Condition

Rhabdomyosarcoma clinical trials and research

• 29Clinical trials
• 47NIH research projects
• 24Countries

Newest Rhabdomyosarcoma clinical trials

All recruiting trials (29)

• Methionine-Reduced Diet in Solid Tumors (early-phase)

  A small pilot study will test whether a methionine-reduced diet can change blood levels in adults with various solid tumors who are receiving standard cancer therapy.

  Recruiting Early Phase 1 University of California, Irvine · Orange, California · NCT07628634

• Becotatug Vedotin for EGFR-positive relapsed, refractory, or metastatic pediatric solid tumors

  This trial will test Becotatug Vedotin, an EGFR-targeted antibody-drug conjugate, to see if it is safe and helps children aged 2–18 with EGFR-positive relapsed, refractory, or metastatic solid tumors.

  Recruiting Phase 1 Sun Yat-sen University · Guangzhou, Guangdong · NCT07584499

• Adaptive Proton Therapy for Pediatric Solid Tumors and Hodgkin Lymphoma

  This study tests a regular imaging approach to see if proton therapy plans for children with solid tumors or Hodgkin lymphoma should be updated during treatment.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT07514819

• Higher-dose chemo over a shorter time vs lower-dose chemo with maintenance in newly diagnosed intermediate-risk rhabdomyosarcoma

  We’re testing whether a shorter, higher-dose chemo plan works better than a longer, maintenance chemo plan for kids and young people with intermediate-risk rhabdomyosarcoma.

  Recruiting at 85 sites Phase 3 Children's Oncology Group · Birmingham, Alabama and 86 other locations · NCT07466316

• Personalized B7-H3 CAR T cells combined with PRAME antigen-specific T cells for relapsed embryonal tumors

  This trial tests a combined personalized B7-H3 CAR T cell and PRAME-specific T cell infusion after lymphodepleting chemotherapy in children and young adults with relapsed or refractory embryonal tumors.

  Recruiting Phase 1 Children's National Research Institute · Washington D.C., District of Columbia and 1 other locations · NCT07172958

• GPC3-targeted CAR T cells armored with IL-15 and IL-21 for pediatric non‑CNS solid tumors

  This treatment will try personalized CAR T cells that target GPC3 and are engineered to make IL‑15 and IL‑21 in children and young adults with relapsed or refractory non‑CNS solid tumors that express GPC3.

  Recruiting Phase 1 Seattle Children's Hospital · Seattle, Washington · NCT07148050

• Personalized neoantigen mRNA vaccine for rhabdomyosarcoma

  A personalized mRNA vaccine, given with an anti-PD-1 antibody and CpG adjuvant, is being tested in patients with refractory rhabdomyosarcoma to see if it can help prolong life.

  Recruiting Early Phase 1 Chinese PLA General Hospital · Beijing, Beijing Municipality · NCT06932861

• Identifying molecular signatures in young patients with rhabdomyosarcoma

  This study is trying to find specific proteins in tumor samples from young patients with rhabdomyosarcoma to help understand their cancer better and improve treatment options.

  Recruiting Lille University · Lille, Nord · NCT06924463

• FGFR4-directed CAR T cell therapy for children and young adults with recurrent or refractory rhabdomyosarcoma

  This trial will try a personalized CAR T cell treatment that targets the FGFR4 protein for people aged 3–39 whose rhabdomyosarcoma has returned or not responded after at least two prior treatments.

  Recruiting Phase 1 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT06865664

• Stratified treatment for pediatric rhabdomyosarcoma based on risk factors

  This study is testing a new treatment plan for children with rhabdomyosarcoma to see if combining chemotherapy and radiotherapy helps improve survival based on their risk levels.

  Recruiting Phase 3 Sun Yat-sen University · Guangzhou, Guangdong · NCT06836492

• Targeted radiation therapy for young patients with metastatic sarcoma

  This study is testing if a special type of radiation therapy can help young patients with metastatic sarcoma live longer and have fewer health issues compared to those who don’t receive it.

  Recruiting at 3 sites Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Washington D.C., District of Columbia and 2 other locations · NCT06796543

• Testing a new drug combination for treating relapsed or refractory sarcomas in adolescents and young adults

  This study is testing a new combination of drugs to see if it can help adolescents and young adults with tough-to-treat sarcomas feel better and improve their chances of recovery.

  Recruiting Phase 1/2 Dana-Farber Cancer Institute · Boston, Massachusetts and 2 other locations · NCT06709495

• Treatment of rare advanced tumors with a combination of drugs

  This study is testing a new combination of drugs to see if it can help people with rare advanced tumors live longer and feel better.

