Trial Outcomes & Findings for Safety and Tolerability Study of Allogeneic Mesenchymal Stem Cell Infusion in Adults With Cystic Fibrosis (NCT NCT02866721)

NCT ID: NCT02866721

Last Updated: 2023-01-30

Results Overview

For this study, dose limiting toxicities included the emergence of infusion-related allergic adverse events as well as regimen related toxicities in the first 24 hours after hMSC infusion of a grade ≥ 3 as scored according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. This is the number of participants who experienced a dose limiting toxicity during the study.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 14 participants
• Primary outcome timeframe: 24 hours post infusion
• Results posted on: 2023-01-30

Participant Flow

Participant milestones

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenous infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells: A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the mesenchymal stem cells (MSCs) were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis (CF) lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenous infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells: A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the mesenchymal stem cells (MSCs) were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis (CF) lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenous infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells: A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the mesenchymal stem cells (MSCs) were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis (CF) lung infection and inflammation.
Overall Study STARTED	3	3	8
Overall Study COMPLETED	3	3	8
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Tolerability Study of Allogeneic Mesenchymal Stem Cell Infusion in Adults With Cystic Fibrosis

Baseline characteristics by cohort

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenous Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis (CF) lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenous Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis (CF) lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenous Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis (CF) lung infection and inflammation.	Total n=14 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	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants	14 Participants n=4 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Continuous	28.3 years STANDARD_DEVIATION 10.7 • n=5 Participants	28.0 years STANDARD_DEVIATION 4.7 • n=7 Participants	31.7 years STANDARD_DEVIATION 12.7 • n=5 Participants	30.2 years STANDARD_DEVIATION 10.6 • n=4 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	4 Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants	10 Participants n=4 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants	2 Participants n=7 Participants	8 Participants n=5 Participants	13 Participants n=4 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	1 Participants n=7 Participants	0 Participants n=5 Participants	1 Participants n=4 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Region of Enrollment United States	3 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants	14 Participants n=4 Participants
Genotype F508del Homozygous	1 Participants n=5 Participants	0 Participants n=7 Participants	3 Participants n=5 Participants	4 Participants n=4 Participants
Genotype F508del Heterozygous	1 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants	7 Participants n=4 Participants
Genotype Other	1 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants
Forced Expiratory Volume in the first second (FEV1) % Predicted Distribution <70%	0 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	5 Participants n=4 Participants
Forced Expiratory Volume in the first second (FEV1) % Predicted Distribution >=70-84%	1 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	3 Participants n=4 Participants
Forced Expiratory Volume in the first second (FEV1) % Predicted Distribution >=85%	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants	6 Participants n=4 Participants
Sweat Chloride at Diagnosis	107.0 mmol/L STANDARD_DEVIATION 7.5 • n=5 Participants	82.7 mmol/L STANDARD_DEVIATION 26.6 • n=7 Participants	99.4 mmol/L STANDARD_DEVIATION 20.8 • n=5 Participants	97.3 mmol/L STANDARD_DEVIATION 20.6 • n=4 Participants
Height	167.8 cm STANDARD_DEVIATION 1.6 • n=5 Participants	169.6 cm STANDARD_DEVIATION 18.7 • n=7 Participants	173.6 cm STANDARD_DEVIATION 8.6 • n=5 Participants	171.5 cm STANDARD_DEVIATION 10.0 • n=4 Participants
Weight	60.1 kg STANDARD_DEVIATION 13.8 • n=5 Participants	63.2 kg STANDARD_DEVIATION 7.4 • n=7 Participants	73.0 kg STANDARD_DEVIATION 14.1 • n=5 Participants	68.2 kg STANDARD_DEVIATION 13.4 • n=4 Participants
Body Mass Index (BMI)	21.3 kg/m^2 STANDARD_DEVIATION 4.7 • n=5 Participants	22.2 kg/m^2 STANDARD_DEVIATION 3.3 • n=7 Participants	24.3 kg/m^2 STANDARD_DEVIATION 4.9 • n=5 Participants	23.2 kg/m^2 STANDARD_DEVIATION 4.4 • n=4 Participants

PRIMARY outcome

Timeframe: 24 hours post infusion

Population: Number of participants with a dose limiting toxicity. All subjects receiving the human mesenchymal stem cell infusing were included.

For this study, dose limiting toxicities included the emergence of infusion-related allergic adverse events as well as regimen related toxicities in the first 24 hours after hMSC infusion of a grade ≥ 3 as scored according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. This is the number of participants who experienced a dose limiting toxicity during the study.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Number of Participants With a Dose Limiting Toxicity (DLT), Triggered by Occurrence in the First 24 Hours After Human Mesenchymal Stem Cell (hMSC) Infusion of Grade ≥3 Infusion-related Allergic Toxicities	0 Participants	0 Participants	0 Participants

PRIMARY outcome

Timeframe: 1 year

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Participants were followed for 12 months after human mesenchymal stem cell infusion. All events from the infusion date through the end of follow-up were included. This shows the total number of adverse events or serious adverse events occurring in each dosing cohort.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Number of Serious Adverse Events and Number of Non-Serious Adverse Events Serious Adverse Event	3 number of adverse events	3 number of adverse events	3 number of adverse events
Number of Serious Adverse Events and Number of Non-Serious Adverse Events Non-Serious Adverse Event	16 number of adverse events	31 number of adverse events	68 number of adverse events

PRIMARY outcome

Timeframe: 1 year

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Participants were followed for 12 months after human mesenchymal stem cell infusion. All events occurring from the infusion date through the end of follow-up were included. This shows the number of pulmonary exacerbations requiring intravenous antibiotics in each dosing cohort.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Number of Pulmonary Exacerbations Requiring Intravenous Antibiotics	2 pulmonary exacerbations	3 pulmonary exacerbations	2 pulmonary exacerbations

PRIMARY outcome

Timeframe: Baseline and 30 minutes, 4 hours, 24 hours, 7 days, 14 days, 28 days, 3 months, 6 months Post Human Mesenchymal Stem Cell Infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Lung function was followed for 6 months post human mesenchymal stem cell infusion. This shows the mean forced expiratory volume in the first second (FEV1) percent predicted for each cohort throughout the study.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 at baseline	83.2 FEV1 percent predicted Standard Deviation 8.5	71.4 FEV1 percent predicted Standard Deviation 13.5	77.7 FEV1 percent predicted Standard Deviation 20.2
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 30 minutes post infusion	79.7 FEV1 percent predicted Standard Deviation 9.9	69.0 FEV1 percent predicted Standard Deviation 9.6	75.3 FEV1 percent predicted Standard Deviation 20.3
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 4 hours post infusion	83.1 FEV1 percent predicted Standard Deviation 14.3	69.0 FEV1 percent predicted Standard Deviation 14.2	75.2 FEV1 percent predicted Standard Deviation 21.1
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 24 hours post infusion	86.4 FEV1 percent predicted Standard Deviation 15.6	75.1 FEV1 percent predicted Standard Deviation 13.7	77.4 FEV1 percent predicted Standard Deviation 21.2
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 7 days post infusion	83.6 FEV1 percent predicted Standard Deviation 4.3	64.5 FEV1 percent predicted Standard Deviation 9.8	76.5 FEV1 percent predicted Standard Deviation 18.7
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 14 days post infusion	85.6 FEV1 percent predicted Standard Deviation 8.0	67.5 FEV1 percent predicted Standard Deviation 8.4	75.6 FEV1 percent predicted Standard Deviation 19.9
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 28 days post infusion	85.4 FEV1 percent predicted Standard Deviation 11.4	69.8 FEV1 percent predicted Standard Deviation 12.0	77.8 FEV1 percent predicted Standard Deviation 19.7
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 3 months post infusion	89.4 FEV1 percent predicted Standard Deviation 17.2	70.9 FEV1 percent predicted Standard Deviation 14.2	76.1 FEV1 percent predicted Standard Deviation 19.5
Forced Expiratory Volume in the First Second (FEV1) % Predicted at Baseline and 30 Minutes, 4 Hours, 24 Hours, 7 Days, 14 Days, 28 Days, 3 Months, 6 Months Post Human Mesenchymal Stem Cell Infusion. FEV1 6 months post infusion	91.3 FEV1 percent predicted Standard Deviation 18.7	70.0 FEV1 percent predicted Standard Deviation 11.1	76.1 FEV1 percent predicted Standard Deviation 20.7

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including Calprotectin were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit for detection for calportectin was 88.3 picogram per millilitre.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Calportectin Measurements at Baseline, Day 7, and Day 28 Baseline	9735.2 pg/ml Standard Deviation 16708.9	88.3 pg/ml Standard Deviation 0	93.1 pg/ml Standard Deviation 5.1
Serum Inflammatory Markers - Calportectin Measurements at Baseline, Day 7, and Day 28 Day 7	88.3 pg/ml Standard Deviation 0	94.2 pg/ml Standard Deviation 10.2	93.1 pg/ml Standard Deviation 5.1
Serum Inflammatory Markers - Calportectin Measurements at Baseline, Day 7, and Day 28 Day 28	88.3 pg/ml Standard Deviation 0	88.3 pg/ml Standard Deviation 0	91.1 pg/ml Standard Deviation 5.1

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit for detection for GM-CSF was 2.6 picograms per milliliter.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Measurements at Baseline, Day 7, and Day 28 Baseline	3.3 pg/ml Standard Deviation 1.3	3.5 pg/ml Standard Deviation 1.2	7.1 pg/ml Standard Deviation 3.1
Serum Inflammatory Markers - Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Measurements at Baseline, Day 7, and Day 28 Day 7	5.2 pg/ml Standard Deviation 1.5	4.3 pg/ml Standard Deviation 0.1	6.2 pg/ml Standard Deviation 3.7
Serum Inflammatory Markers - Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Measurements at Baseline, Day 7, and Day 28 Day 28	4.6 pg/ml Standard Deviation 2.4	2.6 pg/ml Standard Deviation 0	6.7 pg/ml Standard Deviation 3.3

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including interleukin -1 (IL-1) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit for detection for IL-1 was 13.1 picograms per milliliter.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Interleukin-1 (IL-1) Measurements at Baseline, Day 7, and Day 28 Baseline	15.9 pg/ml Standard Deviation 4.8	13.1 pg/ml Standard Deviation 0	13.1 pg/ml Standard Deviation 0
Serum Inflammatory Markers - Interleukin-1 (IL-1) Measurements at Baseline, Day 7, and Day 28 Day 7	13.1 pg/ml Standard Deviation 0	13.1 pg/ml Standard Deviation 0	13.1 pg/ml Standard Deviation 0
Serum Inflammatory Markers - Interleukin-1 (IL-1) Measurements at Baseline, Day 7, and Day 28 Day 28	13.1 pg/ml Standard Deviation 0	13.1 pg/ml Standard Deviation 0	13.1 pg/ml Standard Deviation 0

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including interleukin -17 (IL-17) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit for detection for IL-17 was 13.5 picograms per milliliter.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Interleukin-17 (IL-17) Measurements at Baseline, Day 7, and Day 28 Baseline	13.5 pg/ml Standard Deviation 0	13.8 pg/ml Standard Deviation 0.6	18.0 pg/ml Standard Deviation 26.7
Serum Inflammatory Markers - Interleukin-17 (IL-17) Measurements at Baseline, Day 7, and Day 28 Day 7	20.7 pg/ml Standard Deviation 12.6	15.7 pg/ml Standard Deviation 3.8	18.0 pg/ml Standard Deviation 26.7
Serum Inflammatory Markers - Interleukin-17 (IL-17) Measurements at Baseline, Day 7, and Day 28 Day 28	22.0 pg/ml Standard Deviation 11.5	14.9 pg/ml Standard Deviation 2.5	17.3 pg/ml Standard Deviation 24.5

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including interleukin -6 (IL-6) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit for detection for IL-6 was 13.5 picograms per milliliter.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Interleukin-6 (IL-6) Measurements at Baseline, Day 7, and Day 28 Baseline	4.1 pg/ml Standard Deviation 1.6	4.6 pg/ml Standard Deviation 2.7	4.8 pg/ml Standard Deviation 5.4
Serum Inflammatory Markers - Interleukin-6 (IL-6) Measurements at Baseline, Day 7, and Day 28 Day 7	2.6 pg/ml Standard Deviation 0.3	5.7 pg/ml Standard Deviation 5.9	4.6 pg/ml Standard Deviation 4.7
Serum Inflammatory Markers - Interleukin-6 (IL-6) Measurements at Baseline, Day 7, and Day 28 Day 28	3.1 pg/ml Standard Deviation 0.8	3.7 pg/ml Standard Deviation 2.0	5.4 pg/ml Standard Deviation 5.5

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including interleukin -8 (IL-8) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit for detection for IL-8 was 13.9 picograms per milliliter.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Interleukin-8 (IL-8) Measurements at Baseline, Day 7, and Day 28 Baseline	2225.6 pg/ml Standard Deviation 3824.4	22.9 pg/ml Standard Deviation 15.5	24.9 pg/ml Standard Deviation 26.7
Serum Inflammatory Markers - Interleukin-8 (IL-8) Measurements at Baseline, Day 7, and Day 28 Day 7	21.5 pg/ml Standard Deviation 6.0	17.0 pg/ml Standard Deviation 5.4	32.7 pg/ml Standard Deviation 35.5
Serum Inflammatory Markers - Interleukin-8 (IL-8) Measurements at Baseline, Day 7, and Day 28 Day 28	22.9 pg/ml Standard Deviation 7.9	18.7 pg/ml Standard Deviation 8.3	21.8 pg/ml Standard Deviation 13.6

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including myeloperoxidase (MPO) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Myeloperoxidase (MPO) Measurements at Baseline, Day 7, and Day 28 Baseline	74168.3 pg/ml Standard Deviation 40518.7	245158.5 pg/ml Standard Deviation 88431.6	245928.3 pg/ml Standard Deviation 122070.0
Serum Inflammatory Markers - Myeloperoxidase (MPO) Measurements at Baseline, Day 7, and Day 28 Day 7	122043.5 pg/ml Standard Deviation 58466.6	305665.3 pg/ml Standard Deviation 85217.9	225988.7 pg/ml Standard Deviation 128113.3
Serum Inflammatory Markers - Myeloperoxidase (MPO) Measurements at Baseline, Day 7, and Day 28 Day 28	93838.6 pg/ml Standard Deviation 26655.9	250501.1 pg/ml Standard Deviation 205136.2	226155.5 pg/ml Standard Deviation 142171.8

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: All subjects who received human mesenchymal stem cells were included in the analysis.

Serum inflammatory markers including tumor necrosis factor alpha (TNF-a) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with standard deviation are shown. The lower limit of detection for TNF-a was 12.9 picograms per millilter.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Serum Inflammatory Markers - Tumor Necrosis Factor Alpha (TNF-a) Measurements at Baseline, Day 7, and Day 28 Baseline	12.9 pg/ml Standard Deviation 0	12.9 pg/ml Standard Deviation 0	19.5 pg/ml Standard Deviation 6.6
Serum Inflammatory Markers - Tumor Necrosis Factor Alpha (TNF-a) Measurements at Baseline, Day 7, and Day 28 Day 7	12.9 pg/ml Standard Deviation 0	14.2 pg/ml Standard Deviation 2.3	19.2 pg/ml Standard Deviation 6.8
Serum Inflammatory Markers - Tumor Necrosis Factor Alpha (TNF-a) Measurements at Baseline, Day 7, and Day 28 Day 28	12.9 pg/ml Standard Deviation 0	12.9 pg/ml Standard Deviation 0	19.2 pg/ml Standard Deviation 6.8

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including granulocyte-macrophage colony-stimulating factor (GM-CSF) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Measurements at Baseline, Day 7, and Day 28 Baseline	—	1.796 pg/ml Interval 1.753 to 1.839	1.653 pg/ml Interval 1.209 to 2.097
Sputum Inflammatory Markers - Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Measurements at Baseline, Day 7, and Day 28 Day 7	—	2.755 pg/ml Interval 1.624 to 3.885	1.481 pg/ml Interval 1.209 to 1.753
Sputum Inflammatory Markers - Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF) Measurements at Baseline, Day 7, and Day 28 Day 28	—	2.971 pg/ml Interval 2.488 to 3.453	38.799 pg/ml Interval 1.209 to 76.388

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including interleukin-1 (IL-1) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Interleukin-1 (IL-1) Measurements at Baseline, Day 7, and Day 28 Baseline	—	1267.24 pg/ml Interval 1053.1 to 1481.37	907.90 pg/ml Interval 529.62 to 1286.18
Sputum Inflammatory Markers - Interleukin-1 (IL-1) Measurements at Baseline, Day 7, and Day 28 Day 7	—	916.96 pg/ml Interval 439.02 to 1394.9	2432.30 pg/ml Interval 753.93 to 4110.67
Sputum Inflammatory Markers - Interleukin-1 (IL-1) Measurements at Baseline, Day 7, and Day 28 Day 28	—	1137.81 pg/ml Interval 1109.64 to 1165.98	2501.65 pg/ml Interval 2153.39 to 2849.91

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including interleukin-10 (IL-10) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Interleukin-10 (IL-10) Measurements at Baseline, Day 7, and Day 28 Baseline	—	1.614 pg/ml Interval 1.614 to 1.614	0.979 pg/ml Interval 0.724 to 1.233
Sputum Inflammatory Markers - Interleukin-10 (IL-10) Measurements at Baseline, Day 7, and Day 28 Day 7	—	1.614 pg/ml Interval 1.614 to 1.614	1.424 pg/ml Interval 1.233 to 1.614
Sputum Inflammatory Markers - Interleukin-10 (IL-10) Measurements at Baseline, Day 7, and Day 28 Day 28	—	1.614 pg/ml Interval 1.614 to 1.614	1.233 pg/ml Interval 1.233 to 1.233

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including interleukin-17 (IL-17) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Interleukin-17 (IL-17) Measurements at Baseline, Day 7, and Day 28 Baseline	—	1.076 pg/ml Interval 1.019 to 1.132	0.752 pg/ml Interval 0.643 to 0.861
Sputum Inflammatory Markers - Interleukin-17 (IL-17) Measurements at Baseline, Day 7, and Day 28 Day 7	—	1.144 pg/ml Interval 0.961 to 1.427	0.480 pg/ml Interval 0.318 to 0.643
Sputum Inflammatory Markers - Interleukin-17 (IL-17) Measurements at Baseline, Day 7, and Day 28 Day 28	—	1.167 pg/ml Interval 0.906 to 1.427	0.643 pg/ml Interval 0.643 to 0.643

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including interleukin-6 (IL-6) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Interleukin-6 (IL-6) Measurements at Baseline, Day 7, and Day 28 Baseline	—	4.908 pg/ml Interval 4.837 to 4.979	4.253 pg/ml Interval 4.072 to 4.434
Sputum Inflammatory Markers - Interleukin-6 (IL-6) Measurements at Baseline, Day 7, and Day 28 Day 7	—	4.188 pg/ml Interval 3.448 to 4.927	4.501 pg/ml Interval 1.64 to 7.362
Sputum Inflammatory Markers - Interleukin-6 (IL-6) Measurements at Baseline, Day 7, and Day 28 Day 28	—	6.733 pg/ml Interval 4.396 to 9.069	4.118 pg/ml Interval 1.64 to 6.596

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including interleukin-8 (IL-8) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Interleukin-8 (IL-8) Measurements at Baseline, Day 7, and Day 28 Baseline	—	1814.44 pg/ml Interval 1508.23 to 2120.64	7862.76 pg/ml Interval 3412.01 to 12313.5
Sputum Inflammatory Markers - Interleukin-8 (IL-8) Measurements at Baseline, Day 7, and Day 28 Day 7	—	2398.03 pg/ml Interval 2292.14 to 2503.91	5061.17 pg/ml Interval 2592.75 to 7529.59
Sputum Inflammatory Markers - Interleukin-8 (IL-8) Measurements at Baseline, Day 7, and Day 28 Day 28	—	1538.79 pg/ml Interval 1397.77 to 1679.82	9348.03 pg/ml Interval 8361.68 to 10334.38

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including macrophage inflammatory protein-3 alpha (MIP-3a) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Macrophage Inflammatory Protein-3 Alpha (MIP-3a) Measurements at Baseline, Day 7, and Day 28 Baseline	—	18.01 pg/ml Interval 7.78 to 28.23	7.78 pg/ml Interval 7.78 to 7.78
Sputum Inflammatory Markers - Macrophage Inflammatory Protein-3 Alpha (MIP-3a) Measurements at Baseline, Day 7, and Day 28 Day 7	—	16.06 pg/ml Interval 7.78 to 24.34	14.426 pg/ml Interval 7.78 to 21.07
Sputum Inflammatory Markers - Macrophage Inflammatory Protein-3 Alpha (MIP-3a) Measurements at Baseline, Day 7, and Day 28 Day 28	—	17.03 pg/ml Interval 7.78 to 26.28	7.83 pg/ml Interval 7.78 to 7.87

SECONDARY outcome

Timeframe: Baseline, Day 7, Day 28 post human mesenchymal stem cell infusion

Population: Only 4 subjects were able to produce sputum at all time points.

