PLX3397 in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02390752|
Recruitment Status : Suspended
First Posted : March 18, 2015
Last Update Posted : July 16, 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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)|