  Recruiting Phase 2 Fudan University · Shanghai, Shanghai Municipality · NCT06684327

• Using Ivocizumab to treat various advanced rare tumors

  This study is testing if a new treatment called Ivocizumab can help people with different types of advanced rare tumors feel better and live longer.

  Recruiting Phase 2 Fudan University · Shanghai, Shanghai Municipality · NCT06683846

• Alpha/beta T‑Cell and B‑Cell Depletion plus Zoledronic Acid for Pediatric and Young Adult Solid Tumors

  This study will try removing alpha/beta T cells and B cells from donor stem cell grafts and giving zoledronic acid to see if it helps children and young adults (6 months to 25 years) with relapsed or high‑risk solid tumors tolerate allogeneic transplant and reduce relapse.

  Recruiting Phase 1/2 University of Florida · Gainesville, Florida · NCT06625190

• Using silmitasertib with chemotherapy for relapsed solid tumors

  This study is testing a new drug called silmitasertib combined with chemotherapy to see if it helps people with relapsed solid tumors feel better and manage their cancer.

  Recruiting at 24 sites Phase 1/2 Milton S. Hershey Medical Center · Birmingham, Alabama and 23 other locations · NCT06541262

• Combining Pucotenlimab with standard chemotherapy for pediatric rhabdomyosarcoma

  This study is testing if adding a new drug called Pucotenlimab to standard chemotherapy can help children and teens with a type of cancer called rhabdomyosarcoma.

  Recruiting Phase 1/2 Sun Yat-sen University · Guangzhou, Guangdong · NCT06456892

• Lutetium Lu 177 Edotreotide dose-finding for children with SSTR-positive tumors

  This test gives Lutetium Lu 177 Edotreotide to children ages 2 to under 18 with somatostatin receptor–positive tumors to find a safe pediatric dose and see how the drug behaves in the body.

  Recruiting at 9 sites Phase 1 ITM Solucin GmbH · Palo Alto, California and 8 other locations · NCT06441331

• IL-15 and IL-21–armored GPC3-CAR T cell therapy for adults with GPC3-positive solid tumors

  This trial tests whether CAR T cells engineered to target GPC3 and fortified with IL-15 and IL-21 can treat adults with GPC3-positive liver and other solid tumors.

  Recruiting Phase 1 Baylor College of Medicine · Houston, Texas · NCT06198296

• Personalized cancer vaccine for patients with fusion-driven sarcomas

  This study is testing a personalized cancer vaccine for patients with certain types of sarcomas to see if it can help their immune system fight the cancer and improve their chances of living longer.

  Recruiting Phase 1/2 University Hospital Tuebingen · Essen and 3 other locations · NCT06094101

• Treatment for children with newly diagnosed rhabdomyosarcoma using targeted chemotherapy

  This study is testing a new combination of chemotherapy drugs to see if it helps children with newly diagnosed rhabdomyosarcoma live longer and healthier lives.

  Recruiting at 2 sites Phase 2 St. Jude Children's Research Hospital · Palo Alto, California and 2 other locations · NCT06023641

• Using HER2 CAR T cells with checkpoint inhibitors for advanced sarcoma

  This study is testing a new treatment that combines personalized CAR T cells with immune boosters to see if it helps people with advanced HER2-positive sarcoma.

  Recruiting Phase 1 Baylor College of Medicine · Houston, Texas · NCT04995003

• B7-H3 CAR T-cell therapy for pediatric solid tumors

  This study is testing a new type of immune therapy using specially modified T cells to see if it can help children with tough-to-treat solid tumors.

  Recruiting Phase 1 St. Jude Children's Research Hospital · Memphis, Tennessee · NCT04897321

• Using engineered T cells to treat pediatric solid tumors

  This study is testing a new treatment using specially modified immune cells to see if they can help children with certain types of solid tumors, like liver cancer and rhabdomyosarcoma, feel better.

  Recruiting Phase 1 Baylor College of Medicine · Houston, Texas · NCT04715191

• Treatment options for children and adults with rhabdomyosarcoma

  This study is testing different treatment options for children and adults with rhabdomyosarcoma to see which ones work best for newly diagnosed and relapsed cases.

  Recruiting at 120 sites Phase 1/2 University of Birmingham · Brisbane and 127 other locations · NCT04625907

• Combining vorinostat with chemotherapy for relapsed solid tumors and CNS cancers

  This study is testing if adding a drug called vorinostat to chemotherapy can help people with relapsed solid tumors and brain cancers feel better and improve their treatment outcomes.

  Recruiting Phase 1 New York Medical College · Valhalla, New York · NCT04308330

• Investigating genetic factors in childhood cancer

  This study is trying to find out if certain genes passed down in families can increase the risk of childhood cancer by looking at samples from kids who have had cancer and their relatives.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT03050268

• Transplantation with modified stem cells and Zoledronate for pediatric cancers

  This study is testing a new way to use modified stem cells and a drug called Zoledronate to see if it can safely help children with tough-to-treat cancers.

  Recruiting Phase 1 University of Wisconsin, Madison · Madison, Wisconsin · NCT02508038

• Combination therapy with MM-398 liposome irinotecan and cyclophosphamide in children with solid tumors

  This study tests whether a higher dose of MM-398 (liposomal irinotecan) can be given safely when used with cyclophosphamide in children with recurring or treatment-refractory solid tumors.

  Recruiting at 5 sites Phase 1 South Plains Oncology Consortium · Cincinnati, Ohio and 4 other locations · NCT02013336

NIH-funded research (newest 40 of 47)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Targeting the PAX3-FOXO1 protein in a deadly childhood cancer

  This study is looking at a specific protein that helps a tough childhood cancer called alveolar rhabdomyosarcoma, with the goal of finding new ways to treat kids who have this illness.

  NIH research Duke University · Durham, United States · NIH-9833682

• Targeting how RMS cells fail to mature to slow or stop pediatric muscle cancer spread

  A project to understand why a pediatric muscle cancer called rhabdomyosarcoma spreads, and to test ideas that could slow or stop it by helping cancer cells mature properly.

  NIH research Purdue University · West Lafayette, United States · NIH-11609892

• Genetically engineered mouse model to study and treat high-risk pediatric rhabdomyosarcoma

  A research project aims to create mouse models that mimic high-risk pediatric rhabdomyosarcoma and test therapies that target the NOTCH1 pathway to slow or stop tumor growth.

  NIH research University of Oklahoma Hlth Sciences Ctr · Oklahoma City, United States · NIH-11592251

• Understanding infantile rhabdomyosarcoma biology and new treatment targets

  A new grant is studying why infantile rhabdomyosarcoma develops and what drugs or strategies could specifically target it in young children.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11473226

• Targeting CLK2 to Fight Rhabdomyosarcoma

  Aims to see if blocking CLK2 can weaken rhabdomyosarcoma cancer cells, potentially offering a new treatment for kids and young adults.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11454705

• Epitranscriptomic Crosstalk in Alveolar Rhabdomyosarcoma

  A pediatric cancer study that looks at how chemical marks on messenger RNA may influence the behavior of alveolar rhabdomyosarcoma and point toward newer treatment strategies.

  NIH research University of Minnesota · Minneapolis, United States · NIH-11438954

• Understanding infantile rhabdomyosarcoma biology and new treatment targets

  A study to learn why a rare cancer in very young children grows and to find ways to stop it with targeted therapies.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11411145

• Chemical ways to block PAX3-FOXO1 in a dangerous childhood cancer

  A study to find small molecules that can interfere with the cancer-driving PAX3-FOXO1 fusion protein in alveolar rhabdomyosarcoma, aiming to slow or stop tumor growth in affected children.

  NIH research Massachusetts Institute of Technology · Cambridge, United States · NIH-11401672

• Targeting mutant-MYOD1 driven rhabdomyosarcoma in kids

  Aiming to find new treatments for a deadly childhood cancer driven by MYOD1 mutations by building a fast, collaborative pipeline from lab discoveries to patient care.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11365879

• Explore and target the epigenetic weak spots in PAX3-FOXO1 driven rhabdomyosarcoma

  Researchers are studying how a specific cancer-causing fusion protein changes epigenetics in rhabdomyosarcoma to find new therapies for affected children and teens.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11362175

• Targeting mTORC1 to treat fusion-positive rhabdomyosarcoma in children

  Aims to slow or stop the cancer-causing activity of PAX3-FOXO1 in childhood rhabdomyosarcoma by using a new class of drugs that block protein production at the start of protein-making.

