Safety and efficacy of human umbilical cord-derived mesenchymal stem cells in patients with Alzheimer′s disease: study protocol for an open-label self-control trial

Abstract Background: Bronchopulmonary dysplasia (BPD) is a complex lung pathological lesion secondary to multiple factors and one of the most common chronic lung diseases with a poor prognosis, especially in preterm infants. However, effective therapies for this disease are lacking. Stem cell therapy is a promising way to improve lung injury and abnormal alveolarization, and the human umbilical cord (hUC) is a good source of mesenchymal stem cells (MSCs), which have demonstrated efficacy in other diseases. We hypothesized that intravenous allogeneic hUC-MSCs are safe and effective for severe BPD. Methods: The MSC-BPD trial is a randomized single-center open-label dose-escalation phase II trial designed to investigate the safety and efficacy of hUC-MSCs in children with severe BPD. In this study, 72 patients will be enrolled and randomly divided into two intervention groups and one control group. Patients in the intervention groups will receive a low dose of hUC-MSCs (n = 24; 2.5 million cells/kg) or a high dose of hUC-MSCs (n = 24; 5 million cells/kg) in combination with traditional supportive treatments for BPD. The patients in the control group (n = 24) will be treated with traditional supportive treatments alone without hUC-MSCs. The primary outcome measures will be cumulative duration of oxygen therapy. Follow-up assessments will be performed at 1, 3, 6, 12, and 24 months post-intervention, and the key outcome during follow-up will be changes on chest radiography. Statistical analyses will evaluate the efficacy of the hUC-MSC treatment. Discussion: This will be the first randomized controlled trial to evaluate the safety and efficacy of intravenous hUC-MSCs in children with severe BPD. Its results will provide a new evidence-based therapy for severe BPD.

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Allogeneic human umbilical cord-derived mesenchymal stem cells for moderate and severe bronchopulmonary dysplasia in children: study protocol for a randomized controlled trial (the MSC-BPD Trial)

10.21203/rs.2.381/v1 ◽

2019 ◽

Author(s):

Xian Wu ◽

Yunqiu Xia ◽

Ou Zhou ◽

Yan Song ◽

Xianhong Zhang ◽

...

Keyword(s):

Stem Cells ◽

Randomized Controlled Trial ◽

Mesenchymal Stem Cells ◽

Umbilical Cord ◽

Bronchopulmonary Dysplasia ◽

Controlled Trial ◽

Control Group ◽

Human Umbilical Cord ◽

Safety And Efficacy ◽

Randomized Controlled

Abstract Background Bronchopulmonary dysplasia (BPD) is a complex lung pathological lesion secondary to multiple factors, and one of the most common chronic lung diseases with poor prognosis, especially in preterm infants with moderate and severe BPD. However, there is lack of effective therapies for this disease. Stem cell therapy has been shown a promising way for improving lung injury and abnormal alveolarization, and human umbilical cord (hUC) is a good resource for mesenchymal stem cells (MSCs), which have demonstrated in some other diseases. We hypothesis that intravenous allogeneic hUC-MSCs is safe and effective for moderate and severe BPD. Methods/design The MSC-BPD trial is a randomized single-center open-label and dose-escalation phase II trial designed to investigate the safety and efficacy of hUC-MSCs in children with moderate and severe BPD. In this study, 57 patients will be enrolled and randomly divided into two intervention groups and one control group. Patients in the intervention groups will be receive a low dose of hUC-MSCs (n=19, 1 million cells/kg) or a high dose of hUC-MSCs (n=19, 5 million cells/kg) in combination with traditional supportive treatments for BPD. The patients in the control group (n=19) will be treated with traditional supportive treatments alone without receiving hUC-MSCs treatment. The primary outcome measures will be the accumulative duration of oxygen therapy. Follow-up assessments will be performed at 1, 3, 6, 12, 24 months after interventions. Statistical analyses will evaluate the efficacy of the hUC-MScs treatment. Discussion This study will be the first randomized controlled trial to evaluate the safety and efficacy of intravenous hUC-MSCs in children with moderate and severe BPD. Results of the trial will provide a new evidence-based therapy for moderate and severe BPD. Trial Registration ClinicalTrials.gov, NCT03601416. Registered on 26th July 2018.

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Allogeneic human umbilical cord-derived mesenchymal stem cells for severe bronchopulmonary dysplasia in children: Study protocol for a randomized controlled trial(the MSC-BPD Trial)

10.21203/rs.2.381/v4 ◽

2019 ◽

Author(s):

Xian Wu ◽

Yunqiu Xia ◽

Ou Zhou ◽

Yan Song ◽

Xianhong Zhang ◽

...

