The Use of 5-aminolevulinic Acid (ALA) as an Intraoperative Tumor Marker for Resection of Pediatric Central Nervous System (CNS) Tumors

NCT02050243 | Unknown Phase 1

• 1 other identifier: TASMC-13-JR-528-CTIL
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

Surgery is the cornerstone treatment of most pediatric CNS tumors, including astrocytomas, ependymomas, medulloblastomas, and many other pathologies. In most pediatric CNS tumors, the aim of surgery is maximal tumor resection, while preserving neurological function. Extent of tumor residual has been shown to be a major prognostic factor for progression free survival (PFS), and survival in several malignant and low-grade tumors such as medulloblastomas, ependymomas, and astrocytic tumors. 5-aminolevulinic acid (5-ALA) has been shown to be valuable in intraoperative marking of various cancers. Following oral admission, during surgery, the tumor tissue is illuminated by blue light. Tumor cells tend to metabolize 5-ALA to a porphyrin named protoporhyrin IX (PpIX). PpIX reacts with the blue light and emits a pinky color (- fluorescence). This enables the surgeon to better identify tumor cells and perform a more extensive resection. Over recent years, many studies have proven the efficacy using 5-ALA for resecting various intracranial and spinal tumors, thus achieving a better tumor control. In the suggested study, we propose using the same technique for various pediatric central nervous system tumors. We will focus on the correlation between various pathologies and the fluorescence, trying to deduce the role of 5-ALA in resection of specific pathologies. Also, we will study the safety of 5-ALA use in the pediatric population.

Conditions

Central Nervous System Tumor, Pediatric

Keywords

pediatrics; brain tumor; CNS; fluorescence; 5ALA

Outcome Measures

Primary Outcomes (2)

• sensitivity of 5ALA fluorescence to intraoperatively detect pediatric CNS tumor tissue

  about 3hour following the 5ALA admission, the patient undergoes surgery. during surgery, global impression of tumor fluorescence will be appreciated (none, inhomogenous, intense homogenous) additionally, tumor samples will be taken from various regions, including various fluorescence regions, to try and correlate tumor regions (e.g. necrosis and viable tumor) with fluorescence (and measure the sensitivity of the 5ALA to the specific tumor)

  up to 2 weeks

• number of patients with 5ALA related side effects

  5ALA is known to cause skin hypersensitivity reaction following direct sun light exposure, during the first 48 hours after admission. also, transient elevation in liver enzymes have been documented. the investigators will supervise these reactions during the postoperative phase

  2 weeks

Study Arms (1)

5ALA

EXPERIMENTAL

All included patients will receive 20mg/kg of 5ALA (oral suspension) about 3 hours prior to surgery

Drug: 5ALA

Interventions

5ALA DRUG

20mg/kg of oral suspension of 5ALA

5ALA

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 3 -18 years old
• Any CNS related pathology (including intraaxial and extraaxial tumors, intracranial or spinal intradural) that is planned for either open (microscopic) resection (or biopsy), or lesions undergoing stereotactic biopsies.
• Parental consent
• No personal or familial (1st degree) history of porphyria
• Liver function test within normal limits (alanine aminotransferase (ALT), aspartate aminotransferase (AST)\<2 \* upper normal limit)
• Normal renal function (Cr \<2)

You may not qualify if:

• Surgery with no microscopic use (i.e. purely endoscopic surgeries)
• History of hepatic disease within last 12 months
• History of cutaneous photosensitivity, hypersensitivity to porphyrins, photodermatosis, exfoliative dermatitis, or porphyria
• Inability to comply with photosensitivity precautions

Sponsors & Collaborators

• Tel-Aviv Sourasky Medical Center: lead

Study Sites (1)

Department of pediatric neurosurgery

Tel Aviv, Israel, 64239, Israel

Location

MeSH Terms

Conditions

Central Nervous System Neoplasms; Brain Neoplasms

Condition Hierarchy (Ancestors)

Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Nervous System Diseases; Brain Diseases; Central Nervous System Diseases

Central Study Contacts

Jonathan Roth, MD

CONTACT

972-524262095; jonaroth@gmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: research and development director

Study Record Dates

• First Submitted: November 20, 2013
• First Posted: January 30, 2014
• Study Start: February 1, 2014
• Primary Completion: February 1, 2016
• Study Completion: February 1, 2017
• Last Updated: January 30, 2014

Record last verified: 2014-01

Locations

IL (1)