International Pleuropulmonary Blastoma (PPB) Treatment and Biology Registry Protocol
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 156
- 试验地点
- 1
- 主要终点
- Event-free survival
研究概览
简要总结
Pleuropulmonary Blastoma (PPB) is very rare and there is no established "standard" or "best" therapy. For many years, children with PPB around the world have been treated according to decisions made case-by-case in many different hospitals by many different physicians. No treatment has been tested in a large group of PPB patients.
The goal is to treat many children with one treatment program and to learn the results of the treatment.
详细描述
This Registry will collect family medical history and establishes a collection of specimens from PPB patients and their parents for future research. Primary goals of the biology research are (1) to identify new scientific ways to learn about PPB and the best way to treat it in the future and (2) to establish a collection of PPB biologic specimens for future research.
Background: PPB is a rare cancer of the lung presenting in early childhood, mostly from birth to age ~72 months. PPB occurs within the lung or between the lung and the chest wall. There are three forms of PPB called Types I, II, and III PPB. Children with Type I PPB require surgery and sometimes chemotherapy. Children with Types II and III PPB, require surgery, chemotherapy and sometimes radiation therapy. Because there has never been a consensus as to the best treatment for PPB, the Registry will provide treatment guidelines. It is the goal of the Treatment and Biology Registry that by offering specific regimens the Registry will be able to prospectively review and analyze the regimen for the first time.
Design:
- Diagnostic central pathology review will be required for patients who will be prospectively evaluated.
- This Treatment Registry will offer surgery and chemotherapy regimen guidelines, and optional radiation therapy guidelines for prospectively enrolled patients.
- The Treatment and Biology Registry will create a PPB biologic specimens reference bank for tumor.
Methods:
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Health Services Research
- 盲法
- None
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age : Patients from birth to < 21 years of age at the time of diagnosis will be included in the Treatment and Biology Registry.
- •Patients of any age will be included in the Associated Diseases arm of this study.
- •Pathology Diagnosis: Patients with newly-diagnosed PPB Types I, II or III. Diagnosis is made by the local pathologist. Real-time central pathology review is encouraged but is not required. All cases must be submitted for central pathology review. Only centrally-reviewed cases confirmed as PPB will be analyzed prospectively.
- •Cases in which the initial diagnosis is "suggestive" or "supportive" of PPB, but not diagnostic, and in which later resection specimens, including resections following chemotherapy, confirm a PPB diagnosis will be included. Patients diagnosed by fine needle aspiration biopsy will be included only if a later resection specimen, including resections following chemotherapy, is diagnostic of PPB.
- •Diagnostic pathology for cases of diseases associated with PPB will also require registry central pathology review.
- •Prior Therapy: PPB Type I: All patients are eligible and will be followed in the study.
- •PPB Types II or III: Newly-diagnosed Types II and III PPB patients will be included in the Treatment and Biology Registry.
- •DICER1-related condition and DICER1 gene mutation: all patients are eligible and will be followed in the study.
- •Prior corticosteroid therapy is allowed.
- •Patients who have received other chemotherapy regimens or radiation therapy will not be statistical analysis.
- •Types II and III PPB patients with PRIOR Type I PPB diagnosis: Types II and III PPB cases which are recurrences of an earlier Type I PPB are included.
- •Informed consent by patient or parent/guardian. (also, where appropriate: assent and HIPPA consent)
排除标准
- •Inability of patient, or parent/guardian to obtain informed consent.
- •Patients who have their PPB diagnosed ruled out by Registry central pathology review.
研究组 & 干预措施
Type I PPB therapy
PPB Type I therapy: All patients will be treated with surgery. Chemotherapy after surgery is per the treating physician(s) discretion. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with Vincristine, Dactinomycin, Cyclophosphamide (VAC).
干预措施: Vincristine (Drug)
Type I PPB therapy
PPB Type I therapy: All patients will be treated with surgery. Chemotherapy after surgery is per the treating physician(s) discretion. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with Vincristine, Dactinomycin, Cyclophosphamide (VAC).
干预措施: Dactinomycin (Drug)
Type I PPB therapy
PPB Type I therapy: All patients will be treated with surgery. Chemotherapy after surgery is per the treating physician(s) discretion. If chemotherapy is used the Registry will suggest that it be combination chemotherapy with Vincristine, Dactinomycin, Cyclophosphamide (VAC).
干预措施: Cyclophosphamide (Drug)
Types II and III PPB therapy
Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.
干预措施: Vincristine (Drug)
Types II and III PPB therapy
Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.
干预措施: Dactinomycin (Drug)
Types II and III PPB therapy
Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.
干预措施: Cyclophosphamide (Drug)
Types II and III PPB therapy
Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.
干预措施: Ifosfamide (Drug)
Types II and III PPB therapy
Combination chemotherapy with Ifosfamide, Vincristine, Dactinomycin and Doxorubicin ("IVADo"). Second look and possible 3rd look surgery may be required. Radiation therapy is recommended only for residual disease after maximum surgery.
干预措施: Doxorubicin (Drug)
结局指标
主要结局
Event-free survival
时间窗: 5 years
The primary endpoint for statistical analysis will be time from start treatment to an event, defined as the occurrence of progression or recurrence of PPB, occurrence of a second malignant neoplasm, or death from any cause that is at least possibly related to the original disease or treatment.
次要结局
- Overall response to chemotherapy, and survival(5 years)
研究者
Kris Ann Schultz
M.D.
Children's Hospitals and Clinics of Minnesota