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Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).

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ClinicalTrials.gov Identifier: NCT01688401
Recruitment Status : Completed
First Posted : September 19, 2012
Last Update Posted : March 28, 2019
Sponsor:
Collaborator:
Solving Kids' Cancer
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Tracking Information
First Submitted Date  ICMJE September 10, 2012
First Posted Date  ICMJE September 19, 2012
Last Update Posted Date March 28, 2019
Actual Study Start Date  ICMJE March 8, 2013
Actual Primary Completion Date May 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 26, 2019)
Technical safety as determined by number of participants with toxicity [ Time Frame: 60 days ]
Number of participants with grades 3-5 intracranial hemorrhage, grades 3-5 stroke, as defined by the Nervous system disorder CTCAE, and requirement of blood transfusion.
Original Primary Outcome Measures  ICMJE
 (submitted: September 14, 2012)
Technical safety [ Time Frame: 60 days ]
In this feasibility study, we are going to perform a qualitative evaluation of whether or not we can safely deliver IA chemotherapy to patients with progressive DIPG. Assessment of non-hematologic toxicity will include grading according to the Nervous system disorder CTCAE, specifically grades 3-5 intracranial hemorrhage and grades 3-5 stroke. Note will be made if the patient requires a blood transfusion.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 26, 2019)
Long-term Efficacy as assessed by progression free survival [ Time Frame: 2 years ]
Number of months until disease progression.
Original Secondary Outcome Measures  ICMJE
 (submitted: September 14, 2012)
Efficacy [ Time Frame: 2 years ]
Analysis of long-term efficacy will be made by assessing progression free survival in terms of months.
Current Other Pre-specified Outcome Measures
 (submitted: March 26, 2019)
  • Immediate Efficacy as assessed by number of participants with decrease in required steroid dose [ Time Frame: 60 days ]
  • Immediate Efficacy as assessed by number of participants with decrease in tumor size on MRI [ Time Frame: 60 days ]
  • Immediate Efficacy as assessed by number of participants with decrease in the degree of enhancement on MRI [ Time Frame: 60 days ]
  • Immediate Efficacy as assessed by number of participants with improved neurological examination [ Time Frame: 60 days ]
Original Other Pre-specified Outcome Measures
 (submitted: September 14, 2012)
  • Efficacy [ Time Frame: 60 days ]
    Analysis of immediate efficacy will be made by assessing for signs of tumor response as evidenced by a decrease in required steroid dose.
  • Efficacy [ Time Frame: 60 days ]
    Analysis of immediate efficacy will be made by assessing for signs of tumor response as evidenced by a decrease in tumor size on MRI.
  • Efficacy [ Time Frame: 60 days ]
    Analysis of immediate efficacy will be made by assessing for signs of tumor response as evidenced by a decrease in the degree of enhancement on MRI.
  • Efficacy [ Time Frame: 60 days ]
    Analysis of immediate efficacy will be made by assessing for signs of tumor response as evidenced by an improved neurologic examination.
 
Descriptive Information
Brief Title  ICMJE Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
Official Title  ICMJE Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG).
Brief Summary The goal of this pilot study is to determine if intra-arterial (IA) chemotherapy is safe in the treatment of progressive diffuse intrinsic pontine gliomas (DIPG). IA administration of the chemotherapeutic agent enhances the regional distribution of the drug, thereby increasing the local delivered dose while minimizing systemic toxicity. It also provides a treatment option for these patients at the time of tumor recurrence.
Detailed Description Delivering the chemotherapeutic agent directly to the tumor via the arterial system avoids the complications and adverse events associated with toxicity from systemic chemotherapy.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Diffuse Intrinsic Pontine Glioma (DIPG)
Intervention  ICMJE Drug: Melphalan hydrochloride

Drug administered intra-arterially (injection in the artery).

Standard dose: Cycle 1: 1 mg, intra-arterial (IA) delivery. Cycle 2: 2 mg, intra-arterial (IA) delivery.

Duration of treatment: Eight weeks total - two cycles of IA chemotherapy, separated by four weeks.

Other Name: Alkeran
Study Arms  ICMJE Experimental: IA melphalan
IA melphalan is administered via the basilar artery.
Intervention: Drug: Melphalan hydrochloride
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 26, 2019)
3
Original Estimated Enrollment  ICMJE
 (submitted: September 14, 2012)
5
Actual Study Completion Date  ICMJE November 26, 2018
Actual Primary Completion Date May 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Pediatric patients of all ages with progressive DIPG.
  • Consensus following presentation of the case at the multidisciplinary Pediatric Neuro-Oncology conference, which includes participation of neuro-oncology, neurosurgery, radiation oncology, interventional neuroradiology and neurology.

Exclusion Criteria:

  • Documented hypercoagulable disorders or vasculopathies

    • INR value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>1 - 1.5 x ULN; >1 - 1.5 times above baseline if on anticoagulation).
    • APTT value more than a Grade 1 toxicity by CTCAE v 4.0 criteria (>ULN - 1.5 x ULN).
  • Platelets less than 50 x 103/mm3
  • Absolute neutrophil count less than 500/ mm3
  • Pregnancy
  • Documented severe allergic reaction to IV iodinated contrast, specifically bronchospasm and anaphylaxis.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01688401
Other Study ID Numbers  ICMJE J11164
NA_00069122 ( Other Identifier: JHMIRB )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Study Sponsor  ICMJE Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Collaborators  ICMJE Solving Kids' Cancer
Investigators  ICMJE
Principal Investigator: Monica Pearl, M.D. Johns Hopkins University
PRS Account Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP