更新于：2024-05-01

Choroid Plexus Neoplasms

脉络丛肿瘤

更新于：2024-05-01

基本信息

别名

Choroid Plexus Neoplasm、Choroid Plexus Neoplasms、Choroid Plexus Neoplasms, Primary

+ [16]

简介

Benign or malignant tumors which arise from the choroid plexus of the ventricles of the brain. Papillomas (see PAPILLOMA, CHOROID PLEXUS) and carcinomas are the most common histologic subtypes, and tend to seed throughout the ventricular and subarachnoid spaces. Clinical features include headaches, ataxia and alterations of consciousness, primarily resulting from associated HYDROCEPHALUS. (From Devita et al., Cancer: Principles and Practice of Oncology, 5th ed, p2072; J Neurosurg 1998 Mar;88(3):521-8)

关联

项与脉络丛肿瘤相关的药物

ONC-206

靶点

CLPP x D2 receptor x D3 receptor

作用机制

CLPP激动剂 [+2]

在研机构

原研机构

在研适应症

非在研适应症

最高研发阶段临床1期

首次获批国家/地区-

首次获批日期1800-01-20

项与脉络丛肿瘤相关的临床试验

NCT06357377 / Not yet recruiting临床1期

An Open Label, Phase 1b Safety, Dose-finding, Brain Tumor Delivery, and Pharmacokinetics Study of Intranasal NEO100 in Patients With Pediatric-type Select Brain Tumors

This is an open label, Phase 1b safety, dose-finding, brain tumor delivery, and pharmacokinetics study of intranasal NEO100 in patients with pediatric-type diffuse high grade gliomas. Patients will receive IN NEO100 that will follow a dose titration design, followed by a standard dose escalation design to establish safety. Brain tumor delivery of NEO100 will be confirmed in each disease sub-type by surgical resection/needle biopsy only if clinically indicated and scheduled for clinical purposes and testing with residual tissue for NEO100 and the major metabolite of NEO100 (Perillic Acid).

开始日期2024-05-01

申办/合作机构

NeOnc Technologies, Inc.

NCT05934630 / Active, not recruitingN/AIIT

CSF Cell-free Tumor DNA (CSF cfDNA) Liquid Biopsies for Pediatric, Adolescent, and Young Adult Patients With Primary Brain Tumors

Recent advances in technology have allowed for the detection of cell-free DNA (cfDNA). cfDNA is tumor DNA that can be found in the fluid that surrounds the brain and spinal cord (called cerebrospinal fluid or CSF) and in the blood of patients with brain tumors. The detection of cfDNA in blood and CSF is known as a "liquid biopsy" and is non-invasive, meaning it does not require a surgery or biopsy of tumor tissue. Multiple studies in other cancer types have shown that cfDNA can be used for diagnosis, to monitor disease response to treatment, and to understand the genetic changes that occur in brain tumors over time. Study doctors hope that by studying these tests in pediatric brain tumor patients, they will be able to use liquid biopsy in place of tests that have more risks for patients, like surgery.
There is no treatment provided on this study. Patients who have CSF samples taken as part of regular care will be asked to provide extra samples for this study. The study doctor will collect a minimum of one extra tube of CSF (about 1 teaspoon or 5 mL) for this study. If the patients doctor thinks it is safe, up to 2 tubes of CSF (about 4 teaspoons or up to 20 mL) may be collected. CSF will be collected through the indwelling catheter device or through a needle inserted into the lower part of the patient's spine (known as a spinal tap or lumbar puncture).
A required blood sample (about ½ a teaspoon or 2 3 mL) will be collected once at the start of the study. This sample will be used to help determine changes found in the CSF. Blood will be collected from the patient's central line or arm as a part of regular care.
An optional tumor tissue if obtained within 8 weeks of CSF collection will be collected if available. Similarities between changes in the DNA of the tissue that has caused the tumor to form and grow with the cfDNA from CSF will be compared. This will help understand if CSF can be used instead of tumor tissue for diagnosis. Up to 300 people will take part in this study.
This study will use genetic tests that may identify changes in the genes in the CSF. The report of the somatic mutations (the mutations that are found in the tumor only) will become part of the medical record. The results of the cfDNA sequencing will be shared with the patient. The study doctor will discuss what the results mean for the patient and patient's diagnosis and treatment. There will not be any germline sequencing results reported and these will not be disclosed to the patient, patient's clinician or be recorded in patient medical record. Patient may be monitored on this study for up to 5 years.

