Clinical Trial of Stem Cell Based Tissue Engineered Laryngeal Implants (RegenVOX)
This study aims to test a new groundbreaking treatment for narrowing of the voicebox and upper windpipe, which can be due to injury, inflammatory disease or cancer treatment. Narrowing of the voicebox or upper windpipe can leave patients dependant on a tracheostomy tube to breath through (a tube or hole in the neck), which can require a high level of care with regular hospital visits and can lead to recurrent chest infections. Regular surgical procedures may be necessary to widen the airway. Speaking may be very difficult or not even possible, breathing is usually a struggle and swallowing can also be affected. Patients feel very tired all the time.
The new treatment tested by this study is an implant that will partially replace the voicebox or upper windpipe in order to cure the narrowing. The implant is based on a human donor voicebox or windpipe that has been processed with detergents and enzymes in order to remove all the cells from the donor, leaving a 'scaffold' of connective tissue. The patient's own stem cells are removed from the bone marrow, then are grown on the scaffold in the laboratory. These cells will form the cartilage in the wall of the scaffold. A split skin graft from the patient may be needed to line the inside of the implant. The implant can be considered 'living' due to the cells grown on it, and this type of treatment is referred to as 'tissue engineering' or 'regenerative medicine'.
Once these cells have attached and started to grow on the scaffold, it is ready to be implanted into the patient, and an operation is performed which occurs in two separate stages. The final stage of the operation involves removing the narrow section of voicebox or upper windpipe and implanting the scaffold to reconstruct it. Patients will be followed up for two years after this operation, with investigations such as CT scans, examination of the voicebox and windpipe with a flexible camera (bronchoscopy) and blood tests performed at specific times.
It is intended that this treatment will significantly improve patients' symptoms resulting in better breathing, swallowing and voice function, reducing the need for repeated hospital visits and procedures and enhancing patients' quality of life.
|Disorder of Upper Respiratory System Laryngostenosis Tracheal Stenosis||Other: Stem cell based tissue engineered partial laryngeal implants||Phase 1 Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||Phase I/IIa Clinical Trial of Stem Cell Based Tissue Engineered Partial Laryngeal Implants in 10 Adult Patients With End-stage Laryngeal Stenosis With 24 Months Follow-up.|
- Safety of tissue engineered laryngotracheal replacement [ Time Frame: 24 months post operative follow up ]The primary outcome measure is safety of the tissue engineered implant as defined by morbidity and mortality and measured by occurrence of adverse events throughout 24 months post operative follow up
- Efficacy of tissue engineered laryngotracheal replacement [ Time Frame: 24 months post operative follow up ]
Secondary outcome is efficacy as determined
- Absence of tracheostomy.
- Absence of non-absorbable stent.
- Improvement in mean airway diameter.
- Improvement in Forced Expiratory Volume in 1 second (FEV1).
- Improvement in global quality of life (EQ-5D).
- Improvement in maximum phonation time (MPT) as measured by Voice Analysis Operavox (VAO).
- Improvement in self assessment of voice handicap (VHI-10).
- Improvement in swallowing function (EAT-10).
- Improvement in airway, dyspnoea, voice, swallowing index (ADVS index).
- Improvement in the penetration-aspiration scale (PAS) as per Video Fluoroscopic Swallow (VFS) or Functional Endoscopic Evaluation of Swallowing (FEES).
- Cost effectiveness of tissue engineered laryngotracheal replacement [ Time Frame: 24 months post operative follow up ]We will also develop new pathways for maximising discovery science and health economic benefit from complex regenerative medicine therapies (a reverse translational route map), with important generic benefits for scientists and clinicians.
|Study Start Date:||August 2015|
|Estimated Study Completion Date:||December 2018|
|Estimated Primary Completion Date:||August 2018 (Final data collection date for primary outcome measure)|
Experimental: Tissue engineered airway construct
Stem cell based tissue engineered partial laryngeal implants:
The final experimental product is a highly tested, recellularised, stem cells based tissue engineered product (airway construct) for operative partial laryngeal implantation into patients with severe laryngotracheal stenosis
Other: Stem cell based tissue engineered partial laryngeal implants
Stem cells from the patient receiving the implant are removed from the bone marrow, and are then grown on the scaffold in the laboratory. These cells will form the cartilage in the wall of the scaffold. The implant can be considered 'living' due to the cells grown on it, and this type of treatment is referred to as 'tissue engineering' or 'regenerative medicine'. Once these cells have attached and started to grow on the scaffold, it is ready to be implanted into the patient and a two separate operative stages can occur. The final stage of the operation involves removing the narrow section of voicebox or upper windpipe and implanting the scaffold to reconstruct it.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01977911
|Contact: Helen Knowles, PhD||+44 (0) email@example.com|
|University College London NHS Foundation Trust (UCLH)||Recruiting|
|London, United Kingdom, NW1 2PG|
|Principal Investigator: Martin Birchall, Prof|
|Principal Investigator:||Martin Birchall, Prof||University College London (UCL) Ear Institute; Royal National Throat, Nose and Ear Hospital|