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SF1126 for Patients With Relapsed or Refractory Neuroblastoma
Trial Status: administratively complete
SF1126 is a novel inhibitor of PI3 kinase and mTOR that includes an active moiety
(consisting of LY294002) linked to an RGDS tetrapeptide that targets the active agent to
integrin expressing tissues. In this first pediatric phase 1 trial of SF1126, dose
escalation will follow a 3+3 dose escalation design. Once a recommended phase 2 pediatric
dose is identified, an expansion cohort of 10 patients with tumors with MYCN
amplification, Mycn expression, or Myc expression will be treated.
Funding Source - FDA OOPD
Inclusion Criteria
Patients must have a diagnosis of neuroblastoma either by histologic verification of neuroblastoma and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
Patients must have high-risk neuroblastoma according to COG risk classification at the time of study enrollment.
Patients must have at least ONE of the following: 1) Recurrent/progressive disease at any time prior to study enrollment, 2) Refractory disease, 3) Persistent disease
Patients must have at least ONE of the following: 1) Bone disease, 2) Any amount of neuroblastoma tumor cells in the bone marrow, 3) At least one soft tissue lesion that meets criteria for a TARGET lesion.
Patients must have a Lansky (< 16 years) or Karnofsky (> 16 years) score of at least 50
Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
Patients must not be receiving any other anti-cancer agents or radiotherapy at the time of study entry or while on study.
Patients must not be receiving other investigational medications (covered under another IND) within 30 days of study entry or while on study.
Patients must not be receiving chronic systemic corticosteroids at doses greater than physiologic dosing (inhaled corticosteroids acceptable).
Patient must meet the organ function requirements as stated in the protocol.
Exclusion Criteria
Pregnancy, breast feeding, or unwillingness to use effective contraception during the study.
Patients status post-allogeneic stem cell transplant are not eligible.
Patients who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
Patients with disease of any major organ system that would compromise their ability to withstand therapy.
Patients who are on hemodialysis.
Patients with an active or uncontrolled infection.
Patients with known intraparenchymal brain metastasis at study entry are excluded due to poor CNS penetration of SF1126.
Known history of human immunodeficiency virus (HIV) infection, hepatitis B, or hepatitis C.
Patient declines participation in NANT 2004-05, the NANT Biology Study.
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT02337309.
Locations matching your search criteria
United States
Colorado
Aurora
Children's Hospital Colorado
Status: Active
Name Not Available
Georgia
Atlanta
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Status: Active
Name Not Available
Inhibitors of the PI3 kinase pathway have demonstrated preclinical activity in
neuroblastoma. This activity may derive in part from destabilizing Mycn protein, impeding
tumor angiogenesis, and/or other effects. SF1126 is a novel inhibitor of PI3 kinase and
mTOR that includes an active moiety (consisting of LY294002) linked to an RGDS
tetrapeptide that targets the active agent to integrin expressing tissues. In preclinical
studies, SF1126 results in marked concentration of LY294002 into tumors. In an adult
phase 1 trial, a maximum tolerated dose of SF1126 was not identified up to doses of 1110
mg/m2 administered intravenously twice weekly on a continuous schedule. In this first
pediatric phase 1 trial of SF1126, dose escalation will follow a 3+3 dose escalation
design. Once a recommended phase 2 pediatric dose is identified, an expansion cohort of
10 patients with tumors with MYCN amplification, Mycn expression, or Myc expression will
be treated. All patients will participate in mandatory pharmacokinetic testing.
Additional optional correlative studies will evaluate potential predictive markers and
potential pharmacodynamic markers, including PTEN and PIK3CA aberrations, Myc / Mycn
expression, and Myc / pS6 levels in peripheral blood mononuclear cells.
Trial PhasePhase I
Trial Typetreatment
Lead OrganizationNew Approaches to Neuroblastoma Therapy (NANT)
