PolymyositisEarly Phase 1NCT04723303
Phase 1 Study of ULSC in Patients With Polymyositis (PM) and Dermatomyositis (DM)
A Early Phase 1 study testing Umbilical Cord Lining Stem Cells in people with Polymyositis and Dermatomyositis, run by University of Florida at 1 site in United States.
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… Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Polymyositis, Dermatomyositis
- What is being tested?
- Umbilical Cord Lining Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Oct 2021to 8 Mar 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
- Conditions
- Polymyositis, Dermatomyositis
- Being tested
- Umbilical Cord Lining Stem Cells
- Medical subject headings
- Polymyositis, Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Umbilical Cord Lining Stem CellsDrug
An IV infusion of ULSCs will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm | Experimental | A single IV infusion of ULSC's in patients with DM or PM Receives: Drug: Umbilical Cord Lining Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.)
Measured: Within 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jan 2021
- Study start6 Oct 2021
- Primary completion12 Dec 2023
- Study completion8 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PolymyositisStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Study Participants will be assigned to one of 3 doses of stem cells depending on when the subject is enrolled. The first three patients will be assigned to the low dose group, the next three patients will be assigned the intermediate dose group and last three patients will receive the high dose of ULSC's. An independent review group will evaluate the response to therapy to each dose group before moving to the next dose. The groups and doses are described below: A low dose group receives an IV infusion of 50 million stem cells. An intermediate dose group receives an IV infusion of 100 million stem cells. A high dose group receives an IV infusion of 200 million stem cells.
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Restem, LLC.
- Responsible party
- Sponsor
- Investigators
- Carl Pepine, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04723303
- Sponsor's protocol ID
- DM/PM V2.0
- Other IDs
- IRB201903442, OCR33722
- Status last verified
- Jan 2025
- First submitted
- 8 Dec 2020
- Last updated
- 24 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Study of Mesenchymal Stromal Cells, Umbilical Cord Lining Stem Cells (ULSC), in Patients With Polymyositis (PM) and Dermatomyositis (DM)
- ClinicalTrials.gov ID
- NCT04723303
- Acronym / sponsor ID
- DM/PM V2.0
- Conditions
- Polymyositis, Dermatomyositis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Restem, LLC.
- Study start
- 6 Oct 2021 (actual)
- Primary completion
- 12 Dec 2023 (actual)
- Study completion
- 8 Mar 2024 (actual)
- First posted
- 25 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Study Participants will be assigned to one of 3 doses of stem cells depending on when the subject is enrolled. The first three patients will be assigned to the low dose group, the next three patients will be assigned the intermediate dose group and last three patients will receive the high dose of ULSC's. An independent review group will evaluate the response to therapy to each dose group before moving to the next dose. The groups and doses are described below: A low dose group receives an IV infusion of 50 million stem cells. An intermediate dose group receives an IV infusion of 100 million stem cells. A high dose group receives an IV infusion of 200 million stem cells.
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Drug: Umbilical Cord Lining Stem CellsA single IV infusion of ULSC's in patients with DM or PM |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (17 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Umbilical Cord Lining Stem CellsAn IV infusion of ULSCs will be administered. | Drug | Treatment Arm |
Primary outcome
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.)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carl Pepine, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Umbilical Cord Lining Stem Cells in people with Polymyositis and Dermatomyositis, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jan 2025.