Germ Cell TumorPhase 2NCT06470464
Thalidomide Combined With Chemotherapy in the Treatment of Relapsed or Refractory Yolk Sac Tumor
A Phase 2 study testing Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin) in people with Germ Cell Tumor and Yolk Sac Tumor, run by Shandong First Medical University at 1 site in China.
Trial summary
Remarkable progress has been made in treating germ-cell tumor (GCT) through the use of platinum-based regimens. However, part of yolk sac tumor (YST) with cisplatin resistance or recurrence is nevertheless prone to relapse after second-line treatment. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Germ Cell Tumor, Yolk Sac Tumor
- What is being tested?
- Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 11 Nov 2021to 16 Jun 2024
- Phase
- Phase 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 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- The child must have histological evidence of an extracranial malignant germ cell tumor (vitelline sac tumor).
- Children must be no more than 18 years old at the time of study participation.
- The patients were children with vitelline cystic tumor who were resistant to drugs after at least two platinum-containing chemotherapy regimes or whose disease recurred within 3 months after chemotherapy regimes, or who recurred again or repeatedly after treatment with Sirolimus combined with TIC chemotherapy regimes.
- The child must have measurable lesions (recorded according to RECIST criteria) or non-evaluable disease with tumor marker AFP greater than 5 times the upper limit of normal.
- Lansky performance status score ≥50.
- The life expectancy of the child must exceed 6 weeks.
Show all 9Show fewer
- The child must have recovered from the response to all previous anticancer treatments.
- No serious organ dysfunction: normal cardiac function (ejection fraction > 50% or BNP < 2000pg/ml); Liver function: alanine aminotransferase increased less than 5 times the upper limit of normal, bilirubin increased less than 3 times the upper limit of normal; Renal function: creatinine and urea nitrogen levels below the normal range; The white blood cells were greater than 3×109/L, and the platelets were greater than 100×109/L.
- Obtain the informed consent of the guardian and sign the informed consent.
Who cannot join
- Patients with other tumors.
- Heart, brain, liver, kidney and other organ failure patients.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Study of Thalidomide Combined With Chemotherapy in the Treatment of Relapsed or Refractory Yolk Sac Tumor in Children
Remarkable progress has been made in treating germ-cell tumor (GCT) through the use of platinum-based regimens. However, part of yolk sac tumor (YST) with cisplatin resistance or recurrence is nevertheless prone to relapse after second-line treatment. This leaves a gap in effective treatment, which needs to be filled by novel therapeutic approaches. This paper is the first one to report the treatment combining thalidomide with nab-paclitaxel, gemcitabine, and epirubicin (T-TGA) for children with repeated relapsed or refractory yolk sac tumor (rrrYST).
Read the detailed description
Thalidomide (THD) is currently widely used in the treatment of immune rheumatic diseases, blood tumors and solid tumors. Solid tumors are mostly used in the treatment of lung cancer, liver cancer, stomach cancer, colorectal cancer, pancreatic cancer, etc., and are limited to patients with advanced or advanced tumors. The main mechanisms of its action are inhibition of angiogenesis, immune regulation, induction of apoptosis, target teratogenesis and so on. However, whether from animal experiments or clinical studies, thalidomide alone or in combination with chemotherapy has not been seen to have a very effective therapeutic effect or exact clinical application in the treatment of these solid tumors.
According to the latest relevant case analysis reports and clinical trial results abroad, TIP regimen is still the priority recommended treatment regimen in the previous data for children with recurrent and refractory malignant germ cell tumors. However, the combination of Sirolimus and TIC regimen (nab-paclitaxel + isocyclophosphamide + carboplatin) previously explored by our research group can greatly improve the remission rate of recurrent and refractory germinal cell tumors, especially for recurrent and refractory yolk sac tumors, a certain proportion can be cured or the opportunity for surgery and radiotherapy can be obtained to achieve cure. However, there are still some children who are insensitive or progress again after 2 cycles of remission. For these children with multiple relapsed or particularly difficult yolk sac tumors, there is no effective treatment plan in the world at present, and there is no relevant clinical trial. However, through our clinical experience and understanding in the treatment process of these children, The results of thalidomide combined with TGA chemotherapy regimen (nab-paclitaxel + gemcitabine + epirubicin) were satisfactory. Therefore, we hope to evaluate the therapeutic effectiveness and adverse reactions of this regimen through clinical trials, so as to provide an effective treatment option for children with recurrent and refractory yolk sac tumor.
This paper is the first one to report the treatment combining thalidomide with nab-paclitaxel, gemcitabine, and epirubicin (T-TGA) for children with repeated relapsed or refractory yolk sac tumor (rrrYST).
