ENgineered Tissue Repair of BronchopleUral FiSTula

NCT04435249 | Withdrawn Phase 1 DMC

• 1 other identifier: UK-2019-004939-24
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

Phase I/II Open-label Study to Assess the Safety and Efficacy of a Novel Tissue Engineered Airway Product, Consisting of Expanded Autologous Bone Marrow (BM) Derived Mesenchymal Stromal Cells (MSC) Seeded on to a Decellularised Allogeneic Patch of an Airway Scaffold in Subjects With Clinically Significant Bronchopleural Fistula.

Conditions

Bronchopleural Fistula

Outcome Measures

Primary Outcomes (2)

• Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair

  Safety: Emergent or worsening Serious Adverse Events (SAEs) related to the BPF implant during the 3 months post-defect repair.

  3 months (90 days)

• Efficacy: Bronchopleural Fistula (BPF) closure at 3 months assessed by visual appearance and no clinical signs of leaks.

  Closure of BPF with no need for further surgical closure. Efficacy will be deemed demonstrated if 2 or more subjects meet the primary endpoint.

  3 months (90 days)

Secondary Outcomes (4)

• Change from Baseline in Physiological and Quality of Life scores at 3, 6, 9, 12 months, Evaluation Questionnaire 5D (EQ-5D), 6-minute walking test (6MWT), ambulatory oxygen (O2).

  36 months

• Safety and tolerability: - Adverse Events (AEs) including pre-defined postoperative AEs of special interest up to 30± 3 days post implantation- Related AEs reported after 3, 6, 9, 12, 24 and 36 months

  36 months

• Viability and the integrity of the repair area (closure) visually via bronchoscope. Integrity assessed by visual appearance of no visible leaks- Structural closure of the defect and visual health of the defect area and surrounding tissue

  36 months

• Absence of other surgical interventions at 6, 9, 12, 24 and 36 months

  36 months

Study Arms (1)

Treatment Arm

EXPERIMENTAL

Patients will have a patch of expanded somatic mesenchymal stromal cells (MSCs) seeded onto a decellularised human tracheal-scaffold surgically implanted to repair bronchial fistula.

Other: BPF-001

Interventions

BPF-001 OTHER

Expanded somatic mesenchymal stromal cells (MSCs) seeded onto a decellularised human tracheal-scaffold

Treatment Arm

Eligibility Criteria

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

You may qualify if:

• Male or female subjects 18 years or older.
• Documented diagnoses of BPF through imaging and bronchoscopic examination.
• BPF which involves the tracheobronchial junction or proximal bronchus.
• Subjects who have failed primary repair.
• Subjects who have no evidence of any primary or recurrent cancer (not limited to the surgical site) at the time of pre-operative screening as evidenced by CT and/or targeted biopsy (except for controlled or controllable basal cell carcinoma.
• Subjects who have signed and dated written informed consent to participate in the study.
• Females of childbearing potential (i.e. not surgically sterilised or post-menopausal for at least 2 years) must have a negative serum or urine pregnancy test.
• Male and female subjects of childbearing potential (i.e. not surgically sterilised or post-menopausal for at least 2 years) must use forms of highly effective methods of contraception, which are defined as hormonal methods of contraception (oral, injection or implant), barrier methods (condom or occlusive cap (diaphragm or cervical/vault caps)) with spermicidal foam/gel/cream/film/suppository, or true abstinence for 1 month following surgery.
• Subjects who have produced viable cells from Bone Marrow Aspirate.

You may not qualify if:

• Subjects who have received previous treatment with another Advanced Technology Medicinal Product (ATMP).
• Subjects with ECOG performance status of 3 or 4.
• Subjects deemed not suitable for surgery by the MDT.
• Uncontrolled diabetes, defined as HbA1c levels above 7.0 %.
• Any medical condition contraindicating the ability to tolerate general anaesthetic in the judgement of a consultant anaesthetist.
• Subjects who have a severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or in the judgment of the investigator, would make the subject inappropriate for entry into this trial.
• Subjects with clinically significant renal and liver impairment.
• Subjects undergoing an immunosuppression regimen or suffering from a primary immunodeficiency syndrome.
• Subjects with any known hypersensitivity to the culture and transport media compounds.
• Subjects with current or recurrent disease that could affect the administration, the action or disposition of the investigational product, or clinical or laboratory assessments.
• Subjects with clinically relevant or recent (within 2 years) history of substance abuse, including alcohol.
• Subject who has participated in any other interventional clinical trial within previous 30 days of the start of this study.
• Subjects with known presence of Human Immunodeficiency Virus (HIV) antibody, Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody.
• Subject who, in the investigator's judgement, is unlikely to complete all protocol required study visits or procedures, including follow-up visits, or comply with the study requirements for participation
• Subjects with any medical condition, that in the investigator's judgement, is likely to interfere with assessment of safety or efficacy of study treatment.

Sponsors & Collaborators

• Videregen Limited: lead
• Cell Therapy Catapult: collaborator
• Papworth Hospital NHS Foundation Trust: collaborator
• Royal Free Hospital NHS Foundation Trust: collaborator

Study Design

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

Study Record Dates

• First Submitted: May 29, 2020
• First Posted: June 17, 2020
• Study Start: October 1, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): November 1, 2028
• Last Updated: April 1, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share