Idiopathic Pulmonary FibrosisPhase 1MSC in IPFNCT01385644
A Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis
A Phase 1 study testing Placental MSC in people with Idiopathic Pulmonary Fibrosis, run by The Prince Charles Hospital at 1 site in Australia.
Trial 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). Read more about this study
- Who is it for?
- Ages 40 to 80 years
- Conditions the study is for
- Idiopathic Pulmonary Fibrosis
- What is being tested?
- Placental MSC
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 1 siteAustralia
- Study dates
- Oct 2010to May 2013
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 40 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female from 40 to 80 years of age (Note: see exclusion 13 regarding women of child-bearing potential).
- 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"
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- 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.
- 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.
- 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).
- Diffusing capacity for carbon monoxide (DLCO) greater than 25% of predicted capacity.
- Ability to perform a 6-Minute Walk Test (6MWT) at screening.
- 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.
Who cannot join
- Diagnosis of an interstitial lung disease (ILD) or restrictive lung disease other than IPF.
- 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
- 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.
- 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.
- Hospitalization within 60 days of screening for an acute exacerbation of IPF (AE-IPF).
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- Chronic heart failure (NYHA class III/IV) or known left ventricular ejection fraction less than 25%.
- 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
- Acute or chronic impairment (other than dyspnea) which limits the ability to comply with study requirements and procedures including the 6MWD
- 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.
- Subject requires hemodialysis, peritoneal dialysis or hemofiltration.
- Systolic blood pressure less than 85 mmHg.
- 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.
- Female who is of child-bearing potential.
- Known history of alcohol abuse within 1 year of enrolment.
- Participation in a clinical study involving another investigational drug or device within 28 days of screening.
- Co-morbid condition or illness limiting life expectancy to less than 1 year at time of screening.
- Serious or active medical or psychiatric illness which, in the opinion of the Investigator, would interfere with treatment, assessment or compliance with the protocol.
- Significant hypoxemia or hypercapnia at rest on room air as defined by a PaO2 less than 55mmHg or PaCO2 greater than 50mmHg.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis
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.
Read the 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.
- Conditions
- Idiopathic Pulmonary Fibrosis
- Being tested
- Placental MSC
- Keywords
- Mesenchymal stem cells, Idiopathic pulmonary fibrosis
- Medical subject headings
- Idiopathic Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placental MSCOther
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 group | Role | What they receive |
|---|---|---|
| 1*10^6 MSC / kg | Experimental | Placental MSC Receives: Other: Placental MSC |
| 2*10^6 MSC / kg | Experimental | Placental MSC Receives: Other: Placental MSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Demonstrated Acute Adverse Events Following 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.
Measured: 4 hours post-infusion
Secondary outcomes (3)
Percentage Change in Lung Function as Assessed by FVC Compared to Baseline
Forced Vital Capacity (FVC) was measured and reported as a percentage of predicted and comapred from 6 months post-infusion to baseline
Measured: 6 months post MSC infusion
Percentage Change in 6 Minute Walk Distance Compared to Baseline
At 6 months 6 Minute Walk Distance was mesured and compared as a percentage to baseline
Measured: Baseline and 6 months post MSC infusion
Percentage Change in Lung Function as Assessed by DLCO Compared to Baseline
DLCO was measured as a percentage of predicted, and the percentage change between 6 months post-infusion and baseline is reported.
Measured: 6 months post MSC infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jun 2011 (estimated)
- Study startOct 2010
- Primary completionMay 2013
- Study completionMay 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Idiopathic Pulmonary FibrosisStudies 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Prince Charles Hospital
- Sponsor type
- Other government
- Collaborators
- Mater Medical Research Institute
- Responsible party
- Principal investigator, Daniel Chambers, The Prince Charles Hospital
- Investigators
- Daniel Chambers, MBBS MRCP FRACP MD, Principal investigator, The Prince Charles Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01385644
- Sponsor's protocol ID
- HREC/09/QPCH/105
- Acronym
- MSC in IPF
- Status last verified
- Nov 2015
- First submitted
- 4 May 2011
- Last updated
- 29 Dec 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis
- ClinicalTrials.gov ID
- NCT01385644
- Acronym / sponsor ID
- MSC in IPF / HREC/09/QPCH/105
- Conditions
- Idiopathic Pulmonary Fibrosis
- Keywords
- Mesenchymal stem cells, Idiopathic pulmonary fibrosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- The Prince Charles HospitalOther government
- Collaborators
- Mater Medical Research Institute
- Study start
- Oct 2010
- Primary completion
- May 2013 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 30 Jun 2011 (estimated)
- Results
- Posted 29 Dec 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1*10^6 MSC / kg | Experimental | Other: Placental MSCPlacental MSC |
| 2*10^6 MSC / kg | Experimental | Other: Placental MSCPlacental MSC |
Who may qualify
- Ages
- 40 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Male or female from 40 to 80 years of age (Note: see exclusion 13 regarding women of child-bearing potential).
- Diagnosis of IPF based on the following criteria in accordance with American Thoracic Society/European Respiratory Society (ATS-ERS) guidelines for diagnosing
- Definite or probable usual interstitial pneumonia confirmed on surgical lung biopsy (SLB)
- or
- In absence of SLB, all of the following "major criteria"
Key exclusion criteria (20 listed)
- Diagnosis of an interstitial lung disease (ILD) or restrictive lung disease other than IPF.
- 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
- 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.
- 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.
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 |
|---|---|---|
| Placental MSCMSC 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. | Other | 1*10^6 MSC / kg, 2*10^6 MSC / kg |
Primary outcome
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.
3 secondary outcomes
Percentage Change in Lung Function as Assessed by FVC Compared to Baseline
Time frame: 6 months post MSC infusion
Percentage Change in 6 Minute Walk Distance Compared to Baseline
Time frame: Baseline and 6 months post MSC infusion
Percentage Change in Lung Function as Assessed by DLCO Compared to Baseline
Time frame: 6 months post MSC infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Daniel Chambers, MBBS MRCP FRACP MD principal investigator
The Prince Charles Hospital
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 Placental MSC in people with Idiopathic Pulmonary Fibrosis, run by The Prince Charles Hospital at 1 site in Australia.
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 20 inclusion and 20 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: finished, as of the sponsor's last update on 29 Dec 2015. 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 Australia. 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?
Yes. Results are posted in the registry (first posted 29 Dec 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Participants Who Demonstrated Acute Adverse Events Following Infusion (time frame: 4 hours post-infusion). 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 Dec 2015.