RecruitingPhase 1NCT04365179

Study of NEROFE, a Novel Hormone-Peptide in Adult Patients With Advanced MDS and AML

An Open-Label Phase 1b Study of NEROFE, a Novel Hormone-Peptide in Adult Patients With Advanced Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)


Sponsor

Immune System Key Ltd

Enrollment

18 participants

Start Date

Jun 18, 2020

Study Type

INTERVENTIONAL

Conditions

Summary

This is an open-label Phase 1b study of NEROFE following a traditional 3+3 design to assess safety and to determine the Recommended Phase 2 Dose (RP2D) of NEROFE in patients with MDS or AML. IV NEROFE will be administered three times per week on alternate days. The exact dosage will be determined using the body surface area (BSA) measured on Day 1 of each cycle.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study is testing an experimental drug called NEROFE — a novel hormone-like peptide — in adults with advanced blood cancers: acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). These are cancers of the bone marrow that affect blood cell production. NEROFE works through a different mechanism than most existing cancer drugs and may offer hope when other treatments have failed. **You may be eligible if:** - You have relapsed or treatment-resistant AML or MDS — meaning your cancer came back or never responded to standard therapies - For AML: no alternative life-prolonging treatments are available, or you have high-risk disease unlikely to respond to other options - For MDS: your disease did not achieve complete remission or has progressed on or after standard treatments - You are in a condition that allows you to receive the study drug **You may NOT be eligible if:** - Your cancer is well-controlled or responding to current treatment - You have other serious conditions that would make participation unsafe - You do not meet the specific diagnostic and treatment history requirements Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGNerofe

Up to 12 cycles of 28 days are planned. If patients are deriving benefit, treatment may continue past 12 cycles. Cycle 1: During Cycle 1, assigned NEROFE doses will be administered by intravenous (IV) infusion over 1 hour on Days 1, 3, 5, 8, 10, 12, 15, 17, 19, 22, 24 and 26. Cycles 2-12: Subsequent cycles will be administered as above, presuming that patients do not meet criteria for study withdrawal during cycle 1.


Locations(1)

University of Miami Hospital and Clinics / Sylvester Comprehensive Cancer Center

Miami, Florida, United States

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NCT04365179


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