Explore Clinical Trials
At Cure JM, we believe that research holds the key to better treatments. Clinical trials can help us get there. Participating in a clinical trial is one way you can contribute to advancing care for juvenile myositis and access new therapies before they’re widely available.
Helpful Resources from the National Institutes of Health
These trusted resources from the National Institutes of Health (NIH) can help you better understand how clinical trials work, especially for children battling rare diseases:
- What Are Clinical Trials?
A simple, engaging video that explains how clinical trials work. - Glossary of Clinical Trial Terms
A helpful reference to understand terms you might hear when learning about clinical trials. - Personal Stories from Clinical Trial Participants and Researchers
Hear real experiences from those who have participated in research studies.
These resources can help you feel informed, confident, and prepared to explore research options for you or your family.
Now Enrolling Clinical Trials
RESET-Myositis Trial: CAR-T Cell Therapy (CABA-201)
Contact Info:
- Email: clinicaltrials@cabalettabio.com
- Phone: +1 (267) 759-3100
A phase 1/ 2 clinical trial, investigating a treatment that uses your own T cells to treat myositis
Who Can Participate:
- Children ages 6+ and adults with active myositis (JDM, JPM, or adult forms)
- Must have active disease
- Learn more here
A study physician will determine final eligibility.
How It Works:
- T cells are extracted from the patient, reengineered into CAR T cells, and returned to the patient to target harmful immune cells that may be causing disease
- All participants in the RESET-Myositis Trial will receive CAR-T cell therapy— there is no placebo group in this study
- Learn more about the CAR-T process and how it works here
The trial is enrolling at multiple locations across the U.S., including:
Sites enrolling pediatric patients age 6-26*
- Chicago, IL (Comer Children’s, University of Chicago)
- San Francisco, CA (UCSF)
- Philadelphia, PA (CHOP)
- Ann Arbor, MI (University of Michigan)
- New York, NY (Hospital for Special Surgery)
- New York, NY (Memorial Sloan Kettering Cancer Center)
- Chicago, IL (Lurie Children’s Hospital)
- The Bronx, New York (Children’s Hospital at Montefiore
- Seattle, WA (Seattle Children’s)
- Aurora, Colorado (Children’s Hospital Colorado)
- Not yet recruiting: Bethesda, Maryland (NIH)
- Not yet recruiting: Pittsburgh, PA (UPMC Children’s Hospital of Pittsburgh)
- More locations listed here
Sites enrolling age 18+
- Orange County, CA (UC Irvine)
- Jacksonville, FL (Mayo Clinic)
- Nashville, TN (Vanderbilt)
- Houston, TX (Houston Methodist & MD Anderson)
- Kansas City, MO (UMKC)
- Rochester, MN (Mayo Clinic)
- Portland, OR (OHSU)
- Atlanta, GA (Emory)
- United Kingdom: London, Manchester, Salford
- Not yet recruiting: Pittsburgh, PA (UPMC Arthritis and Autoimmunity Center)
- More locations listed here
*Each children’s hospital may have slightly different maximum age guidelines, please contact clinicaltrials@cabalettabio.com to learn more
Travel:
- Travel to one of the sites may be covered, please contact to learn more
Want to learn more?
Talk to your doctor or email the study team at clinicaltrials@cabalettabio.com
Read more about this trial on clinicaltrials.gov here
Considering a clinical trial can feel overwhelming. Cure JM can connect families with parents and patients who have participated in clinical trials, offering the opportunity to ask questions and hear directly about their experience.
To learn more or request a connection, contact James Tealy, Family Education Director, at james.tealy@curejm.org.
Descartes-08 for Children, Adolescents, and Young Adults with JDM
Understanding Descartes-08 for Families
View the Descartes-08 Study Flyer
A Phase 1/2 clinical trial evaluating the safety, tolerability, and potential effectiveness of Descartes-08, an investigational cell therapy for children, adolescents, and young adults living with childhood-onset systemic lupus erythematosus (SLE), ANCA-associated vasculitis (AAV), juvenile myasthenia gravis, and juvenile dermatomyositis (JDM).
Study Leaders & Contacts:
Sponsor: Cartesian Therapeutics
Phone: (617) 231-8102
Email: trials@cartesiantx.com
Who Can Participate:
Ages 12 and older
Confirmed diagnosis of childhood-onset juvenile dermatomyositis
Moderate disease activity and prior systemic treatment
Ability (patient or guardian) to provide written informed consent
A study physician will determine final eligibility.
