Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma

NCT06540963 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: BCC022
• Study Type: interventional
• Target: 98
• Locations: 1 country
• Sites: 12

Brief Summary

The purpose of this study is to evaluate the investigational drug, tipifarnib (a pill taken by mouth), in combination with the Food and Drug Administration (FDA) approved drug, naxitimab, administered intravenously (IV; a liquid that continuously goes into your body through a tube that has been placed during a surgery into one of your veins). Naxitamab is FDA approved for pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy, it may not be approved in the type of disease used in this study. The goals of this part of the study are:

• Test the safety and tolerability of tipifarnib in combination with naxitimab in patients with cancer
• To determine the activity of study treatments chosen based on:
• How each subject responds to the study treatment
• How long a subject lives without their disease returning/progressing

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• Determine the Overall Response Rate (ORR) of Participants using INSS Response

  To evaluate the activity of Tipifarnib in combination with Naxitamab based on Overall response rate (ORR)

  6 months

Secondary Outcomes (3)

• Number of participants with progression free survival (PFS) during study

  6 months plus 5 years follow up

• Length of time that participants experience Overall Survival (OS)

  6 months plus 5 years follow up

• Number of Participants with Adverse Events as a Measure of Safety and Tolerability

  6 months plus 30 days

Study Arms (2)

HRNB Bone/Bone Marrow

EXPERIMENTAL

Cycles 1-6: Tipifarnib and Naxitamab Tipifarnib: on days 1-7 and 15-21 of each 28-day cycle. Naxitamab IV on Days 1, 3, and 5 of each cycle.

Drug: Tipifarnib; Drug: Naxitamab

HRNB All others

EXPERIMENTAL

Cycles 1-6: Tipifarnib and Naxitamab Tipifarnib: on days 1-7 and 15-21 of each 28-day cycle. Naxitamab IV on Days 1, 3, and 5 of each cycle.

Drug: Tipifarnib; Drug: Naxitamab

Interventions

Tipifarnib DRUG

Tablet

Also known as: R115777

HRNB All others; HRNB Bone/Bone Marrow

Naxitamab DRUG

IV

Also known as: Danyelza

HRNB All others; HRNB Bone/Bone Marrow

Eligibility Criteria

• Age: 1 Year - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age:
• Subjects must be age ≤ 21 years at initial diagnosis. Subjects must be \>12 months of age at enrollment. Safety Run-In (first 6 subjects) must be age 6 years or older.
• Pathology: All subjects must have a pathologically confirmed diagnosis of neuroblastoma at any point in their treatment.
• Tumor assessment: Disease staging must be performed. This disease assessment is required for eligibility and must be done within a maximum of 4 weeks before first dose of study drug.
• Disease Status: Relapsed/Refractory Neuroblastoma Relapsed disease defined as neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment/neuroblastoma protocol) and has now relapsed and is in any number of relapses.
• Refractory disease defined as High-risk neuroblastoma as defined by the International Neuroblastoma Risk Group Staging System (INRG) that failed to achieve complete response (CR) after at least 4 cycles of aggressive multi-drug induction chemotherapy, progression during upfront therapy, or with disease remaining after standard immunotherapy.
• INRG High Risk NB defined as one of the following:
• Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification
• Age ≥ 547 days and INRG Stage M regardless of biologic features
• Any age initially diagnosed with INRG Stage L1 MYCN amplified neuroblastoma (NBL) who have progressed to Stage M without systemic chemotherapy
• Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to Stage M without systemic chemotherapy
• Measurable Disease: Subjects must be relapsed or refractory with active disease. Subjects must have measurable or evaluable disease, including at least one of the following: Measurable tumor \>10mm by computed tomography scan (CT) or magnetic resonance imaging (MRI); a positive metaiodobenzylguanidine (MIBG) scan or positron emission tomography (PET) scan or Positive bone marrow biopsy/aspirate.
• Subjects with central nervous system (CNS) disease currently taking steroids must have been on a stable dose of steroids for at least one week prior to their biopsy and must not have progressive hydrocephalus at enrollment.
• Timing from prior therapy:
• Subjects must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:

You may not qualify if:

• Subjects who are less than 1 year of age
• BSA of \<0.25 m2
• Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
• Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
• Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
• Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
• Previous Gr.4 allergic or anaphylactic reaction to naxitamab, leading to the discontinuation of naxitamab during prior therapy.

Sponsors & Collaborators

• Giselle Sholler: lead

Study Sites (12)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

RECRUITING

Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

RECRUITING

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96813, United States

RECRUITING

Cardinal Glennon Children's Medical Center

St Louis, Missouri, 63104, United States

RECRUITING

Duke University

Durham, North Carolina, 27708, United States

RECRUITING

Randall Children's Hospital

Portland, Oregon, 97227, United States

RECRUITING

Penn State Milton S. Hershey Medical Center and Children's Hospital

Hershey, Pennsylvania, 17033, United States

RECRUITING

Monroe Carrell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

RECRUITING

Dell Children's Blood and Cancer Center

Austin, Texas, 78723, United States

RECRUITING

Children's Medical Center

Dallas, Texas, 75235, United States

RECRUITING

Related Links

• Beat Childhood Cancer Research Consortium website

MeSH Terms

Conditions

Neuroblastoma

Interventions

tipifarnib; naxitamab

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 Officials

• Valerie Brown, MD, PhD

  Beat Childhood Cancer

  STUDY CHAIR

Central Study Contacts

BCC Enroll

CONTACT

7175310003; BCCEnroll@pennstatehealth.psu.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Beat Childhood Cancer Chair

Study Record Dates

• First Submitted: August 2, 2024
• First Posted: August 7, 2024
• Study Start: December 6, 2024
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2035
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (12)