Clinical Trial of Stem Cell Based Tissue Engineered Laryngeal Implants (RegenVOX)
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|ClinicalTrials.gov Identifier: NCT01977911|
Recruitment Status : Withdrawn (Withdrawal of funding)
First Posted : November 7, 2013
Last Update Posted : June 6, 2018
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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.
|Condition or disease||Intervention/treatment||Phase|
|Disorder of Upper Respiratory System Laryngostenosis Tracheal Stenosis||Other: Stem cell based tissue engineered partial laryngeal implants||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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.|
|Estimated Study Start Date :||April 2018|
|Actual Primary Completion Date :||May 25, 2018|
|Actual Study Completion Date :||May 25, 2018|
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.
- 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.
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Patients aged >=18 years with sufficient numbers of Mesenchymal Stromal Cells (MSCs) in their 8-10ml human Bone Marrow (hBM) aspirate.
Patients with Myer-Cotton Grade 3 or 4* laryngotracheal stenosis or equivalent due to traumatic, inflammatory, iatrogenic, or idiopathic causes who have exhausted conventional therapies.
*The Myer-Cotton grading system for mature, firm, circumferential stenosis, confined to the subglottis describes the stenosis based on the per cent relative reduction in cross-sectional area of the subglottis. Four grades of stenosis:
- grade 1 lesions have less than 50% obstruction
- grade 2 lesions have 51% to 70% obstruction
- grade 3 lesions have 71% to 99% obstruction
- grade 4 lesions have no detectable lumen or complete stenosis
- Patients positive for HIV 1, HIV 2, HCV, HBV, syphilis or HTLV
- Those unable to provide informed consent
- Co-morbid severe chronic obstructive pulmonary disease (according to NICE criteria)
- Patients with active / uncontrolled chronic inflammatory conditions such as granulomatosis with polyangitis (formerly Wegener's granulomatosis) and sarcoidosis
- Any current or previous cancer within 5 years (except non-melanoma skin cancer, adequately treated carcinoma in situ of the uterine cervix, laryngeal malignancy treated locally with no local recurrence and no metastases, or low grade airway malignancy such as chondrosarcoma which may be causing airway obstruction)
- Life expectancy less than 5 years unless this limitation is principally due to the airway obstruction to be treated
- Concurrent enrollment in any other Clinical Trial of Investigational Medicinal Product (CTIMP).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01977911
|University College London NHS Foundation Trust (UCLH)|
|London, United Kingdom, NW1 2PG|
|Principal Investigator:||Martin Birchall, Prof||University College London (UCL) Ear Institute; Royal National Throat, Nose and Ear Hospital|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||University College, London|
|Other Study ID Numbers:||
UCL RD & ID no. 12/0331
2013-004359-18 ( EudraCT Number )
|First Posted:||November 7, 2013 Key Record Dates|
|Last Update Posted:||June 6, 2018|
|Last Verified:||June 2018|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
Pathological Conditions, Anatomical
Respiratory Tract Diseases
Respiratory System Abnormalities