Luveltamab Tazevibulin (STRO-002) in Infants and Children < 12 Years of Age With Relapsed/Refractory CBFA2T3::GLIS2 AML

A Phase 1/2, Open-label Study Evaluating the Efficacy, Safety, and Pharmacokinetics (PK) of Luveltamab Tazevibulin (STRO-002) in Infants and Children < 12 Years of Age With CBFA2T3::GLIS2 Acute Myeloid Leukemia (AML)

This trial will evaluate whether luveltamab tazevibulin is well tolerated and active against a rare form of AML carrying a particular genetic abnormality called CBFA2T3::GLIS2 that arises in infants and children. To be treated in this trial children must have a leukemia which did not respond or recurred after prior treatment. Luveltamab tazevibulin is an antibody-drug conjugate, which brings tazevibulin, an anticancer drug, to a molecule called FOLR1, present on the surface of CBFA2T3::GLIS2 AML cells.

Study Overview

• Status: Terminated
• Conditions: Acute Myeloid Leukemia (AML)
• Intervention / Treatment: Drug: Luveltamab tazevibulin

Detailed Description

This is a registrational international, multicenter, two-part open label Phase 1/2 trial in an extremely rare pediatric disease (around 17 new patients a year in US and 10 in EU). Part 1 randomizes subjects 1:1 to one of two luveltamab tazevibulin dose cohorts (1a and 1b). Part 2 further evaluates the safety and the efficacy of the selected dose. Subjects who achieve complete remission after two cycles of treatment may continue luveltamab tazevibulin as monotherapy, while non-responders at PI discretion may add luveltamab tazevibulin with standard of care (SOC) AML treatments. Luveltamab tazevibulin is given IV every two week as monotherapy and every 4 weeks when given with chemotherapy.

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria, 1090
    • St Anna Kinderspital
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • The Hospital for Sick Children
  • Quebec
    • Montreal, Quebec, Canada, H3T 1C5
      • Centre Hospitalier Universitaire-Sainte Justice
• Denmark
  • Copenhagen, Denmark, 02100
    • Rigshopitalet-University of Copenhagen
• France
  • Lyon, France, 69008
    • Institut d'Hématologie et d'Oncologie Pédiatrique
  • Paris, France, 75012
    • Hôpital Armand Trousseau
• Germany
  • Berlin, Germany, 13353
    • Charite-Universitatsmedizin Berlin
  • Essen, Germany, 45122
    • Universitatsklinikum-Essen
• Italy
  • Monza, Italy, 20900
    • Fondazione IRCCS San Gerardo Dei Tintori
  • Rome, Italy, 00165
    • Children Hospital Bambino Gesu IRCCS
• Netherlands
  • CS Utrecht, Netherlands, 3584
    • Princess Máxima Center for Pediatric Oncology
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitario Vall d'Hebron
  • Madrid, Spain, 28009
    • Hospital Infantil Universitario Nino Jesus-Servicio de Hematologia
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Childrens Hospital of Alabama
  • California
    • Los Angeles, California, United States, 90027
      • Childrens Hospital of Los Angeles
    • Palo Alto, California, United States, 94304
      • Lucile Packard Childrens Hospital-Stanford
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital of Colorado
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Childrens National Hospital
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Hospital of Atlanta-Emory
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann & Robert H. Lurie Childrens Hospital
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Dana-Farber Cancer Institute
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota-Masonic Cancer Center
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
    • Saint Louis, Missouri, United States, 63110
      • Washington University-School of Medicine
  • New York
    • New York, New York, United States, 10032
      • Columbia University Irving Medical Center
  • North Carolina
    • Durham, North Carolina, United States, 27711
      • Duke University Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • St. Jude Children's Research Hospital
  • Texas
    • Fort Worth, Texas, United States, 76104
      • Cook Children's Medical Center
    • Houston, Texas, United States, 77030
      • MD Anderson Cancer Center
    • Houston, Texas, United States, 77030
      • Baylor College of Medicine-Dan Duncan Comprehensive Cancer Center
  • Virginia
    • Richmond, Virginia, United States, 23219
      • Massey Cancer Center-Adult Outpatient Pavillion
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Childrens

