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Trial record 2 of 524 for:    "Neuroblastoma"

Naxitamab for Neuroblastoma With Osteomedullary Disease

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ClinicalTrials.gov Identifier: NCT03363373
Recruitment Status : Recruiting
First Posted : December 6, 2017
Last Update Posted : July 11, 2018
Sponsor:
Information provided by (Responsible Party):
Y-mAbs Therapeutics

Brief Summary:

Children and adults diagnosed with high risk neuroblastoma and refractory osteomedullary disease will be treated for up to 93 weeks with naxitamab and granulocyte-macrophage colony stimulating factor (GM-CSF). Participants will be followed for up to five years after first dose.

Naxitamab, also known as hu3F8 is a humanised monoclonal antibody targeting GD2


Condition or disease Intervention/treatment Phase
Neuroblastoma Biological: GM-CSF + hu3F8 Phase 3

Detailed Description:

Each patient will receive treatment for up to 93 weeks following the first hu3F8 administration and remain in the trial for 101 weeks. After the end of trial visit, each patient will enter a long-term follow-up where they will be monitored for up to 5 years after first treatment cycle.

Each investigational cycle is started with 5 days, days -4 to 0, of Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) administered at 250 µg/m2/day in advance of the start of hu3F8 administration. GM-CSF is thereafter administered at 500 µg/m2/day on days 1 to 5. As standard treatment, hu3F8 is administered at 3 mg/kg/day on days 1, 3, and 5, totalling 9 mg/kg per cycle.

The first 5 cycles are repeated every 4 weeks up to week 21 and after that the frequency decreases to every 8 weeks through a total of 93 weeks. End of treatment will take place around 8 weeks after the last cycle and thereafter long-term follow-up will continue.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 37 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Participants will receive up to 15 cyclels of GM-CSF and hu3F8 for a total of 93 weeks. Safety and efficacy will be investigated with short-term follow-up at 8 weeks after last treatment and with long-term follow-up for up to 5 years following first treatment.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3 Trial of Antibody Naxitamab and Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in High-Risk Neuroblastoma Patients With Primary or Secondary Refractory Osteomedullary Disease
Actual Study Start Date : April 3, 2018
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Arm Intervention/treatment
Experimental: GM-CSF + hu3F8
Each investigational cycle is started with 5 days of GM-CSF administered at 250 µg/m2/day in advance of the start of hu3F8 administration. GM-CSF is thereafter administered at 500 µg/m2/day on days 1 to 5. As standard treatment, hu3F8 is administered at 3 mg/kg/day on days 1, 3, and 5 totalling 9 mg/kg per cycle. After 15 cycles at week 101 participants will enter a long-term follow up for up to five years after first dose.
Biological: GM-CSF + hu3F8
Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) and Humanized IgG1 monoclonal GD2 antibody




Primary Outcome Measures :
  1. Response rate during Naxitamab treatment [ Time Frame: 101 weeks ]
    Overall objective response rate (ORR) during the Naxitamab treatment period that will be centrally assessed according to the International Neuroblastoma Response Criteria (INRC) modified with 123I-MIBG criteria and following the use of 18F FDG-PET for MIBG non-avid lesions.


Secondary Outcome Measures :
  1. Incidence of adverse events and serious adverse events [ Time Frame: 101 weeks ]
    Safety will be evaluated by the incidence of adverse events (AE) and serious adverse events (SAEs) graded according to CTCAE, version 4.0.

  2. Duration of Response (DoR) [ Time Frame: 101 weeks ]
    Length of time from patient response to disease progression.

  3. Progression Free Survival (PFS) [ Time Frame: 101 weeks ]
    The interval from the date of first dose of Naxitamab until the date of disease progression

  4. Overall Survival [ Time Frame: 101 weeks ]
    The interval from the date of first dose of Naxitamab until the date of death due to any cause.

  5. Assessment of peak plasma concentration (Cmax) of Naxitamab [ Time Frame: 101 weeks ]
    Cmax will be calculated and summarized with descriptive statistics.

  6. Assessment of trough plasma concentration (Cmin) of Naxitamab [ Time Frame: 101 weeks ]
    Cmin will be calculated and summarized with descriptive statistics.

  7. Assessment of clearance of Naxitamab [ Time Frame: 101 weeks ]
    Clearance will be calculated and summarized with descriptive statistics.

  8. Assessment of volume of distribution (Vd) of Naxitamab [ Time Frame: 101 weeks ]
    Volume of distribution will be calculated and summarized with descriptive statistics.

  9. Assessment of area under the plasma concentration versus time curve (AUC) of Naxitamab [ Time Frame: 101 weeks ]
    AUC will be calculated and summarized with descriptive statistics.

  10. Assessment of half life (T1/2) of Naxitamab [ Time Frame: 101 weeks ]
    T1/2 will be calculated and summarized with descriptive statistics.

  11. Assessment of HAHA formation [ Time Frame: 101 weeks ]
    Human Anti-Human Antibody formation

  12. Opioid use [ Time Frame: 2 weeks ]
    Intravenous opioid use during cycle 1

  13. Number of hospitalization days [ Time Frame: 2 weeks ]
    Hospitalization days related to Naxitamab infusion during cycle 1



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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of neuroblastoma as defined per International Neuroblastoma Response Criteria
  • High-risk neuroblastoma with either primary refractory or secondary refractory osteomedullary disease (persistent neuroblastoma at osteomedullary sites after prior treatment)
  • Life expectancy ≥ 6 months

Exclusion Criteria:

  • Chemotherapy or immunotherapy within 3 weeks prior to start of hu3F8
  • Evidence of neuroblastoma outside osteomedullary sites (any neuroblastoma outside osteomedullary sites must have been removed prior to trial entry)
  • Existing major organ dysfunction > Grade 2, with the exception of hearing loss, hematological status, kidney and liver function
  • Active life-threatening infection

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03363373


Contacts
Contact: Joris Wilms +4570261414 info@ymabs.com

Locations
United States, California
Childrens Hospital Los Angeles Not yet recruiting
Los Angeles, California, United States, 90027
United States, Florida
University of Florida Not yet recruiting
Gainesville, Florida, United States, 32611
United States, Indiana
Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Angie Myers    317-948-8540    almyers2@iu.edu   
United States, New York
Memorial Sloan Kettering Cancer Center Not yet recruiting
New York, New York, United States, 10065
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Keri Streby, MD       Keri.Streby@nationwidechildrens.org   
United States, Texas
M.D. Anderson Cancer Center Not yet recruiting
Houston, Texas, United States, 77030
Denmark
Rigshospitalet Recruiting
København, Denmark, 2100
Contact: Karsten Nysom, MD       Karsten.Nysom@regionh.dk   
Spain
Hospital Sant Joan de Déu Recruiting
Barcelona, Spain, 08950
Contact: Jaume Mora, MD       Jmora@sjdhospitalbarcelona.org   
United Kingdom
The Harley Street Clinic Not yet recruiting
London, United Kingdom, W1G 7HL
Sponsors and Collaborators
Y-mAbs Therapeutics
Investigators
Study Director: Steen Lisby, MD Chief Medical Officer

Responsible Party: Y-mAbs Therapeutics
ClinicalTrials.gov Identifier: NCT03363373     History of Changes
Other Study ID Numbers: 201
First Posted: December 6, 2017    Key Record Dates
Last Update Posted: July 11, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Y-mAbs Therapeutics:
Antibody, Neuroblastoma, Pediatric, Adult

Additional relevant MeSH terms:
Neuroblastoma
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Antibodies
Immunologic Factors
Physiological Effects of Drugs