Safety and Tolerability Study of Allogeneic Mesenchymal Stem Cell Infusion in Adults With Cystic Fibrosis (CEASE-CF)
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|ClinicalTrials.gov Identifier: NCT02866721|
Recruitment Status : Recruiting
First Posted : August 15, 2016
Last Update Posted : March 1, 2018
This study is being done to test if it is safe to give stem cells to adult patients with Cystic Fibrosis (CF). The kind of stem cells we are studying are called allogeneic human mesenchymal stem cells or MSCs. MSCs are cells in the body that can grow into different types of cells and respond to various environmental situations. Allogeneic means the cells come from another person (a donor).
This study is only looking at whether or not it is safe to give the stem cells to adults with CF and how the infusion is tolerated. In the future, other studies may be done to see if stem cells can be a new therapeutic treatment for CF.
Stem cells, like other medical products that are intended to treat, cure or prevent disease, generally require approval from the U.S. Food and Drug Administration (FDA) before they can be marketed. The FDA has not approved any stem cell-based products for usual medical care, other than some specific blood forming stem cells for certain indications.
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Biological: Mesenchymal Stem Cells||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I, Single Center, Open Label, Single Dose, Dose Escalation Study Assessing the Safety and Tolerability of AllogeneiC MEsenchymAl Stem CEll Infusion in Adults With Cystic Fibrosis-CEASE CF|
|Actual Study Start Date :||August 2016|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 2020|
Experimental: Human Allogeneic Mesenchymal Stem Cells
One time IV Infusion of up to 5 x 10^6 allogeneic hMSCs/kg of body weight. A dose escalation using the "3+3" design will be employed. The three doses are 1 x 10^6, 3 x 10^6, and 5 x 10^6 hMSCs/kg. There is no placebo group. All study participants will receive stem cells.
Biological: 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 will be utilized.
Allogeneic MSCs will be derived from bone marrow aspirates from a healthy donor whose serum tests negative for cytomegalovirus (CMV) antibodies. Healthy donors will undergo tests for infectious disease and screening for 41 common CFTR mutations. In addition, the MSCs will be validated for in vitro and in vivo efficacy and potency using the in vivo murine pre-clinical model of CF lung infection and inflammation.
Other Name: MSCs
- Dose limiting toxicity (DLT), triggered by occurrence in the first 24 hours after hMSC infusion of grade ≥3 infusion-related allergic toxicities [ Time Frame: 24 hours ]
- Incidence and severity of adverse events [ Time Frame: 1 year ]
- Number of pulmonary exacerbations [ Time Frame: 1 year ]
- Diary reports (Cystic Fibrosis Respiratory Symptom Diary (CFRSD)) [ Time Frame: 35 days ]
- Changes in subject reported symptoms as captured by the Respiratory Signs and Symptoms Questionnaire (RSSQ) from Baseline (Visit 2) Day 1 to Baseline (Visit 2) Day 2, Visits 3, 4, 5, 6 and 7 and at times when a pulmonary exacerbation is being considered [ Time Frame: 1 year ]
- Changes in physical examination [ Time Frame: 28 days ]
- Changes in vital signs including oxygen saturation checked throughout infusion [ Time Frame: 24 hours ]
- Changes in spirometry (FEV1 %, FEV1 (Liters), FEF25-75) determined 30 minutes, 4 hours, and 24 hours after completion of infusion and from Baseline (Visit 2) Day 1 to Visits 3, 4, 5, 6 and 7 [ Time Frame: 6 months ]
- Change in sputum quantitative microbiology (bacterial colony forming units between Baseline to Day 7 and Day 28) [ Time Frame: 28 days ]
- Changes in hematology, comprehensive chemistry, ESR, hs-CRP, and urinalysis results [ Time Frame: 6 months ]
- Change in blood inflammatory biomarkers from Baseline to Day 7 and Day 28 [ Time Frame: 28 days ]
- Change in sputum inflammatory biomarkers from Baseline to Day 7 and Day 28 [ Time Frame: 28 days ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02866721
|Contact: Heather Tribout, BS, CCRP||(216) 286-0765||Heather.Tribout@UHhospitals.org|
|Contact: Kathleen Hilliard, BA, CCRC||(216) 844-7489||Kathleen.Hilliard@UHhospitals.org|
|United States, Ohio|
|University Hospitals Cleveland Medical Center||Recruiting|
|Cleveland, Ohio, United States, 44106|
|Contact: Heather Tribout, BS, CCRP 216-286-0765 Heather.Tribout@UHhospitals.org|
|Principal Investigator: James F. Chmiel, MD, MPH|
|Principal Investigator:||James F Chmiel, MD, MPH||University Hospitals Cleveland Medical Center|