Thalidomide Combined With Chemotherapy in the Treatment of Relapsed or Refractory Yolk Sac Tumor

NCT06470464 | Completed Phase 2 DMC

• 1 other identifier: HERO2022
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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. 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).

Conditions

Germ Cell Tumor; Yolk Sac Tumor

Keywords

relapsed or refractory yolk sac tumor; thalidomide

Outcome Measures

Primary Outcomes (1)

• 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.

  Every 3 weeks per cycle, up to 6 cycles

Secondary Outcomes (1)

• Radiographic Tumor Response Evaluation

  Every 6 weeks (i.e., every 2 chemotherapy cycles)

Study Arms (1)

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.

Drug: Thalidomide combined with TGA chemotherapy (nab-paclitaxel + gemcitabine+ epirubicin)

Interventions

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.

thalidomide combined with chemotherapy in the treatment of relapsed or refractory yolk sac tumor

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• Patients with other tumors.
• Heart, brain, liver, kidney and other organ failure patients.

Sponsors & Collaborators

• Shandong First Medical University: lead

Study Sites (1)

Shandong Cancer Hospital and Institute

Jinan, Shandong, 250117, China

Location

MeSH Terms

Conditions

Neoplasms, Germ Cell and Embryonal; Endodermal Sinus Tumor; Recurrence

Interventions

130-nm albumin-bound paclitaxel

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Mesonephroma; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Jingfu Wang, MD

  Shandong Cancer Hospital and Institute

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 17, 2024
• First Posted: June 24, 2024
• Study Start: November 11, 2021
• Primary Completion: June 16, 2024
• Study Completion: June 16, 2024
• Last Updated: September 18, 2025

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)