Childhood RhabdomyosarcomaPhase 1/2NCT03868852
Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children
A Phase 1/2 study testing Apatinib mesylate tablets and Three-dimensional conformal intensity modulation in people with Childhood Rhabdomyosarcoma, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine at 1 site in China.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of radiotherapy combined with apatinib mesylate in the treatment of rhabdomyosarcoma in children. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Childhood Rhabdomyosarcoma
- What is being tested?
- Apatinib mesylate tablets and Three-dimensional conformal intensity modulation
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2019to 1 Apr 2020
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be ≥ 3 and ≤ 18 years of age.
- The predicted survival period is more than 3 months.
- According to RECIST version 1.1, there is at least one measurable lesion.
- Postoperative pathological stage was group II-IV.
- Patients have not received radiotherapy before, and can accept chemotherapy and surgical treatment.
- No other anticancer therapy should be used during radiotherapy.
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- The main organs are functioning normally, which meets the following criteria:
- Blood routine examination standards should be met: (no blood transfusion within 14 days) A.HB>90g/L; B.ANC>1.5*109/L; C.PLT>80*109/L
- Biochemical tests should meet the following criteria:
- BIL < 1.25 times normal upper limit (ULN); B. ALT and AST < 2.5 ULN; C. Serum Cr < 1 ULN, endogenous creatinine clearance > 50 ml/min
- Subjects volunteered to participate in this study, patients or legal guardians signed informed consent through patient consent, good compliance, with follow-up.
- Doctors believe that treatment can benefit patients.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
The Department of Radiation Oncology, Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, 200092
Mawei Jiang, MD, 02125078635, 13524527495@163.com
Mawei Jiang, MD
About this study
The purpose of this study is to evaluate the efficacy and safety of radiotherapy combined with apatinib mesylate in the treatment of rhabdomyosarcoma in children.
Read the detailed description
This study is a Interventional study. The target group was 3-18 years old, and the pathological stage was group II-IV recurrence or distant metastasis. A total of 48 patients were enrolled in this study. The treatment was radiotherapy combined with apatinib. The main outcome measures were ORR, DCR , OS , quality of life and drug safety. The main safety indicators were vital signs, laboratory indicators, adverse events (AE), and severe adverse events (SAE). This study is expected to be enrolled for 6 months.
- Conditions
- Childhood Rhabdomyosarcoma
- Being tested
- Apatinib mesylate tablets, Three-dimensional conformal intensity modulation
- Keywords
- Radiation Therapy, Rhabdomyosarcoma in Children
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Apatinib mesylate tabletsDrug
Apatinib mesylate tablets were orally administered 250 mg QD from 1 week before radiotherapy to the whole radiotherapy period. (Appatinib is no longer used after radiotherapy)
Three-dimensional conformal intensity modulationRadiation
Also called 3D-IMRT
Three-dimensional conformal intensity modulation (IMRT) technique was used for post-operative radiotherapy. The radiation dose was 45-54 Gy.
| Study group | Role | What they receive |
|---|---|---|
| Radiotherapy plus apatinib mesylate | Experimental | All eligible patients signed informed consent. Three-dimensional conformal intensity modulation (IMRT) technique was used to treat the patients with radiation doses 45 Gy - 54 Gy. All patients took apatinib mesylate tablets 250 mg QD orally from 1 week before radiotherapy to the whole radiotherapy period. They were required to take apatinib mesylate tablets with warm boiling water half an hour after meal. The daily medication time should be as consistent as possible. Receives: Drug: Apatinib mesylate tablets; Radiation: Three-dimensional conformal intensity modulation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective response rate (ORR)
Refers to the proportion of patients whose tumors have shrunk to a certain extent and maintained for a certain period of time, including CR and PR cases. Objective remission of solid tumors was assessed by RECIST 1.1. Subjects must be accompanied by measurable tumor lesions at baseline. According to RECIST 1.1, the criteria for evaluating efficacy are complete remission (CR), partial remission (PR), stability (SD), and progression (PD).
Measured: First analysis will occur 1 month after accrual of all patients.
Secondary outcomes (4)
Disease Control Rate (DCR)
The percentage of patients whose tumors shrink or remain stable for a certain period of time
Measured: First analysis will occur 1 month after accrual of all patients.
Total Survival (OS)
The time from enrollment to death for any reason. For those who had lost interviews before death, the last follow-up time was usually calculated as the time of death.
Measured: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 24 months.
Quality of Life Score
Quality of life score refer to EORTC qlq-c30 (version 3, Chinese version). Quality of Life Questionnare-Core 30 prepared by The European O-rganization for Reasearch and Treatment of Cancer.
EORTC qlq-c30 (V3.0) is the core scale for all cancer patients, with a total of 30 items. Items 29 and 30 are rated on a scale of 1 to 7 according to their answer choices. The other items are divided into four levels: totally not, a little bit, a lot and very much, and are scored on a scale of 1 to 4.
The higher the score of functional domain and general health domain, the better the functional status and quality of life. The higher the score of symptom domain, the more symptoms or problems (the worse the quality of life).
Measured: Weekly assessment , assessed up to 1 month.
Incidence and severity of Adverse Events
The safety of the drug was evaluated by NCI-CTCAE 4.0. Common Terminology Criteria Adverse Events Version 4.0 prepared by National Cancer Institute.
Measured: From date of enrollment until 30 days after the last medication.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2019
- Study start1 Jan 2019
- Primary completion1 Apr 2020 (estimated)
- Study completion1 Apr 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Childhood RhabdomyosarcomaStudies of the same condition, open and under way.
- More trials run by Xinhua Hospital, Shanghai Jiao Tong University School of MedicineThe 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 1/2
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Model description
- Simons two-stage design method requires the enrollment of 13 patients in the first stage. If more than 7 patients fail to reach the expected end point, the first stage fails and the patients do not need to enter the second stage. A total of 48 patients were enrolled. If more than 30 patients did not reach the expected end point, the overall trial failed.
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mawei Jiang, MD, Principal investigator, Xin Hua Hospital affiliated to Shanghai Jiaotong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03868852
- Sponsor's protocol ID
- XH-18-018
- Status last verified
- Feb 2019
- First submitted
- 11 Feb 2019
- Last updated
- 11 Mar 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Vatner R, James CD, Sathiaseelan V, Bondra KM, Kalapurakal JA, Houghton PJ. Radiation therapy and molecular-targeted agents in preclinical testing for immunotherapy, brain tumors, and sarcomas: Opportunities and challenges. Pediatr Blood Cancer. 2021 May;68 Suppl 2:e28439. doi: 10.1002/pbc.28439. Epub 2020 Aug 22.
PubMed 32827353
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Radiotherapy Combined With Apatinib Mesylate in the Treatment of Rhabdomyosarcoma in Children
- ClinicalTrials.gov ID
- NCT03868852
- Acronym / sponsor ID
- XH-18-018
- Conditions
- Childhood Rhabdomyosarcoma
- Keywords
- Radiation Therapy, Rhabdomyosarcoma in Children
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Study start
- 1 Jan 2019 (actual)
- Primary completion
- 1 Apr 2020 (estimated)
- Study completion
- 1 Apr 2020 (estimated)
- First posted
- 11 Mar 2019 (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.
The purpose of this study is to evaluate the efficacy and safety of radiotherapy combined with apatinib mesylate in the treatment of rhabdomyosarcoma in children.
Detailed description
This study is a Interventional study. The target group was 3-18 years old, and the pathological stage was group II-IV recurrence or distant metastasis. A total of 48 patients were enrolled in this study. The treatment was radiotherapy combined with apatinib. The main outcome measures were ORR, DCR , OS , quality of life and drug safety. The main safety indicators were vital signs, laboratory indicators, adverse events (AE), and severe adverse events (SAE). This study is expected to be enrolled for 6 months.
Study design
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Simons two-stage design method requires the enrollment of 13 patients in the first stage. If more than 7 patients fail to reach the expected end point, the first stage fails and the patients do not need to enter the second stage. A total of 48 patients were enrolled. If more than 30 patients did not reach the expected end point, the overall trial failed.
| Arm | Type | Receives |
|---|---|---|
| Radiotherapy plus apatinib mesylate | Experimental | Drug: Apatinib mesylate tablets, Radiation: Three-dimensional conformal intensity modulationAll eligible patients signed informed consent. Three-dimensional conformal intensity modulation (IMRT) technique was used to treat the patients with radiation doses 45 Gy - 54 Gy. All patients took apatinib mesylate tablets 250 mg QD orally from 1 week before radiotherapy to the whole radiotherapy period. They were required to take apatinib mesylate tablets with warm boiling water half an hour after meal. The daily medication time should be as consistent as possible. |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Patients must be ≥ 3 and ≤ 18 years of age.
- The predicted survival period is more than 3 months.
- According to RECIST version 1.1, there is at least one measurable lesion.
- Postoperative pathological stage was group II-IV.
- Patients have not received radiotherapy before, and can accept chemotherapy and surgical treatment.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Apatinib mesylate tabletsApatinib mesylate tablets were orally administered 250 mg QD from 1 week before radiotherapy to the whole radiotherapy period. (Appatinib is no longer used after radiotherapy) | Drug | Radiotherapy plus apatinib mesylate |
| Three-dimensional conformal intensity modulationAlso: 3D-IMRTThree-dimensional conformal intensity modulation (IMRT) technique was used for post-operative radiotherapy. The radiation dose was 45-54 Gy. | Radiation | Radiotherapy plus apatinib mesylate |
Primary outcome
Objective response rate (ORR)
Time frame: First analysis will occur 1 month after accrual of all patients.
Refers to the proportion of patients whose tumors have shrunk to a certain extent and maintained for a certain period of time, including CR and PR cases. Objective remission of solid tumors was assessed by RECIST 1.1. Subjects must be accompanied by measurable tumor lesions at baseline. According to RECIST 1.1, the criteria for evaluating efficacy are complete remission (CR), partial remission (PR), stability (SD), and progression (PD).
4 secondary outcomes
Disease Control Rate (DCR)
Time frame: First analysis will occur 1 month after accrual of all patients.
Total Survival (OS)
Time frame: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 24 months.
Quality of Life Score
Time frame: Weekly assessment , assessed up to 1 month.
Incidence and severity of Adverse Events
Time frame: From date of enrollment until 30 days after the last medication.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2019.
- Derived from this study: PubMed 32827353Vatner R, James CD, Sathiaseelan V, Bondra KM, Kalapurakal JA, Houghton PJ. Radiation therapy and molecular-targeted agents in preclinical testing for immunotherapy, brain tumors, and sarcomas: Opportunities and challenges. Pediatr Blood Cancer. 2021 May;68 Suppl 2:e28439. doi: 10.1002/pbc.28439. Epub 2020 Aug 22.
- 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. This study is not recruiting at the moment.
Mawei Jiang, MD principal investigator
Xin Hua Hospital affiliated to Shanghai Jiaotong University School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Apatinib mesylate tablets and Three-dimensional conformal intensity modulation in people with Childhood Rhabdomyosarcoma, run by Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 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 12 inclusion and 0 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: status not confirmed, as of the sponsor's last update on 11 Mar 2019. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Mawei Jiang, MD (021-25078635, 13524527495@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective response rate (ORR) (time frame: First analysis will occur 1 month after accrual of all patients.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03868852, which also lists 1 linked publication.
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 11 Mar 2019.