- Conditions
- Germ Cell Tumor, Yolk Sac Tumor
- Being tested
- Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin)
- Keywords
- relapsed or refractory yolk sac tumor, thalidomide
- Medical subject headings
- Neoplasms, Germ Cell and Embryonal, Endodermal Sinus Tumor, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin)Drug
The oral dose of thalidomide is 1.5-2.5mg/kg/d daily (maximum 28g). Each cycle lasts for 21 days (every three weeks), and if treatment parameters are met as described in the study, cycle two and subsequent cycles begin on day 22. Treatment will be discontinued if there is drug-related dose-limiting toxicity or disease progression. Tumor response assessment will be repeated using AFP and LDH every one cycle and imaging examination every two cycles. Children who complete the trial may continue this regimen or undergo surgery or radiation therapy if their disease goes into remission.
| Study group | Role | What they receive |
|---|---|---|
| thalidomide combined with chemotherapy in the treatment of relapsed or refractory yolk sac tumor | Experimental | This study employed an open single-group clinical laboratory design method (Simon Phase II study) to investigate the efficacy and safety of combining thalidomide with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin) in the treatment of children with recurrent or refractory YST. AFP levels were assessed after each course of treatment, while imaging evaluations were conducted every two courses. The remission rate following chemotherapy in this single-group clinical trial was compared to that reported in the literature for treating recurrent or refractory yolk sac tumor, evaluating overall remission rate (ORR) and event-free survival rate (EFS), as well as monitoring and recording chemotherapy-related side effects. Receives: Drug: Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
Proportion of participants achieving partial or complete response，Measurement: Imaging (CT/MRI) every 2 cycles per RECIST 1.1；Serum AFP reduction ≥90% from baseline.
Measured: Every 3 weeks per cycle, up to 6 cycles
Secondary outcomes (1)
Radiographic Tumor Response Evaluation
Assessment of tumor size and characteristics to evaluate treatment response. Method: Contrast-enhanced computed tomography (CT) or magnetic resonance imaging (MRI) scans Outcome Metrics: Complete response (CR), partial response (PR), stable disease (SD), or progressive disease (PD) based on RECIST 1.1 criteria
Measured: Every 6 weeks (i.e., every 2 chemotherapy cycles)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2024
- Study start11 Nov 2021
- Primary completion16 Jun 2024
- Study completion16 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Germ Cell TumorStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shandong First Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jingfu Wang, MD, Study director, Shandong Cancer Hospital and Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06470464
- Sponsor's protocol ID
- HERO2022
- Status last verified
- Nov 2021
- First submitted
- 17 Jun 2024
- Last updated
- 18 Sep 2025
- Results posted
- No
- Data monitoring committee
- Yes
- 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 18 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Thalidomide Combined With Chemotherapy in the Treatment of Relapsed or Refractory Yolk Sac Tumor in Children
- ClinicalTrials.gov ID
- NCT06470464
- Acronym / sponsor ID
- HERO2022
- Conditions
- Germ Cell Tumor, Yolk Sac Tumor
- Keywords
- relapsed or refractory yolk sac tumor, thalidomide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Shandong First Medical UniversityUniversity, hospital or other
- Study start
- 11 Nov 2021 (actual)
- Primary completion
- 16 Jun 2024 (actual)
- Study completion
- 16 Jun 2024 (actual)
- First posted
- 24 Jun 2024 (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.
Remarkable progress has been made in treating germ-cell tumor (GCT) through the use of platinum-based regimens. However, part of yolk sac tumor (YST) with cisplatin resistance or recurrence is nevertheless prone to relapse after second-line treatment. This leaves a gap in effective treatment, which needs to be filled by novel therapeutic approaches. This paper is the first one to report the treatment combining thalidomide with nab-paclitaxel, gemcitabine, and epirubicin (T-TGA) for children with repeated relapsed or refractory yolk sac tumor (rrrYST).
Detailed description
Thalidomide (THD) is currently widely used in the treatment of immune rheumatic diseases, blood tumors and solid tumors. Solid tumors are mostly used in the treatment of lung cancer, liver cancer, stomach cancer, colorectal cancer, pancreatic cancer, etc., and are limited to patients with advanced or advanced tumors. The main mechanisms of its action are inhibition of angiogenesis, immune regulation, induction of apoptosis, target teratogenesis and so on. However, whether from animal experiments or clinical studies, thalidomide alone or in combination with chemotherapy has not been seen to have a very effective therapeutic effect or exact clinical application in the treatment of these solid tumors.
