67Cu-SARTATE™ Peptide Receptor Radionuclide Therapy Administered to Pediatric Patients With High-Risk, Relapsed, Refractory Neuroblastoma

NCT04023331 | Terminated Phase 1 FDA Drug

• 1 other identifier: CL04
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 8

Brief Summary

The aim of this study is to evaluate the safety and efficacy of 67Cu-SARTATE in pediatric patients with high-risk neuroblastoma.

Conditions

Neuroblastoma; Relapsed Neuroblastoma; Refractory Neuroblastoma

Outcome Measures

Primary Outcomes (5)

• Maximum Tolerated Dose (MTD)/Maximum Feasible Dose (MFD) of 67Cu-SARTATE

  MDT/MFD as determined by cohort observations of Dose Limiting Toxicities

  6 weeks

• Safety and tolerability of 67Cu-SARTATE using Common Terminology Criteria for Adverse Events (CTCAE)

  Safety will be assessed via vital signs, pathology tests (hematology, biochemistry, urinalysis, coagulation), physical examinations, electrocardiograms (ECGs) and spontaneous adverse event (AE) reporting.

  Up to 36 months

• Safety and tolerability of 64Cu-SARTATE using CTCAE

  Safety will be assessed via vital signs, pathology tests (hematology, biochemistry, urinalysis, coagulation), physical examinations, ECGs and spontaneous AE reporting.

  Up to 36 months

• Overall response rate

  As assessed by international neuroblastoma response criteria (INRC).

  Up to 12 months

• Best response

  As assessed by international neuroblastoma response criteria (INRC).

  6 to 8 weeks post final therapy

Study Arms (1)

67Cu-SARTATE

EXPERIMENTAL

64Cu-SARTATE - patients will receive a bolus injection of 64Cu-SARTATE during screening, and following each 67Cu-SARTATE Therapy Cycle, at a rate of 2.0 MBq/kg. 67Cu-SARTATE - In the dose escalation phase, patients will receive a single administration of 67Cu-SARTATE as an IV infusion (dose will be determined based on cohort allocation). In the expansion phase, patients will receive at least 2 administrations of 67Cu-SARTATE at the MTD/MFD level as a slow IV infusion. Participants in either phase of the study that demonstrate therapeutic benefit following treatment with 67Cu-SARTATE at any dose may be offered additional Therapy Cycles (each participant may receive a maximum of 4 Therapy Cycles in total).

Drug: 67Cu-SARTATE; Drug: 64Cu-SARTATE

Interventions

67Cu-SARTATE DRUG

67Cu-labelled MeCOSar-Tyr3-octreotate

Also known as: Cu-67 SARTATE, copper 67 SARTATE

67Cu-SARTATE

64Cu-SARTATE DRUG

64Cu-labelled MeCOSar-Tyr3-octreotate

Also known as: Cu-64 SARTATE, copper 64 SARTATE

67Cu-SARTATE

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant is able and willing to provide informed consent (≥18 years), or informed consent is obtained by the parent or legal guardian for minor participants, with the minor providing age appropriate assent, according to local law and regulations;
• Life expectancy ≥ 12 weeks for patients in the Dose Escalation Phase or ≥ 4 months for patients in the Cohort Expansion Phase;
• Known high-risk neuroblastoma OR previously intermediate-risk neuroblastoma that has relapsed or progressed to high-risk, with failure to achieve complete response with standard therapy (defined as at least 4 cycles of aggressive multi-drug induction chemotherapy with or without radiation and surgery, or according to a standard high-risk treatment/neuroblastoma protocol), OR who are medically ineligible to receive standard treatment OR who are intolerant to standard treatment;
• Adequate recovery from acute toxic effects of any prior therapy, as deemed by the Investigator or treating Sub-Investigator;
• Adequate liver function as defined by the following laboratory values obtained within 28 days prior to administration of 64Cu-SARTATE: Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \< 3.0 x upper limit of normal (ULN);
• Adequate renal function;
• Adequate laboratory parameters: Absolute neutrophil count \> 1.0 x 10 9/L; Platelet count \> 50 x 10 9/L; Total bilirubin \<1.5 x ULN;
• Karnofsky or Lansky performance status ≥50;
• All participants must have a hematopoietic stem cell product available (minimum CD34+ cell dose is ≥2 x 10 6 cells/kg);
• Sexually active participants of reproductive potential must practice an effective method of birth control while participating on this study, to avoid possible damage to the fetus. Abstinence is considered acceptable;
• Cu-SARTATE uptake on the 4 hour scan (SUVmax) of any lesion equal to or higher than that of the liver in order to move on to the therapy phase of the study.

You may not qualify if:

• Participants with disease of any major organ system that would compromise their ability to tolerate therapy, as deemed by the Investigator or treating Sub-Investigator;
• Participants with intracranial tumor affecting the brain parenchyma that has not been previously treated at the time of study enrolment;
• Any other active malignancy, or a history of prior malignancy within the past 3 years;
• History of cardiac failure as evidenced by: dyspnea at rest, exercise intolerance, oxygen requirement, clinically significant cardiac dysfunction;
• Planned administration of chemotherapy, anti-cancer cytokine therapy, immunotherapy or radiotherapy within 2 weeks prior to the administration of 64Cu-SARTATE;
• Administration of therapeutic dose of 131I-MIBG within 8 weeks prior to the administration of 64Cu-SARTATE;
• External beam radiation therapy (EBRT) to both kidneys or a single functioning kidney within 12 months prior to the administration of 64Cu-SARTATE;
• Administration of any investigational agents within 21 days prior to administration of 64Cu-SARTATE;
• Treatment with long acting somatostatin analogues (administered within 28 days prior to the administration of 64Cu-SARTATE), or short acting somatostatin analogues (administered within 24 hours prior to the administration of 64Cu-SARTATE);
• Known sensitivity or allergy to somatostatin analogues;
• Previous peptide receptor radionuclide therapy (PRRT);
• Female participants who are pregnant or lactating;
• Participants who are on hemodialysis;
• QTcF interval ≥ 0.45 seconds as measured by Screening ECG;
• Participants with uncontrolled infection(s);

Sponsors & Collaborators

• Clarity Pharmaceuticals Ltd: lead

Study Sites (8)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Cincinnati Children's Hospital Medical Centre

Cincinnati, Ohio, 45229, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

University of Texas Southwestern Medical Centre

Dallas, Texas, 75390, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

University of Wisconsin

Madison, Wisconsin, 53792, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

64Cu-SARTATE

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Design

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

Study Record Dates

• First Submitted: July 14, 2019
• First Posted: July 17, 2019
• Study Start: August 18, 2020
• Primary Completion: January 24, 2025
• Study Completion: March 25, 2025
• Last Updated: September 16, 2025

Record last verified: 2025-09

Locations

US (8)