  NIH research University of California, San Francisco · San Francisco, United States · NIH-11357692

• Chromatin regulators of stemness and therapy resistance in rhabdomyosarcoma

  A study to understand why rhabdomyosarcoma tumors resist treatment and how stem-like cells contribute to relapse, with findings that could guide better therapies for affected children.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11321227

• What drives aggressive fusion-negative rhabdomyosarcoma and its spread

  Looking at the cancer stem cells that make fusion-negative rhabdomyosarcoma grow and spread so new treatments can stop them.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11312633

• Targeted drug strategies for RAS-driven childhood rhabdomyosarcoma

  This project will try combinations of drugs that block RAF-MEK signaling to treat children with RAS-mutant embryonal rhabdomyosarcoma.

  NIH research Oregon Health & Science University · Portland, United States · NIH-11303331

• Why MYOD1‑mutant rhabdomyosarcoma acts more aggressively

  This project looks at whether a specific MYOD1 gene change (L122R) makes some children's rhabdomyosarcoma grow faster and resist treatment.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11286812

• Immature cancer cells in childhood rhabdomyosarcoma

  Researchers are using advanced gene-level and computer analyses to find immature tumor cell groups that may drive relapse in children with rhabdomyosarcoma.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11284094

• Blocking the AVIL gene in rhabdomyosarcoma

  Using new drugs that block the AVIL cancer gene to try to stop rhabdomyosarcoma growth in children, adolescents, and young adults.

  NIH research University of Virginia · Charlottesville, United States · NIH-11258871

• How the protein TAK1 may drive rhabdomyosarcoma growth

  This work tests whether blocking the protein TAK1 can stop rhabdomyosarcoma cells from growing and help them turn into normal muscle, with the goal of helping children and adults with RMS.

  NIH research University of Houston · Houston, United States · NIH-11258041

• New mouse models that mirror high-risk childhood rhabdomyosarcoma

  Researchers are creating mouse versions of high-risk childhood rhabdomyosarcoma to test whether blocking the NOTCH1 pathway can slow tumor growth.

  NIH research Sanford Research/usd · Sioux Falls, United States · NIH-11247133

• Targeting PAK4 to treat high-risk rhabdomyosarcoma in children and teens

  This project looks at whether blocking a protein called PAK4 can help existing drugs work better for children and adolescents with aggressive rhabdomyosarcoma.

  NIH research Emory University · Atlanta, United States · NIH-11233151

• Investigating how certain genes affect treatment resistance in childhood cancer

  This study is looking at rhabdomyosarcoma, a type of cancer that affects kids and can be tough to treat, to find out why some tumors don't respond well to standard treatments, with the hope of discovering better ways to help kids fight this cancer.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-11231976

• Infant rhabdomyosarcoma — finding what drives it and new ways to target it

  Researchers will use animal models and patient tumor samples to find the molecular drivers and potential drug targets for infant rhabdomyosarcoma in young children.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11211683

• Identifying weaknesses in a specific type of childhood cancer

  This study is looking at a tough type of cancer called rhabdomyosarcoma that affects kids and young adults, and it aims to find new ways to treat it by understanding the genetic changes caused by a specific protein that drives the disease.

  NIH research Duke University · Durham, United States · NIH-11200239

• New drugs to block the PAX3‑FOXO1 driver in fusion‑positive alveolar rhabdomyosarcoma

  Small‑molecule approaches aimed at stopping the PAX3‑FOXO1 cancer driver for children with fusion‑positive alveolar rhabdomyosarcoma.

  NIH research Massachusetts Institute of Technology · Cambridge, United States · NIH-11187191

• Infantile rhabdomyosarcoma: causes and new treatment targets

  This project looks into the genes behind aggressive infant rhabdomyosarcoma to find possible drug targets for babies and young children.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11145032

• Investigating ways to reduce a cancer gene's impact in childhood muscle cancer.

  This study is looking at a tough childhood muscle cancer called alveolar rhabdomyosarcoma and how a specific gene makes it harder to treat with chemotherapy; the researchers want to see if a drug called entinostat can help make chemotherapy work better by blocking that gene, which could lead to better outcomes for kids

  NIH research Children's Cancer Therapy Develop/inst · Beaverton, United States · NIH-11136866

• Improving Treatments for Childhood Rhabdomyosarcoma

  This research looks for new ways to stop a type of childhood cancer called rhabdomyosarcoma from growing and becoming resistant to current medicines.

  NIH research University of California, San Francisco · San Francisco, United States · NIH-11126020

• Understanding and targeting a weakness in childhood rhabdomyosarcoma

  This project aims to find new ways to treat a serious childhood cancer called rhabdomyosarcoma by understanding its unique weaknesses.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11122258

• Understanding the causes of Rhabdomyosarcoma and its aggressive behavior

  This study is looking at a tough type of cancer called fusion-negative rhabdomyosarcoma to find out how certain cancer cells keep growing and spreading even when treated, with the goal of creating better treatments to help patients live longer and healthier lives.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11078783

• Understanding how certain cells turn into rhabdomyosarcoma, a type of cancer.

