Study of BLZ-100 in Pediatric Subjects With CNS Tumors

NCT02462629 | Completed Phase 1 DMC

• 1 other identifier: BB-004
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Central Nervous System Tumors

Keywords

central nervous system tumor; BLZ-100; pediatrics; Tumor Paint; brain cancer; tozuleristide

Outcome Measures

Primary Outcomes (2)

• Number of participants with adverse events

  Safety will be assessed by physical examination and measurement of vital signs and laboratory safety parameters.

  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.

  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

  At least 1 hour post-dose

• Extent of resection (expansion cohort only)

  At completion of surgery for surgeon assessment and up to 1 week after surgery for MRI assessment

Study Arms (1)

BLZ-100

EXPERIMENTAL

Drug: BLZ-100

Interventions

BLZ-100 DRUG

Also known as: Tumor Paint, tozuleristide

BLZ-100

Eligibility Criteria

• Age: 1 Month - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Blaze Bioscience Inc.: lead

Study Sites (1)

Seattle Children's

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Central Nervous System Neoplasms; Brain Neoplasms

Interventions

tozuleristide

Condition Hierarchy (Ancestors)

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

Study Officials

• Sarah Leary, MD, MS

  Seattle Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2015
• First Posted: June 4, 2015
• Study Start: June 1, 2015
• Primary Completion: July 1, 2018
• Study Completion: August 1, 2018
• Last Updated: February 21, 2023

Record last verified: 2023-02

Locations

US (1)