Clinical trialNCT02676323TerminatedPhase 1Interventional

Panobinostat With Fludarabine and Cytarabine for Treatment of Children With Acute Myeloid Leukemia or Myelodysplastic Syndrome

SponsorSt. Jude Children's Research HospitalEnrollment19Acute Myeloid LeukemiaMyelodysplastic Syndrome

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

Cancer is the uncontrolled growth of human cells. The growth of normal human cells is controlled by multiple mechanisms. Panobinostat belongs to a class of chemotherapy drugs called "histone deacetylase (HDAC) inhibitors." HDAC inhibitors like panobinostat block enzymes known as histone deacetylases, which stops cancer cells from dividing and causes them to die. Fludarabine and cytarabine are chemotherapy drugs that are commonly used to treat pediatric patients with refractory or relapsed acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). The purpose of this study is to test the safety of panobinostat and to find the highest dose of panobinostat that can be given safely when it is combined with fludarabine and cytarabine. This pilot study will be done in two parts: The goal of Part 1 of the study is to find the highest tolerable dose of panobinostat that can be given to patients with AML or MDS, when it is combined with fludarabine and cytarabine. Once that dose is determined, participants will be enrolled on Part 2: Dose Expansion, to look at the effect of the panobinostat/fludarabine/cytarabine combination in patients with leukemia/MDS. PRIMARY OBJECTIVE: * Determine a tolerable dose of panobinostat when given in combination with fludarabine and cytarabine in pediatric patients with relapsed or refractory AML or MDS. SECONDARY OBJECTIVES: * Characterize the pharmacokinetics of panobinostat after the first dose and at steady-state. * Estimate the overall response rate to the combination of panobinostat, fludarabine, and cytarabine.

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Investigators

The overall officials registered on this study. Names that resolve to a findmyKOL profile link to it; the rest are shown as registered.

Jeffrey E. Rubnitz, MD,PhD
St. Jude Children's Research Hospital
Principal Investigator
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

7 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

Children'S Hospital Los Angeles
Los Angeles, California, United States
Lucile Packard Children'S Hospital Stanford University
Palo Alto, California, United States
Rady Children'S Hospital and Health Center
San Diego, California, United States
Children'S Hospital of Michigan
Detroit, Michigan, United States
St. Jude Children'S Research Hospital
Memphis, Tennessee, United States
Cook Children'S Medical Center
Fort Worth, Texas, United States
Seattle Children'S Hospital
Seattle, Washington, United States

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

CytarabineFludarabineIntrathecal TriplesLeucovorinPanobinostat

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 3 May 2016
Primary completion: 9 Apr 2018
Completion: 9 Apr 2018
Last update posted: 14 Nov 2018
Registered enrollment: 19
Registry id: NCT02676323

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.