Treatment Trials

11 Clinical Trials for Various Conditions

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ACTIVE_NOT_RECRUITING
Testing GDC-0449 (Vismodegib) as Potentially Targeted Treatment in Cancers With Smoothened or Patched 1 Mutant Tumors (MATCH - Subprotocol T)
Description

This phase II MATCH treatment trial tests how well GDC-0449 (vismodegib) works for treating patients with solid tumors, lymphoma, or multiple myeloma that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) or that does not respond to treatment (refractory) and who have a smoothened or patched 1 genetic mutation. Vismodegib is a type of medication called a hedgehog signaling pathway antagonist and works by blocks a type of protein involved in tissue growth and repair and may block the growth of cancer cells.

COMPLETED
Pilot LDE225 in Locally Advanced or Metastatic BCC + Previously Tx Non-LDE225 Smoothened Inhibitors
Description

This is a prospective single-center, open label, pilot study to investigate the safety and efficacy of LDE225 in patients with locally advanced or metastatic basal cell carcinoma. Primary Objectives: • To explore the effects of oral LDE225 on the Progression Free Survival (PFS) of individuals with locally advanced or metastatic BCC who have been previously treated with a non-LDE225 Smo inhibitor. Secondary Objectives: * To evaluate the effect of oral LDE225 on tumor tissue biomarkers of BCC activation (Gii 1, 2, Patched 1,2 and Ki67) in individuals which are non-na"ive to Smo inhibitors other than LDE225, at baseline and at end-of-treatment * To describe adverse effects of oral LDE225 in individuals with a history of non-LDE225 Smo inhibitor usage * To assess the overall survival rates of individuals with locally advanced BCC or metastatic BCC who have previously taken a non-LDE225 Smo inhibitor after treatment with LDE225

TERMINATED
Phase 2 Dasatinib Combo With Smoothened (SMO) Antagonist (BMS-833923)
Description

The purpose of the study is to compare response rates in newly diagnosed Chronic Phase (CP) CML subjects treated with dasatinib plus BMS-833923 versus dasatinib alone.

Conditions
COMPLETED
Dasatinib Combination Therapy With the Smoothened (SMO) Inhibitor BMS-833923 in Chronic Myeloid Leukemia (CML)
Description

The purpose of the study is to determine the safety and tolerability of the combination of BMS-833923 plus dasatinib in patients with chronic myeloid leukemia.

Conditions
COMPLETED
A Phase 1 Study of BMS-833923 (XL139) in Subjects With Advanced or Metastatic Cancer
Description

The purpose of this study is to determine the safety of BMS-833923 (XL139) in patients with advanced or metastatic cancers and determine the recommended phase 2 dose range and schedule

ACTIVE_NOT_RECRUITING
A Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients With Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations
Description

This study employs a 2-stage design that aims to evaluate the efficacy and safety of ENV- 101, a potent Hedgehog (Hh) pathway inhibitor, in patients with refractory advanced solid tumors characterized by loss of function (LOF) mutations in the Patched-1 (PTCH1) gene. Stage 1 of this study will enroll approximately 44 patients randomized between two dose levels. As appropriate, Stage 2 of the study will expand enrollment based on the results of Stage 1.

COMPLETED
SJDAWN: St. Jude Children's Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults With Recurrent Brain Tumors
Description

Approximately 90% of children with malignant brain tumors that have recurred or relapsed after receiving conventional therapy will die of disease. Despite this terrible and frustrating outcome, continued treatment of this population remains fundamental to improving cure rates. Studying this relapsed population will help unearth clues to why conventional therapy fails and how cancers continue to resist modern advances. Moreover, improvements in the treatment of this relapsed population will lead to improvements in upfront therapy and reduce the chance of relapse for all. Novel therapy and, more importantly, novel approaches are sorely needed. This trial proposes a new approach that evaluates rational combination therapies of novel agents based on tumor type and molecular characteristics of these diseases. The investigators hypothesize that the use of two predictably active drugs (a doublet) will increase the chance of clinical efficacy. The purpose of this trial is to perform a limited dose escalation study of multiple doublets to evaluate the safety and tolerability of these combinations followed by a small expansion cohort to detect preliminary efficacy. In addition, a more extensive and robust molecular analysis of all the participant samples will be performed as part of the trial such that we can refine the molecular classification and better inform on potential response to therapy. In this manner the tolerability of combinations can be evaluated on a small but relevant population and the chance of detecting antitumor activity is potentially increased. Furthermore, the goal of the complementary molecular characterization will be to eventually match the therapy with better predictive biomarkers. PRIMARY OBJECTIVES: * To determine the safety and tolerability and estimate the maximum tolerated dose/recommended phase 2 dose (MTD/RP2D) of combination treatment by stratum. * To characterize the pharmacokinetics of combination treatment by stratum. SECONDARY OBJECTIVE: * To estimate the rate and duration of objective response and progression free survival (PFS) by stratum.