开始日期2023-07-12

申办/合作机构

National Cancer Institute

[+1]

NCT04994977 / Recruiting临床1期IIT

Intra-Arterial (IA) Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma (ACPP) and Choroid Plexus Carcinoma (CPC) Prior to Second-Look Surgery

This study will test the safety and efficacy of intra-arterial chemotherapy in subjects with newly diagnosed, residual, or recurrent atypical choroid plexus papilloma and choroid plexus carcinoma prior to a second surgery.
It is believed that intra-arterial chemotherapy will be safe and feasible for this population and will result in decreased tumor size, which may further improve the goals of a second-look surgery.

开始日期2023-05-04

申办/合作机构

Weill Medical College of Cornell University

100 项与脉络丛肿瘤相关的临床结果

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100 项与脉络丛肿瘤相关的转化医学

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0 项与脉络丛肿瘤相关的专利（医药）

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3,960

项与脉络丛肿瘤相关的文献（医药）

2024-02-20·Clinical endocrinology

Evaluation of quality of life in children with precocious puberty.

Article

作者: İlkay N Yılmazer ; Sema Nilay Abseyi ; Gizem Şenyazar ; Merih Berberoğlu ; Zeynep Şıklar ; Zehra Aycan

OBJECTIVE:

This study aimed to investigate the quality of life of patients with central precocious puberty (CPP) who required treatment and premature thelarche (PT) followed up without treatment and to compare the groups with and without treatment among themselves and with healthy children.

DESIGN, PATIENTS AND MEASUREMENT:

This study is designed as a case-control study. A total of 193 children including 59 children with CPP, 53 children with PT, 81 healthy children and their parents were included in the study. A questionnaire was applied to evaluate the sociodemographic characteristics that would affect the quality of life. The 'Pediatric Quality of Life Inventory (PedsQL)' was used to assess the quality of life.

RESULTS:

The PedsQL total scale score was 78.10 ± 17.13, 79.35 ± 11.54 and 79.52 ± 14.65, the psychosocial health summary score was 78.86 ± 16.83, 79.40 ± 12.54 and 79.94 ± 14.94 and physical health summary score was 75.81 ± 20.69, 79.41 ± 15.04 and 78.25 ± 17.52 in CPP, PT and control groups, respectively; however, there was no statistical difference (p > .05). In the scale administered to the parents, scores were similar in the three groups. No difference was found between CPP, PT and control groups in terms of sociodemographic data in the study (p > .05).

CONCLUSION:

Unlike previous studies, in this study the effects of sociodemographic characteristics and whether treatment was initiated or not on quality of life were investigated. Although the scale scores of the CPP group were lower than the PT and control group, there was no statistically significant difference, indicating that quality of life was not negatively affected in the CPP group receiving treatment.

2024-02-17·Journal of endocrinological investigation

Novel variants ensued genomic imprinting in familial central precocious puberty.

Article

作者: V Karaman ; E Karakilic-Ozturan ; S Poyrazoglu ; M Y Gelmez ; F Bas ; F Darendeliler ; Z O Uyguner

INTRODUCTION:

Central precocious puberty (CPP) is characterized by the early onset of puberty and is associated with the critical processes involved in the pubertal switch. The puberty-related gene pool in the human genome is considerably large though few have been described in CPP. Within those genes, the genomic imprinting features of the MKRN3 and DLK1 genes add additional complexity to the understanding of the pathologic pathways. This study aimed to investigate the molecular etiology in the CPP cohort.

METHODS:

Eighteen familial CPP cases were investigated by Sanger sequencing for five CPP-related genes; DLK1, KISS1, KISS1R, MKRN3, and PROKR2. Segregation analysis was performed in all patients with pathogenic variants. Using an ELISA test, the functional pathogenicity of novel variants was also investigated in conjunction with serum delta-like 1 homolog (DLK1) concentrations.

RESULTS:

In three probands, a known variant in the MKRN3 gene (c.982C>T/p.(Arg328Cys)) and two novel variants in the DLK1 gene (c.357C>G/p.(Tyr119Ter) and c.67+78C>T) were identified. All three were inherited from the paternal allele. The individuals carrying the DLK1 variants had low detectable DLK1 levels in their serum.

CONCLUSIONS:

The frequencies were 5.5% (1/18) for MKRN3 11% (2/18) for DLK1, and none for either KISS1, KISS1R, and PROKR2. Low serum DLK1 levels in affected individuals supported the relationship between here described novel DLK1 gene variants with CPP. Nonsense nature of c.357C>G/p.(Tyr119Ter) and an alteration in the evolutionarily conserved nucleotide c.67+78C>T suggested the disruptive nature of the variant's compatibility with CPP.

2024-02-15·Endocrine

Polymorphism rs5780218, rs12998 and rs10158616 in KISS1 gene among the Hubei province Chinese girls with the central precocious puberty.

Article

作者: Lijuan Dong ; Weiguang Zhou ; Zhongchao Lin ; Lingyu Tang ; Xinyi Deng ; Bifeng Chen ; Wei Huang ; Qiantao Xiong

PURPOSE:

Central precocious puberty (CPP) results from early activation of the hypothalamic-pituitary-gonadal (HPG) axis. To elucidate the molecular genetic basis of CPP, we here investigated the effects of polymorphism rs5780218, rs12998 and rs10158616 in KISS1 gene on CPP susceptibility.

METHODS:

The three KISS1 gene polymorphisms were genotyped by the polymerase chain reaction-restriction fragment length polymorphism (PCR-RFLP) and Sanger sequencing in 422 healthy Hubei Chinese girls and 384 Hubei Chinese girls with CPP.

RESULTS:

Single-locus analysis demonstrated that rs5780218 and rs12998 were significantly associated with CPP susceptibility in Hubei Chinese girls. Haplotype analysis exhibited that the AGG carrying the risk allele A of rs5780218 and the -GG carrying the protective allele - of rs5780218 were associated with increased and decreased CPP susceptibility in Hubei Chinese girls, respectively. The following meta-analysis confirmed the contribution of rs5780218 and rs12998 on CPP susceptibility in Chinese girls.

CONCLUSIONS:

rs5780218 and rs12998 in the KISS1 gene may participate in genetic susceptibility to CPP in Chinese girls, and the KISS1 gene rs5780218 may serve as a genetic biomarker of CPP. However, the present findings should be validated in future studies with larger sample sizes in other ethnic populations.

项与脉络丛肿瘤相关的新闻（医药）

2024-04-01

·PRNewswire

Foresee Pharmaceuticals Receives Positive Recommendation from DSMB to Continue the Casppian Study, a Phase 3 Clinical Trial of Leuprolide (FP-001) Injectable Emulsion for the Treatment of Central Precocious Puberty

The independent Data and Safety Monitoring Board (DSMB) recommended the continuation of patient enrollment as planned. There has been no safety concern with the use of Leuprolide (FP-001) injectable Emulsion in pediatric CPP patients. TAIPEI, April 1, 2024 /PRNewswire/ -- Foresee Pharmaceuticals (TPEx: 6576), ("Foresee") today announces the positive safety review by the independent Data and Safety Monitoring Board for its Casppian Phase 3 registration study. The DSMB recommended that Foresee Pharmaceuticals continue the trial with a minor amended protocol. The Casppian Phase 3 study is an open-label, multicenter, multinational clinical trial to evaluate the efficacy, safety, and pharmacokinetics of Leuprolide (FP-001) 42 mg-controlled release in patients with central (Gonadotropin-Dependent) precocious puberty (CPP). Foresee's Leuprolide injectable emulsion, 42 mg (marketed under the brand name CAMCEVI®), is currently approved for the treatment of adult patients with advanced prostate cancer and has demonstrated positive efficacy and safety results with statistically significant treatment effects in adult male patients with advanced prostate cancer. "We are pleased to receive the positive recommendation from the DSMB to continue our clinical trial. Ensuring the safety of the children participating in our trial is paramount and we continue to be dedicated to maintaining the highest standards of safety and ethical conduct throughout this clinical trial. The top-line results from this trial are anticipated in 2025.", said Bassem Elmankabadi, M.D., Senior Vice President of Clinical Development at Foresee Pharmaceuticals. "Foresee's Leuprolide 42 mg formulation (FP-001) dosed subcutaneously every 6 months, has the potential to become the only ready-to-use Leuprolide 6-month long-acting injection on the market, providing benefit to stakeholders in the CPP community. This positive recommendation brings us closer to our goal of delivering safe and effective treatment for children in need," said Dr. Ben Chien, Foresee's Chairman and CEO. "We look forward to establishing commercial partnership soon for the potential future commercialization of FP-001, in CPP indication." About CPP GnRH-dependent CPP is a condition that causes early sexual development in girls and boys, as their "hypothalamus - pituitary gland - gonadal axis" is activated too early, causing children to enter puberty prematurely, between 2 years and 9 years of age. CPP patients are at risk of having significantly short stature as adults in addition to social, psychological, and emotional issues, including lower self-esteem, stress, anxiety, and depression, which may negatively impact their quality of life. According to the NORD (National Organization for Rare Disorders) website, CPP occurs in 1 out of 5,000 to 10,000 children. It is estimated that approximately 80% - 90% of CPP cases are idiopathic, especially in females, with a female-to-male ratio of around 20. Gonadotropin-releasing hormone (GnRH) agonists, including leuprolide, are the most widely used treatment for CPP. About Foresee Pharmaceuticals Co., Ltd. Foresee is a Taiwan and US-based biopharmaceutical company listed on the Taipei Exchange (TPEx: 6576). Foresee's R&D efforts are focused on two key areas, namely its unique Stabilized Injectable Formulation (SIF) long-acting injectable technology with derived drug products targeting specialty markets and secondly, its transformative preclinical and clinical first-in-class NCE programs targeting rare and severe disease areas with high unmet needs. Foresee's product portfolio includes late and early-stage programs. CAMCEVI® 42 mg, for the treatment of advanced prostate cancer, is now approved in the U.S., Canada, EU, and Taiwan and launched in the U.S. in April 2022. Additionally, U.S. and EU regulatory submissions are being prepared for CAMCEVI® 21 mg. The second indication of CAMCEVI® 42 mg – central precocious puberty (CPP), the Casppian phase 3 clinical study, has been initiated. FP-025 – a highly selective oral MMP-12 inhibitor targeting inflammatory and fibrotic diseases, a Phase 2 proof-of-concept study in allergic asthmatic patients has been completed with positive outcomes, with future development in rare immune-fibrotic diseases. FP-020, a follow-on oral MMP-12 inhibitor currently in Phase 1, with development targeted in severe asthma and COPD. FP-045 – a highly selective oral small molecule allosteric activator of ALDH2, a mitochondrial enzyme, for which the FuschiA Phase 1b/2 Fanconi Anemia study is currently being initiated, and a Phase 2 study in pulmonary hypertension-interstitial lung disease (PH-ILD) patients is in planning. SOURCE Foresee Pharmaceuticals Co., Ltd.

