Hope for rhabdomyosarcoma: vitamin a derivative joins chemo in new trial
NCT ID NCT07355855
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 2 trial tests whether adding all-trans retinoic acid (a vitamin A derivative) to standard VAC chemotherapy improves outcomes for 106 people aged 14-60 with intermediate-to-high-risk rhabdomyosarcoma, a rare muscle cancer. Participants will receive the drug combo in 21-day cycles, and researchers will track survival, tumor response, and side effects. The study is not yet recruiting.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- all-trans retinoic acid (ATRA) combined with VAC chemotherapy (vincristine, dactinomycin, cyclophosphamide)
- What this could lead to
- If this works, it could improve survival and disease control for people with intermediate-to-high-risk rhabdomyosarcoma, offering a more effective treatment option.
- What could go wrong
- This is an early Phase 2 trial with only 106 participants, so results may not apply to everyone. The combination may cause side effects like those from standard chemotherapy, and it is not yet proven to be better than existing treatments.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 106 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jan 2026
An estimate. Start dates often move.
- Expected to finish
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Jan 2029
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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14 to 60 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Age ≥ 14 years old and ≤ 60 years old; 2. Histologically confirmed medium to high risk rhabdomyosarcoma (excluding pleomorphic rhabdomyosarcoma); 3. The physical fitness status score of the Eastern Cancer Collaboration Group (ECOG) is 0-1; 4. Have not received any anti-tumor drug treatment in the past; 5. Expected survival time ≥ 3 months; 6. Possess sufficient organ and bone marrow function, with laboratory test values meeting the following requirements within 7 days prior to enrollment (no blood components, cell growth factors, albumin, or other corrective treatment drugs are allowed within 14 days prior to obtaining laboratory tests), as follows Blood routine: Absolute neutrophil count (ANC) ≥ 1.5 × 109/L, platelet count (PLT) ≥ 100 × 109/L, hemoglobin (HGB) ≥ 100 g/L (no transfusion or erythropoietin dependence within 14 days) Liver function: serum total bilirubin ≤ 1.25 times the upper limit of normal (ULN); ALT and AST ≤ 2.5 x ULN (≤ 5x ULN for patients with liver metastases); Serum albumin ≥ 30 g/L; Alkaline phosphatase (ALP) ≤ 5 × ULN. Renal function: Serum creatinine (Cr) ≤ 1.25 × ULN, or creatinine clearance rate ≥ 60 mL/min (using the standard Cockcroft Gault formula): Urine routine results show urinary protein\<2+; For patients whose baseline urine routine test shows urinary protein ≥ 2+, 24-hour urine collection should be performed with a 24-hour urine protein quantification of\<1g. Coagulation function: International normalized ratio (INR) or prothrombin time (PT) ≤ 1.5 times ULN; If the subject is receiving anticoagulant therapy, as long as the INR is within the intended range of use of the anticoagulant drug. 7. For female subjects of childbearing age, a urine or serum pregnancy test should be conducted 3 days before receiving the first study drug and the result should be negative; 8. Participants and their sexual partners are required to use a medically approved contraceptive measure (such as intrauterine devices, birth control pills, or condoms) during the study treatment period and within 6 months after the end of the study treatment period. Exclusion Criteria: 1. Previously received anti-tumor treatment other than surgery and radiation therapy for any malignant tumor; 2. Subjects who cannot accept or tolerate this chemotherapy regimen for various reasons; 3. Biopsy confirmed a patient with bone marrow infiltration; 4. Patients who have undergone major surgical procedures unrelated to medium to high risk rhabdomyosarcoma within the 4 weeks prior to enrollment, or who have not fully recovered from such surgical procedures; 5. Serious heart disease or discomfort, including but not limited to the following diseases: * Diagnosed history of heart failure or systolic dysfunction (LVEF\<50%); * High risk uncontrolled arrhythmias, such as atrial tachycardia, resting heart rate\>100bpm, significant ventricular arrhythmias (such as ventricular tachycardia), or higher-level atrioventricular block (i.e. Mobitz II second or third degree atrioventricular block); * Angina requiring treatment with anti angina drugs; * Clinically significant heart valve disease; * ECG shows transmural myocardial infarction; * Poor control of hypertension (systolic blood pressure\>180mmHg and/or diastolic blood pressure\>100mmHg) 6. Individuals with a known history of allergies to the components of this medication regimen; 7. The researcher believes that the patient is not suitable to participate in any other circumstances of this study.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Fudan university cancer hospital
Shanghai, China
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