NeuroblastomaPhase 2NCT06540963
Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
A Phase 2 study testing Tipifarnib and Naxitamab in people with Neuroblastoma, run by Giselle Sholler at 12 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- Tipifarnib and Naxitamab
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 98 participants
- Where
- 12 sitesUnited States
- Study dates
- 6 Dec 2024to 1 Dec 2035
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age:
- Participants must be age ≤ 21 years at initial diagnosis. Participants must be >12 months of age at enrollment. Safety Run-In (first 6 participants) must be age 6 years or older. As of 09Jun2026 the safety run-in is complete.
- Pathology: All participants 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.
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- 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: Participants must be relapsed or refractory with active disease. Participants 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.
- Cohort 1- High-risk neuroblastoma patients with disease limited to bone and/or bone marrow at enrollment. Participant must have stable disease, minor response, or partial response to their most recent therapy.
- Cohort 2- All other high-risk relapsed or refractory neuroblastoma patients not eligible for Cohort 1, including participants with soft tissue disease.
- Participants 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:
- Participants must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:
- Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).
- Hematopoietic growth factors: At least 5 days since the completion of therapy with a growth factor.
- Small Molecule Inhibitors (anti-neoplastic agent): At least 7 days since the completion of therapy with a small molecule inhibitor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the Study Chair.
- Immunotherapy: At least 4 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells, anti-GD2 Monoclonal antibodies (ex. naxitamab, dinutuximab, etc.).
- XRT (Radiotherapy): At least 30 days since the last treatment except for radiation delivered with palliative intent to a non-target site.
- Stem Cell Transplant:
- Allogeneic: No evidence of active graft vs. host disease
- Allo/Auto: ≥ 2 months must have elapsed since transplant.
- MIBG Therapy: At least 6 weeks since treatment with MIBG therapy.
- Participants must have a Lansky or Karnofsky Performance Scale score of ≥ 50
- Participants must have adequate organ function at the time of enrollment:
- Hematological: Hematological recovery as defined by absolute neutrophil count (ANC) ≥750/μL, platelets ≥30/μL (may be transfused).
- Liver: Normal liver function as defined by Aspartate transferase (AST), Alanine transaminase (ALT), and total bilirubin (TBL) all within upper limit of normal
- Renal:
- For participants < 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: [(0.413) X (Height in cm)] / SCr
- For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: [(140-age) x (Wt in kg) x (0.85 if female)] / (72 x SCr)
- Cardiac: Participants must have a QTcF ≤ 470 msc.
- Participants of childbearing potential must have a negative pregnancy test. Participants of childbearing potential must agree to use an effective birth control method.
- Participants who are lactating must agree to stop breast-feeding. (NOTE: breast milk cannot be stored for future use while the mother is being treated on study.)
- Written informed consent in accordance with institutional and FDA guidelines must be obtained from all participants (or participants' legal representative).
Who cannot join
- Participants who are less than 1 year of age
- BSA of <0.25 m2
- Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
- Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
- Participants 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.
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
United States12 sites, 12 recruiting
- Recruiting
Dell Children's Blood and Cancer Center
Austin, Texas, 78723
Rhea Robinson, mrobinson@ascension.org
Virginia Harrod
- Recruiting
Children's Medical Center
Dallas, Texas, 75235
Rachel Nam, rachel.nam@childrens.com
Tanya Watt
- Recruiting
Duke University
Durham, North Carolina, 27708
Morgan Low, morgan.low@duke.edu
Jessica Sun
- Recruiting
Connecticut Children's Hospital
Hartford, Connecticut, 06106
Adam Barselau, Abarselau@connecticutchildrens.org
Michael Isakoff
- Recruiting
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033
Penn State Clinical Trials Group Email, ExtractClinicalTrials@pennstatehealth.psu.edu
Valerie Brown
- Recruiting
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96813
Andrea Siu, andrea.siu@kapiolani.org
Kelly Hutchins
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Susan Hall, hallsf@archildrens.org
Michael Bishop
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
Aixa Guadarrama, Aixa.Guadarrama@Nicklaushealth.org
Guillermo De Angulo
- Recruiting
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
Aida Constantinescu, aida.constantinescu@vumc.org
Daniel Benedetti
- Recruiting
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Maria Frankos, marie.frankos@orlandohealth.com
Jamie Libes-Bander
- Recruiting
Randall Children's Hospital
Portland, Oregon, 97227
Aaron White, AJWHITE@lhs.org
Jason Glover
- Recruiting
Cardinal Glennon Children's Medical Center
St Louis, Missouri, 63104
Gina Martin, gina.martin@health.slu.edu
William Ferguson
About this study
Official title: Phase II Trial of Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
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 participants with cancer
- To determine the activity of study treatments chosen based on:
- How each participant responds to the study treatment
- How long a participant lives without their disease returning/progressing
- Conditions
- Neuroblastoma
- Being tested
- Tipifarnib, Naxitamab
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TipifarnibDrug
Also called R115777
Tablet
NaxitamabDrug
Also called Danyelza
IV
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Tipifarnib; Drug: Naxitamab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 6 months
Secondary outcomes (3)
Number of participants with progression free survival (PFS) during study
To evaluate the activity of Tipifarnib in combination with Naxitamab based on Progression free survival (PFS)
Measured: 6 months plus 5 years follow up
Length of time that participants experience Overall Survival (OS)
To evaluate the efficacy of Tipifarnib in combination with Naxitamab based upon Overall Survival (OS)
Measured: 6 months plus 5 years follow up
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
To evaluate the safety and tolerability profile of Tipifarnib in combination with Naxitamab in pediatric and young adult participants.
Measured: 6 months plus 30 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2024
- Study start6 Dec 2024
- Primary completion1 Dec 2030 (estimated)
- Study completion1 Dec 2035 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 98 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Giselle Sholler
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Giselle Sholler, Milton S. Hershey Medical Center
- Investigators
- Valerie Brown, MD, PhD, Study chair, Beat Childhood Cancer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06540963
- Sponsor's protocol ID
- BCC022
- Status last verified
- Sep 2026
- First submitted
- 2 Aug 2024
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Trial of Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT06540963
- Acronym / sponsor ID
- BCC022
- Conditions
- Neuroblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 98 participants (estimated)
- Lead sponsor
- Giselle ShollerUniversity, hospital or other
- Study start
- 6 Dec 2024 (actual)
- Primary completion
- 1 Dec 2030 (estimated)
- Study completion
- 1 Dec 2035 (estimated)
- First posted
- 7 Aug 2024 (actual)
- 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.
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 participants with cancer
- To determine the activity of study treatments chosen based on:
- How each participant responds to the study treatment
- How long a participant lives without their disease returning/progressing
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HRNB Bone/Bone Marrow | Experimental | Drug: Tipifarnib, Drug: NaxitamabCycles 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. |
| HRNB All others | Experimental | Drug: Tipifarnib, Drug: NaxitamabCycles 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. |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (37 listed)
- Age:
- Participants must be age ≤ 21 years at initial diagnosis. Participants must be >12 months of age at enrollment. Safety Run-In (first 6 participants) must be age 6 years or older. As of 09Jun2026 the safety run-in is complete.
- Pathology: All participants 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.
Key exclusion criteria (7 listed)
- Participants who are less than 1 year of age
- BSA of <0.25 m2
- Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
- Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
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 |
|---|---|---|
| TipifarnibAlso: R115777Tablet | Drug | HRNB All others, HRNB Bone/Bone Marrow |
| NaxitamabAlso: DanyelzaIV | Drug | HRNB All others, HRNB Bone/Bone Marrow |
Primary outcome
Determine the Overall Response Rate (ORR) of Participants using INSS Response
Time frame: 6 months
To evaluate the activity of Tipifarnib in combination with Naxitamab based on Overall response rate (ORR)
3 secondary outcomes
Number of participants with progression free survival (PFS) during study
Time frame: 6 months plus 5 years follow up
Length of time that participants experience Overall Survival (OS)
Time frame: 6 months plus 5 years follow up
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Time frame: 6 months plus 30 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Beat Childhood Cancer Research Consortium websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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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.
Valerie Brown, MD, PhD study chair
Beat Childhood Cancer
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Tipifarnib and Naxitamab in people with Neuroblastoma, run by Giselle Sholler at 12 sites 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 37 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact BCC Enroll (7175310003, BCCEnroll@pennstatehealth.psu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the Overall Response Rate (ORR) of Participants using INSS Response (time frame: 6 months). 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 3 Sep 2026; AzTrials last refreshed its copy on 6 Nov 2026.