Pediatric Central Nervous System TumorPhase 2/3NCT03579602
Study of Tozuleristide and the Canvas Imaging System in Pediatric Subjects With CNS Tumors Undergoing Surgery
A Phase 2/3 study testing tozuleristide and Canvas System in people with Pediatric Central Nervous System Tumor, run by Blaze Bioscience Inc. at 9 sites in United States.
Trial summary
Many types of cancer are primarily treated with surgery and patient survival is directly related to the extent to which the tumor is able to be removed. It is often difficult for surgeons to distinguish tumor tissue from normal tissue or to detect tumor cells that have spread from the original tumor site, resulting in… Read more about this study
- Who is it for?
- Ages 1 month to 30 years
- Conditions the study is for
- Pediatric Central Nervous System Tumor
- What is being tested?
- tozuleristide and Canvas System
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 118 participants
- Where
- 9 sitesUnited States
- Study dates
- 26 Nov 2018to 13 Jun 2022
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 1 month to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Subjects must be >1 month and ≤30 years of age at the time of study enrollment
- Subjects must have MRI obtained within 30 days of study enrollment documenting a measurable lesion consistent with a pediatric primary CNS tumor for which maximal safe resection is indicated
- Adequate renal function
- Adequate liver function
- Prior therapy: Subjects with prior therapy are eligible provided they have recovered from any acute toxic effects of prior therapy and have sufficient time interval prior to enrollment:
- Radiation therapy: subjects may not have had radiation therapy to the area of tumor planned to be resected within 28 days of study enrollment
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- Chemotherapy: at least 14 days from any myelosuppressive chemotherapy (28 days if prior nitrosourea) and if prior chemotherapy, must have an absolute neutrophil count recovery of ≥ 1000/mm3 following count nadir
- Biologic: at least 7 days from any anti-neoplastic biologic agent (at least 3 half-lives since last administration of monoclonal antibodies)
- Immunotherapy: at least 42 days after completion of any cellular immunotherapy, such as CAR-T cell therapy
- Prior surgery for CNS tumors is allowed
- Prior tozuleristide: at least 1 week after prior dose of tozuleristide if previously treated
- Written informed consent must be obtained from the subject or parent or legal guardian prior to the conduct of study activities. Routine clinical tests, e.g., MRI, clinical laboratory studies, may be used for screening requirements. Assent, when appropriate, will be obtained according to institutional guidelines.
- The risks of treatment with tozuleristide during pregnancy have not been evaluated. Female subjects of child-bearing potential must agree not to attempt to become pregnant or undergo in vitro fertilization and, if participating in sexual activity that could lead to pregnancy, must use 2 reliable methods of contraception simultaneously for 30 days after surgery. Male subjects must agree not to attempt to father a child and, if participating in sexual activity that could lead to pregnancy, must use 2 reliable methods of contraception simultaneously for 30 days after surgery if their partner is of child-bearing potential.
Who cannot join
- Pregnancy and contraception: Subjects who are pregnant or breast-feeding or planning to conceive a child within 30 days are not eligible. Males and females of childbearing potential must agree to use 2 effective forms of contraception from the time of enrollment until 30 days post-surgery
- Subjects with on-going serious medical conditions (poorly controlled asthma, diabetes, heart disease) such that participation in the study could put the subject at increased risk of worsening their condition
- Subjects planned to undergo only a diagnostic biopsy procedure, without intent to resect tissue for therapeutic purposes (e.g., stereotactic pontine biopsy)
- Subjects who in the opinion of the investigator are not willing or able to comply with randomization procedures or other study-required study procedures and observations. Subjects previously enrolled and randomized to Arm 1 (control) are not eligible for re-enrollment unless a second surgery is required by standard of care.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Johns Hopkins University
Baltimore, Maryland, 21287
Children's Hospital of Michigan
Detroit, Michigan, 48201
University of Florida Shands Hospital
Gainesville, Florida, 32608
Chlidren's Hospital Los Angeles
Los Angeles, California, 90027
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
Children's Minnesota
Saint Paul, Minnesota, 55102
University of California, San Francisco
San Francisco, California, 94158
Seattle Children's Hospital
Seattle, Washington, 98105
Washington University St. Louis Children's Hospital
St Louis, Missouri, 63110
About this study
Official title: A Randomized, Blinded Study of Fluorescence Detection of Pediatric Primary Central Nervous System Tumors in Subjects Receiving Tozuleristide and Imaged With the Canvas System
Many types of cancer are primarily treated with surgery and patient survival is directly related to the extent to which the tumor is able to be removed. It is often difficult for surgeons to distinguish tumor tissue from normal tissue or to detect tumor cells that have spread from the original tumor site, resulting in incomplete removal of the tumor and reduced patient survival. In some sites, such as the brain, it is critical to avoid damage to normal tissue around the tumor to prevent adverse effects of surgery on function. Tozuleristide is a drug that is thought to attach to tumor tissue and then fluoresces (glows) when a special light from the Canvas is shined on it. It is hypothesized that tozuleristide, when imaged with the Canvas, will improve surgical outcomes by allowing surgeons to visualize the edges of the tumor or other ambiguous tissue in real-time as they operate. The purpose of this study is to evaluate how well tozuleristide imaged with Canvas work at helping to distinguish between tumor and normal tissue during surgery in pediatric primary central nervous system tumors.
Read the detailed description
Subjects who provide voluntary written informed consent, or have it provided by their legally acceptable representative, will be screened for eligibility. Subjects meeting all of the inclusion and none of the exclusion criteria will be eligible to participate.
Surgical excision will occur at least 1 hour and no more than 36 hours after tozuleristide administration. Surgery will be performed by a neurosurgeon and the Canvas will be operated by a designated Imaging Operator. Fluorescence of tumor and ambiguous tissue during surgery will be assessed and scored. Biopsy samples of these tumor and ambiguous tissues will be collected for pathology analysis.
All subjects will be monitored for safety during their participation in the study.
- Conditions
- Pediatric Central Nervous System Tumor
- Being tested
- tozuleristide, Canvas System
- Keywords
- Tumor Paint, tozuleristide, Canvas imaging system, BLZ-100, CNS tumor, brain cancer, pediatrics, fluorescence guided surgery
- 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.
tozuleristideDrug
Also called BLZ-100, Tumor Paint
Tozuleristide will be administered at least 1 hour and no more than 36 hours prior to planned surgical excision of their tumor.
Canvas SystemDevice
Also called mSIRIS
All subjects enrolled in the study will have their tissue imaged with the Canvas System.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 (no tozuleristide) | Active comparator | Subjects randomized to Arm 1 (~9% of subjects) will not receive tozuleristide but will undergo standard of care neurosurgery and will have imaging performed with the Canvas System. Receives: Device: Canvas System |
| Arm 2 (tozuleristide treated) | Experimental | Subjects randomized to Arm 2 (~ 91% of subjects) will be administered tozuleristide at a dose of 15 mg/m^2 at least 1 hour and no more than 36 hours prior to surgery. They will undergo standard of care neurosurgery and will have imaging performed with the Canvas System. Receives: Drug: tozuleristide; Device: Canvas System |
What is being measured
The primary outcome is the main question the study is designed to answer.
Sensitivity and Specificity of Tozuleristide Fluorescence to Detect Tumor in Equivocal (Ambiguous) Tissue During Surgery When Imaged With the Canvas System
Sensitivity and specificity of tozuleristide imaged with the Canvas system to fluorescently identify tumor in equivocal tissue will be evaluated based on central pathology consensus assessment of tumor and by imaging operator fluorescence assessment. These measures will be compared to the sensitivity and specificity of surgical designation of tumor (more likely tumor or less likely tumor) under white light. Sensitivity reflects the probability that tumor tissue evaluated is fluorescent. Specificity reflects the probability that normal tissue evaluated is not fluorescent. Equivocal tissues are those that the surgeon considers abnormal but ambiguous as tumor vs. not tumor under normal (white light) conditions.
Measured: During surgery (which occurs at least 1 hour post tozuleristide administration)
Ratio of Tozuleristide Sensitivity Compared to Surgeon Sensitivity
Tozuleristide sensitivity as assessed by imaging operator fluorescence assessment compared to sensitivity of surgeon designation of tumor under white light in equivocal tissue biopsies. Sensitivity reflects the probability that tumor tissue evaluated is fluorescent. Equivocal tissues are those that the surgeon considers abnormal but ambiguous as tumor vs. not tumor under normal (white light) conditions.
Measured: During surgery (which occurs at least 1 hour post tozuleristide administration)
Ratio of Tozuleristide Specificity Compared to Surgeon Specificity
Tozuleristide specificity as assessed by imaging operator fluorescence assessment compared to specificity of surgeon designation of tumor under white light in equivocal tissue biopsies. Specificity reflects the probability that normal tissue evaluated is not fluorescent. Equivocal tissues are those that the surgeon considers abnormal but ambiguous as tumor vs. not tumor under normal (white light) conditions.