Keyword(s):

Stem Cells ◽

Mesenchymal Stem Cells ◽

Umbilical Cord ◽

Bronchopulmonary Dysplasia ◽

Controlled Trial ◽

Control Group ◽

Human Umbilical Cord ◽

Safety And Efficacy ◽

Randomized Controlled

Abstract Background: Bronchopulmonary dysplasia (BPD) is a complex lung pathological lesion secondary to multiple factors and one of the most common chronic lung diseases with a poor prognosis, especially in preterm infants. However, effective therapies for this disease are lacking. Stem cell therapy is a promising way to improve lung injury and abnormal alveolarization, and the human umbilical cord (hUC) is a good source of mesenchymal stem cells (MSCs), which have demonstrated efficacy in other diseases. We hypothesized that intravenous allogeneic hUC-MSCs are safe and effective for severe BPD. Methods: The MSC-BPD trial is a randomized single-center open-label dose-escalation phase II trial designed to investigate the safety and efficacy of hUC-MSCs in children with severe BPD. In this study, 72 patients will be enrolled and randomly divided into two intervention groups and one control group. Patients in the intervention groups will receive a low dose of hUC-MSCs (n = 24; 2.5 million cells/kg) or a high dose of hUC-MSCs (n = 24; 5 million cells/kg) in combination with traditional supportive treatments for BPD. The patients in the control group (n = 24) will be treated with traditional supportive treatments alone without hUC-MSCs. The primary outcome measures will be cumulative duration of oxygen therapy. Follow-up assessments will be performed at 1, 3, 6, 12, and 24 months post-intervention, and the key outcome during follow-up will be changes on chest radiography. Statistical analyses will evaluate the efficacy of the hUC-MSC treatment. Discussion: This will be the first randomized controlled trial to evaluate the safety and efficacy of intravenous hUC-MSCs in children with severe BPD. Its results will provide a new evidence-based therapy for severe BPD. Trial registration: ClinicalTrials.gov (NCT03601416; registered on July 26, 2018) Keywords: bronchopulmonary dysplasia, human umbilical cord-derived mesenchymal stem cells, clinical trial, protocol

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Allogeneic human umbilical cord-derived mesenchymal stem cells for severe bronchopulmonary dysplasia in children: study protocol for a randomized controlled trial(the MSC-BPD Trial)

10.21203/rs.2.381/v2 ◽

2019 ◽

Author(s):

Xian Wu ◽

Yunqiu Xia ◽

Ou Zhou ◽

Yan Song ◽

Xianhong Zhang ◽

...

Keyword(s):

Stem Cells ◽

Mesenchymal Stem Cells ◽

Umbilical Cord ◽

Bronchopulmonary Dysplasia ◽

Controlled Trial ◽

Control Group ◽

Human Umbilical Cord ◽

Safety And Efficacy ◽

Randomized Controlled

Abstract Background : Bronchopulmonary dysplasia (BPD) is a complex lung pathological lesion secondary to multiple factors, and one of the most common chronic lung diseases with poor prognosis, especially in preterm infants with severe BPD. However, there is lack of effective therapies for this disease. Stem cell therapy has been shown a promising way for improving lung injury and abnormal alveolarization, and human umbilical cord (hUC) is a good resource for mesenchymal stem cells (MSCs), which have demonstrated in some other diseases. We hypothesis that intravenous allogeneic hUC-MSCs is safe and effective for severe BPD. Methods/design: The MSC-BPD trial is a randomized single-center open-label and dose-escalation phase II trial designed to investigate the safety and efficacy of hUC-MSCs in children with severe BPD. In this study, 48 patients will be enrolled and randomly divided into two intervention groups and one control group. Patients in the intervention groups will be receive a low dose of hUC-MSCs (n=16, 2.5 million cells/kg) or a high dose of hUC-MSCs (n=16, 5 million cells/kg) in combination with traditional supportive treatments for BPD. The patients in the control group (n=16) will be treated with traditional supportive treatments alone without receiving hUC-MSCs treatment. The primary outcome measures will be the cumulative duration of oxygen therapy. Follow-up assessments will be performed at 1, 3, 6, 12, 24 months after interventions, and the key outcome during follow-up will be the chest radiography changes. Statistical analyses will evaluate the efficacy of the hUC-MScs treatment. Discussion: This study will be the first randomized controlled trial to evaluate the safety and efficacy of intravenous hUC-MSCs in children with severe BPD. Results of the trial will provide a new evidence-based therapy for severe BPD. Trial registration: ClinicalTrials.gov, NCT03601416. Registered on 26th July 2018. Key words: bronchopulmonary dysplasia, human umbilical cord-derived mesenchymal stem cells, clinical trial, protocol.

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