How It Works:
Descartes-08 is a cell-based therapy designed to modulate the immune system and reduce autoimmune inflammation.
The study is interventional, meaning participants receive an investigational drug under close supervision to assess safety and response.
Locations:
- St. Louis, Missouri, United States, 63110
- Washington D.C., District of Columbia, United States, 20010
Participant Benefits:
Study-related care and monitoring at no cost
Access to an emerging therapy being investigated for several childhood autoimmune conditions
Contribution to research that may improve future treatments for JDM and related diseases
Read more about this trial on clinicaltrials.gov here
Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis
Sponsor: The Cleveland Clinic
What This Study Is About
A Phase 2 clinical trial evaluating whether topical ruxolitinib 1.5% cream can safely and effectively treat refractory cutaneous dermatomyositis — skin disease that has not improved with standard topical and systemic treatments.
Who Can Participate
Adults 18–89 years old with:
Classic dermatomyositis (CD), juvenile‑onset dermatomyositis (JD), or amyopathic dermatomyositis (AD)
Active skin disease (CDASI activity score >6; BSA 1–20%)
Prior systemic treatment (e.g., steroids, methotrexate, MMF, IVIG, etc.)
Prior failure of at least one topical medication
Stable systemic regimen for ≥60 days
A study physician will determine final eligibility.
What the Study Involves
Open‑label, single‑arm pilot study
Participants apply ruxolitinib 1.5% cream to active skin lesions
Evaluations at 8 and 12 weeks using CDASI (Cutaneous Dermatomyositis Activity and Severity Index)
Why This Matters
Cutaneous dermatomyositis can be painful, persistent, and difficult to treat. This study explores whether a targeted topical JAK inhibitor can reduce inflammation and improve skin symptoms for patients who haven’t responded to other options.
Study Contacts
Anthony Fernandez, MD, PhD 216‑445‑8776 dermatologyresearch@ccf.org
Rothy Rim 216‑444‑7277 dermatologyresearch@ccf.org
Read more about this trial on clinicaltrials.gov here
FAST for DM (Fatty Acid Supplementation Trial for Dermatomyositis)
The FAST for DM Study is evaluating whether omega-3 fatty acids from fish oil, combined with a healthy diet based on the Dietary Guidelines for Americans, can help reduce inflammation and improve muscle strength, function, and quality of life for people living with Dermatomyositis (DM) and Juvenile Dermatomyositis (JDM). Participants will receive dietary coaching and take either fish-oil or placebo capsules for six months, with an optional open-label 12-week extension.
Study Leaders & Contacts
Sponsor: National Institutes of Health (NIH)
Principal Investigator: Lisa G. Rider, M.D.
Recruitment Office: NIH Clinical Center Office of Patient Recruitment (OPR)
Phone: 800-411-1222 TTY: 711
Email: ccopr@nih.gov
Email: fastdmsupport@nih.gov
Who Can Participate:
Adults 18–60 years old living in the United States who can read English and have mild to moderately active DM or JDM. Participants must be able to travel to Bethesda, Maryland for study visits, have access to the internet, and be willing to follow a healthy diet and take study capsules for 6 months. People with overlap connective-tissue diseases, severe myositis, meat or fish/shellfish allergies are not eligible.
How It Works:
Participants will be randomly assigned to receive either fish-oil or placebo capsules, taken twice daily.
All participants will follow a healthy diet with the support of a registered dietitian and attend five onsite visits over six months. These include physical exams, lab tests, imaging scans, and muscle strength assessments. Participants will also wear an activity-tracking watch for two 7-day periods.
After 24 weeks, participants may continue for an optional 12-week extension in which all receive fish-oil supplements.
Where:
National Institutes of Health Clinical Center Bethesda, Maryland 20892 View on Map
Participant Benefits:
Access to expert care and regular health assessments Personalized dietary coaching at no cost Opportunity to contribute to research on nutrition-based therapies for DM and JDM All study procedures, tests, and supplements provided free of charge.
Read more about this trial on clinicaltrials.gov here
JASMINE Study: Anifrolumab for PM, DM, and JDM
A phase 3 clinical trial, investigating a subcutaneous medication (injection) called anifrolumab, designed to reduce inflammation.
