PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)
|ClinicalTrials.gov Identifier: NCT02390752|
Recruitment Status : Suspended
First Posted : March 18, 2015
Last Update Posted : February 28, 2018
- Some people with cancer have solid tumors. Others have refractory leukemia. This doesn t go away after treatment. Researchers want to see if a drug called PLX3397 can shrink tumors or stop them from growing.
- To find the highest safe dose and side effects of PLX3397. To see if it helps treat certain types of cancer.
- People ages 3 22 with a solid tumor or leukemia that has returned or not responded to cancer therapies.
- For Phase II, people ages 3 31 with a Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibroma (PN) that cannot be removed with surgery.
- Participants will be screened with:
- Medical history
- Physical exam
- Blood and urine tests
- Heart tests
- Scans or other tests of the tumor
- Participants will take PLX3397 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. Participants will keep a diary of symptoms.
- Participants with solid tumors will have scans or x-rays.
- Participants with NF1 PN will have MRI scans.
- Participants with leukemia will have blood tests. They may have a bone marrow sample taken.
- Some participants may have a biopsy.
- When finished taking PLX3397, participants will have follow-up visits. They will repeat the screening tests and note side effects.
- Phase II will follow the same procedures as Phase I above, but participants will also fill out questionnaires about their pain and quality of life.
|Condition or disease||Intervention/treatment||Phase|
|Neurofibroma, Plexiform Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Prolymphocytic, Acute Sarcoma||Drug: PLX3397||Phase 1 Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Sequential Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I/II Trial of PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)|
|Study Start Date :||March 17, 2015|
|Estimated Primary Completion Date :||December 1, 2018|
|Estimated Study Completion Date :||December 1, 2018|
Take oral drug daily for 28 day cycle
Take oral drug daily for a 28 day cycle
- Phase I: determine a phase II dose of PLX3397 [ Time Frame: First cycle ]Evaluate the safety and tolerability of PLX3397
- Phase II: determine antitumor activity of PLX3397 in patients with NF1 PN [ Time Frame: At each response evaluation ]Objective response rate (ORR) will be defined as the proportion ofpatients with partial response (PR =PN volume decrease greater than or equal to 20% determined by volumetric MRI analysis).
- To characterize the pharmacokinetic profile [ Time Frame: Cycles 1 and 2 ]Preliminarily determine the antitumor activity within the confines of a phase 1 study for recurrent or refractory pediatric solid tumors and leukemia (AML and ALL)
- Correlative analysis of immune endpoints with response [ Time Frame: Before C1 and then C1D7 and then at each restaging evaluation ]Determine effect of PLX 3397 on circulating biomarkers
- Tolerability [ Time Frame: Each cycle ]Evaluate biologic activity and extended tolerability of PLX3397
- Safety [ Time Frame: Prior to cycles 3,5,9,13 and every 6 cycles ]Evaluate patient reported and functional outcomes
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02390752
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike|
|Bethesda, Maryland, United States, 20892|
|Principal Investigator:||Rosandra N Kaplan, M.D.||National Cancer Institute (NCI)|