Sputum inflammatory markers including tumor necrosis factor alpha (TNF-a) were collected at baseline and at the visits 7 days and 28 days post human mesenchymal stem cell (hMSC) infusion. Mean values with range are shown.

Outcome measures

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=2 Participants One time intravenouos Infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Sputum Inflammatory Markers - Tumor Necrosis Factor Alpha (TNF-a) Measurements at Baseline, Day 7, and Day 28 Baseline	—	216.572 pg/ml Interval 12.591 to 420.553	14.606 pg/ml Interval 13.693 to 15.518
Sputum Inflammatory Markers - Tumor Necrosis Factor Alpha (TNF-a) Measurements at Baseline, Day 7, and Day 28 Day 7	—	45.069 pg/ml Interval 15.892 to 74.245	25.567 pg/ml Interval 10.2 to 40.933
Sputum Inflammatory Markers - Tumor Necrosis Factor Alpha (TNF-a) Measurements at Baseline, Day 7, and Day 28 Day 28	—	26.858 pg/ml Interval 11.052 to 42.664	21.861 pg/ml Interval 7.225 to 36.497

Adverse Events

1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram

Serious events: 2 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 participants at risk One time intravenouos infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 participants at risk One time intravenouos infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 participants at risk One time intravenouos infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Respiratory, thoracic and mediastinal disorders Cystic Fibrosis Pulmonary Exacerbation	33.3% 1/3 • Number of events 2 • 1 year	66.7% 2/3 • Number of events 3 • 1 year	25.0% 2/8 • Number of events 2 • 1 year
Gastrointestinal disorders Constipation	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Vascular disorders Right Common Iliac Thrombus	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year

