Ignite Creation Date:
2024-05-06 @ 3:56 PM
Last Modification Date:
2024-10-26 @ 2:00 PM
Study NCT ID:
NCT04811261
Status:
COMPLETED
Last Update Posted:
2023-08-02
First Post:
2021-03-19
Brief Title:
u-STOP LVAD Bleed Utilization of Umbilical Cord Lining Stem Cells ULSCs To Prevent Left Ventricular Assist Device LVAD Associated Angiodysplastic Bleeding
Sponsor:
University of Florida
Organization:
University of Florida
Study Overview
Official Title:
u-STOP LVAD Bleed Utilization of Umbilical Cord Lining Stem Cells ULSCs To Prevent Left Ventricular Assist Device LVAD Associated Angiodysplastic Bleeding
Status:
COMPLETED
Status Verified Date:
2023-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a Phase I single ascending dose first in human open-label non-controlled dose-escalation trial that will investigate intravenous infusions of Umbilical cord lining stem cells UCLSCs in Left Ventricular Assist Device LVAD patients
This study will recruit patients from the Advanced Heart Failure and LVAD Clinic at the University of Florida including recently implanted patients as well as patients on chronic long-term support with the goal of enrolling up to 9 subjects to participate in the study
This study will recruit patients from the Advanced Heart Failure and LVAD Clinic at the University of Florida including recently implanted patients as well as patients on chronic long-term support with the goal of enrolling up to 9 subjects to participate in the study
Detailed Description:
Subjects will be entered into a 33 dose escalation study a standard design for initial trials to evaluate tolerability of a therapeutic agent and identify a maximal tolerated dose if indeed toxicities are identified in the pre-specified dosing range The first 3 subjects will be administered 50 x 106 ULSCs reconstituted in PBS with 1 human serum albumin in a volume of 250 ml and monitored for adverse events or toxicities immediately following dosing and again at 30 days Safety endpoints will be reviewed by the DSMB and depending on the response the dose will be escalated to 100 x 106 and 200 x 106 ULSC in a constant 250 ml volume We will enroll only 9 subjects in the case that no subject experiences toxicity only expanding the cohort in the event that a subject experiences toxicity at a particular dose
This specific dose escalation approach is based on the following considerations
There is not yet a standard dosing regimen for cell therapy in heart failure patients The previous MSC trial in LVAD patients provided 150 million cells The RIMECARD trial Bartolucci et al Circ Res 2017 Oct 27121101192-1204 PMID 28974553 provided umbilical cord-derived MSC for patients with heart failure via intravenous infusions of 1x106 cellskg Similarly another Phase IIa trial of MSCs in HFrEF used 15x106 cellskg Butler et al Circ Res 2017 Jan 201202332-340 PMID 27856497 Since our LVAD patients range in size from 50 to 100kg we believe the selected range of doses is reasonable for this patient population
In addition in ongoing studies in COVID 19 ARDS IND 19979 we have provided 100 x 106 ULSCs with no adverse safety signals Moreover in our non-clinical animal models we demonstrated safety at doses ranging from a high dose of 1 x 106 cellsmouse which is equivalent to approximately 5 x 107 cellskg in humans and a low dose of 5 x 104 cellsmouse which is equivalent to approximately 25 x 106 cellskg in humans The dose tested in the high dose preclinical study would scale to administration of over 25 billion cells in an individual weighing 100 pounds
This specific dose escalation approach is based on the following considerations
There is not yet a standard dosing regimen for cell therapy in heart failure patients The previous MSC trial in LVAD patients provided 150 million cells The RIMECARD trial Bartolucci et al Circ Res 2017 Oct 27121101192-1204 PMID 28974553 provided umbilical cord-derived MSC for patients with heart failure via intravenous infusions of 1x106 cellskg Similarly another Phase IIa trial of MSCs in HFrEF used 15x106 cellskg Butler et al Circ Res 2017 Jan 201202332-340 PMID 27856497 Since our LVAD patients range in size from 50 to 100kg we believe the selected range of doses is reasonable for this patient population
In addition in ongoing studies in COVID 19 ARDS IND 19979 we have provided 100 x 106 ULSCs with no adverse safety signals Moreover in our non-clinical animal models we demonstrated safety at doses ranging from a high dose of 1 x 106 cellsmouse which is equivalent to approximately 5 x 107 cellskg in humans and a low dose of 5 x 104 cellsmouse which is equivalent to approximately 25 x 106 cellskg in humans The dose tested in the high dose preclinical study would scale to administration of over 25 billion cells in an individual weighing 100 pounds
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| OCR40194 | OTHER | UF OnCore | None |
| IRB202100247 | OTHER | None | None |