A Phase 2a, Open-Label, Two Stage Study of Nerofe or Nerofe With Doxorubicin in Subjects With AML or MDS
- Conditions
- Acute Myelogenous LeukemiaMyelodysplastic Syndromes
- Interventions
- Registration Number
- NCT03059615
- Lead Sponsor
- Immune System Key Ltd
- Brief Summary
This is a Phase 2a, Open-label, one arm study in which the eligible patients will be treated with IV Nerofe, three times a week in 28 days cycles (up to 12 cycles).
Evaluation will include safety procedures, blood level of study drug in certain time points, immune system response and tests checking the mechanism of the drug action.
- Detailed Description
Nerofe is a first-in-class hormone peptide with cancer suppressive properties. It works in three mechanisms of action. The purpose of this research is to study Nerofe's effect in patients diagnosed with AML and MDS.
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Nerofe 48mg/m2 Nerofe 48mg/m2 IV Nerofe - three times a week Nerofe 48mg/m2 + Doxorubicin 10mg/m2 Nerofe 48mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week Nerofe 96mg/m2 Nerofe 96mg/m2 IV Nerofe - three times a week Nerofe 96mg/m2 + Doxorubicin 10mg/m2 Nerofe 96mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week Nerofe 48mg/m2 + Doxorubicin 10mg/m2 Doxorubicin 48mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week Nerofe 96mg/m2 + Doxorubicin 10mg/m2 Doxorubicin 96mg/m2 IV Nerofe + Doxorubicin 10mg/m2 - once a week
- Primary Outcome Measures
Name Time Method Assessing change in IWG Criteria to evaluate response to Nerofe treatment (with or without Doxorubicin) for AML subjects At end of Cycles 2, 4, 6, 8, 10, 12 (Cycle length 28 Days) Bone Marrow samples and CBC will be done every 2 cycles
Assessing changes in R-IPSS (Revised International Prognostic Scoring System) Score to evaluate response to Nerofe treatment (with or without Doxorubicin) for MDS patients. A calculation of several variables. At end of Cycles 2, 4, 6, 8, 10, 12 (Cycle length 28 Days) Measuring cytogenetic abnormalities. Range from very good (0) to very poor (4)
Safety as determined by frequency, nature and severity of adverse events 13 months Per CTCAE v4.0
- Secondary Outcome Measures
Name Time Method Pharmacodynamic analysis of changes from baseline in PBMCs' T1/ST2 receptor expression Every cycle (Cycle length 28 days) At Cycle 1 and 2 on Day 1 and Day 15 and on day 1 of each consecutive cycle (up to 12 cycles)
Pharmacokinetic behavior of Nerofe: Maximum Plasma Concentration (Cmax) At cycles 1 and 2 (Cycle length 28 days) Done at Cycle 1 and 2: pre-dose, 15 minutes, 1, 2, 4, 6, 8 and 24 hours.
Pharmacokinetic behavior of Nerofe: Tmax At cycles 1 and 2 (Cycle length 28 days) Done at Cycle 1 and 2: pre-dose, 15 minutes, 1, 2, 4, 6, 8 and 24 hours.
Pharmacodynamic analysis of changes from baseline in levels of circulating cytokines Every cycle (Cycle length 28 days) At Cycle 1 and 2 on Day 1 and Day 15 and on day 1 of each consecutive cycle (up to 12 cycles)
Pharmacodynamic analysis of changes from baseline in levels of soluble T1/ST2 receptor Every cycle (Cycle length 28 days) At Cycle 1 and 2 on Day 1 and Day 15 and on day 1 of each consecutive cycle (up to 12 cycles)
Pharmacokinetic behavior of Nerofe: Minimum Plasma Concentration (Cmin) At cycles 1 and 2 (Cycle length 28 days) Done at Cycle 1 and 2: pre-dose, 15 minutes, 1, 2, 4, 6, 8 and 24 hours.
Pharmacokinetic behavior of Nerofe: Area Under the Curve (AUC) At cycles 1 and 2 (Cycle length 28 days) Done at Cycle 1 and 2: pre-dose, 15 minutes, 1, 2, 4, 6, 8 and 24 hours.
Trial Locations
- Locations (2)
Kaplan Medical Center
🇮🇱Reẖovot, Israel
Rabin Medical Center
🇮🇱Petach Tikva, Israel