NCT02749708

Study of IRX5183 in Relapsed and Refractory Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

Phase I/II Study of IRX5183 in Relapsed and Refractory Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MDS)

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 13 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the use of IRX5183 in 1) patients with relapsed and/or refractory AML and 2) patients with high-risk MDS or chronic myelomonocytic leukemia (CMML).

Detailed description

This research is being done to learn about the safety and effectiveness of an investigational drug, IRX5183, in treating acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). IRX5183 is a derivative of Vitamin A. IRX5183 is a drug that is designed to cause cancer cells to mature and then die. It is thought that it will stop the uncontrolled growth of leukemia cells. These are 2 parts to this study. The purpose of the first part is to test the safety of IRX5183 at different dose levels. The investigators want to find out what effects, good and/or bad, it has on AML or MDS. Once the investigators figure out the best and safest dose of IRX5183, the investigators will enter the second part of the study using this dose. The purpose of the second part is to see if IRX5183 is effective as a treatment for AML or MDS. To date, IRX5183 has been used in about 25 people. The first part of the study is called a "Phase I" study. Phase I studies use drugs starting at low doses and slowly increase the amounts of the study drugs that are given to people until the side effects of the drugs are too high to give more. This means that not all people in the study will get the same dose of IRX5183. Doses at the beginning of the study will be lower than doses at the end of the study. Because of the design of the study, some people may get doses that are too low to have an effect, and other people will probably get doses that cause side effects. People with AML, MDS, or CMML that has 1) not responded to standard therapy or 2) has returned after a previous response may join.

Conditions

• Acute Myeloid Leukemia (AML)
• Myelodysplastic Syndrome (MDS)
• Chronic Myelomonocytic Leukemia (CMML)

Interventions

Type	Name	Description
DRUG	IRX5183	IRX5183 will be administered orally daily on days 1-28 of each cycle for 2 cycles of induction. In the phase I part of the study, there will be 3 dose levels (dose level 1 \[DL1\] with 50 mg, DL2 with 75 mg, and DL3 with 100 mg), with 1 additional dose level to be only used if excessive toxicity noted at the DL1. There will be no intra-patient dose escalation. A phase II portion of the study was originally planned, but the study was terminated prior to phase II enrollment.

Timeline

• Start date: 2017-01-30
• Primary completion: 2018-08-14
• Completion: 2018-08-14
• First posted: 2016-04-25
• Last updated: 2019-04-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study