A Study to Assess the Safety, Tolerability and Potential Efficacy of a Tracheal Replacement Consisting of a Tissue-engineered Tracheal Scaffold With Seeded Mesenchymal Cells
|ClinicalTrials.gov Identifier: NCT02949414|
Recruitment Status : Suspended
First Posted : October 31, 2016
Last Update Posted : March 29, 2018
|Condition or disease||Intervention/treatment||Phase|
|Tracheomalacia Tracheal Stenosis||Procedure: Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells||Phase 1|
This is a phase I study to evaluate the safety, tolerability and potential efficacy of a novel tracheal replacement therapy using a cadaveric tracheal scaffold and patients own mesenchymal cells isolated from a sample of the patients own bone marrow. The study is aimed at treating patients who suffer from severe tracheal stenosis or malacia and for whom conventional therapies are no longer adequate. A total of 4 patients will be treated during the course of this study.
A hospital multi disciplinary team will review the medical history and available treatment options for all potential patients and recommend whether they are suitable for the study. Once patients are approved they will enter an 8 week screening period. During this period bone marrow from the patient will be harvested and the manufacturing of the final graft tissue will start. The manufacturing facility will use a cadaveric donated decellularised tracheal scaffold (supplied by the NHS blood and transplant body) and the patients own cells to make the final investigational product (graft).
The product will be surgically grafted into the patient in place of the damaged tracheal section. The graft will be supported by a stent for the first 6 months with replacements of this stent occurring at week 8 and week 16 post-surgical procedure. Hospitalization for a number of days will be required during this replacement steps. The patient will be followed frequently post surgery to capture any safety and efficacy measures. Long term follow up will continue up to 5 years post surgery.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Open-label Study to Assess the Safety, Tolerability and Potential Efficacy of a Novel Tracheal Replacement Consisting of a Tissue-engineered Decellularised Tracheal Scaffold With Seeded Autologous Mesenchymal Cells in Subjects With Severe Tracheal Stenosis or Malacia|
|Study Start Date :||September 2016|
|Estimated Primary Completion Date :||September 2019|
|Estimated Study Completion Date :||September 2024|
Experimental: Tracheal Replacement
Each patient will receive surgery to implant the cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells and all follow-up procedures.
Procedure: Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells
The tracheal graft will be manufactured from cadaveric tracheal scaffold and bone marrow derived mesenchymal cells.
- Number of treatment related SAE's as assessed by CTCAE grades as defined in version 4.0 [ Time Frame: Up to 8 months post graft ]
- Efficacy: Post Implant Stent free period [ Time Frame: Up to 5 years post implant ]
- Efficacy: Post Implant Tracheostomy free period [ Time Frame: Up to 5 years post implant ]Absence of other surgical interventions
- Efficacy: Mean Airway Diameter evaluations as captured by CT scans [ Time Frame: Up to 5 years post implant ]
- Efficacy: Changes in lung function results using a combination of FEV1, FEF50 and PEFR results [ Time Frame: Up to 5 years post implant ]
- Number of treatment related AEs as assessed by CTCAE grading version 4.0 [ Time Frame: Upto 5 years post implant ]
- Changes in Quality of Life assessed using EQ-5D questionnaires [ Time Frame: Up to 5 years post graft. ]
- Change in Airway Dyspnoea Voice swallowing Index (ADVS) [ Time Frame: Up to 5 years post graft. ]
- Medical Resource Utilisation (MRU) assessed by number and type of tracheal related procedures and treatments post tracheal replacement. [ Time Frame: Up to 5 years post graft. ]
- Change in maximum phonation time (MPT) measured in seconds [ Time Frame: Up to and including 5 years post graft ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02949414
|Royal Nose Throat and Ear Institute|
|London, United Kingdom, WC1X 8DA|
|Principal Investigator:||Martin Birchall, MD, PhD||University College, London|