Appendix Cancer, Pseudomyxoma Peritonei
Conditions
Keywords
Appendix Cancer, Appendiceal Cancer, Appendiceal Neoplasm, Appendiceal Neoplasms, Appendiceal Adenocarcinoma, Pseudomyxoma Peritonei, PMP, Peritoneal Surface Malignancy, Peritoneal Surface Malignancies, Patient Registry, Rare Cancer, Natural History Study, Natural History, Goblet Cell, Signet Ring Cell, LAMN, Low-Grade Appendiceal Mucinous Neoplasm, High-Grade Appendiceal Mucinous Neoplasm, HAMN
Brief summary
ACPMP's first-ever patient-powered global patient registry/natural history study collects patient and caregiver information and data to fuel clinical trials and drive breakthroughs for appendix cancer and PMP. We've invested the time and resources to make our Registry regulatory grade. Our Registry operates under active, ongoing Institutional Review Board (IRB) oversight by independent ethicists. It is powered by NORD's IAMRARE®, an industry gold standard platform built to be consistent with FDA's best thinking on natural history studies.
Detailed description
The ACPMP Research Foundation Appendix Cancer/PMP Patient Registry is a global, patient-powered natural history study designed to systematically collect longitudinal information from individuals affected by appendix cancer and Pseudomyxoma Peritonei (PMP). The primary aim of the Registry is to improve understanding of these rare diseases, including their characteristics, clinical course, treatment patterns, and progression over time. Information collected through the Registry will help characterize the population affected by appendix cancer and PMP, support the development of recommendations and standards of care, and provide a resource for researchers studying disease biology, treatment outcomes, and accelerate the discovery of new treatments. Participants, caregivers, and other authorized representatives provide information through a series of online surveys that include a prompt for patients to upload pathology reports, genomic sequencing, ctDNA reports and other relevant medical documents. Retrospective and prospective data collected include sociodemographic information, medical and diagnostic history, treatments, disease progression, management of care, and quality of life. Participants are asked to return periodically to update their information, allowing changes in disease and patient experience to be evaluated over time. The Registry also serves as a resource to support future research and clinical trial development. With appropriate review and approval, Registry data may be used by researchers conducting retrospective studies or designing prospective studies of novel treatments. The Registry may also facilitate communication with participants about research opportunities and clinical trials for which they may be eligible. The Registry is sponsored and managed by the Appendix Cancer Pseudomyxoma Peritonei (ACPMP) Research Foundation and is hosted on the National Organization for Rare Disorders (NORD) IAMRARE® platform. The study is conducted under Institutional Review Board (IRB) oversight and follows established procedures for the collection, management, protection, and appropriate research use of participant data.
Interventions
None listed
Sponsors
Appendix Cancer Pseudomyxoma Peritonei Research Foundation
National Organization for Rare Disorders
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Diagnosis of appendix cancer. * Meets the study inclusion criteria for participation. * Participant or, when applicable, a legally authorized representative (LAR) is able to provide informed consent and enter information into the Registry. * A designated representative may provide retrospective information for an individual who died from appendix cancer, as permitted by the study protocol.
Exclusion criteria
* Individuals who do not meet the study inclusion criteria. * Individuals for whom required informed consent cannot be obtained.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Natural history and clinical course of appendix cancer and Pseudomyxoma Peritonei (PMP) | Up to 5 years | Characterization of the natural history and clinical course of appendix cancer and Pseudomyxoma Peritonei (PMP) using participant- and caregiver-reported data collected through the ACPMP Patient Registry. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Disease Characteristics | Up to 5 years | Disease characteristics of appendix cancer and pseudomyxoma peritonei (PMP), including disease type, extent, and other participant-reported disease characteristics. |
| Treatment History | Up to 5 years | Cancer treatment history, including surgical procedures, chemotherapy, and other treatments received for appendix cancer and PMP. |
| Diagnosis | Up to 5 years | Characteristics and timing of diagnosis of appendix cancer and PMP, including initial diagnosis and diagnostic findings. |
| Disease Progression | Up to 5 years | Disease progression over time, based on participant- and caregiver-reported information regarding changes in disease status. |
| Management of Care | Up to 5 years | Changes in management of care for appendix cancer and PMP, including treatments, procedures, and changes in care over time. |
| Signs and Symptoms | Up to 5 years | Changes in signs and symptoms associated with appendix cancer and PMP over time, based on participant- and caregiver-reported data. |
Countries
United States
Contacts
CONTACTMargaret Najarian
PRINCIPAL_INVESTIGATORDeborah Shelton
ACPMP
STUDY_CHAIRDr. Erin P. Ward, MD
University of Utah
Outcome results
None listed