- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04723303
Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)
June 7, 2023 updated by: University of Florida
Phase 1 Study of Mesenchymal Stromal Cells, Umbilical Cord Lining Stem Cells (ULSC), in Patients With Polymyositis (PM) and Dermatomyositis (DM)
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.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
10
Phase
- Early Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Carl Pepine, MD
- Phone Number: 352-273-9082
- Email: Carl.Pepine@medicine.ufl.edu
Study Contact Backup
- Name: Chassidi Rogers
- Phone Number: 352-273-8946
- Email: Chassidi.Garrett@medicine.ufl.edu
Study Locations
-
-
Florida
-
Gainesville, Florida, United States, 32610
- University of Florida
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
16 years to 88 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Treatment Arm
A single IV infusion of ULSC's in patients with DM or PM
|
An IV infusion of ULSCs will be administered.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Subjects with Dose Limiting Toxicity (DLT) that begins during or following ULSC infusion as assessed within 24 hours.
Time Frame: 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
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Carl Pepine, MD, University of Florida
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 6, 2021
Primary Completion (Actual)
March 21, 2023
Study Completion (Estimated)
April 8, 2024
Study Registration Dates
First Submitted
December 8, 2020
First Submitted That Met QC Criteria
January 22, 2021
First Posted (Actual)
January 25, 2021
Study Record Updates
Last Update Posted (Actual)
June 8, 2023
Last Update Submitted That Met QC Criteria
June 7, 2023
Last Verified
June 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- DM/PM V2.0
- IRB201903442 (Other Identifier: UF IRB)
- OCR33722 (Other Identifier: UF OnCore)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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