Cholangiocarcinoma
Conditions
Brief summary
The purpose of this study is to see if the investigational drug, RRx-001, an epigenetic agent, which turns on a number of beneficial genes that the tumor has silenced, can resensitize the tumor(s), in other words make it/them re-respond to gemcitabine and cisplatin, which, hopefully, will translate to a longer lifespan. The name of the open-label study, which means that patients will know what treatments they are receiving, is EPIC, a hybrid or combination of EPIgenetic for the mechanism of action of RRx-001 and Cholangiocarcinoma. The study treatment is divided into two stages. During the first stage, patients will receive RRx-001, which is administered intravenously weekly, for a fixed time period of six weeks. At that time the second stage starts in which cisplatin and gemcitabine are reintroduced for as long as the tumors respond to them to determine whether resensitization has occurred. The primary objective of this clinical trial is to evaluate the progression-free survival (PFS) of patients at 9 weeks after the reintroduction of gemcitabine and cisplatin.
Interventions
DRUGRRx-001
RRx-001 (10 mg/m2) intravenously twice weekly for up to six weeks followed by retreatment with Gemcitabine 1000 mg/m2 and Cisplatin 30 mg/m2 on Days 1 and 8 of a 21 Day Cycle until tumor progression
DRUGGemcitabine and cisplatin
RRx-001 (20 mg) intravenously weekly for up to six weeks followed by retreatment with Gemcitabine 1000 mg/m2 and Cisplatin 25 mg/m2 on Days 1 and 8 of a 21 Day Cycle until tumor progression
Sponsors
EpicentRx, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Histologically or cytologically confirmed diagnosis of biliary tract adenocarcinoma/cholangiocarcinoma * Must have locally advanced or distant metastatic disease that is not surgically curable * Failed first-line chemotherapy * Age ≥ 18 years * Life expectancy of at least 12 weeks (3 months) * Performance status 0 or 1 * Adequate liver, kidney, and bone marrow function
Exclusion criteria
* Symptomatic metastatic brain or meningeal tumors * Investigational compound within 4 weeks of enrollment * History of needing to permanently discontinue prior gemcitabine/ cisplatin regimen for reasons other than progression (i.e. toxicity) * Any medical condition which, in the investigator's opinion, makes the patient unsuitable for participation * Pregnant or nursing * Substance abuse, medical, psychological or social conditions that may interfere with the subject's participation in the study or evaluation of the study results * Albumin \<2.8 * Uncontrolled or clinically relevant ascites * Absolute contraindication for MRI imaging such as intracorporeal metal or pacemaker
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Objective Response (RECIST) | Baseline and every 6-8 weeks while on treatment until progression. Estimated 8 -24 weeks. | Overall objective response (RECIST) |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| RRx-001 the Cisplatin and Gemcitabine Patients with advanced and metastatic biliary tract adenocarcinoma (cholangiocarcinoma) who had been treated with and failed first-line chemotherapy will be treated with RRx-001 (20 mg) intravenously weekly for up to six weeks followed by retreatment with Gemcitabine 1000 mg/m2 and Cisplatin 25 mg/m2 on Days 1 and 8 of a 21 Day Cycle until tumor progression
RRx-001: RRx-001 (10 mg/m2) intravenously twice weekly for up to six weeks followed by retreatment with Gemcitabine 1000 mg/m2 and Cisplatin 30 mg/m2 on Days 1 and 8 of a 21 Day Cycle until tumor progression
Gemcitabine and cisplatin: RRx-001 (20 mg) intravenously weekly for up to six weeks followed by retreatment with Gemcitabine 1000 mg/m2 and Cisplatin 25 mg/m2 on Days 1 and 8 of a 21 Day Cycle until tumor progression | 4 |
| Total | 4 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
Baseline characteristics
| Characteristic | RRx-001 the Cisplatin and Gemcitabine |
|---|---|
| Age, Continuous | 65.5 years STANDARD_DEVIATION 5.8 |
| Race/Ethnicity, Customized American Indian or Alaska Native | 1 Participants |
| Race/Ethnicity, Customized Black or African American | 1 Participants |
| Race/Ethnicity, Customized White | 2 Participants |
| Region of Enrollment United States | 4 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 4 |
| other Total, other adverse events | 4 / 4 |
| serious Total, serious adverse events | 2 / 4 |
Outcome results
Primary
Overall Objective Response (RECIST)
Overall objective response (RECIST)
Time frame: Baseline and every 6-8 weeks while on treatment until progression. Estimated 8 -24 weeks.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| RRx-001 the Cisplatin and Gemcitabine | Overall Objective Response (RECIST) | 3 Participants |