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)
Immune System Key Ltd
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.
Description
NEROFE monotherapy may be administered for a maximum of 12 cycles provided that the patient tolerates treatment and there is evidence of clinical benefit. If patients are receiving clinical benefit, they may continue past 12 cycles. Patients will be followed for a minimum of 30 days after the last dose of NEROFE monotherapy. After this 30 day period, patients will only be followed for the resolution of any ongoing adverse events.
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Confirmed diagnosis of one of the following: 1. Relapsed/refractory Acute Myelogenous Leukemia (AML) where no alternative life prolonging therapy exists. Adverse genetic risk treatment naïve patients may also be considered eligible if, in the opinion of the investigator, these patients are unlikely to benefit from alternative therapy (e.g., an older patient with adverse risk MDS who progresses to AML after failing treatment for MDS (i.e. hypomethylating agent (HMA) or HMA and Venetoclax and is not a candidate for traditional AML induction chemotherapy). 2. Relaps…
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.
Location
- University of Miami Hospital and Clinics / Sylvester Comprehensive Cancer CenterMiami, Florida