Central Nervous System TumorsPhase 1NCT02462629
Study of BLZ-100 in Pediatric Subjects With CNS Tumors
A Phase 1 study testing BLZ-100 in people with Central Nervous System Tumors, run by Blaze Bioscience Inc. at 1 site 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
- Central Nervous System Tumors
- What is being tested?
- BLZ-100
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2015to Aug 2018
- Phase
- Phase 1
- 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 1 month to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age: >1 month and <30 years at time of enrollment
- Diagnosis: Must have MRI documenting a measurable lesion w/in central nervous system consistent with primary central nervous system tumor, for which maximal safe surgical resection is indicated
- Timing of surgery: Must be anticipated to take place at least 1 hour after BLZ-100 administration
- Prior therapy: Must have recovered from acute toxic effects of prior anti-cancer therapy (durations relative to date of enrollment):
- Radiation therapy: may not have had radiation therapy to area of tumor planned to be resected w/in 28 days
- Chemotherapy: ≥14 days from any myelosuppressive chemotherapy and abs neutrophil ct ≥1000/mm3 , 42 days if prior nitrosurea
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- Biologic: ≥7 days from anti-neoplastic biologic agent
- Immunotherapy: ≥42 days after completion of immunotherapy
- Monoclonal antibody: ≥3 half-lives of antibody since last admin.
- Organ function requirements:
- Adequate renal function defined as:
- serum creatinine w/in normal limits or
- calculated creatinine clearance > 100 mL/min/1.73 m2
- Adequate liver function defined as:
- bilirubin <2x upper limit of normal for age
- alanine aminotransferase <3x upper limit of normal for age (<135 U/mL)
- serum albumin >2g/dL
- Adequate coagulation defined as:
- no evidence of active/clinically significant bleeding. May be evidence of punctate hemorrhage w/in tumor as long as not considered clinically significant to warrant urgent surgical evacuation
- internal normalized ratio and partial thromboplastin time <1.5x upper limit of normal
- Informed Consent: Written informed consent must be obtained from subject or parent/legal guardian prior to enrollment. Assent, when appropriate, will be obtained according to institutional guidelines
Who cannot join
- Pregnancy and contraception: Subjects who are pregnant, breast-feeding or planning to conceive within 30 days are not eligible
- Concomitant medications:
- Corticosteroids: no restrictions
- Investigational drugs: must not be receiving other investigational (from other studies) drugs at time of enrollment and must not be planning to take other investigational drugs during DLT period
- Anti-cancer agents: must not be receiving other anti-cancer agents at time of enrollment and must not be planning to take other anti-cancer agents during DLT period
- Anticoagulation: if currently receiving therapeutic anticoagulation with heparin, low-molecular weight heparin, or Coumadin, not eligible
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- Anti-platelet agents: if currently receiving aspirin, ibuprofen or other non-steroidal anti-inflammatory or anti-platelet agents, not eligible
- Photosensitizing drugs, medications which might generate fluorescence or according to label, might generate photochemical reaction. These include hematoporphyrin derivatives and purified fractions; Photofrin®; and precursors of protoporphyrin IX (5-Aminolevulinic acid) used in Gliolan or Hexvix
- Subjects must not have received BLZ-100 within 30 days prior to re-treatment
- Infection: Subjects with uncontrolled infection not eligible
- Bleeding and Thrombosis:
- If active bleeding requiring acute surgical intervention, not eligible
- Subjects with stroke, arterial or venous thrombosis within 6 months not eligible
- Subjects requiring anticoagulation not eligible
- Acuity of surgical needs: Subjects with acute neurologic compromise, symptoms of impending cerebral herniation, or other condition(s) necessitating urgent or emergent neurosurgical intervention to be planned within 2 hours not eligible. NOTE: If subject is enrolled on study, receives study medication and subsequently condition worsens such that urgent surgical intervention is felt to be in best interest of subject, best interest of subject should always take precedence over timing between study medication and surgery
- Required observations: Subjects who in opinion of investigator may not be able to comply with required safety and monitoring requirements are not eligible
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Dose-escalation and Expansion Study of BLZ-100 in Pediatric Subjects With Primary Central Nervous System Tumors
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. The investigators hypothesize that BLZ-100 will improve surgical outcomes by allowing surgeons to visualize the edges of the tumor and small groups of cancer cells that have spread to other sites in real-time as they operate. This is a safety study to assess the safety of BLZ-100 in pediatric patients with 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.
Subjects will be required to arrive at the hospital for dosing at least 1 hour before the planned surgical excision. Following dosing, subjects will be monitored for safety and surgical excision will occur at least 1 hour after study product administration.
- Conditions
- Central Nervous System Tumors
- Being tested
- BLZ-100
- Keywords
- central nervous system tumor, BLZ-100, pediatrics, Tumor Paint, brain cancer, tozuleristide
- 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.
