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N2001-02: I-MIBG With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma

This study has been completed.
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Nant Operations Center, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier:
NCT00253435
First received: November 11, 2005
Last updated: August 23, 2016
Last verified: August 2016
Results First Received: August 25, 2014  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Neuroblastoma
Interventions: Biological: Filgrastim
Drug: Carboplatin
Drug: Etoposide
Drug: Melphalan
Procedure: Peripheral blood stem cell infusion
Radiation: 131I-MIBG
Radiation: Radiation therapy

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations

Opened to accrual on 12/10/04 & closed to accrual on 07/21/10; suspended 6 times for amendments. Opened at 12 NANT institutions.

1st patient started on treatment on 03/11/05. 50 patients enrolled; all eligible & evaluable for toxicity. 49 patients were evaluable for response.


Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Poor Risk Patients Poor risk patients are those who experienced a Minor response (MR) or No Response (NR) to induction therapy or progressive disease (PD) during or following induction.
Good Risk Patients Good risk patients are those who experienced a Partial Response (PR) following 4 cycles of induction.

Participant Flow:   Overall Study
    Poor Risk Patients   Good Risk Patients
STARTED   42 [1]   8 [2] 
1st Interim Analysis   15 [3]   8 [4] 
2nd Interim Analysis   10 [5]   0 [2] 
3rd Interim Analysis   10 [6]   0 [2] 
COMPLETED   42 [7]   8 [8] 
NOT COMPLETED   0   0 
[1] Total number is 42
[2] This cohort opened as long as the poor risk group is open to accrual.
[3] First interim analysis: need 2+ responses in first 15 patients. Goal met study continued.
[4] Only interim analysis: need 1+ response in first 10 patients.
[5] Second interim analysis: need 3+ responses in first 25 patients. Goal met study continued.
[6] Third interim analysis: need 4+ responses in first 35 patients. Goal met study continued.
[7] Forty-two evaluable poor risk patients enrolled.
[8] Eight evaluable patients enrolled.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Number of patients who signed the consent form and started treatment.

Reporting Groups
  Description
Poor Risk Patients Poor risk patients are those who experienced a Minor response (MR) or No Response (NR) to induction therapy or progressive disease (PD) during or following induction.
Good Risk Patients Good risk patients are those who experienced a Partial Response (PR) following 4 cycles of induction.
Total Total of all reporting groups

Baseline Measures
   Poor Risk Patients   Good Risk Patients   Total 
Overall Participants Analyzed 
[Units: Participants]
 42   8   50 
Age [1] 
[Units: Years]
Median (Full Range)
 6.0 
 (1.1 to 22.8) 
 7.7 
 (4.3 to 16.4) 
 6.0 
 (1.1 to 22.8) 
[1] Age (in years) at time of treatment start.
Gender 
[Units: Participants]
     
Female   10   2   12 
Male   32   6   38 
Ethnicity (NIH/OMB) 
[Units: Participants]
     
Hispanic or Latino   4   3   7 
Not Hispanic or Latino   38   5   43 
Unknown or Not Reported   0   0   0 
Race (NIH/OMB) 
[Units: Participants]
     
American Indian or Alaska Native   0   0   0 
Asian   3   0   3 
Native Hawaiian or Other Pacific Islander   0   0   0 
Black or African American   4   0   4 
White   33   8   41 
More than one race   0   0   0 
Unknown or Not Reported   2   0   2 
GFR Status [1] 
[Units: Participants]
     
Normal GFR   30   7   37 
Low GFR   12   1   13 
[1]

Pre-therapy glomerular filtration rate:

Normal GFR: >= 100 ml/min/1.73 m2 & Low GFR: 60-99 ml/min/1.73 m2



  Outcome Measures
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1.  Primary:   Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion   [ Time Frame: Response assessed 60 days post stem cell infusion ]

2.  Secondary:   Event-free Survival (EFS) at 3 Years   [ Time Frame: 3 years since start of treatment ]

3.  Secondary:   Engraftment DLT   [ Time Frame: From treatment start until 60 days post stem cell infusion ]

4.  Secondary:   Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS   [ Time Frame: Between start of MIBG treatment and 60 days post stem cell infusion ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Araz Marachelian, MD, NANT Medical Director
Organization: Chidlren's Hospital Los Angeles
phone: 323-3691-5687
e-mail: amarachelian@chla.usc.edu



Responsible Party: Nant Operations Center, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT00253435     History of Changes
Other Study ID Numbers: CDR0000450148
P01CA081403 ( US NIH Grant/Contract Award Number )
N2001-02 ( Other Identifier: NANT Consortium )
Study First Received: November 11, 2005
Results First Received: August 25, 2014
Last Updated: August 23, 2016
Health Authority: United States: Food and Drug Administration