Tipifarnib and Naxitamab for Relapsed/Refractory Neuroblastoma
ConditionNeuroblastoma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-21
SponsorGiselle Sholler
About this trial
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
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M with MYCN amplification
Disqualifiers
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.
Trial design
Design model
Parallel
Treatments tested in this trial
Tipifarnib
DrugTablet
Naxitamab
DrugIV
Treatment groups
98 Participants
are divided into 2 treatment groupsGroup A: HRNB Bone/Bone MarrowExperimental treatment 2 interventions
Group B: HRNB All othersExperimental treatment 2 interventions
Trial outcomes
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)
Time frame
6 monthsSecondary outcomes
1
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)
Time frame
6 months plus 5 years follow up2
Length of time that participants experience Overall Survival (OS)
To evaluate the efficacy of Tipifarnib in combination with Naxitamab based upon Overall Survival (OS)
Time frame
6 months plus 5 years follow up3
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.
Time frame
6 months plus 30 daysSponsors and contacts
Click on the lead sponsor to view all of their trials.
Giselle Sholler
Lead sponsor
Milton S. Hershey Medical Center
Sponsor institution
Source ClinicalTrials.gov