iCare 2: Personalized Genomic Mutation Informed Treatment of Patients With Myelodysplastic Syndromes

Summary

This open-label, randomized, parallel group phase II study will investigate the efficacy of computational biology-informed treatment vs. standard of care treatment for patients with relapsed or refractory myelodysplastic syndromes (MDS).

Conditions

• Myelodysplastic Syndromes

Interventions

DRUG

FDA-approved drug or combination of drugs

Patients assigned to this arm will receive an FDA-approved drug or combination of drugs. Dosing and treatment schedule will follow the package insert for the selected drug(s).

DRUG

FLAG induction

Patients will receive 30 mg/m2 per day intravenously of fludarabine for 5 days and 2000 mg/m2 per day intravenously of cytarabine for 5 days. 5 mg/kg per day of granulocyte colony stimulating factor (G-CSF) may be given subcutaneously beginning on Day 1 of each treatment until absolute granulocyte count \> 500/ microliter for 3 days.

DRUG

7 + 3 induction

Patients will receive 100-200 mg/m2 per day intravenously of cytarabine for 7 days, plus either 45-60 mg/m2 per day intravenously of daunorubicin or 9-12 mg/m2 per day intravenously of idarubicin for 3 days.

DRUG

Low-dose cytarabine

Patients will receive 20 mg/m2 per day subcutaneously of cytarabine for 10 days every 28 days.

OTHER

Supportive care alone

Patients will receive one or more of the following: blood product transfusions, antibiotics, granulocyte colony-stimulating factor (G-CSF), erythropoietic stimulating factors, and iron chelation.

DEVICE

Computational biology simulations software

Genetic testing results for each patient randomized to this arm will be used by a computational biology simulations software program to generate a personalized map of dysregulated metabolic pathways contributing to the patient's disease. This map will then be used to digitally screen for potentially therapeutic FDA-approved drugs or drug combinations to target the dysregulated metabolic pathways.

Sponsors & Collaborators

• Gateway for Cancer Research

  collaborator OTHER

• Cellworks Group Inc.

  collaborator INDUSTRY

• University of Florida

  lead OTHER

Principal Investigators

• Christopher Cogle, MD · University of Florida

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

99 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2019-05-31

Primary Completion

2021-08-31

Completion

2022-09-30

FDA Device

Yes

Countries

• United States

Study Locations