Clinical Study of Chemotherapy in the Treatment of Recurrent/Refractory Yolk Sac Tumor in Children

NCT06341998 | Completed Phase 2 DMC

• 1 other identifier: HERO2020
• Study Type: interventional
• Target: 32
• 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 sirolimus with nab-paclitaxel, ifosfamide, and carboplatin (S-TIC) for children with repeated relapsed or refractory yolk sac tumor (rrrYST).

Conditions

Germ Cell Tumor; Yolk Sac Tumor

Keywords

multiply relapsed or cisplatin refractory yolk sac tumor; sirolimus; germ cell tumor; mTOR

Outcome Measures

Primary Outcomes (1)

• ORR: Objective Response Rate Allocation 'Non-Randomized' implies that this is a multi-arm study, but only one arm has been specified. Objective Response Rate

  Each treatment cycle spans 21 to 28 days (equivalent to every 3 to 4 weeks). Provided that the treatment criteria outlined in the study are satisfied, the second and subsequent cycles will commence on either the 22nd or 29th day. In the event of severe drug-related adverse reactions, treatment will be discontinued, and the patient will be classified as having PD. Tumor response assessment will entail the re-evaluation of AFP and LDH levels every cycle, alongside imaging examinations every two cycles. Should AFP levels demonstrate a continuous increase over two consecutive monitoring sessions following the initial treatment cycle, or if imaging results indicate tumor progression, a diagnosis of PD will be made, resulting in the termination of the clinical trial. For pediatric patients whose trials have been terminated, alternative anti-tumor therapies will be administered in accordance with their individual clinical conditions.

  Each treatment cycle spans 3 to 4 weeks, 1-4 cycles, last at lest 3 to 16 weeks.

Secondary Outcomes (1)

• Secondary endpoints included the progression-free survival (PFS) rate, overall survival (OS), and safety profile.

  After each patient completed the clinical trial, they underwent imaging assessment examinations at 3/6/9/12/18/24/30/36/48/60 months after the end of the treatment.

Study Arms (1)

sirolimus combined with chemotherapy treat recurrent/refractory yolk sac tumor in children

EXPERIMENTAL

This study employed an open single-group clinical laboratory design method (Simon Phase II study) to investigate the efficacy and safety of combining sirolimus with TIC chemotherapy (albumin-paclitaxel + isocyclophosphamide + carboplatin) in the treatment of children with recurrent/refractory vitellocystoma. 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/refractory yolk sac tumor, evaluating overall remission rate (ORR) and progresion-free survival rate (PFS) and overall survival (OS), as well as monitoring and recording chemotherapy-related side effects.

Drug: sirolimus combined with chemotherapy in the treatment of recurrent/refractory yolk sac tumor in children

Interventions

sirolimus combined with chemotherapy in the treatment of recurrent/refractory yolk sac tumor in children DRUG

The planned treatment protocol comprised four cycles of the S-TIC regimen, which included sirolimus administered orally at a dose of 1 mg/m² from day 1 to day 21-28, nab-paclitaxel at a dose of 200 mg/m² on day 1, ifosfamide at a dose of 1200 mg/m² from day 2 to day 5, and carboplatin at a dose of 400 mg/m² on day 2. Additionally, mesna and sulfamethoxazole were prescribed to prevent hemorrhagic cystitis and Pneumocystis Jirovecii Pneumonia (PJP), respectively. Each cycle spanned a duration of three to four weeks. AFP levels were evaluated at the end of each cycle, and radiological assessments were conducted every two cycles. Surgical intervention decisions were made based on the results of these assessments.

sirolimus combined with chemotherapy treat recurrent/refractory yolk sac tumor in children

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 pediatric patient is required to present histological confirmation of an extracranial yolk sac tumor.
• At the time of enrollment, the patient must be 18 years of age or younger, with no restrictions based on gender
• The patient must exhibit disease progression following a minimum of two platinum-based chemotherapy regimens or experience a relapse after the completion of treatment
• The patient must have measurable lesions, documented in accordance with the RECIST criteria, or possess an unassessable disease with alpha-fetoprotein (AFP) levels exceeding five times the upper limit of normal
• The Lansky performance status score must be 50 or higher, and the patient's life expectancy should be greater than 12 weeks
• The patient must have fully recovered from any prior adverse effects related to anti-cancer treatments

You may not qualify if:

• a history of previous tumors
• organ failure, specifically involving the heart, brain, liver, or kidneys.

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

Interventions

Drug Therapy

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Mesonephroma

Intervention Hierarchy (Ancestors)

Therapeutics

Study Officials

• Jingfu Wang, MD

  Shandong Cancer Hospital and Institute

  PRINCIPAL INVESTIGATOR

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: March 26, 2024
• First Posted: April 2, 2024
• Study Start: June 1, 2020
• Primary Completion: January 1, 2024
• Study Completion: February 1, 2024
• Last Updated: September 19, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)