A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients With Germ Cell Tumors

Study of Active Surveillance and Chemotherapy for Germ Cell Tumors

NCT: NCT03067181 · Status: RECRUITING · Phase: Phase 3 · Sponsor: Children's Oncology Group · Started: 2017-05-25 · Est. Completion: 2027-06-30

Plain English Summary

Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors is a Phase 3 clinical trial sponsored by Children's Oncology Group studying Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7. Tests active surveillance vs. chemotherapy with carboplatin or cisplatin for low and standard risk germ cell tumors. For patients aged 1 month and older, with newly diagnosed germ cell tumors, who meet specific histological and biomarker criteria. Participation involves regular check-ups and blood tests; chemotherapy is optional based on risk level. Alternatives include standard chemotherapy regimens like bleomycin, etoposide, and cisplatin. The trial aims to enroll 1780 participants.

Official Summary

This phase III trial studies how well active surveillance help doctors to monitor subjects with low risk germ cell tumors for recurrence after their tumor is removed. When the germ cell tumor has spread outside of the organ in which it developed, it is considered metastatic. Chemotherapy drugs, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. The trial studies whether carboplatin or cisplatin is the preferred chemotherapy to use in treating metastatic standard risk germ cell tumors.

Who Can Participate

Here is what you need to know about eligibility for this trial. Ages: No age limit for low risk, 1-24 years for standard risk. Health: Must have normal organ function, especially kidney function for chemotherapy. Conditions: Must have a primary extracranial germ cell tumor, confirmed by biopsy or elevated tumor markers. This trial is studying Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

Primary outcomes measure survival and recurrence, which are crucial for patients' long-term health and quality of life. The specific primary outcome measures are: Overall survival (Two years post enrollment); Event-free survival (Two years post enrollment). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses a critical gap in treatment options for germ cell tumors, offering a less toxic alternative for low-risk patients. As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7, where improved treatment options are needed.

Investor Insight

The large market size and competitive landscape suggest a high probability of approval, given the unmet need for less toxic treatments. Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage. The large enrollment target of 1780 participants suggests significant investment in this program.

Is This Trial Right for Me?

Ask your doctor about your eligibility and the risks involved. Participation involves regular check-ups and blood tests, with the option to receive chemotherapy. This trial is currently recruiting participants. The trial is being conducted at 20 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

  • Study Type: INTERVENTIONAL
  • Allocation: RANDOMIZED
  • Model: PARALLEL
  • Masking: NONE
  • Enrollment: 1,780 participants

Interventions

  • OTHER: Best Practice — Undergo observation
  • PROCEDURE: Biopsy Procedure — Undergo a tumor biopsy
  • PROCEDURE: Biospecimen Collection — Undergo blood sample collection
  • BIOLOGICAL: Bleomycin Sulfate — Given IV
  • DRUG: Carboplatin — Given IV

Primary Outcomes

  • Overall survival (Two years post enrollment)
  • Event-free survival (Two years post enrollment)

Secondary Outcomes

  • Presence of hearing loss (8 weeks after the last dose of platin therapy)
  • Adolescents and Young Adults-Hearing Screen (AYA HEARs) (Baseline, end of therapy, 2 months post-end of therapy, and 1 year after enrollment)
  • Whole body lean body mass (At diagnosis, at end of therapy, and up to 1 year after end of therapy)

Full Eligibility Criteria

Inclusion Criteria:

* There is no age limit for the low risk stratum (stage I ovarian immature teratoma and stage I non-seminoma or seminoma malignant GCT \[all sites\])
* Standard risk 1: Patients must be \< 11 years of age at enrollment
* Standard risk 2: Patients must be \>= 11 and \< 25 years of age at enrollment
* Patients enrolling on one of the low risk arms must be newly diagnosed with a stage I germ cell tumor; for the standard risk arms, patients must be newly diagnosed with malignant germ cell tumor (stage II or higher).

