An Open Label Dose Escalation Safety Study of Convection-Enhanced Delivery of IL13-PE38QQR in Patients With Progressive Pediatric Diffuse Infiltrating Brainstem Glioma and Supratentorial High-grade Glioma
|First Received Date ICMJE||April 10, 2009|
|Last Updated Date||November 22, 2014|
|Start Date ICMJE||April 2009|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE
||1) Feasibility of perfusing specific sites within the CNS with IL13-PE38QQR, administered concurrently with gd-DTPA, 2) Safety and tolerability of escalating doses of IL13-PE38QQR via CED to pediatric patient with DIPGs and HGGs|
|Original Primary Outcome Measures ICMJE
||1) Feasibility of perfusing specific sites within the CNS with IL13-PE38QQR, administered concurrentlywith gd-DTPA @@@2) Safety and tolerability of escalating doses of IL13-PE38QQR via CED to pediatric patient with DIPGs and HGGs|
|Change History||Complete list of historical versions of study NCT00880061 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE
||Determine effect of IL13-PE38QQR on MRI tumor measurements, symptom improvement or worsening, changes on clinical exam, radiographic changes, steroid dosing, QOL testing and survival of pediatric patients with DIPG and recurrent HGG|
|Original Secondary Outcome Measures ICMJE||Same as current|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||An Open Label Dose Escalation Safety Study of Convection-Enhanced Delivery of IL13-PE38QQR in Patients With Progressive Pediatric Diffuse Infiltrating Brainstem Glioma and Supratentorial High-grade Glioma|
|Official Title ICMJE||An Open Label Dose Escalation Safety Study of Convection-Enhanced Delivery of IL13-PE38QQR in Patients With Progressive Pediatric Diffuse Infiltrating Brainstem Glioma and Supratentorial High-Grade Glioma|
Clinic visits 4 and 8 weeks after the treatment, and then every 8 weeks for up to 1 year.
Objective: The primary purpose of this study is to test the safety and feasibility of giving a new experimental agent, called IL13-PE38QQR, directly into regions of the brain in patients with diffusely infiltrating pontine glioma (DIPG) or with recurrent or progressive supratentorial high-grade glioma (HGG) using a technique called convection-enhanced delivery or CED. CED uses continuous pressure to push large molecules through the membranes protecting the brain to reach brain tumors. At the same time, we can watch where the molecules go in the brain by attaching a tracer, gadolinium-DTPA, to the IL13-PE38QQR, which can then be seen in the brain with magnetic resonance imaging (MRI). Because we do not know the best dose to use in patients with DIPG or HGG, we will give increasing amounts of IL13-PE38QQR to small groups of patients with each type of brain tumor, known as a dose escalation study. Secondary purposes of this study include determining the effects of this experimental therapy on the tumor, and evaluating the physical changes in the tumor before and after the therapy.
Study Population: Twenty-right pediatric patients with recurrent or progressive DIPG or supratentorial HGG that have undergone standard treatment and who meet all the Inclusion and Exclusion Criteria may be enrolled. Eighteen patients will receive treatment; an additional ten patients may be screening failures or unevaluable.
Design: We propose a Phase I single institution, open label, dose escalation (doses of 0.125, 0.25 and 0.5 micrograms/ml), safety and tolerability study of IL13-PE38QQR infused via CED into patients with either DIPG (up to 9 patients) or recurrent HGG (up to 9 patients). IL13-PE38QQR will be administered to regions of tumor determined by radiographic findings. Escalating dose levels will be evaluated in the following dose cohorts (3 patients per Cohort): Cohort 1 = 0.125 micrograms/ml, Cohort 2 = 0.25 micrograms/ml and Cohort 3 = 0.5 micrograms/ml.
Outcome Measures: To assess the safety, tolerability and potential efficacy of CED of IL13-PE38QQR, we will use detailed clinical and radiographic examinations. These will be performed at baseline and on post-infusion days 1, 28 and 60. After post-infusion day 60, clinical and radiographic studies will then be performed every 8 weeks until imaging or clinical evidence of recurrence/progressive disease or new treatment is initiated.
|Study Type ICMJE||Interventional|
|Study Phase||Phase 1|
|Study Design ICMJE||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Intervention ICMJE||Drug: IL13-PE38QQR
|Study Arm (s)||Not Provided|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Active, not recruiting|
|Completion Date||Not Provided|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
|Ages||up to 17 Years|
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Location Countries ICMJE||United States|
|NCT Number ICMJE||NCT00880061|
|Other Study ID Numbers ICMJE||090117, 09-N-0117|
|Has Data Monitoring Committee||Not Provided|
|Responsible Party||National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )|
|Study Sponsor ICMJE||National Institute of Neurological Disorders and Stroke (NINDS)|
|Collaborators ICMJE||Not Provided|
|Information Provided By||National Institutes of Health Clinical Center (CC)|
|Verification Date||September 2014|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP