Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)

NCT ID: NCT04723303

Last Updated: 2025-01-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: EARLY_PHASE1
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-06
• Study Completion Date: 2024-03-08

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.

Conditions

Polymyositis; Dermatomyositis

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment Arm

A single IV infusion of ULSC's in patients with DM or PM

Group Type: EXPERIMENTAL

Umbilical Cord Lining Stem Cells

Intervention Type: DRUG

An IV infusion of ULSCs will be administered.

Interventions

Umbilical Cord Lining Stem Cells

An IV infusion of ULSCs will be administered.

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 90 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Restem, LLC.

INDUSTRY

Sponsor Role: collaborator

University of Florida

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Carl Pepine, MD

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

University of Florida

Gainesville, Florida, United States

Countries

United States

Other Identifiers

IRB201903442

Identifier Type: OTHER

Identifier Source: secondary_id

OCR33722

Identifier Type: OTHER

Identifier Source: secondary_id

DM/PM V2.0

Identifier Type: -

Identifier Source: org_study_id