临床3期临床1期临床2期上市批准临床结果

2023-10-18

·信达生物

【学术前沿】信迪利单抗发挥在儿童肿瘤免疫治疗价值，中山大学肿瘤防治中心最新研究成果在国际期刊发表

转载自：中山大学肿瘤防治中心儿童肿瘤科编者按目前，儿童肿瘤患者的标准治疗主要包括手术、化疗和放疗。令人遗憾的是，对于儿童实体瘤的预后仍然不容乐观，PD-1等新型免疫检查点抑制剂在儿童肿瘤的应用值得探索。国家一类创新药信迪利单抗率先针对儿童晚期及难治肿瘤进行了积极的尝试，探索儿童肿瘤免疫治疗的潜力。中山大学肿瘤防治中心张翼鷟教授团队在该方面的研究结果现已刊发国际权威期刊《Signal Transduction and Targeted Therapy》，研究显示，信迪利单抗不论是作为单一药物疗法还是与其他药物联合治疗的一部分，都为儿童肿瘤治疗提供了初步依据。近日，中山大学肿瘤防治中心张翼鷟教授团队在国际权威期刊《Signal Transduction and Targeted Therapy》（IF：39.3）在线发表了题为“信迪利单抗在儿童晚期或复发难治肿瘤中安全性及有效性的I期临床试验”的研究结果。儿童肿瘤患者的标准治疗主要包括手术、化疗和放疗。令人遗憾的是，对于儿童肿瘤患者中复发或晚期实体肿瘤的预后仍然不容乐观，免疫检查点抑制剂等新型治疗在儿童肿瘤的应用值得探索。KEYNOTE-051和ADVL1412研究PD1抗体在儿童肿瘤中的疗效，但在亚洲儿童人群中，PD-1抗体的前瞻性研究非常有限。Pembrolizumab和Nivolumab等药物因为费用高昂阻碍了它们在国内的广泛应用，导致儿童肿瘤领域国内PD-1抗体数据的巨大空白。本研究首次探索了国产PD-1抗体信迪利单抗治疗儿童晚期及难治复发的肿瘤的安全性和有效性。研究纳入了29名受试者，分为三个递增剂量的队列：1 mg/kg、3 mg/kg和10 mg/kg。主要的研究目标包括确定剂量限制性毒性并评估治疗的安全性和耐受性。次要研究终点包括客观反应率（ORR）、无进展生存期（PFS）以及总生存期（OS）。受试者的临床病理类型包括除了霍奇金淋巴瘤以外，也包括一些罕见的儿童肿瘤病理亚型，例如原发性纵隔大B细胞淋巴瘤， NK/T细胞淋巴瘤，恶性间皮瘤，间叶原性恶性肿瘤，间变室管膜瘤，上皮样肉瘤，胸腺瘤，卵黄囊瘤，非典型脉络丛乳头状瘤等。安全性方面，信迪利单抗耐受性表现良好，29名患者中有89.7%患者至少出现一种治疗相关不良事件。包括：贫血（34.5%）；疲劳（6.9%）；甲状腺功能减退（17.2%）；甲状腺功能亢进（13.8%）等。3名患者（10.3%）出现了三级以上的治疗相关不良反应，包括转移酶升高和肌炎，以及血小板计数降低。安全数据没有显示出剂量（1-10 mg/kg）相关的特征。未发生治疗相关的严重不良事件（SAEs）。疗效方面，霍奇金淋巴瘤患者中，总体ORR和DCR分别为60.0%和100%，20%患者最佳疗效为CR。在除霍奇金淋巴瘤以外的肿瘤中观察到2例CR（NK/T细胞淋巴瘤和间变室管膜瘤），3例PR（包括1例弥漫性大B细胞淋巴瘤、1例尤文肉瘤和1例胸腺瘤）3例SD (包括1例上皮样肉瘤、1例非典型脉络丛乳头状瘤和1例原发性纵隔大B细胞淋巴瘤）ORR 为29.4%，DCR 为47.1%。另外，对患儿的肿瘤标本进行外显子测序结果发现G 蛋白偶联受体 (GPCR) 是参与信号传递的最大的细胞膜相关分子基因家族，与治疗抵抗和免疫逃避有关，结果表明对免疫检查点抑制剂的无反应与 GPCR 途径的激活有关。使用WES对受试者的FFPE肿瘤和匹配的外周血样本进行基因组分析综上，研究结果显示信迪利单抗在儿童中的耐受性良好。虽然在这项研究中，信迪利单抗在大多数儿童实体瘤中并未表现出明显的单药疗效，但观察到了对霍奇金淋巴瘤的抗肿瘤活性。这一研究为未来进一步探讨PD-1/PD-L1抑制剂在儿童肿瘤治疗中的潜力，不论是作为单一药物疗法还是与其他药物联合治疗的一部分，提供了初步依据。中山大学肿瘤防治中心张翼鷟教授、路素英副教授为本研究的共同通讯作者。中山大学肿瘤防治中心阙旖医师、王娟主治医师，孙斐斐主治医师以及重庆医科大学附属儿童医院王珊教授是本研究的共同第一作者。文献：Yi Que, Juan Wang, Feifei Sun, Shan Wang, Jia Zhu, Junting Huang, Zhenzhen Zhao, Li Zhang, Juan Liu1, Jiaqian Xu,Zijun Zhen, Xiaofei Sun, Suying Lu1 and Yizhuo Zhang. Safety and clinical efficacy of sintilimab (anti-PD-1) in pediatric patients with advanced or recurrent malignancies in a phase I study. Signal Transduction and Targeted Therapy (2023) 8:392 . https://doi.org/10.1038/s41392-023-01636-9.点击蓝字阅读原文

