scholarly journals Mesenchymal Stem Cells for Bronchopulmonary Dysplasia: Phase 1 Dose-Escalation Clinical Trial

2014 ◽  
Vol 164 (5) ◽  
pp. 966-972.e6 ◽  
Author(s):  
Yun Sil Chang ◽  
So Yoon Ahn ◽  
Hye Soo Yoo ◽  
Se In Sung ◽  
Soo Jin Choi ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Bronchopulmonary Dysplasia ◽  
Dose Escalation ◽  
Phase 1

scholarly journals AB0370 SAFETY OF CS20AT04, A HAPLOIDENTICAL ALLOGENEIC BONE MARROW-DERIVED MESENCHYMAL STEM CELLS, IN A PHASE 1 STUDY IN LUPUS NEPHRITIS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1485.2-1485
Author(s):  
C. B. Choi ◽  
T. Y. Lee ◽  
K. S. Kim ◽  
S. C. Bae
Keyword(s):  
Stem Cells ◽  
Bone Marrow ◽  
Mesenchymal Stem Cells ◽  
Lupus Nephritis ◽  
Dose Escalation ◽  
Phase 1 ◽  
Allogeneic Bone Marrow ◽  
Additional Patient ◽  
Allogeneic Bone ◽  
Dose Limiting Toxicity

Background:Mesenchymal stem cells are known to have immunomodulatory properties and may potentially have therapeutic effect in lupus nephritis. Mesenchymal stem cells form a haploidentical donor are an attractive cell sourceObjectives:CS20AT04, a haploidentical allogeneic bone marrow-derived mesenchymal stem cell, was evaluated in patients with lupus nephritis for safety and tolerability.Methods:This was a single-arm phase 1 dose-escalation trial of CS20AT04 in adult patients with lupus nephritis (NCT03174587). A 3 + 3 design was used for dose escalation. The starting dose was 2.0 x 106 cells/kg and was escalated to 3.0 x 106 cells/kg if there no dose-limiting toxicity. The primary objective was to determine the maximum tolerated dose and evaluate the safety and tolerability at 28 days after the infusion.Results:Seven patients were enrolled in the study. Patients received CS20AT04 through intravenous infusion. The initial dose of 2.0 x 106 cells/kg was administered for the first 3 patients without any dose limiting toxicity. There was 1 patient who were not administered the full 2.0 x 106 cells/kg dose due to technical error during infusion. The patient did not show dose limiting toxicity, but 1 additional patient was enrolled to have 3 patients who received the full 2.0 x 106 cells/kg dose before escalating to the next level dose. The dose of 3.0 x 106 cells/kg was administered for the next 3 patients without any dose limiting toxicity. Three adverse events were reported (1 diarrhea, 1 toothache, and 1 arthralgia) and they were all NCI-CTC grade I events.Conclusion:CS20AT04 was well tolerated in single dose up to 3.0 x 106 cells/kg in patients with lupus nephritis.Acknowledgments:This research was supported by a grant of the Korea Health Technology R&D Project through the Korea Health Industry Development Institute (KHIDI), funded by the Ministry of Health & Welfare, Republic of Korea (grant number: HI15C0778).Disclosure of Interests:Chan-Bum Choi: None declared, Tae Yong Lee Shareholder of: Corestem Inc, Employee of: Corestem Inc, Kyung Suk Kim Shareholder of: Corestem Inc, Employee of: Corestem Inc, Sang-Cheol Bae: None declared

An open-label phase 1 clinical trial of the allogeneic side population adipose-derived mesenchymal stem cells in SMA type 1 patients

2021 ◽  
Author(s):  
Rashin Mohseni ◽  
Amir Ali Hamidieh ◽  
Alireza Shoae-Hassani ◽  
Masood Ghahvechi-Akbari ◽  
Anahita Majma ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Side Population ◽  
Phase 1 ◽  
Open Label ◽  
Phase 1 Clinical Trial ◽  
Open Label Phase

Clinical outcome of the Intrathecal administration of the allogeneic side population Adipose Derived Mesenchymal Stem Cells in Werdnig Hoffman patients: An open-label Phase I clinical trial

2020 ◽  
Author(s):  
Rashin Mohseni ◽  
Amir Ali Hamidieh ◽  
Alireza Shoae-Hassani ◽  
Masood Ghahvechi-Akbari ◽  
Anahita Majma ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Stem Cells ◽  
Mesenchymal Stem Cells ◽  
Stem Cell ◽  
Neurodegenerative Disorder ◽  
Tibial Nerve ◽  
Side Population ◽  
Phase 1 ◽  
Intervention Group ◽  
Open Label

Abstract Background Werdnig Hoffman (WH), a hereditary neurodegenerative disorder of lower motoneurons associated with progressive muscle weakness is the most common genetic cause of infant mortality. There is no effective treatment for WH exists. The field of translational research is active now, and clinical trials or case studies are ongoing. We present a phase 1 clinical trial in patients with WH who received side population adipose-derived mesenchymal stem cells (SPADMSCs). Methods The intervention group administered with three intrathecal administrations of escalating doses of SPADMSCs. The safety analysis was assessed by controlling the vital signs and efficacy analysis performed by the Ballard score and EMG test. These tests were performed previous to treatment and at the end of the follow-up. Results The treatment well-tolerated, without any adverse event related to the stem cell administration. Patients showed significant improvement in the amplitude response of motor in the tibial nerve (0.56 mV; p: 0.029). The weight of patients, ventilation days, and number of hospitalizations were not meaningful parameters in the response of patients in the intervention and control groups. One patient in the intervention group is still alive after 36 months. He gained a normal weight and has a normal growth rate. The patient can breathe without ventilator aid. Conclusion The present study for stem cell therapy shows safety and efficacy in WH patients, mainly in the recovery of the tibial nerve, respiratory system, and length of life.

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