Clinical Trials

Clinical trials currently enrolling patients with appendix cancer and/or pseudomyxoma peritonei from appendiceal origin are provided below. (Updated 11/17/21)

The ACPMP Research Foundation attempts to keep this list as updated and complete as possible.  Please note, however, that the primary public sources from which this list is compiled do not always reflect the most recent changes to enrollment status, and there may be changes to the enrollment status or new trials added that are not yet reflected in the periodic updates provided.  Nevertheless, the below list of clinical trials, and the contact information provided for each, is intended to provide sufficient baseline information for use by any individual interested in further exploring potential eligibility and participation.

For more information on clinical trials for appendix cancer and/or pseudomyxoma peritonei or to update any of the information below, please contact Deborah Shelton.

CLINICAL TRIALS OVERVIEW

Clinical studies (often also commonly referred to as clinical trials or clinical research) can have many different objectives, depending on the specific study.  Such objectives may include, for example, developing new treatments, identifying causes of a disease or condition, studying trends, and evaluating ways in which genetics may be related to a disease or condition.

Typically, a new therapy or procedure is first tested in the laboratory and in animal studies, and, if the results merit further investigation, it is then moved into a clinical trial (ie., tested in humans).  The clinical trial testing is conducted in phases (generally Phase 1, 2, and 3, each of which is briefly explained below).  Depending on the particular phase, clinical trials are designed to obtain more detailed information about the investigational therapy, including its safety profile, risks, and effectiveness.

Sometimes a patient decides to participate in a clinical trial because none of the treatments otherwise available have worked, or they find them to have side effects that are not tolerable.  In this way, clinical trials can sometimes provide another option when standard therapies have failed.  Other patients participate in a clinical trial because they want to contribute to the advancement of medical knowledge.  Often, an individual that decides to participate in a clinical trial does so with both goals in mind.

The study of an investigational drug in humans proceeds in phases, with the information learned from each phase used to build upon the next: generally Phase 1, 2, and 3.

Clinical trials – particularly for new cancer therapies – are sometimes collapsed.  In other words, some researchers design these trials to combine to phases into a single study protocol (e.g., Phase 1/2, or Phase 2/3).  The reason for this type of design is to help facilitate a more seamless transition between phases, potentially allowing research questions to be answered more quickly or with fewer patients.

The National Cancer Institute (NCI) provides the following examples of benefits and risks of participating in a clinical trial that should be considered in deciding whether a clinical trial may be right for you:

Potential Benefits: may include having access to a new therapy that is not otherwise available; close monitoring by the clinical research team; being among the first to benefit from a therapy that is determined to be more effective than the standard treatment; and helping researchers learn more about your type of cancer; and benefitting the larger patient population in the future.

Potential Risks: may include the investigational treatment may be inferior to the standard treatment, or may not work for the individual patient at all; there may be unanticipated side effects or worse side effects than the standard treatment; increased doctors visits and associated expenses, including travel and logistical support; extra tests may be required that may be both time-consuming and uncomfortable; and a patient’s health insurance may not cover all patient costs in a clinical trial.

For additional information, see NIH's Clinical Trials Information for Patients and Caregivers.

CURRENT CLINICAL TRIALS

The compilation of clinical-trial information provided is organized as follows: (1) the name of the clinical trial, and the study identifier and link to the clinicaltrials.gov registry providing more detailed information; (2) a brief summary of the protocol/objectives of the trial, (3) key baseline eligibility criteria, and (4) study contact information.  Please note that the eligibility criteria for any given clinical trial are typically extensive, and thus not detailed in the list of clinical trials provided here. All of the inclusion and exclusion criteria can be accessed, however, through the clinicaltrials.gov link provided for that particular trial and should be discussed further with your physician and/or the contact person listed for that clinical trial.  Examples of common eligibility for a clinical study of a new investigational cancer drug include (a) having a specific type or stage of cancer; (b) having received or not received a certain kind of prior therapy; (c) having specific genetic changes in your tumor; (d) medical history; and (e) current health status.

Each clinical trial is assigned a specific identifier; the identifier is referred to formally as the NCT number in the U.S. and is the registry number as provided in the listing for that trial at ClinicalTrials.gov.  The NCT number and the corresponding link to the full listing on ClinicalTrials.gov is provided for each clinical trial listed here where applicable.

The following is a list of current clinical trials but may be of interest to AC/PMP patients. This list is organized by (1) phase of trial, and then (2) within each phase, the type of trial (i.e., (A) Appendix Cancer-Specific; (B) Tumor Agnostic; and (C) Unspecified Solid Tumor). For your additional information, each of these types of trials is described briefly below:

Appendix Cancer-Specific:
These trials are focused, at least in part, directly on Appendix Cancer and PMP.

Specific Tumor Mutations/Characteristics:
Specific Tumor Mutations/Characteristics clinical trials focus on the study of what is sometimes referred to as tumor-agnostic treatments. Tumor-agnostic treatments are drugs that are used to treat any type of cancer, regardless of the organ or type of tissue from which the cancer originally developed. Instead of the primary site of tumor origin, a tumor-agnostic treatment focuses on the tumor’s genetic make-up, including specific mutations or biomarkers. In other words, this type of therapy can be used when a person’s tumor has a very specific molecular alteration that is targeted by the drug or predicts that the drug is likely to work.

Unspecified Solid Tumor:
Although some of the trials listed below are so-called tumor-agnostic trials, some of the trials listed below involve solid tumors generally rather than appendix cancer specifically or tumors with specific mutations. We call these trials “other non-specific tumor” clinical trials.

Temporarily Suspended:
Clinical trials that are not enrolling at this time.  They are currently suspended, pending the Sponsors' analysis of results from the dose-escalation phase (Part 1) to establish the eligibility criteria for the dose-expansion phase (Part 2).  We will try to monitor and update with any changes.

The first group of tumor-agnostic (and other non-specific tumor) trials listed below are Phase 2 trials. The second group of tumor-agnostic listed beneath those are Phase 1/2 trials.

PHASE 1 CLINICAL TRIALS

Appendix Cancer-Specific

Specific Tumor Mutations/Characteristics

Unspecified Solid Tumor

PHASE 2 CLINICAL TRIALS

Specific Tumor Mutations/Characteristics

Other – Virtual Tumor Board Recommended Therapies/Clinical Trials Based on Genomic Sequencing Done as Part of this Study

Unspecified Solid Tumor

Temporarily Suspended

PHASE 1/2 CLINICAL TRIALS

Appendix Cancer-Specific

Specific Tumor Mutations/Characteristics

Unspecified Solid Tumor

Temporarily Suspended

PHASE 3 CLINICAL TRIALS

None currently identified.  

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