Trial Outcomes & Findings for A Study to Evaluate the Potential Role of Mesenchymal Stem Cells in the Treatment of Idiopathic Pulmonary Fibrosis (NCT NCT01385644)

Results Overview

Acute adverse events following infusion was defined as the development of anaphalaxis and/or a 25% increase or decrease from baseline of hemodynamic measurements.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

4 hours post-infusion

Results posted on

2015-12-29

Participant Flow

All Participants known to doctors through standard of care appointments in clinic.

All patients meet all inclusion criteria and none of the exclusion criteria.

Participant milestones

Participant milestones
Measure	1*10^6 MSC / kg Placental MSC 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.	2*10^6 MSC / kg Placental MSC 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.
Overall Study STARTED	4	4
Overall Study COMPLETED	4	4
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	1*10^6 MSC / kg n=4 Participants Placental MSC 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.	2*10^6 MSC / kg n=4 Participants Placental MSC 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.	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Age, Continuous	64.1 years n=5 Participants	66.2 years n=7 Participants	64.1 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Region of Enrollment Australia	4 participants n=5 Participants	4 participants n=7 Participants	8 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 hours post-infusion

Population: Data from 8 participants was analyzed. (4 from each group)

Acute adverse events following infusion was defined as the development of anaphalaxis and/or a 25% increase or decrease from baseline of hemodynamic measurements.

Outcome measures

Outcome measures
Measure	1*10^6 MSC / kg n=4 Participants Placental MSC 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.	2*10^6 MSC / kg n=4 Participants Placental MSC 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.
Number of Participants Who Demonstrated Acute Adverse Events Following Infusion	0 participants	0 participants

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	1*10^6 MSC / kg n=4 Participants Placental MSC 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.	2*10^6 MSC / kg n=4 Participants Placental MSC 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.
Percentage Change in Lung Function as Assessed by FVC Compared to Baseline	99 percentage of baseline Interval 92.0 to 104.0	94 percentage of baseline Interval 90.0 to 97.0

SECONDARY outcome

Timeframe: Baseline and 6 months post MSC infusion

At 6 months 6 Minute Walk Distance was mesured and compared as a percentage to baseline

Outcome measures

Outcome measures
Measure	1*10^6 MSC / kg n=4 Participants Placental MSC 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.	2*10^6 MSC / kg n=4 Participants Placental MSC 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.
Percentage Change in 6 Minute Walk Distance Compared to Baseline	104 percentage of baseline Interval 103.0 to 114.0	104 percentage of baseline Interval 90.0 to 120.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	1*10^6 MSC / kg n=4 Participants Placental MSC 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.	2*10^6 MSC / kg n=4 Participants Placental MSC 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.
Percentage Change in Lung Function as Assessed by DLCO Compared to Baseline	117 percentage of baseline Interval 96.0 to 138.0	86 percentage of baseline Interval 81.0 to 96.0

Adverse Events

1*10^6 MSC / kg

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

2*10^6 MSC / kg

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	1*10^6 MSC / kg n=4 participants at risk Placental MSC 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.	2*10^6 MSC / kg n=4 participants at risk Placental MSC 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.
Gastrointestinal disorders Small Bowel Obstruction	25.0% 1/4 • Number of events 1 • Adverse Events were collected for the duration of the study participation time which was 12 months.	0.00% 0/4 • Adverse Events were collected for the duration of the study participation time which was 12 months.

Other adverse events

Other adverse events
Measure	1*10^6 MSC / kg n=4 participants at risk Placental MSC 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.	2*10^6 MSC / kg n=4 participants at risk Placental MSC 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.
Respiratory, thoracic and mediastinal disorders Halitosis	100.0% 4/4 • Number of events 4 • Adverse Events were collected for the duration of the study participation time which was 12 months.	100.0% 4/4 • Number of events 4 • Adverse Events were collected for the duration of the study participation time which was 12 months.

Additional Information

Assoc Professor Daniel Chambers

The Prince Chalres Hospital

Phone: +61 7 3139 4000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place