Safety and Potential Efficacy of Human Mesenchymal Stem Cells in Non-Cystic Fibrosis Bronchiectasis

NCT02625246 | Completed Phase 1 DMC

• 1 other identifier: 20150627
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

To demonstrate the safety of bone marrow-derived allogeneic human Mesenchymal Stem Cells (hMSCs) in patients with bronchiectasis receiving standard of care therapy, and to explore treatment efficacy

Conditions

Bronchiectasis

Keywords

bronchiectasis; mesenchymal stem cell; lung; pulmonary; interstitial lung disease

Outcome Measures

Primary Outcomes (1)

• Number of Participant with treatment emergent serious adverse events

  incidence of any treatment-emergent serious adverse events defined as the composite of death, non-fatal pulmonary embolism, stroke, hospitalization for worsening dyspnea and clinically significant laboratory test abnormalities

  Week 4 post infusion

Secondary Outcomes (5)

• Difference in Colony Forming Units (CFUs) in semiquantitative culture of sputum

  Participants will be followed from 1 week to an expected average of 24 weeks following infusion.

• rate of decline of lung function

  Participants will be followed from 12 weeks to an expected average of 24 weeks following infusion.

• frequency of acute exacerbations

  Participants will be followed from 12 weeks to an expected average of 48 weeks following infusion.

• reported dyspnea and quality of life assessment

  Participants will be followed from 4 weeks to an expected average of 48 weeks following infusion.

• death from any cause

  Participants will be followed for the duration of the trial, which is an expected average of 48 weeks.

Study Arms (2)

Group 1

EXPERIMENTAL

3 patients will receive a single administration of allogeneic hMSCs: 20 x106 (20 million) cells delivered via peripheral intravenous infusion

Biological: hMSCs

Group 2

EXPERIMENTAL

3 patients will receive a single administration of allogeneic hMSCs: 1 x108 (100 million) cells delivered via peripheral intravenous infusion

Biological: hMSCs

Interventions

hMSCs BIOLOGICAL

intravenous infusion of bone marrow-derived allogeneic stem cells

Also known as: allogeneic mesenchymal stem cell

Group 1; Group 2

Eligibility Criteria

• Age: 30 Years - 87 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Provide written informed consent,
• be between 30 and 87 years old at the time of signing the Informed Consent,
• weight over 45 and under 150 kg,
• have a clinical diagnosis of non-CF bronchiectasis prior to screening,
• Have had at least 2 exacerbations in the past year as documented by physician office or hospital visits (Use of antibiotics of at least one time in the last year),
• Show a baseline FEV1 between 25% and 85% predicted and over or equal to 1 L and a baseline diffusion capacity of lung for carbon monoxide (DLCO) over or equal to 30% (corrected for hemoglobin but not alveolar volume),
• Have a normal Right Ventricular function, as documented by Doppler echo or right heart catheterization,
• if a female of childbearing potential, agree to abide by contraception rules defined below.
• Subjects may receive nondrug therapies including oxygen supplementation not greater than 4 Liters per minute and pulmonary rehabilitation.
• Subjects may be on chronic macrolide or inhaled antibiotic treatment bronchiectasis

You may not qualify if:

• be unable to perform any of the assessments required for endpoint analysis (report safety or tolerability concerns, perform Pulmonary Function Tests (PFT) or HRCT, undergo blood draws, read and respond to questionnaire
• If a female of childbearing potential, have a follicle stimulating hormone (FSH) under 25.8 IU/L
• be actively treated for an acute infectious exacerbation of bronchiectasis
• Have an active infection that is not treated
• Have had active infections occurring within a minimum of 4 weeks of study treatment
• Be currently on treatment for NTM infections
• Have had positive sputum cultures for nontuberculous mycobacterial (NTM) within the past 6 months
• Have a history of drug or alcohol abuse within the past 24 months.
• Be currently receiving (or have received within four weeks of screening) experimental agents for the treatment of bronchiectasis or have been enrolled in clinical trials within the previous 30 days
• Be actively listed (or expect future listing) for transplant of any organ.
• Have clinically important abnormal screening laboratory values.
• Have a serious comorbid illness that, in the opinion of the investigator, may compromise the safety or compliance of the patient or preclude successful completion of the study.
• Have any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or preclude successful completion of the study.
• Have known allergies to penicillin or streptomycin.
• Be an organ transplant recipient.

Sponsors & Collaborators

• Marilyn Glassberg: lead

Study Sites (1)

University of Miami Hospitals & Clinics

Miami, Florida, 33136, United States

Location

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MeSH Terms

Conditions

Bronchiectasis; Lung Diseases, Interstitial

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases

Study Officials

• Glassberg K Marilyn, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Medicine, Surgery, and Pediatrics

Study Record Dates

• First Submitted: December 4, 2015
• First Posted: December 9, 2015
• Study Start: February 4, 2016
• Primary Completion: May 15, 2019
• Study Completion: May 15, 2019
• Last Updated: August 28, 2019

Record last verified: 2019-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)