  * Histologic confirmation of a primary extracranial germ cell tumor in any of the categories outlined below is required of all patients at enrollment , with the following exceptions:

    * Among patients were initially diagnosed with completely resected non-seminoma malignant GCT and later recur during observation post surgery, a diagnostic biopsy is not required for enrollment if elevated tumor markers rise to \> 5 x upper limit of normal (ULN) on at least 2 measurements taken at least 1 week apart. The pathology report of initial surgery should be provided
    * Patients may be enrolled without histologic or cytologic confirmation in the rare case where there are exceptionally raised tumor markers (alpha fetoprotein \[AFP- ≥ 500 ng/mL or HCG ≥ 500 IU/L) and radiologic features consistent with GCT. In addition, the treating clinician must deem that the patient's tumor is not suitable for upfront resection and that a biopsy is not in the patient's best interest; or that there is a need to start therapy urgently
* Low risk immature teratoma (IT); site: ovarian; stage: any; grade: any; histology: pure immature teratoma, mixed immature and mature teratoma, (may contain microscopic foci of yolk sac tumor \[\< 3 mm\], but no other pathological evidence of MGCT); tumor markers: alpha-FP =\< 1,000 ng/mL, beta-HCG institutional normal; all ages
* Low risk stage I non-seminoma MGCT; site: ovarian, testicular, or extragonadal; stage: COG stage I, FIGO stage IA and IB, American Joint Committee on Cancer (AJCC) testicular stage IA, IB and IS; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma (pure or mixed); all ages
* Low risk stage I seminoma-MGCT; site: testicular; stage: COG stage I; AJCC testicular stage IA IB, and IS; histology: must contain only seminoma; may contain immature/mature teratoma; may NOT contain yolk sac tumor, embryonal carcinoma, or choriocarcinoma; all ages
* Standard risk 1 (SR1); site: ovarian, testicular, or extragonadal; stage: COG stage II-IV, FIGO stage IC-IV, (International Germ Cell Consensus Classification \[IGCCC\] criteria DO NOT apply); histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) \< 11
* Standard risk 2 (SR2)

  * Site: ovarian; stage: COG stage II, III, and III-X, FIGO stage IC, II and III; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma; age (years) \>= 11 and \< 25
  * Site: testicular; stage: COG stage II-IV, AJCC stage II, III, IGCCC good risk; histology: must contain at least one of the following: yolk sac tumor, embryonal carcinoma, or choriocarcinoma: must be IGCCC good risk; post op: alpha-FP \< 1,000 ng/mL, beta-HCG \< 5,000 IU/mL and lactate dehydrogenase (LDH) \< 3.0 x normal; age (years) \>= 11 and \< 25
* Notes:

  * IGCCC criteria only apply to SR2 patients with a testicular primary tumor
  * Use post-op tumor marker levels to determine IGCCC risk group
  * Pure seminoma patients are not eligible for the standard risk arms of the study
  * For the low risk stage I non-seminoma MGCT and the standard risk arms, components of yolk sac tumor, embryonal carcinoma, or choriocarcinoma can be mixed with other forms of GCT, such as seminoma or mature or immature teratoma; if yolk sac tumor is the only malignant component present, then it must be deemed by the pathologist to be greater than a "microscopic component" of yolk sac tumor
* Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, 2 or 3; use Karnofsky for patients \> 16 years of age and Lansky for patients =\< 16 years of age
* Organ function requirements apply ONLY to patients who will receive chemotherapy (SR1 and SR2 patients)
* Adequate renal function defined as:
* Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 (within 7 days prior to enrollment) OR
* A serum creatinine based on age/sex as follows (within 7 days prior to enrollment): (mg/dL)

  * 1 month to \< 6 months male: 0.4 female: 0.4
  * 6 months to \< 1 year male: 0.5 female: 0.5
  * 1 to \< 2 years male: 0.6 female: 0.6
  * 2 to \< 6 years male: 0.8 female: 0.8
  * 6 to \< 10 years male: 1 female: 1
  * 10 to \< 13 years male: 1.2 female: 1.2
  * 13 to \< 16 years: male: 1.5 female: 1.4
  * \>= 16 years male: 1.7 female

