Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT02390752
Phase
PHASE1
Status
Recruiting
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Conditions

  • Leukemia, Promyelocytic, Acute
  • Neurofibroma, Plexiform
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Sarcoma

Eligibility Criteria

Sex
ALL
Age
3 Years - 35 Years
Healthy Volunteers
Not accepted

Interventions

  • TURALIO(R) — DRUG
    take oral drug daily for a 28 day cycle

Study Details

Background: \- Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing. Objectives: \- To find the highest safe dose and side effects of TURALIO(R). To see if it helps treat certain types of cancer. Eligibility: \- People ages 3-35 with a solid tumor or leukemia that has returned or not responded to cancer therapies. Design: * Individuals will be screened with: * Medical history * Physical exam * Blood and urine tests * Heart tests * Scans or other tests of the tumor * Individuals will take TURALIO(R) as a capsule once daily for a 28-day cycle. They can do this for up to 2 years. * During the study, participants will have many tests and procedures. They include repeats of the screening tests. Individuals will keep a diary of symptoms. * Individuals with solid tumors will have scans or x-rays. * Individuals with leukemia will have blood tests. They may have a bone marrow sample taken. * Some individuals may have a biopsy. * When finished taking TURALIO(R), individuals will have follow-up visits. They will repeat the screening tests and note side effects.

Key Dates

Start date
Apr 29, 2015
Status verified
Jun 2026
Primary completion
Dec 31, 2026
Completion
Dec 31, 2028

Study Design

Enrollment
54 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Phase I
    take oral drug daily for a 28 day cycle

Primary Outcome Measure

Phase I: determine a phase II dose of TURALIO(R) [ Time Frame: first cycle ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
Jo Hurtt
[email protected]
240-858-7012
Rosandra N. Kaplan, M.D.
[email protected]
(240) 760-6198

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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