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A Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis (MSC in IPF)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01385644
Recruitment Status : Completed
First Posted : June 30, 2011
Results First Posted : December 29, 2015
Last Update Posted : December 29, 2015
Sponsor:
Collaborator:
Mater Medical Research Institute
Information provided by (Responsible Party):
Daniel Chambers, The Prince Charles Hospital

Tracking Information
First Submitted Date  ICMJE May 4, 2011
First Posted Date  ICMJE June 30, 2011
Results First Submitted Date  ICMJE October 20, 2015
Results First Posted Date  ICMJE December 29, 2015
Last Update Posted Date December 29, 2015
Study Start Date  ICMJE October 2010
Actual Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 24, 2015)
Number of Participants Who Demonstrated Acute Adverse Events Following Infusion [ Time Frame: 4 hours post-infusion ]
Acute adverse events following infusion was defined as the development of anaphalaxis and/or a 25% increase or decrease from baseline of hemodynamic measurements.
Original Primary Outcome Measures  ICMJE
 (submitted: June 29, 2011)
To provide evidence of safe delivery of MSC in doses as per protocol. [ Time Frame: 6 months post MSC infusion ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 24, 2015)
  • Percentage Change in Lung Function as Assessed by FVC Compared to Baseline [ Time Frame: 6 months post MSC infusion ]
    Forced Vital Capacity (FVC) was measured and reported as a percentage of predicted and comapred from 6 months post-infusion to baseline
  • Percentage Change in 6 Minute Walk Distance Compared to Baseline [ Time Frame: Baseline and 6 months post MSC infusion ]
    At 6 months 6 Minute Walk Distance was mesured and compared as a percentage to baseline
  • Percentage Change in Lung Function as Assessed by DLCO Compared to Baseline [ Time Frame: 6 months post MSC infusion ]
    DLCO was measured as a percentage of predicted, and the percentage change between 6 months post-infusion and baseline is reported.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 29, 2011)
  • Evidence of improvement in or stabilisation of lung function (FVC, TLC, DLCO). [ Time Frame: Baseline and 6 months post MSC infusion ]
  • Evidence of improvement in or stabilisation of exercise capacity as assessed by 6MWD. [ Time Frame: Baseline and 6 months post MSC infusion ]
  • Evidence of improvement in or stabilisation of gas exchange as assessed by resting PaO2 and pulse oximetry during exercise testing. [ Time Frame: Baseline and 6 months post MSC infusion ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis
Official Title  ICMJE A Phase I Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis
Brief Summary

The primary objective of this study is to establish the feasibility and safety of infusions of placental Mesenchymal Stem Cells (MSC) from related or unrelated HLA identical or HLA mismatched donors in the treatment of Idiopathic Pulmonary Fibrosis (IPF).

The secondary objectives are to document changes in lung function, 6 minute walk distance (6MWD), gas exchange and radiological appearance following infusion of MSC over a six month evaluation period.

Detailed Description This is a Phase I, open-label, single centre, non-randomized dose-escalation evaluation of the safety and feasibility of MSC treatment for subjects diagnosed with IPF. The first 4 patients will receive a dose of 1 x 10^6 placenta-derived MSC/kg. An interim safety analysis will be carried out by the Data Safety Management Board (DSMB) when these first 4 patients have all undergone their 3 month study visit. Should no serious adverse events be documented due, or likely due, to the MSC infusion, a subsequent 4 patients will receive an IV infusion of 2 x 10^6 placenta-derived MSC/kg. Therefore a total of up to eight (8) subjects who meet all eligibility criteria and who provide written informed consent will be enrolled in the study.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Idiopathic Pulmonary Fibrosis
Intervention  ICMJE Other: Placental MSC
MSC will be derived from mothers donating their term placenta for clinical trial research purposes at Mater Mothers Hospital, Brisbane. The donation, isolation and expansion of placental-derived MSC for research purposes has been approved by the Mater Health Services (MHS) Human Research Ethics Committee (Reference No. 1292A). These volunteer donor mothers are unrelated to and will be HLA-unmatched with the IPF recipients.
Study Arms  ICMJE
  • Experimental: 1*10^6 MSC / kg
    Placental MSC
    Intervention: Other: Placental MSC
  • Experimental: 2*10^6 MSC / kg
    Placental MSC
    Intervention: Other: Placental MSC
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 29, 2011)
8
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE May 2013
Actual Primary Completion Date May 2013   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Male or female from 40 to 80 years of age (Note: see exclusion 13 regarding women of child-bearing potential).
  2. Diagnosis of IPF based on the following criteria in accordance with American Thoracic Society/European Respiratory Society (ATS-ERS) guidelines for diagnosing

    IPF:

