Gene transfer study in patients with late onset Pompe diseaseTrial ID 7517-CL-1101

试验摘要

The purpose of this study is to evaluate the safety, tolerability, and clinical response of ASP7517, and determine the Recommended Phase 2 Dose (RP2D) and/or the Maximum Tolerated Dose (MTD) of ASP7517 when administered as a single agent and in combination with pembrolizumab.

This study will also evaluate other measures of anticancer activity of ASP7517 when administered as a single agent and in combination with pembrolizumab based on central and local assessment.

谁可以参加

Every clinical study has its own set of rules for who can join, known as eligibility criteria. These might cover things like your age, gender, general health, the type and stage of your condition, and your past treatments. But, only the study team can decide if you’re a good fit to take part.

预期结果

If the study is right for you, and you’d like to join, you will be randomly assigned to receive either one of the study medications or a placebo. In this study, 4 out of 5 people will receive a study medicine and 1 out of 5 people will receive a placebo. A placebo does not have any medicine in it but looks just like the medicine being studied. Whilst you are free to leave the study at any time, staying in a study until the end really helps scientists and doctors to discover new treatments for people living with eczema.

研究治疗持续时间

10 Months

研究访问次数

Approximately 13

地点

未找到任何位置。

试验目的

In a world where innovation meets care, we invite you to explore the possibilities of groundbreaking treatments. Imagine a scenario where you could be part of a journey that transforms lives.

Picture this: 80% of participants will receive a revolutionary treatment, while 20% will experience a placebo that mirrors the real thing. Your involvement could pave the way for new discoveries in eczema care. Remember, you can opt out at any moment, but your commitment until the end can significantly contribute to the advancement of medical science.

研究文件

Plain Language Summary

可用语言: English

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Protocol

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Protocol and Statistical Analysis Plan

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Clinical Study Report

可用语言: English

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Press Release

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关于正在招募参与者的 Study of ASP7517 Alone and with Pembrolizumab in Participants with Advanced Solid Tumors Expressing WT1 Antigen，您是否想了解更多临床试验中心相关信息？请在右侧填写您的信息联系我们，我们将予以回复。

试验信息

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参与者年龄

18 岁及以上

疾病

晚期/转移性肿瘤

参与者性别

女性和男性

试验开展时间

2021年7月 - 2027年11月

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入选标准

Participant has locally-advanced (unresectable) or metastatic solid tumor malignancy that is confirmed by available pathology records or current biopsy. Participant must also have received all standard therapies (unless the therapy is contraindicated or intolerable) appropriate to provide clinical benefit for his/her specific tumor type. However, participants with metastatic melanoma who have not received checkpoint inhibitors [CPIs] (i.e., CPIs naive) may enroll in the Phase 2 Combination Therapy Arm Dose Expansion Cohort to receive CPI: Pembrolizumab.
Participant must be diagnosed with solid tumor known to express WT1 antigen such as, but not limited to melanoma, ovarian cancer or Colorectal Cancer (CRC).
Participant consents to provide an archival tumor specimen in a tissue block or unstained serial slides, if available, prior to study treatment.
Participant has an Eastern Cooperative Oncology Group (ECOG) Performance Status of ≤ 2.
Participant’s last dose of prior antineoplastic therapy, including any immunotherapy, was 21 days or 5 half-lives, whichever is shorter, prior to initiation of IP administration. A participant with BRAF gene, epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) mutation positive non-small cell lung carcinoma is allowed to remain on epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) or anaplastic lymphoma kinase (ALK) or BRAF inhibitor therapy until 4 days prior to the start of Investigational Product (IP) administration.
Participant has completed any radiotherapy (including stereotactic radiosurgery) at least 2 weeks prior to IP administration.
Participant’s Adverse Events (AEs) (excluding alopecia) from prior therapy have improved to grade 1 or baseline within 14 days prior to start of IP.
Participant has adequate organ function prior to start of IP. If a participant has received a recent blood transfusion, the laboratory tests must be obtained ≥ 4 weeks after any blood transfusion.
A female participant is eligible to participate if she is not pregnant and at least 1 of the following conditions applies:
- 非有生育能力的女性 (WOCBP) 或者
- WOCBP who agrees to follow the contraceptive guidance from the time of informed consent through at least 6 months after the final IP administration.
Female participant must agree not to breastfeed starting at screening and throughout the IP and for 180 days after the final IP administration.
Female participant must not donate ova starting at screening and throughout the IP and for 180 days after the final IP administration.
A male participant with female partner(s) of childbearing potential (including breastfeeding partner) must agree to use contraception throughout the treatment period and for at least 180 days after the final IP administration.
Male participant must not donate sperm during the treatment period and for 180 days after the final IP administration.
Male participant with pregnant partner(s) must agree to remain abstinent or use a condom for the duration of the pregnancy throughout the study period and for 180 days after the final IP administration.
Participant agrees not to participate in another interventional study while receiving IP.

Additional Inclusion Criteria for Participants in the Expansion Cohorts:

Participant meets one of the following:
- Participant has the tumor type for which a confirmed response was observed in a monotherapy or combination therapy dose escalation (for melanoma, ovarian cancer and Colorectal Cancer [CRC] only) cohort; OR
- For tumor specific expansion cohorts of ASP7517 or ASP7517 with pembrolizumab, participant has the applicable tumor type melanoma, ovarian cancer, CRC.
根据 RECIST v1.1，受试者至少有 1 个可测量病灶。如果已证实既往放疗部位的病灶出现进展，将该病灶视为可测量病灶。
Participant consents to provide a tumor specimen in a tissue block or unstained serial slides obtained within 56 days prior to first dose of IP. If a recent tissue sample cannot be provided due to medical or safety concerns, enrollment into the study must be discussed with the medical monitor.
Participant consents to undergoing a tumor biopsy (core tissue biopsy or excision) during the treatment period as indicated in the schedule of assessments.

排除标准

Participant weighs < 45 kg at screening.
Participant has received investigational therapy (other than an investigational EGFR TKI in a participant with EGFR activating mutations or ALK or BRAF inhibitor in a participant with an ALK mutation) within 21 days or 5 half-lives, whichever is shorter, prior to start of IP.
Participant requires or has received systemic steroid therapy or any other immunosuppressive therapy within 14 days prior to Cycle 1 Day 1. Participants using a physiologic replacement dose of hydrocortisone or its equivalent (defined as up to 30 mg per day of hydrocortisone up to 10 mg per day of prednisone) are allowed.
受试者出现症状性中枢神经系统 (CNS) 转移或受试者存在不稳定 CNS 转移的证据，即使无症状（例如，通过扫描发现进展）。Participants with previously treated CNS metastases are eligible, if they are clinically stable and have no evidence of CNS progression by imaging for at least 4 weeks prior to start of IP and are not requiring immunosuppressive doses of systemic steroids (> 30 mg per day of hydrocortisone or > 10 mg per day of prednisone or equivalent) for longer than 2 weeks.
Participant has an active autoimmune disease. Participants with type 1 diabetes mellitus, endocrinopathies stably maintained on appropriate replacement therapy or skin disorders (e.g., vitiligo, psoriasis or alopecia) not requiring systemic treatment are allowed.
Participant was discontinued from prior immunomodulatory therapy due to a grade ≥ 3 toxicity that was mechanistically related (e.g., immune related) to the agent.
Participant has known history of serious hypersensitivity reaction to a known ingredient of ASP7517 or pembrolizumab or severe hypersensitivity reaction to treatment with another monoclonal antibody.
Participant has a known history of human immunodeficiency virus.
Participant with known history of positive hepatitis B surface antigen or isolated hepatitis B core antibody (including acute HBV or chronic HBV) or hepatitis C ([HCV] ribonucleic acid [RNA] detected by qualitative assay). Hepatitis C RNA testing is not required in participants with negative hepatitis C antibody testing.
Participant has received a live vaccine against infectious diseases within 28 days prior to initiation of IP.
Participant has a history of drug-induced pneumonitis (interstitial lung disease), a history of (non-infectious) pneumonitis that required steroids, radiation pneumonitis or currently has pneumonitis.
Participant has an infection requiring systemic therapy within 14 days prior to IP.
Participant has received a prior allogeneic bone marrow or solid organ transplant.
Participant is expected to require another form of antineoplastic therapy while on IP.
Participant has had a myocardial infarction or unstable angina within 6 months prior to the start of IP or currently has an uncontrolled illness including, but not limited to symptomatic congestive heart failure, clinically significant cardiac disease, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
Participant has a clinically significant abnormal electrocardiogram at screening.
Participant has symptomatic cardiovascular disease within the preceding 12 months unless cardiology consultation and clearance has been obtained for study participation, including but not limited to the following: significant coronary artery disease (e.g., requiring angioplasty or stenting), acute myocardial infarction or unstable angina pectoris < 3 months prior screening, uncontrolled hypertension, clinically significant arrhythmia or congestive heart failure (New York Heart Association grade ≥ 3).
Any condition that makes the participant unsuitable for study participation.
Participant has had a major surgical procedure and has not completely recovered within 28 days prior to the start of IP.
Participant has a prior malignancy active (i.e., requiring treatment of intervention) within the previous 2 years prior to the screening visit, except for locally curable malignancies that have been apparently cured, such as basal or squamous cell skin cancer, superficial bladder cancer or carcinoma in situ of the cervix or breast. Participants with organ confined prostate cancer with no evidence of recurrent or progressive disease are eligible if hormonal therapy has been initiated or the malignancy has been surgically removed or treated with definitive radiotherapy.
Participant has International Normalized Ration (INR) > 1.5 x Upper Limits of Normal (ULN) and/or activated partial thromboplastin time (aPTT) > 1.5 x institutional normal limits.

