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

NCT02949414 | Suspended Phase 1 DMC

• 1 other identifier: D-00173-CT2013002
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Tracheomalacia; Tracheal Stenosis

Keywords

Malacia; Stenosis; Trachea; Transplant

Outcome Measures

Primary Outcomes (1)

• Number of treatment related SAE's as assessed by CTCAE grades as defined in version 4.0

  Up to 8 months post graft

Secondary Outcomes (5)

• Efficacy: Post Implant Stent free period

  Up to 5 years post implant

• Efficacy: Post Implant Tracheostomy free period

  Up to 5 years post implant

• Efficacy: Mean Airway Diameter evaluations as captured by CT scans

  Up to 5 years post implant

• Efficacy: Changes in lung function results using a combination of FEV1, FEF50 and PEFR results

  Up to 5 years post implant

• Number of treatment related AEs as assessed by CTCAE grading version 4.0

  Upto 5 years post implant

Other Outcomes (4)

• Changes in Quality of Life assessed using EQ-5D questionnaires

  Up to 5 years post graft.

• Change in Airway Dyspnoea Voice swallowing Index (ADVS)

  Up to 5 years post graft.

• Medical Resource Utilisation (MRU) assessed by number and type of tracheal related procedures and treatments post tracheal replacement.

  Up to 5 years post graft.

Study Arms (1)

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.

Procedure: Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells

Interventions

Cadaveric decellularised tracheal scaffold seeded with autologous mesenchymal cells PROCEDURE

The tracheal graft will be manufactured from cadaveric tracheal scaffold and bone marrow derived mesenchymal cells.

Tracheal Replacement

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Cell Therapy Catapult: lead
• University College, London: collaborator
• Videregen Limited: collaborator

Study Sites (1)

Royal Nose Throat and Ear Institute

London, WC1X 8DA, United Kingdom

Location

MeSH Terms

Conditions

Tracheomalacia; Tracheal Stenosis; Constriction, Pathologic; Tracheal Diseases

Condition Hierarchy (Ancestors)

Tracheobronchomalacia; Cartilage Diseases; Musculoskeletal Diseases; Respiratory Tract Diseases; Musculoskeletal Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Martin Birchall, MD, PhD

  University College, London

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 15, 2016
• First Posted: October 31, 2016
• Study Start: September 1, 2016
• Primary Completion: September 1, 2019
• Study Completion: September 1, 2024
• Last Updated: March 29, 2018

Record last verified: 2018-03

Data Sharing

• IPD Sharing: Will share

Locations

GB (1)