RhabdomyosarcomaPhase 2NCT07355855
A Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma
A Phase 2 study testing ATRA+VAC and ATRA+VAC+Anlotinib in people with Rhabdomyosarcoma, run by Fudan University at 1 site in China.
Trial summary
This study is a prospective, multi-cohort, multi-center clinical trial targeting patients with intermediate-to-high-risk rhabdomyosarcoma who have not previously received systemic anti-tumor treatment. It aims to evaluate the efficacy and safety of all-trans retinoic acid combined with VAC chemotherapy. Read more about this study
- Who is it for?
- Ages 14 to 60 years
- Conditions the study is for
- Rhabdomyosarcoma
- What is being tested?
- ATRA+VAC and ATRA+VAC+Anlotinib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 106 participants
- Where
- 1 siteChina
- Study dates
- Jan 2026to Jan 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 14 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age ≥ 14 years old and ≤ 60 years old;
- Histologically confirmed medium to high risk rhabdomyosarcoma (excluding pleomorphic rhabdomyosarcoma);
- The physical fitness status score of the Eastern Cancer Collaboration Group (ECOG) is 0-1;
- Have not received any anti-tumor drug treatment in the past;
- Expected survival time ≥ 3 months;
- 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.
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- 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.
- 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;
- 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.
Who cannot join
- Previously received anti-tumor treatment other than surgery and radiation therapy for any malignant tumor;
- Subjects who cannot accept or tolerate this chemotherapy regimen for various reasons;
- Biopsy confirmed a patient with bone marrow infiltration;
- 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;
- Serious heart disease or discomfort, including but not limited to the following diseases:
- Diagnosed history of heart failure or systolic dysfunction (LVEF<50%);
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- 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)
- Individuals with a known history of allergies to the components of this medication regimen;
- The researcher believes that the patient is not suitable to participate in any other circumstances of this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Fudan university cancer hospital
Shanghai
xin Liu, 021-64175590-88503, jeanettexin@hotmail.com
About this study
Official title: A Multicenter, Multi-cohort, Prospective Phase II Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma
This study is a prospective, multi-cohort, multi-center clinical trial targeting patients with intermediate-to-high-risk rhabdomyosarcoma who have not previously received systemic anti-tumor treatment. It aims to evaluate the efficacy and safety of all-trans retinoic acid combined with VAC chemotherapy.
- Conditions
- Rhabdomyosarcoma
- Being tested
- ATRA+VAC, ATRA+VAC+Anlotinib
- Medical subject headings
- Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATRA+VACDrug
Cohort 1: Intermediate-risk RMS: ATRA+VAC treatment
The specific medication is:
Vincristine (V), administered intravenously, at a dose of 1.5 mg/m2 (with a maximum of 2 mg), on days 1, 8, and 15, every three weeks.
Dactinomycin (A), intravenous infusion, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w.
Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. All-trans-retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd. Capsules, 10mg/capsule, 20mg bid, orally, administered continuously every day, with 21 days constituting one course of treatment. If safety is confirmed, the dosage can be increased to 30mg bid.
ATRA+VAC+AnlotinibDrug
Cohort 2: High risk group RMS+: ATRA+VAC, ATRA+anlotinib maintenance therapy
The specific medication is:
Vincristine (V), static push, 1.5 mg/m2 (maximum not exceeding 2mg), D1, 8, 15, q3w.
Dactinomycin (A), intravenous drip, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w.
Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. After chemotherapy, Anlotinib, 12mg, d1-d14, po, q3w+ATRA maintenance therapy. Both are maintained for a period of time until disease progression or toxicity is intolerable, or for at least 2 years.
All trans retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd., capsule, 10mg/capsule, 20mg bid orally, administered continuously daily for 21 days as a course of treatment. If safety is ensured, the dosage will be increased to 30mg bid.
| Study group | Role | What they receive |
|---|---|---|
| ATRA+VAC | Experimental | Not described Receives: Drug: ATRA+VAC |
| ATRA+VAC+Anlotinib | Experimental | Not described Receives: Drug: ATRA+VAC+Anlotinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
2-year event-free survival (EFS) rate
Measured: up to 2 years
Secondary outcomes (6)
Overall survival (OS)
Measured: up to 2 years
Disease control rate (DCR)
Measured: up to 2 years
Objective response rate (ORR)
Measured: up to 2 years
adverse event
Measured: up to 2 years
Quality of life (Functional Assessment of Cancer Therapy - General)
Measured: up to 2 years
The correlation between the gene status of PAX3/7-FOXO1 and therapeutic efficacy
Subgroup analysis of the effect of PAX3/7-FOXO1 gene expression on the 2-year EFS rate
Measured: up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2026
- Study startJan 2026 (estimated)
- Primary completionJan 2029 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RhabdomyosarcomaStudies of the same condition, open and under way.