Other adverse events

Measure	1 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 participants at risk One time intravenouos infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	3 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=3 participants at risk One time intravenouos infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.	5 x 10^6 Human Allogeneic Mesenchymal Stem Cells Per Kilogram n=8 participants at risk One time intravenouos infusion of up to 5 x 10\^6 allogeneic human mesenchymal stem cells per kilogram body weight (hMSCs/kg). A dose escalation using the "3+3" design was employed. The three doses were 1 x 10\^6, 3 x 10\^6, and 5 x 10\^6 hMSCs/kg. There was no placebo group. All study participants received stem cells. Mesenchymal Stem Cells (MSCs): A single dose, one time infusion (in the vein) of one of the following doses of hMSCs: 1 x 10\^6, 3 x 10\^6 or 5 x 10\^6 hMSCs/kg body weight during Visit 2. A traditional 3+3 design was utilized. Allogeneic MSCs were derived from bone marrow aspirates from a healthy donor whose serum tested negative for cytomegalovirus (CMV) antibodies. The healthy donor underwent tests for infectious disease and screening for 41 common cystic fibrosis transmembrane conductance regulator (CFTR) mutations. In addition, the MSCs were validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of cystic fibrosis lung infection and inflammation.
Vascular disorders Right leg pain	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Gastrointestinal disorders Vomiting	33.3% 1/3 • Number of events 1 • 1 year	33.3% 1/3 • Number of events 2 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Gastrointestinal disorders Abdominal Pain	33.3% 1/3 • Number of events 1 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Gastrointestinal disorders Nausea	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 2 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Eye disorders Constipation	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Gastrointestinal disorders Diarrhea	33.3% 1/3 • Number of events 1 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Gastrointestinal disorders Hemorrhoids	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 2 • 1 year
Gastrointestinal disorders Change in Appetite	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Gastrointestinal disorders Bleeding Hemorrhoids	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Gastrointestinal disorders Slow Weight Gain	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
General disorders Fatigue	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	25.0% 2/8 • Number of events 2 • 1 year
General disorders Non-cardiac Chest Pain	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
General disorders Risk of Pathogen Exposure	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Immune system disorders Febrile Viral Illness	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Infections and infestations Upper Respiratory Infection	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	25.0% 2/8 • Number of events 2 • 1 year
Infections and infestations Influenza	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Infections and infestations Urinary Tract Infection	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Injury, poisoning and procedural complications Fracture	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Gastrointestinal disorders Weight Loss	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Metabolism and nutrition disorders Hypomagnesemia	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Musculoskeletal and connective tissue disorders Myalgia	33.3% 1/3 • Number of events 1 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Musculoskeletal and connective tissue disorders Bilateral Lower Extremity Pain	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Musculoskeletal and connective tissue disorders Body Aches	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Musculoskeletal and connective tissue disorders Generalized Pain	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Musculoskeletal and connective tissue disorders Right Heel Pain	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Nervous system disorders Headahche	0.00% 0/3 • 1 year	66.7% 2/3 • Number of events 2 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Nervous system disorders Aphasia	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Nervous system disorders Dizziness	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Nervous system disorders Lethargy	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Nervous system disorders Memory Impairment	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Nervous system disorders Syncope	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year
Psychiatric disorders Anxiety	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Psychiatric disorders Confusion	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Psychiatric disorders Depression	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Psychiatric disorders Suicidal Ideation	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Renal and urinary disorders Cystoscopy	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Renal and urinary disorders Hematuria	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Renal and urinary disorders Increased Creatinine	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Renal and urinary disorders Ureter Stricture	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Renal and urinary disorders Urinary Hesitancy	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Renal and urinary disorders Urinary Retention	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Renal and urinary disorders Urology Procedure	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Reproductive system and breast disorders Breast Swelling	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Respiratory, thoracic and mediastinal disorders Increased Sputum Production	33.3% 1/3 • Number of events 1 • 1 year	66.7% 2/3 • Number of events 2 • 1 year	37.5% 3/8 • Number of events 3 • 1 year
Respiratory, thoracic and mediastinal disorders Cystic Fibrosis Pulmonary Exacerbation	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 3 • 1 year
Respiratory, thoracic and mediastinal disorders Cough	33.3% 1/3 • Number of events 2 • 1 year	66.7% 2/3 • Number of events 5 • 1 year	37.5% 3/8 • Number of events 4 • 1 year
Respiratory, thoracic and mediastinal disorders Decreased FEV1	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	25.0% 2/8 • Number of events 3 • 1 year
Respiratory, thoracic and mediastinal disorders Nasal Congestion	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	50.0% 4/8 • Number of events 5 • 1 year
Respiratory, thoracic and mediastinal disorders Sore Throat	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	25.0% 2/8 • Number of events 4 • 1 year
Respiratory, thoracic and mediastinal disorders Hemoptysis	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	12.5% 1/8 • Number of events 2 • 1 year
Respiratory, thoracic and mediastinal disorders Sinus Congestion	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	25.0% 2/8 • Number of events 4 • 1 year
Respiratory, thoracic and mediastinal disorders Shortness of Breath	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Skin and subcutaneous tissue disorders Cold Sore	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Skin and subcutaneous tissue disorders Left Buttock Condyloma	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Skin and subcutaneous tissue disorders Left Shoulder Cyst Removal	0.00% 0/3 • 1 year	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/8 • 1 year
Skin and subcutaneous tissue disorders Mild Pruritus Ani	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Skin and subcutaneous tissue disorders Mild Non-Tender, Non-Fluctuant Erythema Around IV Insertion Site	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Vascular disorders Hematoma	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 2 • 1 year
Vascular disorders Right leg edema	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Surgical and medical procedures PICC Line Insertion	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Surgical and medical procedures CT Scan of Right Lower Extermity	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Surgical and medical procedures Left Popliteal Venogram and Lysis Catheter Placement	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Surgical and medical procedures Lysis Catheter Removal	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Surgical and medical procedures PICC Line	0.00% 0/3 • 1 year	0.00% 0/3 • 1 year	12.5% 1/8 • Number of events 1 • 1 year
Surgical and medical procedures PICC Line Right Upper Extremity	33.3% 1/3 • Number of events 1 • 1 year	0.00% 0/3 • 1 year	0.00% 0/8 • 1 year

Additional Information

Erica Roesch, MD

University Hospitals, Rainbow Babies and Children's Hospital

Phone: (216) 844-3267

Email: Erica.Roesch@UHHospitals.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place