    Definite or probable usual interstitial pneumonia confirmed on surgical lung biopsy (SLB)

    or

    In absence of SLB, all of the following "major criteria"

    • High resolution CT scan (HRCT) showing definite findings for IPF (bibasilar reticular abnormalities with minimal ground glass opacities)
    • Absence of other causes of IPF including drug toxicities, environmental exposure and connective tissue disease
    • Abnormal pulmonary function tests including evidence of a restrictive ventilatory impairment and impaired gas exchange
    • Transbronchial biopsy or BAL suggesting no features of an alternative diagnosis and three of four of the following "minor criteria"
    • Age greater than 50 years
    • Insidious onset of otherwise unexplained dyspnea on exertion
    • Duration of illness greater than 3 months
    • Bibasal, inspiratory crackles

    Within 90 days of study enrolment, diagnosis must be confirmed by HRCT chest.

  3. Honeycombing greater than 5% in 0 - 3 lung zones (each lung divided into 3 zones - 1) at the level of the carina 2) highest point of right hemi diaphragm and 3) mid way between these two levels) as assessed on HRCT.
  4. Forced vital capacity (FVC) greater than 50 of predicted with a ratio of forced expiratory volume in 1 second to FVC (FEV1/FVC) greater than 0.7 (Pulmonary function tests must be completed no more than 90 days before screening).
  5. Diffusing capacity for carbon monoxide (DLCO) greater than 25% of predicted capacity.
  6. Ability to perform a 6-Minute Walk Test (6MWT) at screening.
  7. Competency to understand the information given in the Human Research and Ethics Committee (HREC) approved ICF and must sign the form prior to the initiation of any study procedures.

Exclusion Criteria:

  1. Diagnosis of an interstitial lung disease (ILD) or restrictive lung disease other than IPF.
  2. Obstructive lung disease as determined by evidence of airflow obstruction on HRCT or physiologic criteria including:

    FEV1/FVC ratio less than 0.7 Residual volume (RV) greater than 120% by plethysmography or significant (verified by radiologist) emphysema on HRCT if plethysmography not available Evidence of reactive airway disease by change in FEV1 of greater than 12% following bronchodilator challenge

  3. Evidence of sustained improvement of IPF condition defined as improvement from pre-therapy pulmonary function tests (PFTs) observed with two or more successive post-therapy PFTs over the year prior to randomization.
  4. Active or recent (less than 60 days prior to enrolment) significant respiratory tract infection, or a history of frequent (greater than 2 per year for the last 2 years) infective exacerbations of IPF.
  5. Hospitalization within 60 days of screening for an acute exacerbation of IPF (AE-IPF).
  6. Chronic heart failure (NYHA class III/IV) or known left ventricular ejection fraction less than 25%.
  7. Chronic treatment with the following drugs prescribed for IPF (within 4 weeks of randomization):

    oral corticosteroids (greater than 20 mg/day of prednisone or equivalent), immunosuppressive or cytotoxic drugs, antifibrotic drugs, chronic use of N-acetylcysteine

  8. Acute or chronic impairment (other than dyspnea) which limits the ability to comply with study requirements and procedures including the 6MWD
  9. Chronic treatment with immunosuppressive, cytotoxic, or antifibrotic drugs including pirfenidone, D-penicillamine, colchicine, cyclosporine A, TNF-alpha antagonists, imatinib, interferon-gamma, cyclophosphamide, or azathioprine within 30 days of randomization.
  10. Subject requires hemodialysis, peritoneal dialysis or hemofiltration.
  11. Systolic blood pressure less than 85 mmHg.
  12. History of malignancies within the past 5 years, with the exception of squamous or basal cell carcinoma of the skin or successfully treated in situ carcinoma of the cervix.
  13. Female who is of child-bearing potential.
  14. Known history of alcohol abuse within 1 year of enrolment.
  15. Participation in a clinical study involving another investigational drug or device within 28 days of screening.
  16. Co-morbid condition or illness limiting life expectancy to less than 1 year at time of screening.
  17. Serious or active medical or psychiatric illness which, in the opinion of the Investigator, would interfere with treatment, assessment or compliance with the protocol.
  18. Significant hypoxemia or hypercapnia at rest on room air as defined by a PaO2 less than 55mmHg or PaCO2 greater than 50mmHg.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 40 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01385644
Other Study ID Numbers  ICMJE HREC/09/QPCH/105
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Daniel Chambers, The Prince Charles Hospital
Study Sponsor  ICMJE The Prince Charles Hospital
Collaborators  ICMJE Mater Medical Research Institute
Investigators  ICMJE
Principal Investigator: Daniel Chambers, MBBS MRCP FRACP MD The Prince Charles Hospital
PRS Account The Prince Charles Hospital
Verification Date November 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP