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Clinical Trials

As part of our mission to eliminate cancer, MD Anderson researchers conduct hundreds of clinical trials. Below are some supported by the Cancer Neuroscience Program, sorted by cancer type.

Brain Tumors

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2015-0775 NCT02754544	Using ECoGgrid mapping in predicting eloquent cortex and post-op functional outcomes in patients with brain tumors			Active
2022-0736	Intraoperative analysis of human brain cancer tissue during surgery utilizing a handheld mass spectrometry system	lab/tissue trial	n/a	Active

Glioblastoma

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2019-1035 NCT04555577	Phase I trial of DNA-PK inhibitor (M3814) in combination with radiation and adjuvant temozolomide in newly diagnosed MGMT unmethylated glioblastoma	Phase I	M3814 (peposertib), temozolomide	Active
2019-0216 NCT03970447	GBM AGILE Global Adaptive Trial Master Protocol: An International, Seamless Phase II/III Response Adaptive Randomization Platform Trial Designed To Evaluate Multiple Regimens In Newly Diagnosed and Recurrent glioblastoma (GBM)	Phase II/III	TEMOZOLOMIDE, Bi-functional Alkylating Agent VAL-083, Troriluzole, VT1021 and LOMUSTINE	Active
2022-0717 NCT05109728	A phase 1b dose finding study assessing safety and activity of 177LuLu-DOTA-TATE in newly diagnosed glioblastoma in combination with radiotherapy with or without Temozolomide and in recurrent glioblastoma as single agent	Phase I	AAA601 (177Lu-dotatate)	Active
2022-0990 NCT05879367	An open-label, Phase 1b study to evaluate the safety and tolerability of eflornithine plus temozolomide in patients with newly diagnosed glioblastoma	Phase I	Eflornithine, TEMOZOLOMIDE	Active
2019-0216 NCT03970447	GBM AGILE Global Adaptive Trial Master Protocol: An International, Seamless Phase II/III Response Adaptive Randomization Platform Trial Designed To Evaluate Multiple Regimens In Newly Diagnosed and Recurrent Glioblastoma (GBM)	Phase II/III	Bi-functional Alkylating Agent VAL-083, LOMUSTINE, TEMOZOLOMIDE, Troriluzole, VT1021 and LOMUSTINE	Active
2020-0758 NCT05053880	A Phase 1b/2a Study of ACT001 and anti-PD-1 in Patients with Surgically Accessible Recurrent Glioblastoma Multiforme	Phase I/II	ACT001 and Pembrolizumab	Active
2021-0041 NCT05039281	Phase I/II Study to Evaluate the Safety and Clinical Efficacy of Atezolizumab (anti-PD-L1) in Combination with Cabozantinib in Patients with Recurrent Glioblastoma (rGBM)	Phase I/II	Atezolizumab, Cabozantinib	Active
2022-0931 NCT05765812	A Phase 1/2 open label study of Debio 0123 in combination with temozolomide in adult participants with recurrent or progressive glioblastoma and of Debio 0123 in combination with temozolomide and radiotherapy in adult participants with newly-diagnosed glioblastoma	Phase I/II	Debio 0123, Temozolomide
2020-0960 NCT04991870	A Phase I clinical trial with a window-of-opportunity component of engineered NK cells containing deleted TGF-ßR2 and NR3C1 in recurrent glioblastoma	Phase I	CB-NK-TGF-Î²R2-/NR3C1	Active
2022-0717 NCT05109728	A phase 1b dose finding study assessing safety and activity of 177LuLu-DOTA-TATE in newly diagnosed glioblastoma in combination with radiotherapy with or without temozolomide and in recurrent glioblastoma as single agent	Phase I	AAA601 (177Lu-dotatate)	Active
2024-0330 NCT04910022	A phase I/II combination study of NMS-03305293 and temozolomide in adult patients with recurrent glioblastoma	Phase I/II		Active
2022-0901 NCT05383872	Safety and effectiveness of Exablate Model 4000 using microbubble resonators to temporarily mediate blood-brain barrier disruption (BBBD) for liquid biopsy in subjects with glioblastoma brain tumors		n/a	Active+B40C36A36:F47

Glioma

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2023-0004 NCT05580562	ONC201 for the treatment of newly diagnosed H3 K27M-mutant diffuse glioma following completion of radiotherapy: A randomized, double-blind, placebo-controlled, multicenter study	Phase III		Active
2020-1154 NCT04617002	Intermediate-size Expanded Access to ONC201 for Patients with H3 K27M-mutant and/or Midline Gliomas	Phase II	ONC201	Active
2022-0650 NCT05484622	A Phase 1, safety lead-in and randomized, open-label, Perioperative Study of Vorasidenib in combination with Pembrolizumab in subjects with recurrent or progressive enhancing IDH-1 mutant Astrocytomas	Phase I	AG-881, Pembrolizumab	Active
2012-0441	Prospective Assessment of Correlative and Tissue Biomarkers in Glioma Patients (The PROACTIVE-Glioma Program) - A Biomarker-Driven Program for Clinical Trial Development for Newly Diagnosed and Recurrent Glioma Patients			Active
PA18-1154	Longitudinal CSF and Blood Monitoring of Genomic Alterations in Patients with Glioma: A Companion Study to the PROACTIVE Program (2012-0441)			Active
2018-0097 NCT04479696	Novel Intervention to Reduce Patient and Caregiver Anxiety around Radiation Treatment for Brain Tumors with a Customized Neuro-Imaging Referenced Symptom video (for glioma grade 2, 3 and 4)	Imaging Trial		Active
2018-0564 NCT04186832	Feasibility of Monitoring Step Count as a Measure of Physical Activity in Patients with Newly Diagnosed Glioma Patients Undergoing Radiation Therapy			Active
2020-0124 NCT04553757	Seizure control as a new metric in assessing efficacy of tumor treatment in patients with low grade glioma			Active
2015-0953NCT03896568	Phase I Clinical Trial Of Allogeneic Bone Marrow Human Mesenchymal Stem Cells Loaded With A Tumor Selective Oncolytic Adenovirus, DNX-2401, Administered Via Intra-Arterial Injection In Patients With Recurrent High-Grade Glioma	Phase I	MSC-D24	Hold
PA17-0844NCT04771806	Serial Advanced Magnetic Resonance Imaging (MRI) for Guidance of Personalized Adaptive Radiotherapy for High Grade Glioma	Imaging Trial	n/a	Active
NRG-BN005 NCT03180502	A Phase II Randomized Trial of Proton vs. Photon Therapy (IMRT) for Cognitive Preservation in Patients with IDH Mutant, Low to Intermediate Grade Gliomas*	Phase II	Proton vs Photon 2:1 randomization	CNPE

*Grade II and III IDH mutant gliomas

Brain Metastases

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2020-1111 NCT04365374	A Phase 3 Randomized Controlled Trial of Post-Surgical Stereotactic Radiotherapy (SRT) versus Surgically Targeted Radiation Therapy (STaRT) with Gamma Tile for Treatment of Newly Diagnosed Metastatic Brain Tumors	Phase III	Surgery + SRT, or Surgery + GT
2023-0131 NCT06224543	Evaluate Artificial Intelligence (AI) Synthesized MR Images from Low Dose Gadobutrol CNS Images vs Standard Dose Gadobutrol CNS Images	Imaging Trial	n/a	Active
2021-0899 NCT05673928	A Phase II Study of Tucatinib and Ado-trastuzumab Emtansine (T-DM1) in Patients With HER2-positive Metastatic Solid Tumors and Metastases to Brain (TUCATEMEB)	Phase II	Tucatinib, T-DM1	Open/active
2021-0611 NCT05064280	Ph II Study of Pembrolizumab in combination with Lenvantinib in Pts. w/ TNBC, NSCLC, and other Tumor Types and Brain Metastasis	Phase II	Pembro, Lenvatinib	Open

Lung Cancer with Brain Metastases

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2015-0883 NCT02696993	Phase I/II trial of nivolumab with radiation or nivolumab and ipilimumab with radiation for the treatment of intracranial metastases from non-small cell lung cancer	Phase I/II	Infused chemo- therapy and SRS or WBRT	only Part B Phase 2 open
2024‐0369 NCT04804644	NRG-CC009: Phase III trial of stereotactic radiosurgery (SRS) versus hippocampal-avoidant whole brain radiotherapy (HA-WBRT) for 10 or fewer brain metastases from small cell lung cancer	Phase III	Randomized study: Arm 1 = Stereotactic radiosurgery (SRS/FSRS)3 Arm 2 = Whole brain radiotherapy with hippocampal avoidance (HA-WBRT)+ Memantine	Open
2019-0900 NCT04516070	Stereotactic radiosurgery (SRS) as definitive management for a limited number of small cell lung cancer (SCLC) brain metastasis	Phase II	SSRS	Active

Melanoma with Brain Metastases

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2021-1203 NCT05704647	Phase II Study of Nivolumab in Combination with Relatlimab in Patients with Active Melanoma Brain Metastases	Phase II	nivolumab/relatlimab	Open/active

Renal Cell Carcinoma with Brain Metastases

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2021-0520 NCT05048212	Ph II study of nivolumab with ipilimumab and cabozantinib in patients with untreated renal cell carcinoma brain metastasis	Phase II	Ipi/, Nivo, Cabo	Open

Breast Cancer with Brain Metastases

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2021‐0836NCT04647916	A Phase II Trial of Sacituzumab Govitecan (IMMU-132) (NSC #820016) for Patients With HER2-Negative Breast Cancer and Brain Metastases	Phase II	Sacituzumab Govitecan (IMMU-132)
2022‐0910 NCT05323955	Secondary Brain Metastases Prevention After Isolated Intracranial Progression on Trastuzumab/Pertuzumab or T-DM1 in Patients With Advanced Human Epidermal Growth Factor Receptor 2+ Breast Cancer With the Addition of Tucatinib	Phase II	Trastuzumab, Trastuzumab Emtansine (T-DM1), Pertuzumab, Tucatinib
2022-0638NCT05386108	An Open-label Multicenter Phase 1b-2 Study of Elacestrant as Monotherapy and in Combination with Abemaciclib in Women and Men with Brain Metastasis from Estrogen Receptor Positive, HER-2 Negative Breast Cancer (ELECTRA)	Phase IB/II	Elacestrant, Abemaciclib	Open per slot availability

Advanced Solid Tumors

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2019-0273 NCT03975387	Phase 1/2 Open-Label Study of the Safety, Pharmacokinetics, and Preliminary Activity of ASTX295 in Subjects with Wild-Type TP53 Advanced Solid Tumors	Phase I/II	ASTX295
2019-0595 NCT04137900	First-in-Human, Multicenter, Open-Label, Phase 1 Dose-Escalation and Cohort Expansion Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of TAB004 as Monotherapy and in Combination with Toripalimab in Subjects with Advanced Solid Malignancies	Phase I	TAB004 + Toripalimab
2019-0676 NCT04130516	A Multicenter Phase 1-2A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of LNS8801 in Patients With Advanced Cancer With and Without Pembrolizumab	Phase I/II	LNS8801- expansion
2019-0697 NCT04123366	A Phase 2 Study of Olaparib in Combination with Pembrolizumab in Participants with Previously Treated, Homologous Recombination Repair Mutation (HRRm) and/or Homologous Recombination Deficiency (HRD)-Positive Advanced Cancer	Phase II	Olaparib + Pembrolizumab
2019-1066 NCT04190628	A Phase I, First-In-Human, Multicenter, Open-Label Study of ABM-1310, Administered Orally in Adult Patients with Advanced Solid Tumors	Phase I	ABM-1310
2020-0439 NCT04771520	Phase 2 Study of Avapritinib in Patients with CKIT or PDGFRA Mutation-Positive Malignant Solid Tumors	Phase II	Avapritinib
2020-0564 NCT04879121	A Phase II Basket Study of the ORAL TRK inhibitor Larotrectinib in Subjects with NTRK amplification positive and Pan TRK positive tumor	Phase II	Larotrectinib
2020-0595 NCT04526106	A First-in-human Study of Highly Selective FGFR2 Inhibitor, RLY-4008, in Patients with Intrahepatic Cholangiocarcinoma (ICC) and Other Advanced Solid Tumors	Phase I	RLY-4008
2020-0833 NCT04585750	A Phase 1/2 Open-label, Multicenter Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of PC14586 in Patients with Advanced Solid Tumors Harboring a p53 Y220C Mutation (PYNNACLE)	Phase I/II	PC14586
2020-1238 NCT04762602	A Multicenter, Open-Label Phase 1 Study Evaluating the Safety and Tolerability of HMPL-306 in Subjects with Locally Advanced or Metastatic Solid Tumors with IDH Mutations	Phase I	HMPL-306
2021-0873 NCT05103358	A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes	Phase I	ABI-009
2021-1180 NCT05275478	A Phase 1/2, Multi-Center, Open-Label Study to Evaluate the Safety, Tolerability, and Preliminary Antitumor Activity of TNG908 in Patients with MTAP-deleted Advanced or Metastatic Solid Tumors	Phase I/II	TNG908 Escalation
2022-0845 NCT05584670	A First-in-human, Dose Escalation and Dose Expansion Study of SAR445877 in Adult Participants With Advanced Solid Tumors	Phase I/II	SAR445877
2021-1207 NCT05231746	A Phase 1, Multicenter, Open-label Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of hSTC810 Monotherapy in Subjects with Advanced Solid Tumors	Phase I	hSTC810
2022-0061 NCT05228600	A Multi-Center, Open-Label, Phase I Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Anti-tumor Activity of YL-13027 in Patients with Advanced Solid Tumors	Phase I	YL-13027; Part 1 (Phase Ia) Dose Escalation
NCI10129 NCT03212274	A Phase 2 Study of the PARP Inhibitor Olaparib (AZD2281) in IDH1 and IDH2 mutant Advanced Solid Tumors	Phase II	Olaparib
2022‐0674 NCT05593094	A Phase 1 Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of ZN-A-1041 Enteric Capsules as a Single Agent or in Combination in Patients With HER2-Positive Advanced Solid Tumors*	Phase I	ZN-A-1041
2022‐0764 NCT05868226	PRE-I-SPY TRIAL - PRE-Investigation of Serial Studies to Predict Your Therapeutic Response with Imaging And molecular Analysis: A Phase I/Ib platform trial*	Phase I	Tucatinib 300mg BID Zanidatamab 20mg/kg IV Q 2 weeks	Open

*Specifically, HER2-positive Breast Cancer

Primary Central Nervous System Lymphoma

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2024-0453 NCT03328078	An Open-Label, Dose Escalation and Dose Expansion Trial Evaluating the Safety, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Orally Administered CA-4948 in Patients with Relapsed or Refractory Primary Central Nervous System Lymphoma	Phase I/II	CA-4948 and Ibrutinib	Active
2023-0329 NCT04947319	An Open-label Phase II Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Tirabrutinib in Patients With Primary Central Nervous System Lymphoma (PCNSL)	Phase II	Tirabrutinib	Active

Chordoma

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2020-0217 NCT05041127	This is a multicenter, single arm, phase 2 study designed to evaluate the efficacy and safety of cetuximab for the treatment of advanced (unresectable)/metastatic, chordoma. The target patient population will be any chordoma patient 18 years of age with locally unresectable disease or metastatic disease.	Phase II	Cetuximab	Active

Meningioma

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
ALLIANCEA071401 NCT02523014	Phase II Trial of SMO/AKT/NF2/CDKe Inhibitors in Progressive Meningiomas With SMO/AKT/NF2/CDK Pathway Mutations	Phase II		Active
2016-0684 NCT02693990	A Phase I/II Trial of Increased Dose Intensity Modulated Proton Therapy (IMPT) for meningioma*	Phase I/II	IMPT Proton	Active

*Grade II and Grade III Meningioma

Ependymoma

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2018-0344 NCT03750513	Pilot trial of LET optimized IMPT for pediatric patients with ependymoma*	Phase I	IMPT Proton	Active

*Grade II and III ependymoma

Head and neck squamous cell carcinomas

Protocol number / NCT	Protocol Title	Phase	Treatment	Study Status
2017-0826 NCT03575949	Dual-Time Point (DTP) FDG PET CT for the Post-Treatment Assessment of Head and Neck Tumors Following Definitive Chemoradiation Therapy	Imaging Trial	n/a	Hold

Multiple Cancer Types


Protocol number / NCT
Protocol Title
Disease Subtypes
Phase
Treatment
Study Status
2023-0845
Neuro health initiative: a standardized multiphase approach to prospective data collection for patients undergoing cancer treatments at risk for neurotoxicity (The NHI Program)
Brain and Nervous System
n/a
n/a
Active
2020-0904

NCT03173950
Phase II Trial of the Immune Checkpoint Inhibitor Nivolumab in Patients with Recurrent Select Rare CNS Cancers
Choroid plexus tumor, Gliosarcoma, Medulloblastoma, Neuroblastoma
Phase II
Nivolumab
Active
2022-0170

NCT06200155
TRIP-Treatment to improve depression and/or anxiety using psilocybin-assisted psychotherapy in patients with advanced cancer on maintenance therapy
Depression, Anxiety, Psilocybin-Assisted Psychotherapy, Advanced Cancer


Phase II


Psilocybin
Active
2019-0715

NCT04623931
Phase II Trial of Treatment Intensification for IDH Wildtype, Non-histological Glioblastoma, Gliomas (IDH Wildtype Lower Grade Glioma Treatment Intensification)
Diffuse astrocytoma IDH wildtype (molecular GBM), previously anaplastic astrocytoma, anaplastic oligodendroglioma, or grade II
Phase II


Dose escalation radiation daily to 60Gy
Active


2019-0715

NCT04623931
Phase II Trial of Treatment Intensification for IDH Wildtype, Non-histological Glioblastoma, Gliomas (IDH Wildtype Lower Grade Glioma Treatment Intensification)
Diffuse astrocytoma IDH wildtype (molecular GBM), previously anaplastic astrocytoma, anaplastic oligodendroglioma, or grade II
Phase II


Dose escalation radiation daily to 60Gy
Active


2022-0198

NCT05500508
A Phase 1B/2A Study of the Safety, Tolerability and Initial Efficacy of Oral AMXT 1501 Dicaprate and Intravenous Difluoromethylornithine (DFMO) in Patients with Cancer
Diffuse Midline Glioma, Advanced solid tumors 
Phase I/II
AMXT1501 + DMFO
 
2022-0044

NCT05450562
A Phase 1/2 Open-label, First-in-human, Dose Escalation and Expansion Study for the Evaluation of Safety, Pharmacokinetics, Pharmacodynamics, and Anti-tumor Activity of SAR444200-based Regimen in Participants With Advanced Solid Tumors


Liver (hepatocellular carcinoma), lung (non-small cell carcinoma), NOS, solid tumor
Phase I/II
SAR444200, Cemiplimab
 
2021-0972

NCT05199584
A Phase 2, Multi-Center Study Evaluating the Safety and Efficacy of ENV-101 (Taladegib) in Patients with Advanced Solid Tumors Harboring PTCH1 Loss of Function Mutations
Medulloblastoma, advanced solid tumors
Phase II
ENV-101
 
2024-0173 (SELENA) 

NCT06545682 
Phase Ib Study of AlpeliSib with PEmbroLizumab in Patients with MEtastatic Breast CaNcer or MelanomA (SELENA) 
Melanoma w & w/o brain mets and TNBC w brain mets
Phase IB
Alpelisib + Pembrolizumab
Open
2024-0082

NCT06536504
A Randomized Controlled Trial to Assess the Impact of Live and Recorded Music-Based Interventions on Pre-operative Stress, Mood, Pain and Biomarkers, in Neurosurgical Patients (Music-STAR Trial)


Patients undergoing neurosurgical procedures
 
n/a


Active
PA18-0719

NCT05087888
Investigation of Serial Advanced Magnetic Resonance Imaging (MRI) and Biofluid Biomarkers Predictive of Neurocognitive Decline following Brain Radiation
brain tumors and head and neck tumors, that during radiation, expose the brain to radiation 
Imaging Trial
n/a
Active
2021-1174

NCT05700721
Phase II trial of the PARP inhibitor niraparib and PD-1 inhibitor dostarlimab in patients with advanced cancers with active progressing brain metastases (STARLET)
BRCA1/2, HRR aberrant, NSCLC, SCLC, TNBC
Phase II


Niraparib & Dostarlimab
Open/active
2020-0493

NCT04988009
Patient Perceptions Around Quality of Care through Telemedicine in Neuro-Oncology
all 
 
 
Active
2021-1019
Dosimetry and bio-distribution of 68Ga-RMX-VH in newly diagnosed or recurrent patients with solid tumors: A Phase I exploratory study
 
Imaging

Trial
 
Active
2023-0010
A Phase I Study of the Treatment of Recurrent Malignant Glioma with rQNestin34.5v.2, a Genetically Engineered HSV-1 Virus, and Immunomodulation with Cyclophosphamide


IDH wild-type glial tumor including GBM, grade 3 anaplastic astrocytoma or oligodendroglioma or grade 2 astrocytoma with genetic features consistent with GBM
Phase I
 Six repeat doses of rQNestin34.5v.2


Active
2023-0206

NCT04498026
Adherus™ Dural Sealant in Spinal Procedures


Patients receiving Spinal Duraplasty
Pivotal Trial
n/a
Active
2022-0585

NCT05804227
Window-of-opportunity trial of Ulixertinib for MAPK-activated low-grade gliomas in adults
Glioma, oligodendroglioma
Phase I
Ulixertinib
Active
2018-0552

NCT03741673


A Phase III trial of Pre-operative Stereotactic Radiosurgery (SRS) versus Post-operative SRS for brain metastases
Any metastatic primary eligible for SRS

(but small-cell lung, lymphoma, leukemia, or multiple myeloma)
Phase III
surgery and radiation
Active