免疫疗法临床结果

2023-10-17

·生物谷

中山大学张翼鷟团队发布首个国产PD-1免疫治疗儿童肿瘤临床数据

这项1期临床研究结果显示信迪利单抗在儿童中的耐受性良好。虽然在这项研究中，信迪利单抗在大多数儿童实体瘤中并未表现出明显的单药疗效，但观察到了对霍奇金淋巴瘤的抗肿瘤活性。中山大学肿瘤防治中心张翼鷟教授团队在 Signal Transduction and Targeted Therapy 期刊发表了题为：Safety and clinical efficacy of sintilimab (anti-PD-1) in pediatric patients with advanced or recurrent malignancies in a phase I study（信迪利单抗在儿童晚期或复发难治肿瘤中安全性及有效性的I期临床试验）的研究论文。该研究首次探索了国产PD-1抗体信迪利单抗（Sintilimab）治疗儿童晚期及难治复发的肿瘤的安全性和有效性。该研究纳入了29名受试者，分为三个递增剂量的队列：1 mg/kg、3 mg/kg和10 mg/kg。主要的研究目标包括确定剂量限制性毒性并评估治疗的安全性和耐受性。次要研究终点包括客观反应率（ORR）、无进展生存期（PFS）以及总生存期（OS）。受试者的临床病理类型包括除了霍奇金淋巴瘤以外，也包括一些罕见的儿童肿瘤病理亚型，例如原发性纵隔大B细胞淋巴瘤， NK/T细胞淋巴瘤，恶性间皮瘤,，间叶原性恶性肿瘤，间变室管膜瘤，上皮样肉瘤，胸腺瘤，卵黄囊瘤，非典型脉络丛乳头状瘤等。安全性方面，信迪利单抗耐受性表现良好，29名患者中有89.7%患者至少出现一种治疗相关不良事件。包括：贫血（34.5%）；疲劳（6.9%）；甲状腺功能减退（17.2%）；甲状腺功能亢进（13.8%）等。3名患者（10.3%）出现了三级治疗相关不良反应，包括转移酶升高和肌炎，以及血小板计数降低。安全数据没有显示出剂量（1-10 mg/kg）相关的特征。未发生治疗相关的严重不良事件（SAEs）。疗效方面，霍奇金淋巴瘤患者中，总体ORR和DCR分别为60.0%和100%，20%患者最佳疗效为CR。在除霍奇金淋巴瘤以外的肿瘤中观察到2例CR（NK/T细胞淋巴瘤和间变室管膜瘤），3例PR（包括1例弥漫性大B细胞淋巴瘤、1例尤文肉瘤和1例胸腺瘤），3例SD（包括1例上皮样肉瘤、1例非典型脉络丛乳头状瘤和1例原发性纵隔大B细胞淋巴瘤）ORR 为29.4%，DCR 为47.1%。另外，对患儿的肿瘤标本进行外显子测序结果发现G蛋白偶联受体（GPCR）是参与信号传递的最大的细胞膜相关分子基因家族，与治疗抵抗和免疫逃避有关，结果表明对免疫检查点抑制剂的无反应与GPCR通路的激活有关。使用全外显子测序对受试者的FFPE肿瘤和匹配的外周血样本进行基因组分析综上，这项1期临床研究结果显示信迪利单抗在儿童中的耐受性良好。虽然在这项研究中，信迪利单抗在大多数儿童实体瘤中并未表现出明显的单药疗效，但观察到了对霍奇金淋巴瘤的抗肿瘤活性。这一研究为未来进一步探讨PD-1/PD-L1抑制剂在儿童肿瘤治疗中的潜力，不论是作为单一药物疗法还是与其他药物联合治疗的一部分，提供了初步依据。