Measured: During surgery (which occurs at least 1 hour post tozuleristide administration)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jul 2018
- Study start26 Nov 2018
- Primary completion1 Apr 2022
- Study completion13 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Pediatric Central Nervous System TumorStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Subjects will be randomized 1:10 to 1 of 2 study arms. Subjects in arm 1 (\~9% of subjects) will not receive tozuleristide but will undergo neurosurgery and imaging will be performed with the Canvas. Subjects in arm 2 (\~91% of subjects) will receive tozuleristide and will undergo neurosurgery and imaging will be performed with the Canvas.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 118 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blaze Bioscience Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Pacific Pediatric Neuro-Oncology Consortium
- Responsible party
- Sponsor
- Investigators
- Sarah Leary, MD, Principal investigator, Seattle Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03579602
- Sponsor's protocol ID
- BB-006
- Other IDs
- PNOC012
- Status last verified
- Jun 2024
- First submitted
- 15 Jun 2018
- Last updated
- 27 Jun 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Blinded Study of Fluorescence Detection of Pediatric Primary Central Nervous System Tumors in Subjects Receiving Tozuleristide and Imaged With the Canvas System
- ClinicalTrials.gov ID
- NCT03579602
- Acronym / sponsor ID
- BB-006
- Conditions
- Pediatric Central Nervous System Tumor
- Keywords
- Tumor Paint, tozuleristide, Canvas imaging system, BLZ-100, CNS tumor, brain cancer, pediatrics, fluorescence guided surgery
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 118 participants (actual)
- Lead sponsor
- Blaze Bioscience Inc.Industry (pharma, biotech, devices)
- Collaborators
- Pacific Pediatric Neuro-Oncology Consortium
- Study start
- 26 Nov 2018 (actual)
- Primary completion
- 1 Apr 2022 (actual)
- Study completion
- 13 Jun 2022 (actual)
- First posted
- 6 Jul 2018 (actual)
- Results
- Posted 27 Jun 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Many types of cancer are primarily treated with surgery and patient survival is directly related to the extent to which the tumor is able to be removed. It is often difficult for surgeons to distinguish tumor tissue from normal tissue or to detect tumor cells that have spread from the original tumor site, resulting in incomplete removal of the tumor and reduced patient survival. In some sites, such as the brain, it is critical to avoid damage to normal tissue around the tumor to prevent adverse effects of surgery on function. Tozuleristide is a drug that is thought to attach to tumor tissue and then fluoresces (glows) when a special light from the Canvas is shined on it. It is hypothesized that tozuleristide, when imaged with the Canvas, will improve surgical outcomes by allowing surgeons to visualize the edges of the tumor or other ambiguous tissue in real-time as they operate. The purpose of this study is to evaluate how well tozuleristide imaged with Canvas work at helping to distinguish between tumor and normal tissue during surgery in pediatric primary central nervous system tumors.
Detailed description
Subjects who provide voluntary written informed consent, or have it provided by their legally acceptable representative, will be screened for eligibility. Subjects meeting all of the inclusion and none of the exclusion criteria will be eligible to participate.
Surgical excision will occur at least 1 hour and no more than 36 hours after tozuleristide administration. Surgery will be performed by a neurosurgeon and the Canvas will be operated by a designated Imaging Operator. Fluorescence of tumor and ambiguous tissue during surgery will be assessed and scored. Biopsy samples of these tumor and ambiguous tissues will be collected for pathology analysis.
All subjects will be monitored for safety during their participation in the study.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
- Model description
- Subjects will be randomized 1:10 to 1 of 2 study arms. Subjects in arm 1 (~9% of subjects) will not receive tozuleristide but will undergo neurosurgery and imaging will be performed with the Canvas. Subjects in arm 2 (~91% of subjects) will receive tozuleristide and will undergo neurosurgery and imaging will be performed with the Canvas.
