visits, or comply with the study requirements for participation • Subjects with any medical condition, that in the investigator’s judgement, is likely to interfere with assessment of safety or efficacy of study treatment. • Subject who is pregnant. • Subjects with cancer (except for controlled or controllable basal cell carcinoma). .
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • Safety: To assess the safety of the engineered airway tissue product during 3 months (90 days) post defect repair • Efficacy: To determine BPF closure at 3 months post defect repair. ;Secondary Objective: • To assess impact on the subject’s Quality of Life (QoL) of the engineered airway tissue product patch at 3, 6, 9,12, 24 and 36 months following defect repair. • To assess the viability and the integrity of the repair area visually via bronchoscopy at 6, 9, 12 months. Integrity assessed by visual appearance and no clinical signs of leaks. • To assess safety of the TE airway patch at 6, 9,12, 24 and 36 months following defect repair. • Incidence of interventions at the operative sites up to 6, 9, 12, 24 and 36 months.;Primary end point(s): • Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair. • Efficacy: BPF closure assessed by visual appearance and no clinical signs of leaks.;Timepoint(s) of evaluation of this end point: • Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair. • Efficacy: BPF closure at 3 months assessed by visual appearance and no clinical signs of leaks. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Impact as assessed in change from Baseline in Physiological and QoL scores for each subject at 3, 6, 9, 12, 24 and up to 36 months post defect repair (physiological and QoLs): QoL EQ-5D, 6-minute walking test (6MWT), ambulatory O2 measurements. • Viability of the repair area and integration with vascular supply at 6, 9, 12 months. Adverse events related to the BPF implant procedures reported after 3, 6, 9, 12, 24 and 36 months. • Interventions at the operative sites up to 6, 9, 12, 24 and 36 months. ;Timepoint(s) of evaluation of this end point: • Efficacy: closure of BPF with no need for further surgical closure. The trial will be considered to have demonstrated efficacy if 2 or more subjects meet the primary endpoint. • Safety and tolerability: - AEs including pre-defined postoperative AEs of special interest up to 30 ± 3 days post implantation - Related AEs reported after 3, 6, 9, 12, 24 and 36 months • Viability and the integrity of the repair area (closure) visually via bronchoscopy at 6, 9, 12, 24 and 36 months: - Integrity assessed by visual appearance of no visible leaks - Structural closure of the defect and visual health of the defect area and surrounding • Absence of other surgical interventions at 6, 9, 12, 24 and 36 months | — |
Countries
United Kingdom
Contacts
Public ContactRegulatory Affairs
Cell and Gene Therapy Catapult
Outcome results
None listed