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

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

Study Contact Backup

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:

  1. Adult, male or female, age ≥18 years old
  2. Diagnosis of definite or probable DM or PM, according to the criteria of Bohan and Peter
  3. 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).
  4. Signs informed consent.

Exclusion Criteria:

  1. A diagnosis of inclusion body myositis, juvenile DM or PM, myositis in the context of significant overlap with another systemic autoimmune rheumatologic disease.
  2. Non immune myopathies.
  3. Cancer associated myositis.
  4. Hypersensitivity to study product components. History of hypersensitivity to dimethyl sulfoxide (DMSO).
  5. Pregnant or lactating women.
  6. Concomitant severe cardiac, pulmonary disease, active infection or other conditions that preclude assessment of safety and efficacy of the study product.
  7. 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.
  8. Anticipated need for surgery during the trial period.
  9. A history of prevalent noncompliance with medical therapy.
  10. Recipient of an organ transplant.
  11. Neutropenia (absolute neutrophil count<1,800/mm3 [or <1,000/mm3 in African-American subjects]).
  12. Severe impairment in renal function (estimated glomerular filtration rate <30 ml/kg*min).
  13. Recent or planned use of vaccination with live attenuated viruses.
  14. 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).
  15. Condition that would impair an assessment of muscle strength, including neurological disorders such as Parkinson's disease or severe musculoskeletal condition.
  16. Any other condition that, in the judgment of the Investigator or Sponsor, would be a contraindication to enrollment, study product administration, or follow-up.
  17. 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.

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

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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