Central Nervous System Tumor, PediatricPhase 1/25-ALANCT02050243
The Use of 5-aminolevulinic Acid (ALA) as an Intraoperative Tumor Marker for Resection of Pediatric Central Nervous System (CNS) Tumors
A Phase 1/2 study testing 5ALA in people with Central Nervous System Tumor, Pediatric, run by Tel-Aviv Sourasky Medical Center at 1 site in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Central Nervous System Tumor, Pediatric
- What is being tested?
- 5ALA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 1 siteIsrael
- Study dates
- Feb 2014to Feb 2017
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Israel1 site
Department of pediatric neurosurgery
Tel Aviv, Israel, 64239
Jonathan Roth, MD, 972-524262095, jonaroth@gmail.com
Jonathan Roth, MD
About this study
Official title: The Use of 5-aminolevulinic Acid (ALA) as an Intraoperative Tumor Marker for Resection of Pediatric CNS Tumors
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.
Read more
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.
Read the detailed description
Following the above general description, eligible children will receive 5-aminolevulinic acid (5ALA) prior to surgery.
During surgery, careful attention will be payed to the fluorescence from the tumor, as well as the ability to differentiate between tumor and non tumorous tissue.
Comparisons between fluorescence and pathologies will be performed. Additionally, careful documentation of side effects will be done, to increase the safety of 5ALA use.
- Conditions
- Central Nervous System Tumor, Pediatric
- Being tested
- 5ALA
- Keywords
- pediatrics, brain tumor, CNS, fluorescence, 5ALA
- Medical subject headings
- Central Nervous System Neoplasms, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5ALADrug
20mg/kg of oral suspension of 5ALA
| Study group | Role | What they receive |
|---|---|---|
| 5ALA | Experimental | All included patients will receive 20mg/kg of 5ALA (oral suspension) about 3 hours prior to surgery Receives: Drug: 5ALA |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
Measured: 2 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2014 (estimated)
- Study startFeb 2014
- Primary completionFeb 2016 (estimated)
- Study completionFeb 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Central Nervous System Tumor, PediatricStudies of the same condition, open and under way.
- More trials run by Tel-Aviv Sourasky Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tel-Aviv Sourasky Medical Center
- Sponsor type
- Other government
- Responsible party
- Principal investigator, michal roll, Tel-Aviv Sourasky Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02050243
- Sponsor's protocol ID
- TASMC-13-JR-528-CTIL
- Acronym
- 5-ALA
- Status last verified
- Jan 2014
- First submitted
- 20 Nov 2013
- Last updated
- 30 Jan 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Use of 5-aminolevulinic Acid (ALA) as an Intraoperative Tumor Marker for Resection of Pediatric CNS Tumors
- ClinicalTrials.gov ID
- NCT02050243
- Acronym / sponsor ID
- 5-ALA / TASMC-13-JR-528-CTIL
- Conditions
- Central Nervous System Tumor, Pediatric
- Keywords
- pediatrics, brain tumor, CNS, fluorescence, 5ALA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Tel-Aviv Sourasky Medical CenterOther government
- Study start
- Feb 2014
- Primary completion
- Feb 2016 (estimated)
- Study completion
- Feb 2017 (estimated)
- First posted
- 30 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Following the above general description, eligible children will receive 5-aminolevulinic acid (5ALA) prior to surgery.
During surgery, careful attention will be payed to the fluorescence from the tumor, as well as the ability to differentiate between tumor and non tumorous tissue.
Comparisons between fluorescence and pathologies will be performed. Additionally, careful documentation of side effects will be done, to increase the safety of 5ALA use.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 5ALA | Experimental | Drug: 5ALAAll included patients will receive 20mg/kg of 5ALA (oral suspension) about 3 hours prior to surgery |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 5ALA20mg/kg of oral suspension of 5ALA | Drug | 5ALA |
Primary outcome
sensitivity of 5ALA fluorescence to intraoperatively detect pediatric CNS tumor tissue
Time frame: up to 2 weeks
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)
number of patients with 5ALA related side effects
Time frame: 2 weeks
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 2014.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing 5ALA in people with Central Nervous System Tumor, Pediatric, run by Tel-Aviv Sourasky Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 30 Jan 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Jonathan Roth, MD (972-524262095, jonaroth@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
sensitivity of 5ALA fluorescence to intraoperatively detect pediatric CNS tumor tissue (time frame: up to 2 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jan 2014.