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LymphomaPhase 1NCT02909777

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

A Phase 1 study testing CUDC-907 in people with Lymphoma and Neuroblastoma, run by Dana-Farber Cancer Institute at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 21 years
Conditions the study is for
Lymphoma, Neuroblastoma, Brain Tumor
What is being tested?
CUDC-907
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
26 participants
Where
4 sitesUnited States
Study dates
Oct 2016to Dec 2025
Phase
Phase 1
Are there results?
None posted in the registry

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 > 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:
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  • 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:
  • Myelosuppressive chemotherapy: At least 14 days after the last dose of myelosuppressive chemotherapy (42 days for nitrosourea or mitomycin C).
  • Radiotherapy:
  • At least 14 days after local palliative XRT (small port);
  • At least 90 days must have elapsed after prior TBI, craniospinal XRT or if >50% radiation of pelvis;
  • At least 42 must have elapsed if other substantial BM radiation;
  • At least 42 days must have passed since last MIBG or other radionuclide therapy.
  • Small molecule biologic therapy: At least 7 days following the last dose of a biologic agent. For agents with known adverse events occurring beyond 7 days, this duration must be extended beyond the time in which adverse events are known to occur. If extended duration is required, this should be discussed and approved by the study chair.
  • Monoclonal antibody: At least 21 days after the last dose of anitbody
  • Myeloid growth factors: At least 14 days following the last dose of long-acting growth factor (e.g. Neulasta) or 7 days following short-acting growth factor.
  • Stem Cell Infusion or Cellular Therapies: The patient must have no evidence of graft versus host disease and at least 42 days must have elapsed after transplant, stem cell infusion, or cellular therapy.
  • Major Surgery: At least 3 weeks from prior major surgical procedure. Note: Biopsy and central line placement/removal are not considered major.
  • PI3K and HDAC inhibitors: The patient must not have received prior CUDC-907 therapy. Prior treatment with individual PI3K or HDAC inhibitors is allowed. Patients must not have received therapy with the combination of PI3K and HDAC inhibitors.
  • Participants must have normal organ function as defined below.
  • Bone Marrow Function:
  • Absolute neutrophil count ≥1,000/uL
  • Platelets ≥75,000/uL and transfusion independent, defined as not receiving a platelet transfusion for at least 5 days prior to CBC documenting eligibility.
  • Hepatic Function:
  • Total bilirubin ≤ 1.5 x upper limit of normal for age
  • ALT (SGPT) ≤ 135 U/L For the purpose of this study, the ULN for ALT is 45 U/L
  • Serum albumin > 2 g/dL
  • Renal Function:
  • --A serum creatinine based on age/gender as follows: Age Maximum Serum Creatinine (mg/dL) Male Female
  • to < 2 years 0.6 0.6
  • to < 6 years 0.8 0.8
  • 6 to < 10 years 1 1 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4
  • ≥ 16 years 1.7 1.4 OR
  • --Creatinine clearance ≥ 70 mL/min/1.73 m2 for participants with creatinine levels above institutional normal.
  • Adequate Cardiac Function: QTc < 480 msec
  • Adequate GI Function: Diarrhea < grade 2 by CTCAE version 4
  • Adequate Metabolic Function: Fasting glucose < grade 2 (< 160 mg/dL or < 8.9 mmol/L) without the use of antihyperglycemic agents.
  • Additional Agent-Specific Requirements
  • Patients must be able to swallow either intact capsules or mini-tabs without chewing.
  • In order to limit dose deviations due to rounding, patients must have a body surface area of at least 0.5 m2
  • For patients with CNS tumors (primary or metastatic), any baseline neurologic deficits (including seizure) must be stable for at least one week prior to study enrollment.
  • Ability to understand and/or the willingness of the patient (or parent or legally authorized representative, if minor) to provide informed consent, using an institutionally approved informed consent procedure.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02115

  • Texas Children's, Baylor College of Medicine

    Houston, Texas, 77030

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

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

    San Francisco, California, 94158

Other trials in United States

About this study

Official title: Phase 1 Trial of CUDC-907 in Children and Young Adults With Relapsed or Refractory Solid Tumors, CNS Tumors, or Lymphoma

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.

Read the detailed description

This is a Phase I clinical trial. A Phase I clinical trial tests the safety of an investigational intervention and also tries to find the best dose of the investigational intervention to use for further studies. "Investigational" means that the intervention is being studied.

The FDA (the U.S. Food and Drug Administration) has not approved CUDC-907 as a treatment option for any disease.

This is the first time that CUDC-907 will be given to children.

In this research study, the investigators are evaluating a new drug, CUDC-907, as a potential new treatment for children with solid tumors, lymphomas and brain tumors. CUDC-907 is an oral drug that blocks certain proteins in tumor cells. These proteins may be important in the growth of some cancers. Laboratory experiments and results from adult studies demonstrate that CUDC-907 may stop tumor growth and, in some cases, cause tumor cells to die. CUDC-907 has been tested in adults with cancer to find out about side effects and dosing in adults. The primary goal of this study is to evaluate side effects of CUDC-907 and find the best dose of CUDC-907 when used in children. Other goals of this study are to determine whether this drug may have benefits against the types of cancer seen in children and to measure the effects of CUDC-907 in the blood.

Being tested
CUDC-907
Keywords
Lymphoma, Neuroblastoma, Brain Tumor, Solid Tumor
Medical subject headings
Lymphoma, Neuroblastoma, Brain Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CUDC-907Drug

Study groups in NCT02909777
Study groupRoleWhat they receive
CUDC-907Experimental* 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
Receives: Drug: CUDC-907

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Dose Limiting Toxicity

    Measured: 2 years

Secondary outcomes (6)

  1. Progression Free Survival

    Measured: 2 years

  2. Peak plasma concentration of CUDC-907 in Pediatrics

    Determined using standard methods and reported descriptively in aggregate and by assigned dose level.

    Measured: 2 years

  3. Exposure (AUC) of CUDC-907 in Pediatrics

    Determined using standard methods and reported descriptively in aggregate and by assigned dose level.

    Measured: 2 years

  4. Duration of Response

    Measured: 2 years

  5. Adverse Events per CTCAE Version 4

    Measured: 2 years

  6. Objective Response

    Measured: 2 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Sep 2016 (estimated)
  2. Study startOct 2016
  3. Primary completionDec 2025
  4. Study completionDec 2025

Resources

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02909777
Sponsor's protocol ID
16-371
Status last verified
Feb 2026
First submitted
13 Sep 2016
Last updated
11 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
Individual data sharing
No

Publications

  • 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.

    PubMed 41308010

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CUDC-907 in people with Lymphoma and Neuroblastoma, run by Dana-Farber Cancer Institute at 4 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 50 inclusion and 12 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: finished, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Dose Limiting Toxicity (time frame: 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02909777, which also lists 1 linked publication.

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 11 Feb 2026.

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