Phase I/II Open-label Study to Assess the Safety and Efficacy of a Novel Tissue Engineered Airway Product, Consisting of Expanded Autologous Bone Marrow (BM) Derived Mesenchymal Stromal Cells (MSC) Seeded on to a Decellularised Allogeneic Patch of an Airway Scaffold in Subjects With Clinically Significant Bronchopleural Fistula.
The proposed clinical trial is a phase I/II open-label study to assess the safety and efficacy of a novel tissue engineered airway product, consisting of expanded autologous Bone Marrow (BM) derived Mesenchymal Stromal Cells (MSC) seeded on to a decellularised allogeneic patch of an airway scaffold in subjects with clinically significant bronchopleural fistula. It is a phase I/II open-label study, which is an uncontrolled pilot in 5 subjects.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Expanded somatic mesenchymal stromal cells (MSCs) seeded onto a decellularised human tracheal-scaffold
Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair
Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair.
Time frame: 3 months (90 days)
Efficacy: Bronchopleural Fistula (BPF) closure at 3 months assessed by visual appearance and no clinical signs of leaks.
Closure of BPF with no need for further surgical closure. Efficacy will be deemed demonstrated if 2 or more subjects meet the primary endpoint.
Time frame: 3 months (90 days)
Change from Baseline in Physiological and Quality of Life scores at 3, 6, 9, 12 months, Evaluation Questionnaire 5D (EQ-5D), 6-minute walking test (6MWT), ambulatory oxygen (O2).
Impact as assessed in change from Baseline in Physiological and Quality of Life scores for each subject at 3, 6, 9, 12, 24 and up to 36 months post defect repair: QoL EQ-5D, 6-minute walking test, ambulatory O2 measurements.
Time frame: 36 months
Safety and tolerability: - Adverse Events (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
Adverse events related to the BPF implant procedures reported after 3, 6, 9, 12, 24 and 36 months.
Time frame: 36 months
Viability and the integrity of the repair area (closure) visually via bronchoscope. Integrity assessed by visual appearance of no visible leaks- Structural closure of the defect and visual health of the defect area and surrounding tissue
viability of the repair area and integration with vascular supply at 6, 9, 12, 24 and 36 months.
Time frame: 36 months
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Absence of other surgical interventions at 6, 9, 12, 24 and 36 months
Interventions at the operative sites up to 6, 9, 12, 24 and 36 months
Time frame: 36 months