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Completed

NCT Number: NCT01183897

3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow

The purpose of this study is to see find out what effects, good and/or bad, the combination of 3F8 and GM-CSF has on the patient and the cancer.

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Key information

Conditions

Neuroblastoma Neoplasms Neoplasms by Histologic Type Neoplasms, Germ Cell and Embryonal Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Neoplasms, Neuroepithelial Neuroectodermal Tumors Neuroectodermal Tumors, Primitive Neuroectodermal Tumors, Primitive, Peripheral

Age range

18 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Memorial Sloan-Kettering Cancer Center

New York, 10065, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of NB as defined by a) histopathology (confirmed by the MSKCC Department of Pathology), or b) BM metastases or MIBG-avid lesion(s) plus high urine catecholamine levels.
  • High-risk NB as defined by risk-related treatment guidelines1 and the International NB Staging System,89 i.e., stage 4 with (any age) or without (> or = to 18 months of age) MYCN amplification or MYCN-amplified stage 4S.
  • Patients have primary refractory disease limited to BM, i.e., high-risk NB (defined above) resistant to standard therapy, as evidenced by incomplete response in BM, but no measurable MIBG-avid soft tissue tumor assessable for response and no progressive disease.
  • Signed informed consent indicating awareness of the investigational nature of this program.

Exclusion criteria

  • Creatinine > 3.0 mg/dL
  • ALT, AST and Alkaline Phosphatase > 5.0 times the upper limit of normal
  • Bilirubin > 3.0 mg/dL
  • Patients with grade 3 or higher toxicities (using the CTCAE v 4.0) related to cardiac, neurological, pulmonary or gastrointestinal function as determined by physical exam. Patients must have normal blood pressure for age.
  • Progressive disease
  • History of allergy to mouse proteins.
  • Active life-threatening infection.
  • Human anti-mouse antibody (HAMA) titer >1000 Elisa units/ml.
  • Inability to comply with protocol requirements.

Treatment and study plan

3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic

Biological

This phase II, open-label, single arm trial assesses the anti-NB activity of high-dose 3F8 (80 mg/m2/day), which is used in cycles 1-2, with return to standard 3F8 dosage (20 mg/m2/day) in subsequent cycles.Clinical results will be compared to those in the predecessor trials which used only the standard 3F8 dosage. Starting with A(6), patients no longer receive high-dose 3F8 but receive only standard dose 3F8 (20mg/m2/day) for all cycles The patients have primary refractory BM disease. Protocol treatment is through 24 months.

Real-time quantitative RT-PCR63-65 will be used to assess MRD in BM. 13-cis-retinoic acid is started no later than after cycle 4 (i.e., after response is scored), but sooner if CR is achieved after cycles 1, 2, or 3, or if early HAMA develops.

Primary outcomes

  1. Assess the Activity of High-dose 3F8/GM-CSF

    Time frame: 2 years

    against persistent neuroblastoma in bone marrow of patients who have no other evidence of disease by standard studies.

Secondary outcomes

  1. Apply Real-time Quantitative RT-PCR

    Time frame: 2 years

    to test the hypothesis that the minimal residual disease content of bone marrow after the first treatments with 3F8/GMCSF has significant prognostic impact on progression-free survival.

  2. Monitor Safety of the High-dose Antibody Treatment

    Time frame: 2 years

    to assure no side-effects or noxious sequelae develop or emerge that were not seen in the prior phase I study.

Sponsors and collaborators

Lead sponsor

Memorial Sloan Kettering Cancer Center

Other

Registry information

Official study title

3F8/GM-CSF Immunotherapy Plus 13-Cis-Retinoic Acid for Primary Refractory Neuroblastoma in Bone Marrow: A Phase II Study

Important dates

Study start
2010
Primary completion
2018
Study completion
2018
First posted
Aug 18, 2010
Registry last updated
Aug 13, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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