SF1126 for Patients With Relapsed or Refractory Neuroblastoma

NCT02337309 | Terminated Phase 1 DMC

• 3 other identifiers: NANT 2014-01N14-01R01FD005740
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 11

Brief Summary

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

Conditions

Neuroblastoma

Outcome Measures

Primary Outcomes (1)

• Toxicities, based on the CTCAE criteria, will be used to measure the severity of adverse events

  Toxicity will be graded using the CTCAE criteria, version 4. The CTCAE provides descriptive terminology and a grading scale for each adverse event listed. A copy of the CTCAE can be downloaded from the CTEP home page (http://ctep.cancer.gov).

  6 months

Secondary Outcomes (2)

• Evaluation of response

  After day 1 of week 4 of cycles 2, 4, and 6

• Pharmacokinetics: Parameters include AUC, clearance, Cmax, Tmax, & terminal half-life for SF1101 & SF1174. With rapid conversion of SF1126 to SF1101, only AUC, clearance, Cmax & Tmax are calculated for SF1126.

  Day 1, cycle 1

Study Arms (1)

SF1126

OTHER

Patients will receive SF1126 IV over 90 minutes on Days 1 and 4 of each week during each cycle.

Drug: SF1126

Interventions

SF1126 DRUG

SF1126 in IV form with be given to patients on this study.

SF1126

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• New Approaches to Neuroblastoma Therapy Consortium: lead
• SignalRX Pharmaceuticals, Inc.: collaborator
• University of Southern California: collaborator

Study Sites (11)

Children's Hospital Los Angeles

Los Angeles, California, 90027-0700, United States

Location

UCSF Helen Diller Family Comprehensive Cancer Center

San Francisco, California, 94143, United States

Location

Children Hospital of Colorado

Aurora, Colorado, 80045, United States

Location

AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus

Atlanta, Georgia, 30322, United States

Location

University of Chicago Comer Children's Hospital

Chicago, Illinois, 60637, United States

Location

Childrens Hospital Boston, Dana-Farber Cancer Institute.

Boston, Massachusetts, 02115, United States

Location

C.S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

Cook Children's Healthcare System

Fort Worth, Texas, 76104, United States

Location

Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, 98105, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

SF 1126

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

• Steven DuBois, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2014
• First Posted: January 13, 2015
• Study Start: July 9, 2015
• Primary Completion: May 22, 2018
• Study Completion: May 22, 2018
• Last Updated: August 20, 2018

Record last verified: 2018-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (11)