Clinical trial NCT03446638: iCare 2: Personalized Genomic Mutation Informed Treatment of Patients With Myelodysplastic Syndromes
Official title: iCare 2: Personalized Genomic Mutation Informed Treatment of Patients With Myelodysplastic Syndromes
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Myelodysplastic Syndromes
Keywords: myelodysplastic syndromes
MDS
computational biology
relapsed
refractory
Lead sponsor: University of Florida
Sponsor class: OTHER
Collaborators: name: Gateway for Cancer Research
name: Cellworks Group Inc.
First posted: 2018-02-27
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).

Detailed description: It is hypothesized that personalized treatment informed by computational biology simulation technology will improve treatment outcomes for patients with relapsed or refractory MDS.

Eligibility criteria: Inclusion Criteria:

* Provide written informed consent
* Must be at least 18 years of age
* Diagnosis of MDS, as defined by World Health Organization (WHO) 2008, that has relapsed after any duration of time from last best response or is refractory to induction therapy (defined as 4 cycles of treatment with a hypomethylating agent, 2 cycles of lenalidomide, 1 cycle of low intensity chemotherapy, or 1 cycle of high intensity chemotherapy)
* ECOG performance status of 0-2
* Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) may participate, provided they meet the following conditions:

  1. Must agree to use physician-approved contraceptive methods (e.g., abstinence, intrauterine device, oral contraceptive, double barrier device) throughout the study and for 3 months following the last dose of study treatment; and
  2. Must have a negative serum or urine pregnancy test within 7 days prior to beginning treatment on this trial
* Males with female partners of child-bearing potential must agree to use physician approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout the study and should avoid conceiving children for 6 months following the last dose of study treatment.

Exclusion Criteria:

* Must not have acute myeloid leukemia (AML), as defined by WHO 2008
* Pregnant and nursing subjects are excluded because the effects of study treatments on a fetus or nursing child are unknown
* Must not have had treatment with any anti-cancer therapy (investigational or standard) within the previous 21 days prior to the first dose of study drug or less than full recovery (no worse than CTCAE v4.0 grade 1) from the clinically significant toxic effects of that treatment.

Primary outcomes:
measure: Difference in overall response, as measured by International Working Group (IWG) 2006 criteria for response in MDS
timeFrame: 4 months
Secondary outcomes:
measure: Difference in safety and feasibility, as measured by CTCAE v4.0 criteria
timeFrame: 5 months
measure: Difference in time to death between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 3 years
measure: Difference in time to progression to acute myeloid leukemia (AML), as measured by IWG 2006 criteria for response in MDS, between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 4 months
measure: Difference in time to disease relapse, as measured by IWG 2006 criteria for response in MDS, between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 4 months
measure: Difference in time to best response, as measured by IWG 2006 criteria for response in MDS, between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 4 months
measure: Difference in change in myeloblast percentage between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 4 months
measure: Difference in blood transfusion rate between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 7 months
Other outcomes:
measure: Differences in mutant allele frequencies between patients treated with computational biology-informed therapy and those treated with standard of care regimens
timeFrame: 4 months
measure: Laboratory correlations between computational model and actual intracellular pathway activation status
timeFrame: 4 months
measure: Clinical correlations between pharmacogenotypes and drug efficacy (as measured by IWG 2006 criteria for response in MDS)
timeFrame: 4 months
measure: Clinical correlations between pharmacogenotypes and drug-related adverse events (as measured by CTCAE v4.0 criteria)
timeFrame: 5 months
Locations:
city: Gainesville
country: United States
facility: University of Florida
state: Florida
Study officials:
affiliation: University of Florida
name: Christopher Cogle, MD
role: PRINCIPAL_INVESTIGATOR
References: