NCT05124912NARecruiting
Monteris Medical · UCLA, Los Angeles, California
Randomized, post-market multi-center study investigating the efficacy of two sets of treatment algorithms in brain metastases (BM) patients at the time of first intervention for radiographic progression after stereotactic radiosurgery (SRS), with or without surgery.
Eligibility: 18 Years to · All · In order to be eligible to participate in this study, an individual must meet all of the following criteria:
1. Patients with radiographically proven (by gadolinium-enhanced \[Gd-\] MRI) parenchymal brain metastases from histologically confirmed non-central nervous system (CNS) cancer.
2. Patients with a "targetable", bidimensionally-measurable, intracranial lesion that is radiographically recurrent after previous treatment with SRS +/- surgery (craniotomy or LITT). To classify a lesion as radiographically progressive, the lesion must demonstrate a ≥ 25% increase in size following treatment based on the Neuro-Oncology Criteria of Tumor Response for CNS Tumors. To be "targetable" for this study, the lesion should be coverable through a planned single LITT trajectory and thus have a maximum perpendicular diameter (perpendicular to the laser trajectory) of 3 cm. An intra-operative decision to utilize two trajectories is acceptable and patient may remain on study.
3. Patient must be at least 3 months post initial SRS treatment of the target lesion
4. Target lesion must be amenable to undergo surgical biopsy and LITT treatment as determined by the treating neurosurgeon.
5. Frozen pathology diagnosis must be attainable.
6. Patient must be symptomatically stable for a minimum of 3 days prior to the procedure date on a on a max total daily steroid dose equivalent to 4mg of Dexamethasone.
7. ≥18 years of age
8. KPS ≥70
9. Patient is able and willing to complete study requirements
10. Patients with adequate hematologic parameters (all tests to be performed within \<4 weeks of biopsy):
1. ANC ≥ 1.5 X 109/L
2. Platelet count ≥ 100 x 109/L
11. Blood chemistry laboratory value for serum creatinine \< 1.5 x ULN (test to be performed within \<4 weeks of biopsy)
12. Female patients must have a negative serum pregnancy test at screening. (Not applicable to patients with bilateral oophorectomy and/or hysterectomy or to those patients who are postmenopausal)
13. All patients of reproductive potential must agree to use an effective method of contraception during the study
14. Patients must be accessible for follow-up
NCT05688683Phase IIRecruiting
Fondation Ophtalmologique Adolphe de Rothschild · Fondation Adolphe de Rothschild, Paris
Interactions between the perceptual and motor systems are fundamental to the performance of complex motor tasks and are at the heart of the fine motor control required for the production of complex sounds such as speech production or playing a musical instrument. In such situations, the brain must learn to generate relevant motor commands to a sound-producing system with fixed physical characteristics, such as the vocal tract or a musical instrument.
No study has yet been able to directly test the dynamic aspect of this sensorimotor learning in an acoustic production task with fine motor control.
The Adolphe de Rothschild Foundation Hospital takes care of patients requiring awake surgery. During these procedures, a direct cortical recording, called electro-corticography, is performed in order to better delineate the tumor or epileptogenic resection area. Reference recordings are made in healthy areas at a distance from the lesion site making it possible to record normal brain activity.
In this case, we would propose to the patient to use a tool similar to the theremin (a musical instrument the size of a golf ball whose displacement in space modulates the frequency and the harmonics of a sound). The patient should therefore learn in order to create relevant motor patterns.
Eligibility: 18 Years to · All · * Benefiting from an intracranial brain recording during a programmed surgery in awake condition.
NCT00001595Phase IIRecruiting
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · National Institutes of Health Clinical Center, Bethesda, Maryland
There is a variety of tumors affecting the pituitary gland in childhood; some of these tumors (eg craniopharyngioma) are included among the most common central nervous system tumors in childhood. The gene(s) involved in the pathogenesis of these tumors are largely not known; their possible association with other developmental defects or inheritance pattern(s) has not been investigated. The present study serves as a (i) screening/training, and, (ii) a research protocol.
As a screening and training study, this protocol allows our Institute to admit children with tumors of the hypothalamic-pituitary unit to the pediatric endocrine clinics and wards of the NIH Clinical Center for the purposes of
(i) training our fellows and students in the identification of genetic defects associated with pituitary tumor formation, and
(ii) teaching our fellows and students the recognition, management and complications of pituitary tumors
As a research study, this protocol aims at
(i) developing new clinical studies for the recognition and therapy of pituitary tumors; as an example, two new studies have emerged within the context of this protocol: (a) investigation of a new research magnetic resonance imaging (MRI) tool and its usefulness in the identification of pituitary tumors, and (b) investigation of the psychological effects of cortisol secretion in pediatric patients with Cushing disease. Continuation of this protocol will eventually lead to new, separate protocols that will address all aspects of diagnosis of pituitary tumors and their therapy in childhood.
(ii) Identifying the genetic components of pituitary oncogenesis; those will be investigated by (a) studying the inheritance pattern of pituitary tumors in childhood and their possible association with other conditions in the families of the patients, and (ii) collecting tumor tissues and examining their molecular genetics. As with the clinical studies, the present protocol may help generate ideas for future studies on the treatment and clinical follow up of pediatric patients with tumors of the pituitary gland and, thus, lead to the development of better therapeutic regimens for these neoplasms.
Eligibility: 2 Years to 70 Years · All · * INCLUSION CRITERIA:
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
1. Stated willingness to comply with all study procedures and availability for the duration of the study.
2. Male or female with:
1. Evidence for the existence of a tumor of the hypothalamic-pituitary unit or related disorder, as indicated by previously obtained imaging studies or biochemical investigation of the hypothalamo-hypophyseal function (aged 2 years to 70 years)
or
2. Family members (any age) of patients with a family history of tumors of the hypothalamic-pituitary unit or related disorders as part of the linkage part of the study, or
3. Members (any age) of a kindred suspected of having an inherited form of neoplasia of the hypothalamic-pituitary unit or related disorder, as evidenced by results of a patient enrolled in this protocol, as part of the linkage part of the study
3. Ability of the subject or LAR to understand and the willingness to sign a written informed consent document.
EXCLUSION CRITERIA:
An individual who meets any of the following criteria will be excluded from participation in this study:
1. Pregnancy: Pregnancy will be evaluated only in participants of reproductive age (from 10 years old until 60 years of age unless menopause has already occurred per clinical report of the participant).
For participants enrolled as external participants or under the Linkage study (where research activities include no more than blood draws), any female who could possibly become pregnant will be screened using clinical criteria (history, with pregnancy testing only if indicated) for exclusion and this information will be documented in the consent process note in EMR. If a participant has initially been registered as external location and then presents on-site, then pregnancy test will be performed if within the reproductive age group.
2. Patients with any medical, physical, psychiatric, or social condition, which, in the opinion of the investigators, would make participation in this protocol not in their best interest, will be excluded from the study.
3. Patients who are critically ill, unstable, or with severe organ failure that may affect/limit the endocrine evaluation and place unsustainable demands on CC or NICHD resources may be excluded.
NCT05831631Phase IIRecruiting
Pietro Mortini, MD, Prof. · IRCCS San Raffaele Scientific Institute, Milan, Milan
The goal of this observational study is to characterize the circulating leukocyte profile and the immune T cells distribution within the tumor in patients with malignant brain tumors and to correlate these findings with the oncological outcome.
Participants will be subjected to blood sampling before surgery and for 12 months of follow-up. Additional sampling and analysis will be performed on tumor samples.
Eligibility: 18 Years to · All · Inclusion criteria:
* Adult patients (≥18 years)
* Able to express informed consent
* With primary or secondary malignant brain tumor
* Requiring neurosurgical treatment (radiosurgery, stereotactic biopsy, surgery)
NCT07688356Phase 2Recruiting
Samsung Medical Center · Samsung Medical Center, Seoul
This is a Phase 2, open-label, single-arm clinical study designed to evaluate the efficacy and safety of Belvarafenib in patients with BRAF-altered primary brain tumors (Cohort 1) and metastatic brain tumors (Cohort 2). Eligible patients are those with a confirmed BRAF alteration identified by next-generation sequencing (NGS).
Patients who meet the eligibility criteria will receive a detailed explanation of the study, including its purpose, procedures, potential benefits, and risks. Only patients who voluntarily provide written informed consent will be enrolled.
All enrolled patients will receive Belvarafenib monotherapy at a dose of 450 mg twice daily (BID). The study drug will be taken orally within 30 minutes after meals with at least 200 mL of water, preferably at approximately 12-hour intervals each day. One treatment cycle is defined as 28 consecutive days of continuous dosing without a planned treatment break. Patients will receive treatment for six cycles (approximately six months) as the initial treatment period. Treatment may be extended or discontinued earlier at the investigator's discretion based on clinical benefit, disease status, and tolerability.
During the study, patients will undergo regular clinical evaluations, including physical examinations, vital sign assessments, laboratory tests, and monitoring for adverse events. Radiologic assessments using MRI and/or CT will be performed at scheduled intervals to evaluate tumor response and disease progression. The study aims to determine whether Belvarafenib can control tumor growth, delay disease progression, and improve clinical outcomes in patients with BRAF-altered brain tumors.
If treatment-related toxicities occur, dose reductions are permitted according to the protocol. The dose may be reduced from 450 mg BID to 300 mg BID, and subsequently to 200 mg BID, if clinically indicated. Temporary treatment interruption may also be implemented until toxicity resolves. If unacceptable toxicity persists despite dose modification, treatment will be permanently discontinued.
Study treatment may be discontinued if any of the following occurs: confirmed disease progression, unacceptable toxicity, withdrawal of informed consent, inability to comply with the study protocol, receipt of other anticancer therapies that may interfere with study outcomes, or if the investigator determines that continued treatment is no longer in the patient's best interest. However, if radiologic disease progression is observed but the investigator determines that the patient continues to derive clinical benefit, treatment beyond progression may be considered after discussion with the sponsor, with appropriate documentation of the rationale.
After discontinuation of study treatment, patients will receive the most appropriate subsequent management, including best supportive care (BSC) or other anticancer therapies, as determined by the treating investigator. Follow-up assessments will continue according to the study protocol.
The primary objective of this study is to evaluate the efficacy of Belvarafenib in patients with BRAF-altered primary and metastatic brain tumors, while also assessing its safety profile. The results of this study are expected to provide important clinical evidence supporting the development of new treatment strategies for patients with BRAF-altered brain tumors.
Eligibility: 19 Years to · All · (Applicable to Both Cohorts)
1. Male or female patients aged 19 years or older.
2. Histologically confirmed primary brain tumor or metastatic brain tumor.
3. Documented BRAF mutation, including point mutations (e.g., V600E) or BRAF fusion mutations.
4. Willing and able to provide written informed consent prior to participation in the study.
5. Estimated life expectancy of at least 3 months.
6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
7. Adequate organ function demonstrated by laboratory assessments performed within 14 days prior to the first dose of study treatment, meeting all of the following criteria:
* Absolute neutrophil count (ANC) ≥ 1.5 × 10⁹/L
* Hemoglobin ≥ 9 g/dL
* Platelet count ≥ 100 × 10⁹/L
* PT/INR and aPTT ≤ 1.5 × upper limit of normal (ULN)
* Total bilirubin ≤ 1.5 × ULN (≤ 3 × ULN for patients with Gilbert syndrome)
* AST and ALT ≤ 2.5 × ULN (≤ 5 × ULN in patients with liver metastases)
* Alkaline phosphatase ≤ 2.5 × ULN (≤ 5 × ULN in patients with liver or bone metastases)
* Albumin ≥ 2.5 g/dL
* Amylase ≤ 1.5 × ULN
* Serum creatinine ≤ 1.5 × ULN or calculated creatinine clearance (CrCl) \> 50 mL/min using the Cockcroft-Gault formula
8. Women of childbearing potential (defined as women from menarche until 1 year after menopause unless permanently sterile) and men with partners of childbearing potential must agree to use highly effective contraception throughout the study and for 3 months after the last dose of Belvarafenib.
Women of childbearing potential must have a negative pregnancy test during screening unless surgically sterile.
Acceptable contraceptive methods include:
* Hormonal contraception
* Intrauterine device (IUD) or intrauterine system (IUS)
* Vasectomy or bilateral tubal occlusion
* Complete abstinence Barrier methods (e.g., male or female condoms); if barrier methods are used alone, the use of two complementary barrier methods is recommended.
9. Additional Inclusion Criteria for Cohort 1 (Primary Brain Tumors) 1) Patients with BRAF-mutant primary brain tumors who meet at least one of the following conditions:
\- No available or appropriate local treatment options (e.g., surgery or radiotherapy);
* Radiographic evidence of disease recurrence or progression following standard therapy (including surgery and/or chemoradiotherapy \[CCRT\]), with no further suitable standard treatment available;
* Standard treatment is considered inappropriate or unavailable in the investigator's judgment.
10. Additional Inclusion Criteria for Cohort 2 (Metastatic Brain Tumors)
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1. Patients with brain metastases from BRAF-mutant solid tumors.
2. Patients with radiographic evidence of disease progression after receiving the standard treatment for the primary malignancy, or whose disease is refractory to conventional therapy, regardless of prior exposure to BRAF-targeted therapy, and who have no available or appropriate local treatment options (e.g., surgery or radiotherapy).
3. At least one measurable intracranial lesion, with a maximum of five target lesions, as defined by the Response Assessment in Neuro-Oncology (RANO) criteria.
NCT06351904Phase 1Recruiting
Ractigen Therapeutics. · GenesisCare North Shore, St Leonards, New South Wales
This is an open label, multi-center study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of RAG-01 in patients with NMIBC who have failed BCG therapy.
Eligibility: 18 Years to 75 Years · All · 1. Ability to understand the study and have signed the informed consent form;
2. Any adult ≥ 18 years old;
3. Pathologically confirmed high grade NMIBC defined as grade 2 or grade 3 disease;
4. Expected survival ≥ 6 months;
5. ECOG PS ≤2;
6. Sufficient organ functions, as defined below:
Investigations Hematology Absolute Neutrophil Count (ANC): ≥ 1.5 x 109/L Hemoglobin: ≥ 90 g/L Platelet: ≥ 100 x 109/L Liver Function Serum bilirubin: ≤ 1.25×ULN or 2.5×ULN（with Gilbert syndrome） AST \& ALT: ≤ 2.5×ULN Renal Function Creatinine Clearance (Cockcroft-Gault equation): ≥ 30 mL/min
7. Subject must be able to tolerate catheterization;
8. Female subject of childbearing potential and her spouse must use adopt effective contraception (non-pharmacological contraception required) from signing informed consent to within 6 months after the last instillation;
NCT07359235Phase 1Recruiting
Tongji Hospital · Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei
Recombinant oncolytic adenovirus injection (KD01) is an oncolytic virus product. Its main component is a conditionally replicative recombinant human type 5 adenovirus, where part of the E3 region has been replaced with the gene encoding the tBid apoptotic protein.AK104 is a humanized bispecific antibody co-targeting PD-1 (Programmed Cell Death Protein 1) and CTLA-4 (Cytotoxic T-Lymphocyte-Associated Antigen 4)-two key immune checkpoint receptors. It is designed as a novel tetrameric construct that preferentially binds to tumor-infiltrating lymphocytes (TILs) co-expressing PD-1 and CTLA-4 in the tumor microenvironment (with higher avidity than in peripheral tissues).This is a Phase I clinical study designed to evaluate the safety, tolerability, and preliminary efficacy of intravesical instillation of recombinant oncolytic adenovirus injection (KD01) in patients with bladder cancer.This study consists of Phase Ia and Phase Ib, where Phase Ia is a dose-escalation stage.The Phase Ia will include histopathologically confirmed non-muscle-invasive bladder cancer (NMIBC) patients with high-risk stratification (including extremely high-risk).The Phase Ib study will include two independent cohorts: Cohort A comprises high-risk non-muscle-invasive bladder cancer (NMIBC) patients (carcinoma in situ \[CIS\], with or without Ta/T1 stage lesions) who have shown no response to BCG. Cohort B will include T2-4aN0-1M0 stage bladder cancer patients (clinically localized muscle-invasive bladder urothelial carcinoma).
Eligibility: 18 Years to 75 Years · All · * 1.The Phase Ia will include histopathologically confirmed non-muscle-invasive bladder cancer (NMIBC) patients with high-risk stratification (including extremely high-risk). High-risk stratification criteria are defined as G3/high-grade tumors that meet any of the following criteria: carcinoma in situ (CIS); stage T1; diameter\>3 cm; multiple tumors; or recurrent tumors. The Phase Ib Cohort A will include BCG unresponsive NMIBC patients with carcinoma in situ \[CIS\], with or without Ta/T1 stage lesions, while Cohort B will include histopathologically confirmed muscle-invasive bladder cancer (cT2-4aN0-1M0).Note: BCG non-response is defined as meeting any of the following criteria: 1. Persistent or recurrent CIS within 12 months after completing adequate BCG treatment; 2. Recurrence of high-grade Ta/Tl disease within 6 months after completing adequate BCG treatment; 3. High-grade T1 disease at first evaluation after induction BCG treatment. (Adequate BCG treatment is defined as: Complete at least 5 out of 6 doses in the initial induction course. plus 2 out of 6 doses from the second induction course, or 2 doses from the 3-dose maintenance course.)
* 2.Excluding Phase Ib Cohort B, participants must either be medically ineligible for radical cystectomy as determined by urologists or have declined the procedure.
* 3.Age 18 to 75 (including 18 and 75).
* 4.Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) score of 0 to 1.
* 5.Estimated survival time is ≥2 years.6.No dysfunction of major organs, including but not limited to hematopoietic function and cardiac, pulmonary,hepatic and renal function.
Hematologic system (no history of blood transfusion or hematopoietic growth factor treatment within 14 days)
1. Absolute neutrophil count (ANC)≥1.5×109/L
2. Platelets （PLT）≥75×109/L
3. Hemoglobin （Hb）≥90g/L
Hepatic function
1. Total bilirubin (TBIL)≤1.5× upper limit (ULN)
2. Alanine aminotransferase (ALT)≤3×ULN
3. Aspartate aminotransferase (AST)≤3×ULN
Renal function
1. Creatinine （Cr）≤1.5×ULN
2. Creatinine clearance (Ccr) (Calculation required only if creatinine exceeds 1.5×ULN)\>50 mL/min (calculated using the Cockcroft-Gault formula)
Coagulation function
1. Activated Partial Thromboplastin Time (APTT)≤1.5×ULN
2. International normalized ratio (INR)≤1.5×ULN
* 7.Premenopausal patients must have a negative blood pregnancy test during the screening period;they must agree to use reliable contraceptive methods (barrier contraceptive method or abstinence) with their partners during the trial and for at least 6 months after the last dose.
* 8.Patients must provide informed consent to the trial and voluntarily sign of a written informed consent form before participating in the trial.
NCT07036731NARecruiting
The First Affiliated Hospital with Nanjing Medical University · The First Affiliated Hospital of Nanjing Medical University, Nanjing, Jiangsu 210029, Nanjing, Jiangsu
This study is a prospective, randomized, multicenter clinical trial aiming to enroll 428 patients with non-muscle-invasive bladder cancer (NMIBC) from the Department of Urology at Jiangsu Provincial People's Hospital and its affiliated centers, who have undergone their initial transurethral resection and are scheduled for a second resection. Within 1-6 weeks after the first resection and prior to the second resection, 100ml of routine urine will be collected and sent within 24 hours to Beijing Genetron Health Clinical Laboratory Co., Ltd. for the extraction of urinary sediment DNA, which will then undergo genome-wide low-depth sequencing. Patients with positive test results will proceed with the second resection, while those with negative results will be randomly assigned in a 1:2 ratio to either the intervention group, which will not undergo a second resection, or the control group, which will undergo the second resection. All enrolled patients will be followed up for two years. The study will statistically analyze and compare the two-year recurrence-free survival rates between the two groups and evaluate the predictive capability of the test results for follow-up recurrence.
Eligibility: 18 Years to 80 Years · All · * Patients with NMIBC who have completed the initial transurethral resection and are scheduled for a second resection, as recommended by guidelines or physician's choice:
1. Incomplete initial TURBT due to various reasons (including large tumor size, high risk of intraoperative bleeding, extensive tumor range, etc.), posing a risk of residual tumor;
2. Absence of muscle layer tissue in the initial resection specimen;
3. Post-initial resection pathology indicating T1 stage;
4. Post-initial resection pathology indicating G3/high-grade tumor, excluding pure carcinoma in situ;
* Ability to provide a 100ml routine urine sample within 1-6 weeks after the initial resection and before the second resection;
* Age ≥ 18 years;
* Willingness to provide basic clinical information, pathology, and subsequent - recurrence monitoring results;
* Willingness to sign the informed consent form.
NCT07475403Phase 2Recruiting
Tianjin Medical University Second Hospital · The Second Hospital of Tianjin Medical University, Tianjin
This study evaluates whether urinary tumor DNA (utDNA) testing, together with clinical, pathologic, and radiographic assessment, can help guide treatment discontinuation and active surveillance in patients with unresectable very-high-risk non-muscle-invasive bladder cancer (VHR NMIBC) treated with bladder-sparing systemic immunotherapy.
Participants receive systemic immune checkpoint inhibitor-based therapy every 3 weeks for an initial 3 cycles. Initial response assessment is performed using transurethral resection of bladder tumor (TURBT) and chest and abdominopelvic computed tomography (CT). Participants without progression to muscle-invasive, regional nodal, or distant metastatic disease then undergo post-TURBT urine cytology and urinary tumor DNA (utDNA) testing. Participants with both negative urine cytology and negative utDNA results receive an additional 3 cycles of systemic immunotherapy.
After the additional treatment, participants undergo repeat evaluation using cystoscopy with biopsy, urine cytology, utDNA testing, and chest and abdominopelvic CT. Participants with negative findings on cystoscopic biopsy, urine cytology, and utDNA testing, and without radiographic evidence of nodal or distant metastatic disease, discontinue systemic immunotherapy and enter an active surveillance phase with regular follow-up monitoring. Participants who do not meet these criteria continue further clinical management and follow-up according to institutional practice.
The study aims to determine whether a shortened duration of systemic immunotherapy guided by integrated molecular, clinical, pathologic, and radiographic response assessment can maintain favorable oncologic outcomes while reducing unnecessary treatment exposure in this high-risk population.
Eligibility: 18 Years to · All · 1. Age ≥18 years.
2. Histologically confirmed non-muscle-invasive urothelial carcinoma of the bladder classified as very-high-risk (VHR) according to EAU 2025 guideline criteria.
3. Disease considered unresectable by the investigator and multidisciplinary team, defined as complete tumor eradication by standard transurethral resection of bladder tumor (TURBT) being not feasible or unlikely to achieve adequate local control.
4. Patients who are ineligible or refuse for radical cystectomy, after discussion with the treating team.
5. At least one measurable or evaluable bladder lesion/documented residual disease suitable for response assessment by cystoscopy, TURBT/biopsy, pathology, urine cytology, and urinary tumor DNA (utDNA) testing.
6. ECOG performance status 0-2.
7. Adequate organ function, including:
Hematologic function: Absolute neutrophil count ≥1.5 × 10⁹/L, Platelet count ≥100 × 10⁹/L, Hemoglobin ≥9 g/dL Hepatic function: Total bilirubin ≤1.5 × ULN, AST ≤2.5 × ULN, ALT ≤2.5 × ULN Renal function: Serum creatinine ≤1.5 × ULN or Creatinine clearance ≥60 mL/min.
8. Ability to provide urine samples for utDNA testing and urine cytology during treatment and follow-up.
NCT06394570Phase 1, Phase 2Recruiting
University of Texas Southwestern Medical Center · UT Southwestern Medical Center, Dallas, Texas
STAR-EV will evaluate the combination of enfortumab vedotin plus radiotherapy (RT) as neoadjuvant treatment for muscle invasive bladder cancer prior to radical cystectomy surgery. The study will use "dose escalation" to evaluate the safety and efficacy of study treatment at three dose regimens:
Level 0: EV treatment followed by RT to the bladder Level 1: EV treatment with RT starting on Cycle 2, Day 15 Level 2: EV treatment with RT starting on Cycle 1, Day 15
Following completion of EV+RT neoadjuvant therapy, all subjects will undergo surgery as part of routine care.
Eligibility: 18 Years to · All · 1. Urothelial carcinoma of the urinary bladder stage cT2-4a (AJCC 8th edition) N0M0 planned for radical cystectomy. Mixed cell types with variant histologies (including squamous, plasmacytoid, adenocarcinoma, sarcomatoid, micropapillary, nested, and lipid cell variants) are allowed as long as any urothelial histology is present (i.e. -not 100% variant histology). Small cell/neuroendocrine component is excluded.
2. Ineligibility for cisplatin-based chemotherapy based on treating physician assessment and any of the following "Galsky criteria": renal insufficiency (Creatinine Clearance \<60ml/min by standard institutional calculation method), \>=grade 2 peripheral neuropathy, \>=grade 2 hearing loss, New York Heart Association (NYHA) class III heart failure; a combination of these; or patient refusal.
