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

Dermatomyositis Clinical Trial

Official title:

Phase 1 Study of Mesenchymal Stromal Cells, Umbilical Cord Lining Stem Cells (ULSC), in Patients With Polymyositis (PM) and Dermatomyositis (DM)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04723303
• Other study ID #: DM/PM V2.0
• Secondary ID: IRB201903442OCR3
• Status: Active, not recruiting
• Phase: Early Phase 1
• Start date: October 6, 2021
• Est. completion date: April 8, 2024

Study information

• Verified date: June 2023
• Source: University of Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 10
• Est. completion date: April 8, 2024
• Est. primary completion date: March 21, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 90 Years

Eligibility

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

Related Conditions & MeSH terms

• Dermatomyositis
• Polymyositis

Intervention

Drug:
• Umbilical Cord Lining Stem Cells
An IV infusion of ULSCs will be administered.

Locations

Country	Name	City	State
United States	University of Florida	Gainesville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Florida	Restem, LLC.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.)	Within 24 hours