Clinical Trials


A list of clinical trials currently enrolling patients with appendix cancer and/or pseudomyxoma peritonei from appendiceal origin is provided below.  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.


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. 

Phase 1 Study – A phase 1 study is early testing in humans.  Its purpose is to determine dosing, safety and tolerance of an investigational drug and to gather information about its biological activity within the body. 

Phase 2 Study – Once an acceptable margin of safety is established for the investigational drug, Phase 2 begins.  A Phase 2 study is generally the first study in which primary measurements will be made to determine efficacy of the investigational drug.  In terms of the a study’s objectives, though, the line between a Phase 1 and Phase 2 study can sometimes be somewhat blurred, particularly in the case of oncology trials where there is a small study population and interest in expediting development process while appropriately safeguarding patient safety. 

Phase 3 Study – A Phase 3 study is a large, pivotal safety and effectiveness trial.  Information gathered during this stage includes additional evidence of safety and efficacy, long-term tolerance, drug interactions, etc.

As noted above, 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.


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 registry providing more detailed information; (2) a brief summary of the protocol/objectives of the trial, and (3) key baseline eligibility criteria.  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 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  The NCT number and the corresponding link to the full listing on is provided for each clinical trial listed here where applicable.

This list will be updated as confirmation of enrollment status and eligibility criteria is received.  For any questions about those clinical trials pending addition to this list, please contact ACPMP at

1. Nivolumab and Ipilimumab in Mucinous Colorectal and Appendiceal Tumors (NCT03693846)

Location: University of Pennsylvania, Abramson Cancer Center, Philadelphia, Pennsylvania.

Brief description: This is a Phase 2 clinical trial of patients with mucinous adenocarcinoma of the appendix (or colon, rectum).  A total of 21 patients will be enrolled.  Treatment will consist of nivolumab  (brand name OPDIVO) every 4 weeks and ipilimumab (brand name YERVOY) every 8 weeks until disease progression, unacceptable toxicity, or 2 years of therapy.

Baseline Eligibility Criteria, include, but are not limited to:

  • metastatic disease confined to the peritoneum only;
  • prior systemic chemotherapy with FOLFOX and FOLFIRI unless contraindicated or refused;
  • immunohistochemistry testing demonstrates microsatellite stable and/or mismatch repair proficient;
  • no bowel obstruction within the past 60 days;

Contact Person(s): Nurse Navigator Trish Gambino; Study Nurse Lisa DiCicco.

Note: The physician with primary responsibility for this clinical trial (i.e., the Principal Investigator) Dr. Peter O’Dwyer, or one of the members from his research team, is scheduled to speak about the trial at the Drexel ACPMP Patient-Practitioner Symposium: “Innovations in the Multidisciplinary Management of PMP/Appendiceal Cancer,” Saturday April 6, 2019, 7:30 am to 12:30 pm, Philadelphia, Pennsylvania.  The ACPMP Research Foundation is the co-host and sponsor of this educational event.

2. LCI-GI-APX-NIN-001:  Ninetedanib (brand name OFEVRO) in Metastatic Appendiceal Carcinoma (NCT03287947) 

Location: Levine Cancer Institute, Charlotte, NC (and additional sites in/around Charlotte area).

Brief description: This is a Phase 2 study of patients with metastatic appendix carcinoma.  A total of approximately 39 participants will be enrolled.  Treatment will consist of oral nintedanib (brand name OFEVRO), 200 mg taken twice daily.   

Baseline Eligibility Criteria, include, but are not limited to:

  • metastatic appendiceal carcinoma;
  • failed prior initial systemic chemotherapy with 5-FU chemotherapy (e.g., FOLFOX or FOLFIRI with or without bevacizumab (brand name AVASTIN)).  This may include intolerance of prior chemotherapy disease progression on or within 6 months of last day of treatment with prior chemotherapy.
  • no active brain metastases or CT/MRI evidence of cavitary/necrotic tumors;
  • no tumors with CT/MRI evidence of local invasion of major blood vessels;
  • no bowel obstruction within the past 60 days;
  • no gastrointestinal disorders (e.g., chronic diarrhea) or abnormalities that would interfere with absorption of the study drug.  (Note: Potential exception for patients that tolerate anti-diarrheal medications).
  • no experience of significant weight loss (more than 10% baseline weight) within past 2 months prior to consenting to trial.  (Note: removal of ascites is not to be calculated as weight loss.)

Contact Person(s): Deirdre Maxwell or Nathan Thompson – Tel. 980-442-23353.

3. Phase 1/2 Study to Investigate the Safety, Biologic, and Antitumor Activity of ONCOS-102 in combination with Durvalumab (brand name IMFINZI) in Patients with Peritoneal Malignancies (NCT02963831)

Location(s): Ludwig Cancer Institute - Research Facilities in Miami, Florida; Buffalo, New York; Toledo, Ohio, and Charlottesville, Virginia.

Brief description: This is a Phase 1/2 study in patients with peritoneal disease, including from appendiceal carcinoma.  A total of approximately 78 participants will be enrolled.  Treatment will consist of ONCOS-102 (an investigational drug) administered intraperitoneally once a week for a total of 6 weeks, and durvalumb (brand name IMFINZI) administered by IV infusion once every four weeks for a total of 12 four-week cycles. 

Baseline Eligibility Criteria, include, but are not limited to:

  • peritoneal malignancy, including cancer originating from the appendix;  
  • failed on prior standard chemotherapy with no additional therapy options available known to prolong survival;
  • willing to undergo a core needle biopsy during screening and during Study Week 5;
  • no ongoing bowel perforation or presence of bowel fistular or abscess, or history of small or large bowel obstruction within 3 months of registering for the clinical trial, including persons with palliative gastric drainage catheters.  NOTE: palliative diverting ileostomy or colostomy ARE ALLOWED if patient has been symptom-free for more than 3 months.

Contact Person(s)Lisa Shohara and/or Danielle McCabe, Tel. 212-450-1515.

4. Injection of Bromelain and Acetylcysteine in combination into recurrent mucinous tumor or pseudomyxoma peritonei: a phase 1/2 study (Australian New Zealand Clinical Trials Registry (ANZCTR) Universal Trial Number (UTN) U1111-1203-1657)

Location(s): St. George Hospital – Kogarah, Australia 

Brief description: This is a Phase 1/2 study in patients with mucinous peritoneal tumor, including PMP that are not suitable for cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (CRS/HIPEC) or other potentially beneficial surgery.   A total of approximately 60 participants will be enrolled.  The combination of Bromelain and Acetylcysteine will be injected directly into the tumor or peritoneal cavity via a drain and allowed to dwell for 24 hours with the expectation that the tumor will be dissolved by the drug combination administered.  The tumor will then be drained and a repeat treatment will be considered.  The dose of the drug is dependent on the calculated tumor dimensions and volume.    

Baseline Eligibility Criteria, include, but are not limited to:

  • patients with psuedomyxoma Peritonei (PMP or mucinous soft to intermediate grade tumor who are not candidates for CRS/HIPEC or other surgery;
    • Note:  Having hard tumor in one region does not exclude treatment in another area provided the appearance is mucinous.
  • in the case of a target lesion (versus free intraperitoneal), the tumor must be safely accessible percutaneously;
  • no suspected fistula of the tumor into the GI tract, invading or abutting major vessel or other area of concern;
  • no infected tumor (pus on aspiration or indicated on blood test)

Contact Person(s)Sarah Valle, Tel. +61291132070

Note: Currently enrolling in Australia only; U.S. and European studies may be forthcoming.