3. Age \>=18.
4. Performance status Eastern Cooperative Oncology Group (ECOG) 0-1
5. Adequate organ and marrow function as defined below:
•Hematologic:
-Absolute neutrophil count (ANC) \>=1500/mm3
* Platelet count \>=100x109/L
* Hemoglobin ≥ 9 g/dL
•Hepatic:
* Serum bilirubin ≤ 1.5 × upper limit of normal (ULN) or ≤ 3 × ULN for subjects with Gilbert's disease
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 × ULN
•Renal:
* No end stage renal disease requiring dialysis allowed
6. All men, as well as women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 3 months following completion of study neoadjuvant therapy. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
6a. A female of child-bearing potential is any woman (regardless of sexual orientation, marital status, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
* Has not undergone a hysterectomy or bilateral oophorectomy; or
* Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months).
7.Ability to understand and the willingness to sign a written informed consent.
NCT05951179Phase 2Recruiting
Protara Therapeutics · Urology Centers of Alabama, Homewood, Alabama
TARA-002-101-Ph2 is an open-label study to investigate the safety and efficacy of intravesical instillation of TARA-002 in adults 18 years of age or older with high-grade CIS NMIBC (± Ta/T1).
The purpose of this Phase 2 study (TARA-002-101-Ph2) is to further assess the safety and efficacy of TARA-002 at the RP2D which has been established in the Phase 1a dose finding study (TARA-002-101-Ph1a).
This Phase 2 study includes participants with CIS NMIBC (± Ta/T1) with active disease, defined as disease present at last tumor evaluation (within 3 months) prior to signing the ICF.
Participants will be enrolled into one of 2 cohorts:
Cohort A:
* Participants with CIS (± Ta/T1) who are BCG naive, or
* Participants with CIS (± Ta/T1) who are BCG exposed and have not received intravesical BCG for at least 24 months prior to the most recent CIS diagnosis
Cohort B:
* Participants who are BCG unresponsive
Eligibility: 18 Years to · All · * Male or female participants 18 years of age or older at the time of signing informed consent
* Participants who have voluntarily given written informed consent after the nature of the study has been explained according to applicable requirements prior to study entry
* Central histologic confirmation of high-grade non-muscle invasive CIS (± Ta/T1) with active disease
* Participants who are BCG naive, or participants who are BCG exposed and have not received intravesical BCG for at least 24 months prior to the most recent CIS diagnosis (Cohort A). Participants with CIS (± Ta/T1) who are BCG unresponsive.
NCT06704191Phase 1, Phase 2Recruiting
Mayo Clinic · Mayo Clinic in Florida, Jacksonville, Florida
This phase Ib/II trial compares the safety, tolerability and acceptability of intravesical chemotherapy given at home to in-clinic administration in patients with non-muscle invasive bladder cancer. Chemotherapy drugs, such as bacillus Calmette-Guerin (BCG), gemcitabine, docetaxel, and mitomycin C, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Standard of care chemotherapy for non-invasive bladder cancer is usually given directly into the bladder through a catheter (intravesical). This process requires numerous visits and can be disruptive to the lives of patients and caregivers. Bringing cancer care to the patients with in-home intravesical therapy may help reduce the disruption to daily lives. In-home intravesical chemotherapy may be safe and tolerable and may also be preferable to in-clinic administration in patients with non-muscle invasive bladder cancer.
Eligibility: 18 Years to · All · * PHASE IB ONLY: Female or male patients with histologically confirmed non-muscle invasive bladder cancer (stage Ta, Tis, or T1) who are currently receiving induction therapy with one of the following eligible intravesical treatment regimens
* Gemcitabine
* Sequential gemcitabine/docetaxel
* Bacillus Calmette-Guerin
* Mitomycin C
* PHASE II ONLY: Female or male patients with histologically confirmed non-muscle invasive bladder cancer (stage Ta, Tis or T1) who are receiving maintenance therapy with an eligible regimen
* PHASE IB ONLY: Able to be successfully catheterized and able to tolerate first dost of intravesical therapy in the outpatient clinic
* Residing within the area serviced by supplier network
* Residence either has Wi-Fi or cellular data network connection for virtual telehealth visits
* Age ≥ 18 years at time of registration
* Signed informed consent form by patient
* Willing and able to comply with the study protocol in the investigator's judgment
* Ability to complete questionnaire(s) by themselves or with assistance
* Willingness to follow birth control requirements for females and males of reproductive potential