Trial Locations

  • Children's Hospital of Alabama, Birmingham, Alabama, United States
  • USA Health Strada Patient Care Center, Mobile, Alabama, United States
  • Anchorage Associates in Radiation Medicine, Anchorage, Alaska, United States
  • Anchorage Radiation Therapy Center, Anchorage, Alaska, United States
  • Alaska Breast Care and Surgery LLC, Anchorage, Alaska, United States
  • Alaska Oncology and Hematology LLC, Anchorage, Alaska, United States
  • Alaska Women's Cancer Care, Anchorage, Alaska, United States
  • Anchorage Oncology Centre, Anchorage, Alaska, United States
  • Katmai Oncology Group, Anchorage, Alaska, United States
  • Providence Alaska Medical Center, Anchorage, Alaska, United States
  • ...and 10 more locations

Frequently Asked Questions

What is clinical trial NCT03067181?

NCT03067181 is a Phase 3 INTERVENTIONAL study titled "Active Surveillance, Bleomycin, Etoposide, Carboplatin or Cisplatin in Treating Pediatric and Adult Patients With Germ Cell Tumors." It is currently recruiting and is sponsored by Children's Oncology Group. The trial targets enrollment of 1780 participants.

What conditions does NCT03067181 study?

This trial investigates treatments for Childhood Extracranial Germ Cell Tumor, Extragonadal Embryonal Carcinoma, Germ Cell Tumor, Malignant Germ Cell Tumor, Malignant Ovarian Teratoma, Stage I Ovarian Choriocarcinoma, Stage I Ovarian Embryonal Carcinoma AJCC v6 and v7, Stage I Ovarian Yolk Sac Tumor AJCC v6 and v7, Stage I Testicular Choriocarcinoma AJCC v6 and v7, Stage I Testicular Embryonal Carcinoma AJCC v6 and v7. The primary condition under study is Childhood Extracranial Germ Cell Tumor.

What treatments are being tested in NCT03067181?

The interventions being studied include: Best Practice (OTHER), Biopsy Procedure (PROCEDURE), Biospecimen Collection (PROCEDURE), Bleomycin Sulfate (BIOLOGICAL), Carboplatin (DRUG). Undergo observation

What does Phase 3 mean for NCT03067181?

Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.

What is the current status of NCT03067181?

This trial is currently "Recruiting." It started on 2017-05-25. The estimated completion date is 2027-06-30.

Who is sponsoring NCT03067181?

NCT03067181 is sponsored by Children's Oncology Group. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT03067181?

The trial aims to enroll 1780 participants. The trial is currently recruiting and accepting new participants.

How is NCT03067181 designed?

This is a interventional study, uses randomized allocation, follows a parallel design, employs none masking.

What are the primary outcomes being measured in NCT03067181?

The primary outcome measures are: Overall survival (Two years post enrollment); Event-free survival (Two years post enrollment). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT03067181 being conducted?

This trial is being conducted at 20 sites, including Birmingham, Alabama; Mobile, Alabama; Anchorage, Alaska; Kingman, Arizona and 16 more sites (United States).

Where can I find official information about NCT03067181?

The official record for NCT03067181 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT03067181. This government database provides the most up-to-date and detailed information about the trial.

What is NCT03067181 testing in simple terms?

Tests active surveillance vs. chemotherapy with carboplatin or cisplatin for low and standard risk germ cell tumors. For patients aged 1 month and older, with newly diagnosed germ cell tumors, who meet specific histological and biomarker criteria.

Why is this trial significant?

This trial addresses a critical gap in treatment options for germ cell tumors, offering a less toxic alternative for low-risk patients. As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.

What are the potential risks of participating in NCT03067181?

Key risks include potential recurrence of the tumor and side effects from chemotherapy, such as hearing loss. Side effects from chemotherapy may include nausea, fatigue, and hair loss. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT03067181?

Ask your doctor about your eligibility and the risks involved. Participation involves regular check-ups and blood tests, with the option to receive chemotherapy. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT03067181 signal from an investment perspective?

The large market size and competitive landscape suggest a high probability of approval, given the unmet need for less toxic treatments. This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.

What happens if the treatment in this trial doesn't work?

Participation involves regular check-ups and blood tests; chemotherapy is optional based on risk level. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.