Clinical trial NCT02452970: RRx-001 in Second Line Treatment of Advanced Cholangiocarcinoma Prior to Readministration of First-Line Therapy
Official title: A Phase 2, Single-Arm, Open-label, Study of RRx-001 in Second Line Treatment of Advanced Cholangiocarcinoma Prior to Readministration of First-Line Therapy
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Cholangiocarcinoma
Lead sponsor: EpicentRx, Inc.
Sponsor class: INDUSTRY
First posted: 2015-05-25
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Overall Objective Response (RECIST)
timeFrame: Baseline and every 6-8 weeks while on treatment until progression. Estimated 8 -24 weeks.
Secondary outcomes:
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins Sidney Kimmel Comprehensive Cancer Center
state: Maryland
Study officials:
affiliation: Johns Hopkins School of Medicine
name: Ana De Jesus Acosta, MD
role: PRINCIPAL_INVESTIGATOR
References: