Phase II Study of Intraventricular Methotrexate in Childr... | NCT02684071

Studies
Phase II Study of Intraventricular Methotrexate in Children With Recurrent or Progressive Malignant Brain Tumors

NCT02684071

Sponsor

Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Status

Terminated

Last Update Posted

Jan 10, 2020Actual

Enrollment

3Actual

Phase

Phase 2

Conditions

Recurrent Childhood Medulloblastoma
Recurrent Childhood Ependymoma
Childhood Atypical Teratoid/Rhabdoid Tumor
Embryonal Tumor With Abundant Neuropil and True Rosettes
Metastatic Malignant Neoplasm to the Leptomeninges

Interventions

Intra thecal methotrexate
topotecan
cyclophosphamide

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Status Module

Record Verification Date

Jan 2020

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Lack of additional funding; patients (3) no longer receiving intervention.

Expanded Access Info

Start Date

Feb 2016Actual

Primary Completion Date

Dec 9, 2019Actual

Completion Date

Dec 9, 2019Actual

First Submitted Date

Feb 11, 2016

First Submission Date that Met QC Criteria

Feb 11, 2016

First Posted Date

Feb 17, 2016Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 7, 2020

Last Update Posted Date

Jan 10, 2020Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

Detailed Description

The purpose of this research study is to test an experimental treatment method for recurrent or progressive brain tumors in children aged from 0-22 years. The use of methotrexate and chemotherapy (topotecan and cyclophosphamide) is experimental in this study. This means that their use by themselves or together has not been approved by the U.S. Food and Drug Administration for this usage. This study will attempt to determine via serial MRI scans if methotrexate administration into the lateral or fourth ventricle in combination with systemic intravenous topotecan and cyclophosphamide is effective in reducing tumor burden in the brain and spine for patients with recurrent or progressive brain tumors, including medulloblastoma, ependymoma, PNET (Primitive Neuroectodermal Tumor), atypical teratoid/rhabdoid tumor (AT/RT), and other malignant embryonal tumors. In addition, the study will evaluate the toxicity of the above mentioned experimental treatment during a two-year progression-free survival and survival of children with recurrent malignant brain tumors.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

3Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Intra thecal methotrexate	Experimental	IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide	Drug: Intra thecal methotrexate Drug: topotecan Drug: cyclophosphamide

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Intra thecal methotrexate	Drug	IT methotrexate via Ommaya reservoir with concomitant systemic topotecan and cyclophosphamide	Intra thecal methotrexate

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Tumor response	MRI response	6 months

Secondary Outcomes

Measure	Description	Time Frame
Toxicity of chemotherapy regimen (topotecan and cyclophosphamide)	Laboratory results, MRI, physical examination	6 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Recurrent or progressive supratentorial or posterior fossa tumors with measurable disease on imaging studies of the brain and spine or CSF cytology.
Tumor histology, including: Medulloblastoma , PNET, or other embryonal tumor, Atypical teratoid/rhaboid tumor (AT/RT), and Ependymoma.
Leptomeningeal dissemination of a previously diagnosed CNS tumor.
Diagnosis of tumor pathology will be based upon pathology diagnosis from previous surgeries for patients with recurrent tumors.
Patients must have received prior radiation before current recurrence, unless the patient is less than 36 months old at diagnosis and has progressed after at least one upfront chemotherapy regimen in which case no prior radiation is required.
Patients must have a life expectancy of at least 12 weeks as indicated by the patient's oncologist and/or neurosurgeon.
Lansky or Karnofsky Performance status of at least 50.
Negative pregnancy test.
Specific organ function requirements for: Central Nervous System, Bone Marrow, renal and liver.

Exclusion Criteria:

Patients that do not meet the inclusion criteria above.
Pregnant or lactating female patients.
Patients currently enrolled in another experimental treatment protocol.
Patients with documented allergies to any of the chemotherapy agents used in this study.
Patient/Parent refuses study participation.
Patient is severely somnolent or comatose.
Unable or unwilling to commit to return or to follow-up visits.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

22 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Name	Affiliation	Role
Ziad Khatib, MD	Nicklaus Children's Hospital	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Nicklaus Children's Hospital		Miami	Florida	33155	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
16533779	Background	Gururangan S, Petros WP, Poussaint TY, Hancock ML, Phillips PC, Friedman HS, Bomgaars L, Blaney SM, Kun LE, Boyett JM. Phase I trial of intrathecal spartaject busulfan in children with neoplastic meningitis: a Pediatric Brain Tumor Consortium Study (PBTC-004). Clin Cancer Res. 2006 Mar 1;12(5):1540-6. doi: 10.1158/1078-0432.CCR-05-2094.
9696374

ID	Term
D008527	Medulloblastoma
C531673	Familial ependymoma
D018335	Rhabdoid Tumor
D018242	Neuroectodermal Tumors, Primitive
D055756	Meningeal Carcinomatosis
D004806	Ependymoma

ID	Term
D005910	Glioma
D018302	Neoplasms, Neuroepithelial
D017599	Neuroectodermal Tumors
D009373	Neoplasms, Germ Cell and Embryonal
D009370	Neoplasms by Histologic Type
D009369	Neoplasms
D009375	Neoplasms, Glandular and Epithelial
D009380	Neoplasms, Nerve Tissue
D018193	Neoplasms, Complex and Mixed
D008577	Meningeal Neoplasms
D016543	Central Nervous System Neoplasms
D009423	Nervous System Neoplasms
D009371	Neoplasms by Site
D009422	Nervous System Diseases

ID	Term
D019772	Topotecan
D003520	Cyclophosphamide

ID	Term
D002166	Camptothecin
D000470	Alkaloids
D006571	Heterocyclic Compounds
D010752	Phosphoramide Mustards
D009588	Nitrogen Mustard Compounds
D009150	Mustard Compounds
D006846	Hydrocarbons, Halogenated
D006838	Hydrocarbons
D009930	Organic Chemicals
D063088	Phosphoramides
D009943	Organophosphorus Compounds