Back to resultsPhase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)
Primary Purpose
Polymyositis, Dermatomyositis
Status
Active
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Umbilical Cord Lining Stem Cells
Sponsored by
About this trial
This is an interventional treatment trial for Polymyositis
Eligibility Criteria
Inclusion Criteria:
- Adult, male or female, age ≥18 years old
- Diagnosis of definite or probable DM or PM, according to the criteria of Bohan and Peter
- Patients with PM will either be positive for a myositis-associated antibody or have undergone evaluation to exclude mimics, as deemed appropriate by the Investigator (See Note below).
- Signs informed consent.
Exclusion Criteria:
- A diagnosis of inclusion body myositis, juvenile DM or PM, myositis in the context of significant overlap with another systemic autoimmune rheumatologic disease.
- Non immune myopathies.
- Cancer associated myositis.
- Hypersensitivity to study product components. History of hypersensitivity to dimethyl sulfoxide (DMSO).
- Pregnant or lactating women.
- Concomitant severe cardiac, pulmonary disease, active infection or other conditions that preclude assessment of safety and efficacy of the study product.
- Patients with predominant muscle atrophy secondary to uncontrolled or chronic DM or PM, based on clinical, biochemical, and/or radiologic assessment, despite previous optimized treatment.
- Anticipated need for surgery during the trial period.
- A history of prevalent noncompliance with medical therapy.
- Recipient of an organ transplant.
- Neutropenia (absolute neutrophil count<1,800/mm3 [or <1,000/mm3 in African-American subjects]).
- Severe impairment in renal function (estimated glomerular filtration rate <30 ml/kg*min).
- Recent or planned use of vaccination with live attenuated viruses.
- Active cancer or prior diagnosis of cancer within the past 2 years (patients with basal and squamous cell cancer of skin will not be excluded).
- Condition that would impair an assessment of muscle strength, including neurological disorders such as Parkinson's disease or severe musculoskeletal condition.
- Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up.
- History of Atrial septal defect or ventricular septal defect
Sites / Locations
- University of Florida
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment Arm
Arm Description
A single IV infusion of ULSC's in patients with DM or PM
Outcomes
Primary Outcome Measures
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.)
Secondary Outcome Measures
Full Information
NCT ID
NCT04723303
First Posted
December 8, 2020
Last Updated
June 7, 2023
Sponsor
University of Florida
Collaborators
Restem, LLC.
1. Study Identification
Unique Protocol Identification Number
NCT04723303
Brief Title
Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)
Official Title
Phase 1 Study of Mesenchymal Stromal Cells, Umbilical Cord Lining Stem Cells (ULSC), in Patients With Polymyositis (PM) and Dermatomyositis (DM)
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 6, 2021 (Actual)
Primary Completion Date
March 21, 2023 (Actual)
Study Completion Date
April 8, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Florida
Collaborators
Restem, LLC.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Polymyositis, Dermatomyositis
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Sequential Assignment
Model Description
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.
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment Arm
Arm Type
Experimental
Arm Description
A single IV infusion of ULSC's in patients with DM or PM
Intervention Type
Drug
Intervention Name(s)
Umbilical Cord Lining Stem Cells
Intervention Description
An IV infusion of ULSCs will be administered.
Primary Outcome Measure Information:
Title
Number of Subjects with Dose Limiting Toxicity (DLT) that begins during or following ULSC infusion as assessed within 24 hours.
Description
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.)
Time Frame
Within 24 hours
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
90 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adult, male or female, age ≥18 years old
Diagnosis of definite or probable DM or PM, according to the criteria of Bohan and Peter
Patients with PM will either be positive for a myositis-associated antibody or have undergone evaluation to exclude mimics, as deemed appropriate by the Investigator (See Note below).
Signs informed consent.
Exclusion Criteria:
A diagnosis of inclusion body myositis, juvenile DM or PM, myositis in the context of significant overlap with another systemic autoimmune rheumatologic disease.
Non immune myopathies.
Cancer associated myositis.
Hypersensitivity to study product components. History of hypersensitivity to dimethyl sulfoxide (DMSO).
Pregnant or lactating women.
Concomitant severe cardiac, pulmonary disease, active infection or other conditions that preclude assessment of safety and efficacy of the study product.
Patients with predominant muscle atrophy secondary to uncontrolled or chronic DM or PM, based on clinical, biochemical, and/or radiologic assessment, despite previous optimized treatment.
Anticipated need for surgery during the trial period.
A history of prevalent noncompliance with medical therapy.
Recipient of an organ transplant.
Neutropenia (absolute neutrophil count<1,800/mm3 [or <1,000/mm3 in African-American subjects]).
Severe impairment in renal function (estimated glomerular filtration rate <30 ml/kg*min).
Recent or planned use of vaccination with live attenuated viruses.
Active cancer or prior diagnosis of cancer within the past 2 years (patients with basal and squamous cell cancer of skin will not be excluded).
Condition that would impair an assessment of muscle strength, including neurological disorders such as Parkinson's disease or severe musculoskeletal condition.
Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up.
History of Atrial septal defect or ventricular septal defect
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Carl Pepine, MD
Organizational Affiliation
University of Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)
We'll reach out to this number within 24 hrs