Trial of CUDC-907 in Children and Young Adults With Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma

NCT02909777 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 16-371
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 4

Brief Summary

This research study is evaluating a novel drug called CUDC-907 as a possible treatment for resistant (refractory) pediatric solid tumors (including neuroblastoma), lymphoma, or brain tumors.

Conditions

Lymphoma; Neuroblastoma; Brain Tumor; Solid Tumor

Keywords

Lymphoma; Neuroblastoma; Brain Tumor; Solid Tumor

Outcome Measures

Primary Outcomes (1)

• Dose Limiting Toxicity

  2 years

Secondary Outcomes (6)

• Progression Free Survival

  2 years

• Peak plasma concentration of CUDC-907 in Pediatrics

  2 years

• Exposure (AUC) of CUDC-907 in Pediatrics

  2 years

• Duration of Response

  2 years

• Adverse Events per CTCAE Version 4

  2 years

Study Arms (1)

CUDC-907

EXPERIMENTAL

* CUDC-907 orally administered * CUDC-907 once daily for 5 consecutive days per week followed by two days without dosing * Dose level assigned at registration * Pre-dose pharmacokinetic blood sample will be collected * Dose escalation will follow a standard 3+3 design

Drug: CUDC-907

Interventions

CUDC-907 DRUG

CUDC-907

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 \> 1 years and ≤ 21 years at time of enrollment.
• Karnofsky performance status ≥ 50% for patients ≥16 years of age and Lansky ≥ 50% for patients \<16 years of age (see Appendix A)
• Diagnosis requirement
• For Parts A and B, participants must have evaluable or measurable disease (see Section 11).
• For Part A, participants must have histologically confirmed solid tumors, CNS tumors, or lymphoma based upon biopsy or surgery at initial diagnosis and/or relapse/progression. The only exception to histologic confirmation is for pediatric tumors that are routinely diagnosed exclusively by standard clinical imaging criteria: diffuse intrinsic pontine glioma and optic pathway glioma.
• For Part B, participants must have one of the following diagnoses histologically confirmed:
• Neuroblastoma with evidence of Mycn/Myc positivity based on any of the following:
• MYCN amplification (\> 4 copy amplification) from COG reference laboratory or other CLIA-certified laboratory; or
• Mycn protein expression \> 1+ according to validated assay in Children's Hospital Los Angeles (CHLA) Clinical Pathology Laboratory; or
• Myc expression \> 1+ according to validated assay in CHLA Clinical Pathology Laboratory.
• One of the following mature B cell lymphoma diagnoses:
• Diffuse large B cell lymphoma
• Burkitt lymphoma
• Participants must have disease that is relapsed or refractory and for which standard curative or palliative measures do not exist or are no longer effective.
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy except organ function as noted in Section 3.1.6). Patients must meet the following minimum washout periods prior to enrollment:

You may not qualify if:

• Patients must not be receiving any of the following concomitant medications:
• Pharmacologic doses of systemic corticosteroids unless for CNS metastatic or primary disease. For patients with CNS metastatic or primary tumors receiving corticosteroids, they should be on a stable or decreasing dose over the 7 days prior to registration and meet criteria.
• For all patients, receipt of systemic physiologic replacement steroids, topical and/or inhaled corticosteroids is acceptable.
• Non-steroidal anti-inflammatory drugs, oral anticoagulants, and therapeutic heparins.
• Pregnant participants will not be entered on this study given that the effects of CUDC-907 on the developing human fetus are unknown.
• Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with CUDC-907, breastfeeding mothers are not eligible.
• Participants of child-bearing or child-fathering potential must agree to use adequate contraception (hormonal birth control; intrauterine device; double barrier method; or total abstinence) throughout their participation, including up until 30 days after last dose of CUDC-907.
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to CUDC-907.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. Note that patients who have had prior allogeneic transplantation are required to have CMV PCR testing performed during screening. A positive screen would be evidence of an active infection and would render the patient ineligible.
• Patients with a known history of HIV, hepatitis B, and/or hepatitis C (testing not required as part of screening).
• Patients with a known history of type 1 or type 2 diabetes mellitus.
• Patients with gastrointestinal disease or disorder that could interfere with absorption of CUDC-907, such as bowel obstruction or inflammatory bowel disease.

Sponsors & Collaborators

• Curis, Inc.: collaborator
• Dana-Farber Cancer Institute: lead

Study Sites (4)

University of California, San Francisco, Benioff Children's Hospital

San Francisco, California, 94158, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Texas Children's, Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Shulman DS, Vo KT, Balis FM, Lindsay H, Bagatell R, Place AE, Chi SN, Shusterman S, Ezrre S, Czaplinski J, Bhushan K, Kao PC, London WB, DuBois SG. A Phase 1 Trial of Fimepinostat in Children and Adolescents With Relapsed and Refractory Solid and CNS Tumors. Cancer Med. 2025 Dec;14(23):e71417. doi: 10.1002/cam4.71417.

  PMID: 41308010 DERIVED

MeSH Terms

Conditions

Lymphoma; Neuroblastoma; Brain Neoplasms

Interventions

CUDC-907

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• David S Shulman, 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: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: September 13, 2016
• First Posted: September 21, 2016
• Study Start: October 1, 2016
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: February 11, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)