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New treatment option for Neurofibroma, Plexiform
Official title 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 ...
ClinicalTrials.gov ID: NCT02390752
What this study is testing
What is TURALIO(R)?
TURALIO(R) is an investigational medicine, given as an once-weekly pill taken by mouth, being studied as a potential treatment for neurofibroma, plexiform.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: \- Some people with cancer have solid tumors. Others have refractory leukemia.
- Phase 1: an early, usually small safety study
- Time commitment: about 2 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 35
You may be able to join if
- Diagnosis:
- Individuals must have recurrent or refractory solid tumors or acute leukemia (limited to AML or ALL) or have been intolerant of prior therapies...
- In addition, individuals with NF1 and with malignant peripheral nerve sheath tumor (MPNST).
- Individuals must have relapsed after or be refractory to effective standard therapies. There are no limits on number of prior therapeutic regimens.
- Disease status: Individuals with refractory solid tumors including patients with NF1 and MPNST must have evaluable disease, patients with leukemia...
You likely can't join if
- Individuals who are pregnant or breast feeding or who become pregnant while enrolled on this trial will be excluded from participation, due to the...
- Ongoing treatment with any other cancer therapy or investigational agent, with the exception of IT chemotherapy for leukemia, when indicated.
- Individuals who require therapy with warfarin.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina...
- Active untreated infection.
- Known active hepatitis A, B, C or HIV infection, chronic Hepatitis B or C, or HIV infection or inactive Hepatitis B carrier.
See the full eligibility criteria
Who can join
- Diagnosis:
- Individuals must have recurrent or refractory solid tumors or acute leukemia (limited to AML or ALL) or have been intolerant of prior therapies, confirmed by the Laboratory of Pathology, NCI, e.g., solid tumors...
- In addition, individuals with NF1 and with malignant peripheral nerve sheath tumor (MPNST).
- Individuals must have relapsed after or be refractory to effective standard therapies. There are no limits on number of prior therapeutic regimens.
- Disease status: Individuals with refractory solid tumors including patients with NF1 and MPNST must have evaluable disease, patients with leukemia must have measurable or evaluable disease at the time of enrollment...
- Age \>= 3 and \ = 0.55 m\^2):
- Ability of subject or Legally Authorized Representative [LAR] (the parent/guardian if subject is a minor) to understand and the willingness to sign a written informed consent document.
- Individuals must be able to swallow capsules.
- Performance Status: Karnofsky \>= 50% for patients \> 16 years of age and Lansky \>= 50% for patients \<= 16 years of age. Individuals who are wheelchair bound because of paralysis will be considered "ambulatory" when...
- Prior therapy: Individuals must have fully recovered (to Grade 1) from the acute toxic effects of all prior anti-cancer therapy.
- Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).
- Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the...
- Immunotherapy: At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines.
- Monoclonal antibodies: At least 3 half-lives of the antibody after the last dose of a monoclonal antibody.
- XRT: At least 7 days after local palliative XRT (small port); At least 150 days must have elapsed if prior TBI or if \>= 50% radiation of pelvis; \>= 14 days from whole brain radiation, craniospinal radiation, or...
- HSCT: \>= 56 days from stem cell transplant with no evidence of active graft vs. host disease; must be off immunosuppressive therapy for at least 4 weeks and have no active graft-versus-host disease (GVHD) at the time...
- Surgery: \>= 14 days from surgery
- Others: \>= 7 days from last dose of short active hematopoietic growth factors, i.e. filgrastim, \>= 14 days for long-acting, i.e. pegfilgrastim.
- Steroids: Individuals with CNS tumors who are managed with steroids are eligible if they have no worsening neurologic deficits and are on a stable or decreasing dose of corticosteroids for greater than or equal to 7...
- Absolute neutrophil count \>= 1.5 x 10\^9/L
- Hemoglobin \> 10 g/dL
- Platelet count \>= 100 x 10\^9/L
- AST and ALT \<= upper limit of normal (ULN)
- TBil and DBil \<= ULN with an exception of patients with confirmed Gilbert's syndrome. For patients with confirmed Gilberts syndrome, the TBil should be \<= 1.5 x ULN
- Serum creatinine \<= 1.5 x ULN
- Exceptions:
- Cytopenias due to underlying disease (i.e. potentially reversible with anti-neoplastic therapy); A subject will not be excluded because of cytopenia due to disease, based on the results of bone marrow studies.
- Known active or chronic human immunodeficiency virus (HIV) or hepatitis C virus (HCV) infection, or positive hepatitis B (Hep B) surface antigen. Prior hepatitis infection that has been treated with highly effective...
- Hepatobiliary diseases including biliary tract diseases, autoimmune hepatitis, inflammation, fibrosis, cirrhosis of liver caused by viral, alcohol, or genetic reasons. Gilbert's disease is allowed if TBil is \<= 1.5 x...
- Cardiac ejection fraction \>= 50%, and QTcF \< 450 ms (male) or \<470 ms (female) on ECG at Baseline. (Fridericia's Formula: QTcF = (QT)/RR0.33)
- Contraception: Women of child-bearing potential must agree to use an effective method of birth control during treatment and for 1 month after receiving their last dose of study drug. Fertile men must also agree to use...
What rules you out
- Individuals who are pregnant or breast feeding or who become pregnant while enrolled on this trial will be excluded from participation, due to the unknown effects of TURALIO(R) on a growing fetus or newborn child.
- Ongoing treatment with any other cancer therapy or investigational agent, with the exception of IT chemotherapy for leukemia, when indicated.
- Individuals who require therapy with warfarin.
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations...
- Active untreated infection.
- Known active hepatitis A, B, C or HIV infection, chronic Hepatitis B or C, or HIV infection or inactive Hepatitis B carrier.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to TURALIO(R) or other agents used in study.
- Individuals with PT and/or INR higher than or equal to 1.5 times upper limit of normal, unless patients have lupus anticoagulant in which case they are eligible if cleared by hematology.
- Drugs that strongly inhibit or potentiate CYP3A4, which includes CYP3A4 inducer, UGT inhibitors and acid reducing agents and avoid concomitant use of PPIs:
- Individuals who have received these drugs within 14 days or within 5 half-lives of the drug (whichever is longer) prior to study initiation will be excluded.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.