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

Early Phase 1

Completed

• Conditions: Polymyositis; Dermatomyositis
• Interventions: Drug: Umbilical Cord Lining Stem Cells

• Registration Number: NCT04723303
• Lead Sponsor: University of Florida

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-12-08
• Study First Submitted QC: 2021-01-22
• Study First Posted: 2021-01-25
• Study Start: 2021-10-06
• Primary Completion: 2023-12-12
• Study Completion: 2024-03-08
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-23
• Last Update Posted: 2025-01-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Treatment Arm	Umbilical Cord Lining Stem Cells	A single IV infusion of ULSC's in patients with DM or PM

Primary Outcome Measures

Name	Time	Method
Number of Subjects with Dose Limiting Toxicity (DLT) that begins during or following ULSC infusion as assessed within 24 hours.	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.)

Trial Locations

Locations (1)

University of Florida; 🇺🇸 Gainesville, Florida, United States