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• AML with CBFA2T3::GLIS2 gene fusion centrally confirmed
• Refractory or relapsed disease with ≥ 5% bone marrow involvement with leukemic blasts by morphology
• Age < 12 years.
• Lansky performance of ≥ 50
• Adequate organ functions

Exclusion Criteria:

• Active central nervous system (CNS) disease (CNS3)
• Pre-existing clinically significant corneal disorders or constitutional diseases associated with an increased risk of AML treatment toxicities
• Active or uncontrolled infections or other active severe intercurrent illnesses,
• Prior treatment with a FOLR1- targeting ADCs or with ADCs that contain a tubulin inhibitor
• History of allogeneic hematopoietic stem cell transplant or any organ transplant in the prior 84 days
• Graft versus host disease (GVHD) of any grade or GVHD treatment with exception of low dose steroids

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1; Luveltamab tazevibulin 3.5mg every 2 weeks	Drug: Luveltamab tazevibulin; Luveltamab tazevibulin is an antibody-drug conjugate (ADC) targeting folate receptor α (FRα or FOLR1). It consists of an IgG1 antibody (SP8166) conjugated to cathepsin cleavable 3-aminophenyl hemiasterlin payload, yielding a homogenous ADC with a drug antibody ratio of four. The active warhead (SC209) inhibits tubulin polymerization leading to mitotic arrest and cell death. Other Names: STRO-002; Luvelta; Other Names: STRO-002; Luvelta
Experimental: Cohort 2; Luveltamab tazevibulin 4.3 mg every 2 weeks	Drug: Luveltamab tazevibulin; Luveltamab tazevibulin is an antibody-drug conjugate (ADC) targeting folate receptor α (FRα or FOLR1). It consists of an IgG1 antibody (SP8166) conjugated to cathepsin cleavable 3-aminophenyl hemiasterlin payload, yielding a homogenous ADC with a drug antibody ratio of four. The active warhead (SC209) inhibits tubulin polymerization leading to mitotic arrest and cell death. Other Names: STRO-002; Luvelta; Other Names: STRO-002; Luvelta

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluate efficacy of luveltamab tazevibulin monotherapy	Complete remission rate	Up to 12 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Assess additional efficacy outcome measures	• Duration of CR	Up to 2 years
Evaluate safety measures	Incidence and severity of AEs and clinical laboratory abnormalities per NCI CTCAE v5.0 in patients receiving luveltamab tazevibulin monotherapy.	Up to 2 years
To characterize the PK of luveltamab tazevibulin	Concentration of luveltamab tazevibulin (ADC, TAb, and SC209) in the blood.	Up to 2 years
Assess the immunogenic potential of luveltamab tazevibulin	Incidence of ADAs	Up to 2 years
Assess additional efficacy outcome measures	• Response rate including complete remission with partial hematologic recovery (CRh) rate [CR = CRh]	Up to 2 years
Assess additional efficacy outcome measures	• Event-free survival (EFS)	Up to 2 years
Assess additional efficacy outcome measures	• Relapse-free survival (RFS)	Up to 2 years
Assess additional efficacy outcome measures	• Overall survival (OS)	Up to 2 years

Collaborators and Investigators

• Sponsor: Sutro Biopharma, Inc.
• Collaborators: Children's Oncology Group; Innovative Therapies For Children with Cancer Consortium
• Investigators: Study Director: Craig Berman, MD, Sutro Biopharma

Study record dates

Study Major Dates

• Study Start (Actual): December 4, 2024
• Primary Completion (Actual): June 1, 2025
• Study Completion (Actual): June 1, 2025

Study Registration Dates

• First Submitted: November 2, 2024
• First Submitted That Met QC Criteria: November 6, 2024
• First Posted (Actual): November 7, 2024

Study Record Updates

• Last Update Posted (Actual): August 24, 2025
• Last Update Submitted That Met QC Criteria: August 18, 2025
• Last Verified: November 1, 2024

More Information

Terms related to this study

• Keywords: STRO-002; Pediatric AML; CBFA2T3::GLIS2 Fusion; CBFA2T3::GLIS2 AML; RAM Phenotype (CD56pos); CD45, CD38, HLA-DR weak or absent); REFRaME; AML, Child; REFRaME-P1; Luveltamab; tazevibulin
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: REFRαME P1; 2023-506240-16-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes