RecruitingPhase 1

Testing for Safety and Colorectal Cancer Preventive Effects of ONC201

Phase I Trial of ONC201 for Chemoprevention of Colorectal Cancer

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 13 weeks
  • Procedures mentioned: Biopsies

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Patients receive ONC201 orally (PO) once weekly (QW) or once every 3 weeks (Q3W) for 13 weeks."
  • "Patients also undergo collection of blood, tissue biopsy, and sigmoidoscopy/colonoscopy throughout the study."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This early-stage study tests the safety of different doses of ONC201, also called dordaviprone, and whether it may help prevent colon and rectal cancer. It is for adults with familial adenomatous polyposis (FAP), a condition that causes many colon growths called polyps, or a history of multiple polyps.

The purpose of this phase I trial is to test the safety and cancer preventive effects of different doses of ONC201 in people with familial adenomatous polyposis (FAP) or a history of multiple polyps. People with familial adenomatous polyposis (FAP) or a history of multiple polyps are at higher than average risk of developing colorectal cancer. ONC201, now known as dordaviprone, is a drug that may stop cancer cells from growing. This drug has been shown in previous studies to cause cancer cell death but not harm normal cells. If successful, this study may help us develop a new option for colorectal cancer prevention.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be at least 18 years old, understand the study, and agree in writing to join.
  • Have FAP, or meet these findings on your most recent colonoscopy within 5 years: more than 5 adenomas (a type of polyp) under 1 cm, or at least 3 adenomas with at least one measuring 1 cm or more.
  • Be able to do most daily activities and be expected to live at least 5 years.
  • Have blood cell counts and liver and kidney test results that meet the study’s limits.
  • Be due for a routine colonoscopy, or an approved scope exam if your colon was removed. The exam must find at least 2 adenomas measuring 5 mm or more. At least one must be within reach of a short scope; it will be left in place and marked. Six samples of normal colon lining will also be taken.
  • Agree to another scope exam about 12 weeks after starting ONC201.
  • Be willing and able to follow the visit schedule, treatment plan, lab tests, and other study steps.
  • If you or your partner could become pregnant, use birth control or avoid sex before joining, during the study, and for 4 weeks after treatment. Stop the study drug and tell the study doctor right away if you become pregnant or think you might be.

Who can't join

  • Have Lynch syndrome, an inherited condition that raises cancer risk, or a past colon or rectal cancer that grew into nearby tissue.
  • Have another cancer that has spread into nearby tissue and is growing or needs treatment within 3 years of joining. Some treated skin cancers and early cervical cancer are exceptions. Older treated cancers need review by the lead study doctor.
  • Are taking another experimental drug or took one within the past 4 weeks; or have ever received cell-killing chemotherapy or ONC201.
  • Have had an allergic reaction to a drug similar to ONC201.
  • Have an uncontrolled illness, such as an active infection or serious heart problem, or a mental health or social problem that would keep you from following study steps.
  • Are pregnant or breastfeeding.
  • Take certain medicines that change how the body breaks down ONC201, or medicines that can cause a dangerous heart rhythm, unless they are stopped at least 72 hours before ONC201 as required by the study.
  • Have a heart test showing a QTc over 480 milliseconds (a delay in the heart’s electrical recovery), a history of heart failure or the dangerous rhythm called torsades de pointes, or a family history of long QT syndrome.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * Be identified as high risk for recurrent colorectal adenomas, as defined by: * A diagnosis of FAP AND/OR * Findings of either \> 5 small (less than 1 cm) adenomas OR \>= 3 with at least one \>= 10 mm on most recent colonoscopy performed in the past 5 years * Be \>= 18 years of age on day of signing informed consent * Have an Eastern Cooperative Oncology Group (ECOG) performance status =\< 1 (Karnofsky \>= 70%) * Leukocytes \>= 3,000/microliter * Absolute neutrophil count \>= 1,000/microliter * Platelets \>= 100,000/microliter * Total bilirubin within normal institutional limits * Aspartate aminotransferase (AST) (serum (glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase (\[SGPT\]) =\< 1.5 x institutional upper limit of normal * Creatinine =\< 1.5 x institutional upper limit of normal * Participant is due to undergo a standard of care lower gastrointestinal (GI) colonoscopy (in participants who have undergone prior colectomy and for whom colonoscopy is not feasible, an appropriate standard-of-care lower endoscopic evaluation may be substituted for the baseline colonoscopy at the site investigator's discretion) for detection and removal of colorectal polyps. On this colonoscopy, participant is required to have: * Two (2) adenomatous polyps of at least five (5) mm in size * At least one (1) polyp within reach of a flexible sigmoidoscope (which will be retained in the colon or rectum and marked) * In addition to polypectomy, six (6) biopsies of normal colonic mucosa \>= 1 cm from a collected polyp will also be collected * Willing to undergo a second, research intent endoscopic procedure (either sigmoidoscopy or colonoscopy), approximately 12 weeks after initiating ONC201 treatment * Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures * Life expectancy of at least 5-years * ONC201 is an imipridone agent with the potential for teratogenic or abortifacient effects. For this reason and because imipridones potential teratogenic effects are unknown, men and women of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for four weeks after study treatment is completed. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should STOP the study medication and inform her study physician immediately * Ability to understand and the willingness to sign a written informed consent document Exclusion Criteria: * Prior history of hereditary nonpolyposis colorectal cancer (HNPCC), also known as Lynch syndrome * Participants may not be currently receiving any other investigational agents or have received any investigational agents within the past four weeks * Prior history of invasive colorectal cancer * Prior invasive active neoplasm that is progressing or requires active treatment within 3 years from registration. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. Participants with a history of prior invasive neoplasm diagnosed and treated greater than 3 years form registration may be considered with consultation of the primary investigator * Prior history of exposure to cytotoxic chemotherapy or ONC201 * History of allergic reactions attributed to compounds of similar chemical or biologic composition to ONC201 * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements * Pregnant and women who are nursing are excluded from this study because ONC201 is an imipridone agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events (AEs) in nursing infants secondary to treatment of the mother with ONC201, breastfeeding should be discontinued if the mother is treated with ONC201 * Concomitant use of strong/moderate CYP3A4/5 inducers/inhibitors. These agents must be discontinued at least 72-hours prior to beginning ONC201 * Any of the following cardiac criteria: * Prolongation of corrected QT (QTc) interval (QTc interval \> 480 milliseconds, preferably using Frederica's QT correction formula), confirmed on electrocardiogram (ECG) tracings performed during screening * A history of Torsades de pointes, heart failure, or family history of prolonged QT Syndrome * Concomitant use of drugs that are known to prolong QT and have a known risk of torsade de pointes (TdP) unless they are willing to stop these medications and possibly change to an alternative non-excluded medication to treat the same condition at least 72 hours prior to beginning ONC201

Study sites (5)

MI

MO

OH

RI