According to the latest relevant case analysis reports and clinical trial results abroad, TIP regimen is still the priority recommended treatment regimen in the previous data for children with recurrent and refractory malignant germ cell tumors. However, the combination of Sirolimus and TIC regimen (nab-paclitaxel + isocyclophosphamide + carboplatin) previously explored by our research group can greatly improve the remission rate of recurrent and refractory germinal cell tumors, especially for recurrent and refractory yolk sac tumors, a certain proportion can be cured or the opportunity for surgery and radiotherapy can be obtained to achieve cure. However, there are still some children who are insensitive or progress again after 2 cycles of remission. For these children with multiple relapsed or particularly difficult yolk sac tumors, there is no effective treatment plan in the world at present, and there is no relevant clinical trial. However, through our clinical experience and understanding in the treatment process of these children, The results of thalidomide combined with TGA chemotherapy regimen (nab-paclitaxel + gemcitabine + epirubicin) were satisfactory. Therefore, we hope to evaluate the therapeutic effectiveness and adverse reactions of this regimen through clinical trials, so as to provide an effective treatment option for children with recurrent and refractory yolk sac tumor.
This paper is the first one to report the treatment combining thalidomide with nab-paclitaxel, gemcitabine, and epirubicin (T-TGA) for children with repeated relapsed or refractory yolk sac tumor (rrrYST).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| thalidomide combined with chemotherapy in the treatment of relapsed or refractory yolk sac tumor | Experimental | Drug: Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin)This study employed an open single-group clinical laboratory design method (Simon Phase II study) to investigate the efficacy and safety of combining thalidomide with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin) in the treatment of children with recurrent or refractory YST. AFP levels were assessed after each course of treatment, while imaging evaluations were conducted every two courses. The remission rate following chemotherapy in this single-group clinical trial was compared to that reported in the literature for treating recurrent or refractory yolk sac tumor, evaluating overall remission rate (ORR) and event-free survival rate (EFS), as well as monitoring and recording chemotherapy-related side effects. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- The child must have histological evidence of an extracranial malignant germ cell tumor (vitelline sac tumor).
- Children must be no more than 18 years old at the time of study participation.
- The patients were children with vitelline cystic tumor who were resistant to drugs after at least two platinum-containing chemotherapy regimes or whose disease recurred within 3 months after chemotherapy regimes, or who recurred again or repeatedly after treatment with Sirolimus combined with TIC chemotherapy regimes.
- The child must have measurable lesions (recorded according to RECIST criteria) or non-evaluable disease with tumor marker AFP greater than 5 times the upper limit of normal.
- Lansky performance status score ≥50.
Key exclusion criteria (2 listed)
- Patients with other tumors.
- Heart, brain, liver, kidney and other organ failure patients.
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 |
|---|---|---|
| Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin)The oral dose of thalidomide is 1.5-2.5mg/kg/d daily (maximum 28g). Each cycle lasts for 21 days (every three weeks), and if treatment parameters are met as described in the study, cycle two and subsequent cycles begin on day 22. Treatment will be discontinued if there is drug-related dose-limiting toxicity or disease progression. Tumor response assessment will be repeated using AFP and LDH every one cycle and imaging examination every two cycles. Children who complete the trial may continue this regimen or undergo surgery or radiation therapy if their disease goes into remission. | Drug | thalidomide combined with chemotherapy in the treatment of relapsed or refractory yolk sac tumor |
Primary outcome
Overall Response Rate (ORR)
Time frame: Every 3 weeks per cycle, up to 6 cycles
Proportion of participants achieving partial or complete response，Measurement: Imaging (CT/MRI) every 2 cycles per RECIST 1.1；Serum AFP reduction ≥90% from baseline.
1 secondary outcome
Radiographic Tumor Response Evaluation
Time frame: Every 6 weeks (i.e., every 2 chemotherapy cycles)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2025.
- Individual participant dataSharing planned: No
Related trials for Germ Cell Tumor
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 1/2
Study of INBRX-106 and INBRX-106 in Combination With Pembrolizumab (Keytruda®) in Subjects With Locally Advanced or Metastatic Solid Tumors (Hexavalent OX40 Agonist)
Solid Tumor, Non-Small Cell Lung Cancer
RecruitingPhase 1
IL-17 Blockade to Decrease irAEs (REPLAY)
Metastatic Melanoma
RecruitingPhase 2
A UGT1A1 Genotype-Directed Study of Belinostat Pharmacokinetics and Toxicity
Carcinoma, Neuroendocrine, Tumor, Neuroendocrine
Enrolling by invitationObservational study
Natural History Study of Early Life Exposures in Agriculture (ELEA)
Prostate Cancer, Leukemia
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.
Jingfu Wang, MD study director
Shandong Cancer Hospital and Institute
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 Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin) in people with Germ Cell Tumor and Yolk Sac Tumor, run by Shandong First Medical 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 9 inclusion and 2 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: finished, as of the sponsor's last update on 18 Sep 2025. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: Every 3 weeks per cycle, up to 6 cycles). 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 18 Sep 2025.