  This study is looking into how certain cells that aren't muscle cells can turn into rhabdomyosarcoma, a type of aggressive cancer that affects kids, to help us understand why this happens and find better ways to treat it.

  NIH research St. Jude Children's Research Hospital Graduate School of Biomedical Sciences, LLC · Memphis, United States · NIH-11071727

• Investigating the biology and treatment targets of infantile rhabdomyosarcoma

  This study is looking into a type of cancer called infantile rhabdomyosarcoma that affects young kids, and it's trying to find out how certain genes, especially one called VGLL2-NCOA2, might help us discover better treatments beyond just chemotherapy and surgery.

  NIH research Research Inst Nationwide Children's Hosp · Columbus, United States · NIH-11065573

• Investigating how TAK1 signaling affects Rhabdomyosarcoma growth

  This study is looking at how a protein called TAK1 affects the growth of Rhabdomyosarcoma, a type of cancer that mostly affects kids and young adults, to see if blocking this protein can help the cancer cells become more like normal cells and possibly shrink the tumors.

  NIH research University of Houston · Houston, United States · NIH-11062176

• Creating a mouse model to study and treat high-risk rhabdomyosarcoma in children

  This study is creating special mice that have a type of cancer similar to what some kids get, called rhabdomyosarcoma, to help scientists learn more about what causes it and how it grows, with the hope of finding better treatments for children facing this tough illness.

  NIH research Sanford Research/usd · Sioux Falls, United States · NIH-11044502

• Understanding the aggressive nature of a specific type of pediatric cancer.

  This study is looking at a specific change in a gene that makes rhabdomyosarcoma, a type of cancer in kids, harder to treat, and it aims to find new ways to help kids with this cancer by understanding how this change affects tumor growth and treatment resistance.

  NIH research Massachusetts General Hospital · Boston, United States · NIH-11032733

• Understanding immature tumor cells in pediatric rhabdomyosarcoma

  This study is looking at how certain early-stage cells in rhabdomyosarcoma, a cancer that mainly affects kids, behave during treatment to help understand why some children might have their cancer come back, with the hope of creating better, personalized treatments for them.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11031369

• Developing targeted therapies for children with RAS-driven rhabdomyosarcoma

  This study is working to find better and safer treatments for kids with a tough type of cancer called high-risk rhabdomyosarcoma, especially those with certain gene changes, by exploring new drug combinations that could help them fight the disease more effectively.

  NIH research Oregon Health & Science University · Portland, United States · NIH-11013863

• Understanding why pediatric rhabdomyosarcoma tumors don't mature properly

  This study is looking into why rhabdomyosarcoma, a type of cancer that affects kids, doesn't develop like normal muscle cells do, and it's trying to find new ways to help these tumors grow properly so that treatments can work better and have fewer side effects for children.

  NIH research St. Jude Children's Research Hospital · Memphis, United States · NIH-11008924

• Targeting a new cancer gene in rhabdomyosarcoma

  This study is looking at a new way to treat rhabdomyosarcoma, a common soft-tissue cancer in kids, by targeting a gene called AVIL that helps the cancer grow, with the hope of finding better treatments for children facing this tough diagnosis.

  NIH research University of Virginia · Charlottesville, United States · NIH-11001485

• Finding ways to target a specific protein in a childhood cancer

  This study is looking for new ways to help kids with a tough type of cancer called fusion-positive alveolar rhabdomyosarcoma by finding special chemicals that can block a protein that helps the cancer grow.

  NIH research Massachusetts Institute of Technology · Cambridge, United States · NIH-10987609

• Targeting a specific cancer driver in a type of childhood cancer

  This study is looking at a type of childhood cancer called rhabdomyosarcoma, especially the cases caused by a specific gene change, and it's exploring how a new kind of medicine can help fight this cancer and improve treatment for kids who have it.

  NIH research University of California, San Francisco · San Francisco, United States · NIH-10979766

Related topics

Quick answers

How many Rhabdomyosarcoma clinical trials are recruiting right now?

29, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 24 countries. The most sites are in United States (20 trials), China (6 trials), France (3 trials), Canada (2 trials), Spain (2 trials). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1: 12, Phase 1/2: 6, Phase 2: 3, Phase 3: 2. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

Find the studies that fit you

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