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 Institutos Nacionales de Salud (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.
Opening Soon
Deucravacitinib (JAK-inhibitor) in JDM, DM
“DEFEAT DM/JDM” is an “open label” trial which means all participants receive deucravacitinib. There is no placebo.
Contact Info:
- Contact info will be provided once the study begins recruiting
- If you have a question now, please contact James.Tealy@curejm.org
It is a Phase 2 clinical trial, which means the trial is testing how well the drug works in a smaller group of participants to help determine if the treatment shows enough promise to move on to larger Phase 3 studies.
Who Can Participate:
- Adults (18 and older) who have active JDM that have not responded well to other treatments.
- More specific requirements will be reviewed by the study physician and are listed aquí.
A study physician will determine final eligibility
How It Works:
Deucravacitinib works by calming the overactive immune system that causes inflammation in conditions like dermatomyositis and juvenile dermatomyositis. It does this by blocking TYK2, a key signal that tells the immune system to stay in attack mode. By turning down that signal, the drug can help reduce symptoms like muscle weakness, fatigue, and rash.
What Does Participation Involve:
- This study takes place at the Institutos Nacionales de Salud (NIH) in Bethesda, MD
- 9 clinic visits over 7 months, with each visit possibly lasting up to 6 hours
- You’ll take a pill (deucravacitinib) twice a day at home
- The study includes regular tests and questionnaires about symptoms and daily function
- Ask the study team about travel support or reimbursement
Can Participants Keep Taking the Drug?
Yes — if the drug helps, participants may be able to extend treatment beyond the initial 24 weeks. The study physician will discuss this with you.
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 aquí
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
Where:
The trial is enrolling at multiple locations across the U.S., including:
Sites enrolling pediatric patients age 6-26*
- Chicago, IL (University of Chicago & Northwestern)
- San Francisco, CA (UCSF)
- Philadelphia, PA (CHOP) – coming soon!
- More locations listed aquí
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
- More locations listed aquí
*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
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
- Correo electrónico: information.center@astrazeneca.com
View all Sites in the U.S. and Abroad aquí
One of the sites is George Washington University Medical Center, where many JM patients are treated:
- PI: Dr. Rodolfo Curiel
- Contact: Dr. Joaquin Calderon (jcalderon@mfa.gwu.edu | (202) 741-2632)
- Contact: Haider Nadhem (hnadhem@mfa.gwu.edu | (202) 741-3582)
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 aquí
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
Where:
- All visits take place at GW Medical Faculty Associates (Washington, D.C.)
Participant Benefits:
- Study-related care and treatment at no cost
- Some travel support may be available
CALiPSO-1 Clinical Trial Evaluating CAR-NK Cell Therapy
A phase 1 clinical trial testing CNTY-101, a treatment using CAR-NK (natural killer) cells to target harmful immune cells.
Contact Info:
Correo electrónico: trial_response_century@centurytx.com
¿Quién puede optar?
You may qualify if:
- You are 17 years of age or older
- You have a confirmed diagnosis of DM, PM, JDM, or JPM
- Your condition is considered refractory (not responding to traditional treatment)
- Learn more aquí
A study physician will determine final eligibility.
Where Is the Study Taking Place?
Currently Enrolling Sites:
- Los Angeles, CA (USC)
- Chicago, IL (Lurie Children’s Hospital)
- Houston, TX (Texas Children’s Hospital)
- Salt Lake City, UT (Primary Children’s Hospital)
Coming Soon:
- Sacramento, CA (UC Davis)
- View all study locations aquí
What is CNTY-101?
CNTY-101 is a CAR-NK cell product designed to target CD19, a marker on harmful B cells involved in autoimmune activity.
Interested in Learning More?
Download the CALiPSO-1 Study Flyer
Learn more about the trial aquí.
- All participants Trial will receive CAR-NK cell therapy — there is no placebo group in this study
Preguntas frecuentes
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.
What Outcomes Can I Expect
Every patient is different. Some participants may see improvements in muscle strength, rash, or inflammation. The study doctor will explain what to expect and monitor you closely throughout the trial.
What if I Change My Mind?
You can stop taking part at any time, for any reason.
What are the costs of taking part?
In most cases, it will not cost you anything to take part in this study. The study drug, study visits, tests and assessments are all free of charge.
What happens if I can’t attend a visit?
Contact your study and they will arrange a new visit for you.
Why Consider a Clinical Trial?
- You may gain access to investigational treatments not yet widely available
- Help uncover better ways to manage or treat juvenile myositis (JM)
- Receive expert monitoring and care during the trial
- Contribute to research that could benefit others in the JM community
What Are Clinical Trial Phases?
- Phase 1: Tests whether a treatment is safe and how it behaves in the body (usually small groups)
- Phase 2: Tests how well the treatment works and continues to monitor safety
- Phase 3: Compares the new treatment to current standard treatments in larger groups of people
All trials are reviewed and monitored by medical ethics boards called Institutional Review Boards (IRBs).
How to Get Started
- Contactar con el equipo del estudio – 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