Myelodysplastic SyndromesNCT03446638
iCare 2: Personalized Genomic Mutation Informed Treatment of Patients With Myelodysplastic Syndromes
A study testing FDA-approved drug or combination of drugs, FLAG induction and 7 + 3 induction in people with Myelodysplastic Syndromes, run by University of Florida at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 99 years
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- FDA-approved drug or combination of drugs, FLAG induction and 7 + 3 induction
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- May 2019to Sep 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 99 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- 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
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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).
Read the 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.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- FDA-approved drug or combination of drugs, FLAG induction, 7 + 3 induction, Low-dose cytarabine, Supportive care alone
- Keywords
- myelodysplastic syndromes, MDS, computational biology, relapsed, refractory
- Medical subject headings
- Myelodysplastic Syndromes, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FDA-approved drug or combination of drugsDrug
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).
FLAG inductionDrug
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.
7 + 3 inductionDrug
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.
Low-dose cytarabineDrug
Patients will receive 20 mg/m2 per day subcutaneously of cytarabine for 10 days every 28 days.
Supportive care aloneOther
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.
Computational biology simulations softwareDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| Computational Biology-Informed Treatment | Experimental | Patients randomized to this arm will receive an FDA-approved drug or combination of drugs predicted to have a therapeutic effect based on their individual MDS disease genetic profile by a computational biology simulation software program. The specific drug or combination of drugs that a patient on this arm will receive will be decided jointly by a molecular oncology board comprised of physicians, pharmacists, and nurse coordinators and the treating physician. Patients will receive a minimum of 2 months and a maximum of 4 months of treatment with the selected drug or combination of drugs. Receives: Drug: FDA-approved drug or combination of drugs; Device: Computational biology simulations software |
| Standard of Care Treatment | Active comparator | Patients randomized to this arm will receive either one of three standard of care treatment regimens of the treating physician's choice (low-dose cytarabine, 7 + 3 induction, or FLAG induction) or supportive care alone. Patients will receive a minimum of 2 months and a maximum of 4 months of the selected treatment regimen or of supportive care alone. Receives: Drug: FLAG induction; Drug: 7 + 3 induction; Drug: Low-dose cytarabine; Other: Supportive care alone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in overall response, as measured by International Working Group (IWG) 2006 criteria for response in MDS
Difference in overall response (number of patients who achieve complete response, partial response, stable disease, or hematologic improvement per IWG 2006 criteria) between patients treated with computational biology-informed therapy vs. those treated with standard of care regimens
Measured: 4 months
Secondary outcomes (7)
Difference in safety and feasibility, as measured by CTCAE v4.0 criteria
Difference in safety and feasibility, as measured by CTCAE v4.0 criteria, between patients treated with computational biology-informed treatment and those who receive a standard of care regimen
Measured: 5 months
Difference in time to death between patients treated with computational biology-informed therapy and those treated with standard of care regimens
Measured: 3 years
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
Measured: 4 months
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
Measured: 4 months
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
Measured: 4 months
Difference in change in myeloblast percentage between patients treated with computational biology-informed therapy and those treated with standard of care regimens
Measured: 4 months
Difference in blood transfusion rate between patients treated with computational biology-informed therapy and those treated with standard of care regimens
Measured: 7 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2018
- Study startMay 2019 (estimated)
- Primary completionAug 2021 (estimated)
- Study completionSep 2022 (estimated)
Why it stopped: administratively withdrawn
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Gateway for Cancer Research, Cellworks Group Inc.
- Responsible party
- Sponsor
- Investigators
- Christopher Cogle, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03446638
- Sponsor's protocol ID
- iCare 2
- Other IDs
- OCR17918, IRB201702867
- Status last verified
- Jun 2019
- First submitted
- 20 Feb 2018
- Last updated
- 11 Jun 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 11 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- iCare 2: Personalized Genomic Mutation Informed Treatment of Patients With Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT03446638
- Acronym / sponsor ID
- iCare 2
- Conditions
- Myelodysplastic Syndromes
- Keywords
- myelodysplastic syndromes, MDS, computational biology, relapsed, refractory
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: administratively withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Gateway for Cancer Research, Cellworks Group Inc.
- Study start
- May 2019 (estimated)
- Primary completion
- Aug 2021 (estimated)
- Study completion
- Sep 2022 (estimated)
- First posted
- 27 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Computational Biology-Informed Treatment | Experimental | Drug: FDA-approved drug or combination of drugs, Device: Computational biology simulations softwarePatients randomized to this arm will receive an FDA-approved drug or combination of drugs predicted to have a therapeutic effect based on their individual MDS disease genetic profile by a computational biology simulation software program. The specific drug or combination of drugs that a patient on this arm will receive will be decided jointly by a molecular oncology board comprised of physicians, pharmacists, and nurse coordinators and the treating physician. Patients will receive a minimum of 2 months and a maximum of 4 months of treatment with the selected drug or combination of drugs. |
| Standard of Care Treatment | Active comparator | Drug: FLAG induction, Drug: 7 + 3 induction, Drug: Low-dose cytarabine, Other: Supportive care alonePatients randomized to this arm will receive either one of three standard of care treatment regimens of the treating physician's choice (low-dose cytarabine, 7 + 3 induction, or FLAG induction) or supportive care alone. Patients will receive a minimum of 2 months and a maximum of 4 months of the selected treatment regimen or of supportive care alone. |
Who may qualify
- Ages
- 18 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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:
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FDA-approved drug or combination of drugsPatients 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 | Computational Biology-Informed Treatment |
| FLAG inductionPatients 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 | Standard of Care Treatment |
| 7 + 3 inductionPatients 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 | Standard of Care Treatment |
| Low-dose cytarabinePatients will receive 20 mg/m2 per day subcutaneously of cytarabine for 10 days every 28 days. | Drug | Standard of Care Treatment |
| Supportive care alonePatients will receive one or more of the following: blood product transfusions, antibiotics, granulocyte colony-stimulating factor (G-CSF), erythropoietic stimulating factors, and iron chelation. | Other | Standard of Care Treatment |
| Computational biology simulations softwareGenetic 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. | Device | Computational Biology-Informed Treatment |
Primary outcome
Difference in overall response, as measured by International Working Group (IWG) 2006 criteria for response in MDS
Time frame: 4 months
Difference in overall response (number of patients who achieve complete response, partial response, stable disease, or hematologic improvement per IWG 2006 criteria) between patients treated with computational biology-informed therapy vs. those treated with standard of care regimens
7 secondary outcomes
Difference in safety and feasibility, as measured by CTCAE v4.0 criteria
Time frame: 5 months
Difference in time to death between patients treated with computational biology-informed therapy and those treated with standard of care regimens
Time frame: 3 years
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
Time frame: 4 months
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
Time frame: 4 months
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
Time frame: 4 months
Difference in change in myeloblast percentage between patients treated with computational biology-informed therapy and those treated with standard of care regimens
Time frame: 4 months
Difference in blood transfusion rate between patients treated with computational biology-informed therapy and those treated with standard of care regimens
Time frame: 7 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jun 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher Cogle, MD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing FDA-approved drug or combination of drugs, FLAG induction and 7 + 3 induction in people with Myelodysplastic Syndromes, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 11 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Difference in overall response, as measured by International Working Group (IWG) 2006 criteria for response in MDS (time frame: 4 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jun 2019.