Conditions
Anaplastic AstrocytomaAnaplastic EpendymomaAnaplastic GangliogliomaAnaplastic MeningiomaAnaplastic OligodendrogliomaPleomorphic Xanthoastrocytoma, AnaplasticAtypical Teratoid/Rhabdoid TumorBrain CancerBrain TumorCentral Nervous System NeoplasmsChoroid Plexus CarcinomaCNS Embryonal Tumor With Rhabdoid FeaturesGanglioneuroblastoma of Central Nervous SystemCNS TumorEmbryonal Tumor of CNSEpendymomaGlioblastomaGliomaGlioma, MalignantMedulloblastomaMedulloblastoma; Unspecified SiteMedulloepitheliomaNeuroepithelial TumorNeoplasmsNeoplasms, NeuroepithelialPapillary Tumor of the Pineal Region (High-grade Only)Pediatric Brain TumorPineal Parenchymal Tumor of Intermediate Differentiation (High-grade Only)PineoblastomaPrimitive Neuroectodermal TumorRecurrent MedulloblastomaRefractory Brain TumorNeuroblastoma. CNSGlioblastoma, IDH-mutantGlioblastoma, IDH-wildtypeMedulloblastoma, Group 3Medulloblastoma, Group 4Glioma, High GradeNeuroepithelial Tumor, High GradeMedulloblastoma, SHH-activated and TP53 MutantMedulloblastoma, SHH-activated and TP53 WildtypeMedulloblastoma, Chromosome 9q LossMedulloblastoma, Non-WNT Non-SHH, NOSMedulloblastoma, Non-WNT/Non-SHHMedulloblastoma, PTCH1 MutationMedulloblastoma, WNT-activatedEpendymoma, RecurrentGlioma, Recurrent High GradeGlioma, Recurrent MalignantEmbryonal Tumor, NOSGlioma, Diffuse Midline, H3K27M-mutantEmbryonal Tumor With Multilayered Rosettes (ETMR)Ependymoma, NOS, WHO Grade IIIEpendymoma, NOS, WHO Grade IIMedulloblastoma, G3/G4Ependymoma, RELA Fusion Positive
TERMINATED
Safety/Efficacy Study of LDE225 (Sonidegib) Plus Bortezomib in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma
Description

The purpose of the study is to determine whether the combination of LDE225 (sonidegib) plus bortezomib is safe and effective in the treatment of relapsed or relapsed/refractory multiple myeloma.

TERMINATED
Single-Agent Glasdegib In Patients With Myelofibrosis Previously Treated With Ruxolitinib
Description

A lead-in cohort of \~20 patients with primary or secondary myelofibrosis previously treated with 1 or more Janus kinase inhibitors enrolled to single-agent glasdegib to evaluate safety and tolerability. Following the lead-in, a phase 2, double blind, 2-arm study, randomized 2:1 to oral single-agent glasdegib versus placebo in 201 patients resistant or intolerant to ruxolitinib.

COMPLETED
Phase Ib, Dose Escalation Study of Oral LDE225 in Combination With BKM120 in Patients With Advanced Solid Tumors
Description

The purpose of this study is to determine the maximum dose of LDE225 and BKM120 that can be safely given together to patients and/or the dose that will be used in future studies. This study will also learn more about how the combination of these two investigational drugs may work for patients with certain cancers (specifically metastatic breast cancer, advanced pancreatic adenocarcinoma, metastatic colorectal cancer and recurrent glioblastoma multiforme).

COMPLETED
Dose Finding and Safety of Oral LDE225 in Patients With Advanced Solid Tumors
Description

This first-in-human dose-escalation study is to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in patients with advanced solid tumors.