| Arm | Type | Receives |
|---|---|---|
| Arm 1 (no tozuleristide) | Active comparator | Device: Canvas SystemSubjects randomized to Arm 1 (~9% of subjects) will not receive tozuleristide but will undergo standard of care neurosurgery and will have imaging performed with the Canvas System. |
| Arm 2 (tozuleristide treated) | Experimental | Drug: tozuleristide, Device: Canvas SystemSubjects randomized to Arm 2 (~ 91% of subjects) will be administered tozuleristide at a dose of 15 mg/m^2 at least 1 hour and no more than 36 hours prior to surgery. They will undergo standard of care neurosurgery and will have imaging performed with the Canvas System. |
Who may qualify
- Ages
- 1 month to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Subjects must be >1 month and ≤30 years of age at the time of study enrollment
- Subjects must have MRI obtained within 30 days of study enrollment documenting a measurable lesion consistent with a pediatric primary CNS tumor for which maximal safe resection is indicated
- Adequate renal function
- Adequate liver function
- Prior therapy: Subjects with prior therapy are eligible provided they have recovered from any acute toxic effects of prior therapy and have sufficient time interval prior to enrollment:
Key exclusion criteria (4 listed)
- Pregnancy and contraception: Subjects who are pregnant or breast-feeding or planning to conceive a child within 30 days are not eligible. Males and females of childbearing potential must agree to use 2 effective forms of contraception from the time of enrollment until 30 days post-surgery
- Subjects with on-going serious medical conditions (poorly controlled asthma, diabetes, heart disease) such that participation in the study could put the subject at increased risk of worsening their condition
- Subjects planned to undergo only a diagnostic biopsy procedure, without intent to resect tissue for therapeutic purposes (e.g., stereotactic pontine biopsy)
- Subjects who in the opinion of the investigator are not willing or able to comply with randomization procedures or other study-required study procedures and observations. Subjects previously enrolled and randomized to Arm 1 (control) are not eligible for re-enrollment unless a second surgery is required by standard of care.
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 |
|---|---|---|
| tozuleristideAlso: BLZ-100, Tumor PaintTozuleristide will be administered at least 1 hour and no more than 36 hours prior to planned surgical excision of their tumor. | Drug | Arm 2 (tozuleristide treated) |
| Canvas SystemAlso: mSIRISAll subjects enrolled in the study will have their tissue imaged with the Canvas System. | Device | Arm 1 (no tozuleristide), Arm 2 (tozuleristide treated) |
Primary outcome
Sensitivity and Specificity of Tozuleristide Fluorescence to Detect Tumor in Equivocal (Ambiguous) Tissue During Surgery When Imaged With the Canvas System
Time frame: During surgery (which occurs at least 1 hour post tozuleristide administration)
Sensitivity and specificity of tozuleristide imaged with the Canvas system to fluorescently identify tumor in equivocal tissue will be evaluated based on central pathology consensus assessment of tumor and by imaging operator fluorescence assessment. These measures will be compared to the sensitivity and specificity of surgical designation of tumor (more likely tumor or less likely tumor) under white light. Sensitivity reflects the probability that tumor tissue evaluated is fluorescent. Specificity reflects the probability that normal tissue evaluated is not fluorescent. Equivocal tissues are those that the surgeon considers abnormal but ambiguous as tumor vs. not tumor under normal (white light) conditions.
Ratio of Tozuleristide Sensitivity Compared to Surgeon Sensitivity
Time frame: During surgery (which occurs at least 1 hour post tozuleristide administration)
Tozuleristide sensitivity as assessed by imaging operator fluorescence assessment compared to sensitivity of surgeon designation of tumor under white light in equivocal tissue biopsies. Sensitivity reflects the probability that tumor tissue evaluated is fluorescent. Equivocal tissues are those that the surgeon considers abnormal but ambiguous as tumor vs. not tumor under normal (white light) conditions.
Ratio of Tozuleristide Specificity Compared to Surgeon Specificity
Time frame: During surgery (which occurs at least 1 hour post tozuleristide administration)
Tozuleristide specificity as assessed by imaging operator fluorescence assessment compared to specificity of surgeon designation of tumor under white light in equivocal tissue biopsies. Specificity reflects the probability that normal tissue evaluated is not fluorescent. Equivocal tissues are those that the surgeon considers abnormal but ambiguous as tumor vs. not tumor under normal (white light) conditions.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Sarah Leary, MD principal investigator
Seattle Children's Hospital
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing tozuleristide and Canvas System in people with Pediatric Central Nervous System Tumor, run by Blaze Bioscience Inc. at 9 sites in United States.
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 13 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: finished, as of the sponsor's last update on 27 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Jun 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Sensitivity and Specificity of Tozuleristide Fluorescence to Detect Tumor in Equivocal (Ambiguous) Tissue During Surgery When Imaged With the Canvas System (time frame: During surgery (which occurs at least 1 hour post tozuleristide administration)). The study lists 3 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 27 Jun 2024.