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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 ...

NCT02390752 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 54

Last updated 2026-04-24

No results posted yet for this study

Summary

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.

Conditions

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

Interventions

DRUG

TURALIO(R)

take oral drug daily for a 28 day cycle

Sponsors & Collaborators

  • National Cancer Institute (NCI)

    lead NIH

Principal Investigators

  • Rosandra N Kaplan, M.D. · National Cancer Institute (NCI)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-04-29
Primary Completion
2026-12-31
Completion
2028-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02390752 on ClinicalTrials.gov