Study Leaders & Contacts:
- Name: AstraZeneca Clinical Study Information Center
- Phone Number: 1-877-240-9479
- Email: information.center@astrazeneca.com
Where:
View all Sites in the U.S. and Abroad here
Who Can Join:
- Adults 18+ with PM, DM, or JDM (polymyositis, dermatomyositis, or juvenile dermatomyositis)
- JDM participants must have been diagnosed 10+ years ago
- Moderate to severe disease activity
- Learn more here
A study physician will determine final eligibility.
How It Works:
- Anifrolumab blocks type I interferon pathways involved in immune overactivation
- The JASMINE study is a randomized, double-blind, placebo-controlled Phase 3 trial, which means that neither the doctors or patients will know if you are receiving anifrolumab or a placebo
- A study doctor will talk to you about what this means
Participant Benefits:
- Study-related care and treatment at no cost
- Some travel support may be available
Read more about this trial here
Active Clinical Trials (Not Currently Recruiting)
ALKIVIA Trial: Efgartigimod (SC) for Myositis
ALKIVIA Trial: Efgartigimod (SC) for Myositis
The ALKIVIA study is a Phase 2/3 clinical trial evaluating an investigational treatment called efgartigimod. This medication is designed to target parts of the immune system that may be contributing to inflammation in myositis.
Researchers are studying whether this treatment is safe and effective in improving symptoms such as muscle weakness and overall disease activity.
Who Could Participate
(Not currently recruiting)
Adults ages 18 and older
Diagnosed with an inflammatory myopathy, including:
Dermatomyositis (DM)
Polymyositis (PM)
Immune-mediated necrotizing myopathy (IMNM)
Antisynthetase syndrome
Must have active disease (such as muscle weakness or signs of inflammation)
A study physician determined final eligibility.
How It Works
Participants were randomly assigned to receive either:
The investigational treatment (efgartigimod), or
A placebo (inactive treatment)
The medication is given as a subcutaneous (under the skin) injection
Researchers compare how participants respond to treatment over time
What Researchers Are Measuring
Overall improvement in disease activity
Muscle strength
Patient and physician assessments of disease activity
Time to meaningful improvement
Locations
This study is being conducted at multiple sites across the United States, including major academic and clinical centers. View more information here.
Important Notes
This study includes a placebo group
The trial is ongoing but not currently enrolling new participants
Findings from this study may help inform future treatment options for myositis
Learn More
View full study details on clinicaltrials.gov here
A Phase 2 Study of Empasiprubart in Adults With Dermatomyositis
Sponsor
argenx
Study Type
Interventional, Phase 2 Randomized, double‑blind, placebo‑controlled
Purpose
This clinical trial is evaluating the safety and efficacy of empasiprubart, an investigational therapy, in adults with dermatomyositis. Participants will receive either empasiprubart or placebo for 25 weeks, followed by a long‑term safety follow‑up period.
Who Can Participate
Adults 18 years and older with:
A confirmed diagnosis of dermatomyositis or juvenile‑onset dermatomyositis
Active muscle disease and at least mild skin disease
Stable background DM treatment
Required vaccinations completed before starting the study
Key exclusions include: other inflammatory myopathies, significant interstitial lung disease, active infections, recent malignancy, or major uncontrolled medical conditions.
Study Duration
Approximately 92 weeks total
25‑week treatment period
65‑week safety follow‑up
Primary Outcomes
Incidence of adverse events
Discontinuation due to adverse events
Secondary Outcomes
Total Improvement Score (TIS) based on ACR/EULAR criteria
Locations
28 global sites, including:
United States: California, Florida, Ohio
Europe: Greece, Italy, Poland, Spain
Georgia (country)
Moldova
More Information
View full study details on clinicaltrials.gov here
Frequently Asked Questions
Clinical trials involve potential risks and benefits. A study doctor will help you understand what’s involved and whether a trial may be right for you.
Who Can Join?
Eligibility varies by study. Some are open to children or adults; others focus on treatment-resistant (refractory) or long-standing disease. Contact the study team to learn more.
How to Get Started
- Contact the Study Team – They can confirm eligibility and answer your questions
- Talk to Your Doctor – Share trial info and discuss whether a study may be appropriate
- Stay Informed – Trials evolve quickly; check back regularly for updates or new opportunities
- Contact James Tealy, Cure JM’s Manager of Family & Clinician Education – James is a JM parent and can explain things in a simple, easy-to-understand way.
Contact James at James.Tealy@curejm.org