BLZ-100Drug
Also called Tumor Paint, tozuleristide
| Study group | Role | What they receive |
|---|---|---|
| BLZ-100 | Experimental | Not described Receives: Drug: BLZ-100 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events
Safety will be assessed by physical examination and measurement of vital signs and laboratory safety parameters.
Measured: Seven days after study drug administration
Change in concentration of BLZ-100 in the blood
BLZ-100 levels in blood will be analyzed by chemical means and these data will be used to calculate pharmacokinetic parameters.
Measured: Prior to dosing and at 10 minutes, 30 minutes, and 60 minutes post BLZ-100 administration
Secondary outcomes (2)
Fluorescence signal in excised central nervous system tumors
Fluorescence signal in sections of excised tumor and adjacent normal tissue will be measured using an infrared imaging system.
Measured: At least 1 hour post-dose
Extent of resection (expansion cohort only)
For subjects enrolled in the expansion cohort, an extent of resection assessment will be made by the surgeon and confirmed by post-operative MRI according to standard of care procedures.
Measured: At completion of surgery for surgeon assessment and up to 1 week after surgery for MRI assessment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2015 (estimated)
- Study startJun 2015
- Primary completionJul 2018
- Study completionAug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Central Nervous System TumorsStudies 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 1
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blaze Bioscience Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sarah Leary, MD, MS, Principal investigator, Seattle Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02462629
- Sponsor's protocol ID
- BB-004
- Status last verified
- Feb 2023
- First submitted
- 2 Jun 2015
- Last updated
- 21 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Dose-escalation and Expansion Study of BLZ-100 in Pediatric Subjects With Primary Central Nervous System Tumors
- ClinicalTrials.gov ID
- NCT02462629
- Acronym / sponsor ID
- BB-004
- Conditions
- Central Nervous System Tumors
- Keywords
- central nervous system tumor, BLZ-100, pediatrics, Tumor Paint, brain cancer, tozuleristide
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Blaze Bioscience Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2015 (actual)
- Primary completion
- Jul 2018 (actual)
- Study completion
- Aug 2018 (actual)
- First posted
- 4 Jun 2015 (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.
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. The investigators hypothesize that BLZ-100 will improve surgical outcomes by allowing surgeons to visualize the edges of the tumor and small groups of cancer cells that have spread to other sites in real-time as they operate. This is a safety study to assess the safety of BLZ-100 in pediatric patients with 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.
Subjects will be required to arrive at the hospital for dosing at least 1 hour before the planned surgical excision. Following dosing, subjects will be monitored for safety and surgical excision will occur at least 1 hour after study product administration.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BLZ-100 | Experimental | Drug: BLZ-100 |
Who may qualify
- Ages
- 1 month to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (21 listed)
- Age: >1 month and <30 years at time of enrollment
- Diagnosis: Must have MRI documenting a measurable lesion w/in central nervous system consistent with primary central nervous system tumor, for which maximal safe surgical resection is indicated
- Timing of surgery: Must be anticipated to take place at least 1 hour after BLZ-100 administration
- Prior therapy: Must have recovered from acute toxic effects of prior anti-cancer therapy (durations relative to date of enrollment):
- Organ function requirements:
Key exclusion criteria (16 listed)
- Pregnancy and contraception: Subjects who are pregnant, breast-feeding or planning to conceive within 30 days are not eligible
- Concomitant medications:
- Infection: Subjects with uncontrolled infection not eligible
- Bleeding and Thrombosis:
- Acuity of surgical needs: Subjects with acute neurologic compromise, symptoms of impending cerebral herniation, or other condition(s) necessitating urgent or emergent neurosurgical intervention to be planned within 2 hours not eligible. NOTE: If subject is enrolled on study, receives study medication and subsequently condition worsens such that urgent surgical intervention is felt to be in best interest of subject, best interest of subject should always take precedence over timing between study medication and surgery
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 |
|---|---|---|
| BLZ-100Also: Tumor Paint, tozuleristide | Drug | BLZ-100 |
Primary outcome
Number of participants with adverse events
Time frame: Seven days after study drug administration
Safety will be assessed by physical examination and measurement of vital signs and laboratory safety parameters.
Change in concentration of BLZ-100 in the blood
Time frame: Prior to dosing and at 10 minutes, 30 minutes, and 60 minutes post BLZ-100 administration
BLZ-100 levels in blood will be analyzed by chemical means and these data will be used to calculate pharmacokinetic parameters.
2 secondary outcomes
Fluorescence signal in excised central nervous system tumors
Time frame: At least 1 hour post-dose
Extent of resection (expansion cohort only)
Time frame: At completion of surgery for surgeon assessment and up to 1 week after surgery for MRI assessment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2023.
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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, MS principal investigator
Seattle Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BLZ-100 in people with Central Nervous System Tumors, run by Blaze Bioscience Inc. at 1 site 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 21 inclusion and 16 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 21 Feb 2023. 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 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of participants with adverse events (time frame: Seven days after study drug administration). 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 21 Feb 2023.