Additional Exclusion Criteria for Participants in Combination Expansion Cohorts:

Participants with metastatic CRC with documented microsatellite instability-high (MSI-H) or mismatch repair (MMR) deficient who have received prior treatment with PD-1 or programmed death-ligand (PD-L1) inhibitors such as nivolumab or pembrolizumab.
CPI naïve metastatic melanoma participants who have received PD-1 or PD-L1 inhibitors, such as nivolumab or pembrolizumab.
Participants with metastatic ovarian cancer with documented MSI-H or MMR deficient who have received PD-1or PD-L1 inhibitors, such as nivolumab or pembrolizumab.

常见问题

临床试验是否只针对晚期癌症患者？

虽然某些临床试验可能侧重于更晚期的癌症，但许多试验对处于不同癌症阶段的患者开放。每项研究都有关于参与资格的规定。例如，只有特定年龄段的患者或患有特定类型肿瘤的患者才能参与。

我是否需要停止当前治疗才能参加临床试验？

有时，研究人员希望参与者在临床试验期间继续接受当前治疗。有时，您可能需要暂停当前的治疗。如果研究性治疗无效，您通常可以恢复原有的治疗方案。

我是否应该担心会服用安慰剂？

在癌症临床试验中，只有当该类癌症尚无其他治疗方法时，才会使用安慰剂。这有助于将研究性治疗与安慰剂进行比较。安慰剂在癌症试验中很少使用，因为通常会采用最佳可用疗法，即所谓的“标准治疗方案”。

我是否需要医生转诊才能参加一项研究？

您的医生可能并不了解所有可供您参与的临床试验机会。请与您的医生或其他医疗服务提供者讨论您所找到的临床试验信息。他们可以帮助您判断临床试验是否适合您。If you do not find any options on this website, we recommend you visit an online public registry website like clinicaltrials.gov to see a wide variety of available clinical trials.

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Gene transfer study in patients with late onset Pompe disease

试验摘要

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谁可以参加

预期结果

地点

试验目的

研究文件

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