- More trials run by Fudan UniversityThe 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
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 106 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhiguo Luo, MD, PhD, Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07355855
- Sponsor's protocol ID
- RMS-ATRA-IIT-01
- Status last verified
- Dec 2025
- First submitted
- 30 Dec 2025
- Last updated
- 21 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Multi-cohort, Prospective Phase II Clinical Study on the Efficacy and Safety of All-trans Retinoic Acid Combined With VAC Regimen in the Treatment of Intermediate-to-high-risk Rhabdomyosarcoma
- ClinicalTrials.gov ID
- NCT07355855
- Acronym / sponsor ID
- RMS-ATRA-IIT-01
- Conditions
- Rhabdomyosarcoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 106 participants (estimated)
- Lead sponsor
- Fudan UniversityUniversity, hospital or other
- Study start
- Jan 2026 (estimated)
- Primary completion
- Jan 2029 (estimated)
- Study completion
- Jan 2029 (estimated)
- First posted
- 21 Jan 2026 (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 is a prospective, multi-cohort, multi-center clinical trial targeting patients with intermediate-to-high-risk rhabdomyosarcoma who have not previously received systemic anti-tumor treatment. It aims to evaluate the efficacy and safety of all-trans retinoic acid combined with VAC chemotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ATRA+VAC | Experimental | Drug: ATRA+VAC |
| ATRA+VAC+Anlotinib | Experimental | Drug: ATRA+VAC+Anlotinib |
Who may qualify
- Ages
- 14 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Age ≥ 14 years old and ≤ 60 years old;
- Histologically confirmed medium to high risk rhabdomyosarcoma (excluding pleomorphic rhabdomyosarcoma);
- The physical fitness status score of the Eastern Cancer Collaboration Group (ECOG) is 0-1;
- Have not received any anti-tumor drug treatment in the past;
- Expected survival time ≥ 3 months;
Key exclusion criteria (13 listed)
- Previously received anti-tumor treatment other than surgery and radiation therapy for any malignant tumor;
- Subjects who cannot accept or tolerate this chemotherapy regimen for various reasons;
- Biopsy confirmed a patient with bone marrow infiltration;
- 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;
- Serious heart disease or discomfort, including but not limited to the following diseases:
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 |
|---|---|---|
| ATRA+VACCohort 1: Intermediate-risk RMS: ATRA+VAC treatment The specific medication is: Vincristine (V), administered intravenously, at a dose of 1.5 mg/m2 (with a maximum of 2 mg), on days 1, 8, and 15, every three weeks. Dactinomycin (A), intravenous infusion, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w. Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. All-trans-retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd. Capsules, 10mg/capsule, 20mg bid, orally, administered continuously every day, with 21 days constituting one course of treatment. If safety is confirmed, the dosage can be increased to 30mg bid. | Drug | ATRA+VAC |
| ATRA+VAC+AnlotinibCohort 2: High risk group RMS+: ATRA+VAC, ATRA+anlotinib maintenance therapy The specific medication is: Vincristine (V), static push, 1.5 mg/m2 (maximum not exceeding 2mg), D1, 8, 15, q3w. Dactinomycin (A), intravenous drip, 1.25mg/m2 (maximum not exceeding 2.5mg), D1, q3w. Cyclophosphamide (C), intravenous infusion, 1200mg/m2, D1, q3w. After chemotherapy, Anlotinib, 12mg, d1-d14, po, q3w+ATRA maintenance therapy. Both are maintained for a period of time until disease progression or toxicity is intolerable, or for at least 2 years. All trans retinoic acid: provided free of charge by Shandong Liangfu Pharmaceutical Co., Ltd., capsule, 10mg/capsule, 20mg bid orally, administered continuously daily for 21 days as a course of treatment. If safety is ensured, the dosage will be increased to 30mg bid. | Drug | ATRA+VAC+Anlotinib |
Primary outcome
2-year event-free survival (EFS) rate
Time frame: up to 2 years
6 secondary outcomes
Overall survival (OS)
Time frame: up to 2 years
Disease control rate (DCR)
Time frame: up to 2 years
Objective response rate (ORR)
Time frame: up to 2 years
adverse event
Time frame: up to 2 years
Quality of life (Functional Assessment of Cancer Therapy - General)
Time frame: up to 2 years
The correlation between the gene status of PAX3/7-FOXO1 and therapeutic efficacy
Time frame: up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jan 2026.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ATRA+VAC and ATRA+VAC+Anlotinib in people with Rhabdomyosarcoma, run by Fudan University at 1 site in China.
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 10 inclusion and 13 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: opening soon, as of the sponsor's last update on 21 Jan 2026. 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 China. 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?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact xin Liu (021-64175590-88503, jeanettexin@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
2-year event-free survival (EFS) rate (time frame: up to 2 years). 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 21 Jan 2026.