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| ID | Type | Description | Link |
|---|---|---|---|
| IRB201903442 | Other Identifier | UF IRB | |
| OCR33722 | Other Identifier | UF OnCore |
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| Name | Class |
|---|---|
| Restem, LLC. | INDUSTRY |
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This study will investigate Umbilical Cord Lining Stem Cells (ULSC) as an investigational medicinal product and its use in patients with polymyositis (PM) or dermatomyositis (DM) to see if a single intravenous (IV) infusion of allogeneic umbilical cord lining stem cells (ULSC) safe, tolerable, and feasible to administer.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm | Experimental | A single IV infusion of ULSC's in patients with DM or PM |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Umbilical Cord Lining Stem Cells | Drug | An IV infusion of ULSCs will be administered. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Subjects with Dose Limiting Toxicity (DLT) that begins during or following ULSC infusion as assessed within 24 hours. | Dose Limiting Toxicities are treatment-emergent suspected adverse reactions graded severe, such as severe infusion-related hypersensitivity toxicities of grade ≥3, and any serious adverse event (SAE). (Note: DLT during an infusion will stop that infusion in that subject.) | Within 24 hours |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Carl Pepine, MD | University of Florida | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Florida | Gainesville | Florida | 32610 | United States |
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| ID | Term |
|---|---|
| D017285 | Polymyositis |
| D003882 | Dermatomyositis |
| ID | Term |
|---|---|
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
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Study Participants will be assigned to one of 3 doses of stem cells depending on when the subject is enrolled. The first three patients will be assigned to the low dose group, the next three patients will be assigned the intermediate dose group and last three patients will receive the high dose of ULSC's. An independent review group will evaluate the response to therapy to each dose group before moving to the next dose. The groups and doses are described below:
A low dose group receives an IV infusion of 50 million stem cells.
An intermediate dose group receives an IV infusion of 100 million stem cells.
A high dose group receives an IV infusion of 200 million stem cells.
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| D009422 |
| Nervous System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |