TracheomalaciaPhase 1NCT02949414
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
A Phase 1 study testing Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells in people with Tracheomalacia and Tracheal Stenosis, run by Cell Therapy Catapult at 1 site in United Kingdom.
Trial summary
This is a phase I study to evaluate the safety, efficacy and tolerability of a novel tracheal replacement therapy using cadaveric de-cellularised tracheal scaffold and patients' own mesenchymal cells isolated from a sample of their bone marrow in patients' who suffer from severe tracheal malacia or stenosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Tracheomalacia, Tracheal Stenosis
- What is being tested?
- Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Sep 2016to Sep 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female subjects 18 years or older (all subjects must provide written informed consent)
- Stent or tracheostomy dependent diagnosis of tracheal stenosis or tracheomalacia (Cotton-Myers grade 2 or more)
- Subjects in the above categories for whom further conventional therapies are no longer adequate
Who cannot join
- Pregnancy
- Subjects unable to provide informed consent
- Prior tracheal transplant
- No viable bone marrow cells within the screening period
- Subjects who have conventional treatment options still available that may have additive impact
- Subject diagnosed or treated for a malignancy within 1 year of study entry or who have previously been diagnosed with a malignancy and have any radiographic or biochemical biomarker evidence of malignancy. Subjects with completely resected basal cell carcinoma or squamous cell carcinoma of the skin or in situ malignancy are not excluded
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- Subject with active inflammatory or infectious conditions such as polychondritis, granulomatosis with polyangiitis ('Wegener's'), sarcoidosis or tuberculosis
- Co-morbid moderate or severe chronic obstructive pulmonary disease (COPD) as defined in Global Initiative for COPD, 2011 2, that is unrelated to tracheal stenosis or malacia
- Subjects with known presence of human immunodeficiency virus (HIV) antibody, Hepatitis B surface antigen (HbsAG) or Hepatitis C antibody
- Subject with clinically relevant or recent (within 2 years) history of substance abuse, including alcohol
- Serious medical or psychiatric illness likely to interfere with participation in the study
- Participation in any other clinical trial within previous 30 days of the start of this study or concurrent participation in another clinical trial.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Royal Nose Throat and Ear Institute
London, WC1X 8DA
About this study
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
This is a phase I study to evaluate the safety, efficacy and tolerability of a novel tracheal replacement therapy using cadaveric de-cellularised tracheal scaffold and patients' own mesenchymal cells isolated from a sample of their bone marrow in patients' who suffer from severe tracheal malacia or stenosis.
Read the detailed description
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.
- Conditions
- Tracheomalacia, Tracheal Stenosis
- Being tested
- Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells
- Keywords
- Malacia, Stenosis, Trachea, Transplant
- Medical subject headings
- Tracheomalacia, Tracheal Stenosis, Constriction, Pathologic, Tracheal Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cellsProcedure or surgery
The tracheal graft will be manufactured from cadaveric tracheal scaffold and bone marrow derived mesenchymal cells.
| Study group | Role | What they receive |
|---|---|---|
| Tracheal Replacement | Experimental | Each patient will receive surgery to implant the cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells and all follow-up procedures. Receives: Procedure: Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of treatment related SAE's as assessed by CTCAE grades as defined in version 4.0
Measured: Up to 8 months post graft
Secondary outcomes (5)
Efficacy: Post Implant Stent free period
Measured: Up to 5 years post implant
Efficacy: Post Implant Tracheostomy free period
Absence of other surgical interventions
Measured: Up to 5 years post implant
Efficacy: Mean Airway Diameter evaluations as captured by CT scans
Measured: Up to 5 years post implant
Efficacy: Changes in lung function results using a combination of FEV1, FEF50 and PEFR results
Measured: Up to 5 years post implant
Number of treatment related AEs as assessed by CTCAE grading version 4.0
Measured: Upto 5 years post implant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2016 (estimated)
- Study startSep 2016
- Primary completionSep 2019 (estimated)
- Study completionSep 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for TracheomalaciaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cell Therapy Catapult
- Sponsor type
- University, hospital or other
- Collaborators
- University College, London, Videregen Limited
- Responsible party
- Sponsor
- Investigators
- Martin Birchall, MD, PhD, Principal investigator, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02949414
- Sponsor's protocol ID
- D-00173-CT2013002
- Status last verified
- Mar 2018
- First submitted
- 15 Jan 2016
- Last updated
- 29 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Study and analysis will be published upon completion.
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02949414
- Acronym / sponsor ID
- D-00173-CT2013002
- Conditions
- Tracheomalacia, Tracheal Stenosis
- Keywords
- Malacia, Stenosis, Trachea, Transplant
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Suspended
- Enrolment
- 4 participants (estimated)
- Lead sponsor
- Cell Therapy CatapultUniversity, hospital or other
- Collaborators
- University College, London, Videregen Limited
- Study start
- Sep 2016
- Primary completion
- Sep 2019 (estimated)
- Study completion
- Sep 2024 (estimated)
- First posted
- 31 Oct 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase I study to evaluate the safety, efficacy and tolerability of a novel tracheal replacement therapy using cadaveric de-cellularised tracheal scaffold and patients' own mesenchymal cells isolated from a sample of their bone marrow in patients' who suffer from severe tracheal malacia or stenosis.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Tracheal Replacement | Experimental | Procedure: Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cellsEach patient will receive surgery to implant the cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells and all follow-up procedures. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Male or female subjects 18 years or older (all subjects must provide written informed consent)
- Stent or tracheostomy dependent diagnosis of tracheal stenosis or tracheomalacia (Cotton-Myers grade 2 or more)
- Subjects in the above categories for whom further conventional therapies are no longer adequate
Key exclusion criteria (12 listed)
- Pregnancy
- Subjects unable to provide informed consent
- Prior tracheal transplant
- No viable bone marrow cells within the screening period
- Subjects who have conventional treatment options still available that may have additive impact
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cellsThe tracheal graft will be manufactured from cadaveric tracheal scaffold and bone marrow derived mesenchymal cells. | Procedure or surgery | Tracheal Replacement |
Primary outcome
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
5 secondary outcomes
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
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2018.
- Individual participant dataSharing planned: Yes. Study and analysis will be published upon completion.
Related trials for Tracheomalacia
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Primary Posterior Tracheopexy Prevents Tracheal Collapse
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Martin Birchall, MD, PhD principal investigator
University College, London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells in people with Tracheomalacia and Tracheal Stenosis, run by Cell Therapy Catapult at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: paused, as of the sponsor's last update on 29 Mar 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Mar 2018.