Gilteritinib for Myelodysplastic Syndromes

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
M D Anderson Cancer Center, Houston, TX
Myelodysplastic Syndromes+9 More
Gilteritinib - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating the safety and efficacy of a new drug combination in treating patients with acute myeloid leukemia.

See full description

Eligible Conditions

  • Myelodysplastic Syndromes
  • Myelodysplastic Syndromes (MDS)
  • Refractory Acute Myelogenous Leukemia (AML)
  • Acute Myeloid Leukemia (AML)
  • acute, recurrent Myeloid Leukemia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Myelodysplastic Syndromes

Study Objectives

This trial is evaluating whether Gilteritinib will improve 2 primary outcomes and 6 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of Up to 28 days.

Year 2
Relapse-free survival
Year 2
Overall survival
Day 28
Overall response (OR) rate (Phase II)
Up to 2 years
Complete response rate
Proportion of patients proceeding to hematopoietic stem cell transplantation
To assess minimal residual disease negativity by flow cytometry
Up to 28 days
Maximum tolerated dose (Phase I)
Day 30
Incidence of adverse events

Trial Safety

Safety Progress

1 of 3

Other trials for Myelodysplastic Syndromes

Trial Design

1 Treatment Group

Treatment (decitabine, cedazuridine, venetoclax, gilteritib)
1 of 1
Experimental Treatment

This trial requires 42 total participants across 1 different treatment group

This trial involves a single treatment. Gilteritinib is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Treatment (decitabine, cedazuridine, venetoclax, gilteritib)INDUCTION (CYCLE 1): Patients receive decitabine and cedazuridine PO QD on days 1-5, venetoclax PO QD on days 1-28, and gilteritinib PO QD on days 1-28 in the absence of disease progression or unacceptable toxicity CONSOLIDATION (CYCLES 2-24): Patients receive decitabine and cedazuridine PO QD on days 1-5, gilteritinib PO QD on days 1-28, and venetoclax PO QD on days 1-21. Treatment repeats every 28 days for up to 23 cycles in the absence of disease progression or unacceptable toxicity. MAINTENANCE (CYCLES 24+): Patients receive gilteritinib PO QD on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Venetoclax
FDA approved
Gilteritinib
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years for reporting.

Closest Location

M D Anderson Cancer Center - Houston, TX

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. You must have received newly diagnosed for Myelodysplastic Syndromes or one of the other 9 conditions listed above. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Phase I cohort: Adults >= 18 years with relapsed/refractory FLT3-mutated AML or myelodysplastic syndrome (MDS) that is intermediate-2 or high-risk by the International Prognostic Scoring System
Confirmed newly diagnosed AML with FLT3 mutation
Either age >= 75
Or 18-74 with at least one comorbidity (congestive heart failure [CHF] requiring therapy or ejection fraction [EF] =< 50%, diffusion capacity of the lung for carbon monoxide [DLCO] =< 65% or forced expiratory volume in 1 second [FEV1] =< 65%, or Eastern Cooperative Oncology Group [ECOG] 2 or 3, or other significant co-morbidity precluding use of cytotoxic chemotherapy as approved by the principal investigator (PI)
Phase II cohort B: Adults >= 18 years with relapsed/refractory FLT3-mutated AML or MDS that is intermediate-2 or high-risk by the International Prognostic Scoring System who have received 1 prior therapy
For all cohorts, patients with either FLT3-ITD or FLT3 D835 mutations will be eligible
Performance status =< 3 (Eastern Cooperative Oncology Group [ECOG] scale)
Total serum bilirubin =< 2.5 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the PI
Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) =< 3 x ULN, unless due to the underlying leukemia approved by the PI
Creatinine clearance >= 30 mL/min

Patient Q&A Section

Does gilteritinib improve quality of life for those with leukemia, myeloid, acute?

"In patients who received gilteritinib for CLL, improved QOL was observed as compared with historical controls. Recent findings provide preliminary evidence that gilteritinib is well tolerated in patients with chronic lymphocytic leukaemia and might be an effective therapeutic option." - Anonymous Online Contributor

Unverified Answer

What is the survival rate for leukemia, myeloid, acute?

"Survival rates for leukemia, myeloid, acute were very good with more than 90% surviving after 15 years. There was a significant difference in survival rate based on age where patients over 100 had a significantly lower survival rate than those under the age of 50. Patients with myeloid and acute leukemias were found to live longer than those who had acute lymphocytic leukemia." - Anonymous Online Contributor

Unverified Answer

How does gilteritinib work?

"In vitro and in vivo data support that gilteritinib acts directly on JAK2; this action appears to be mediated by inhibition of downstream signaling events involving STAT3 activation. Combining gilteritinib with other agents targeting PI3K/Akt or STAT3 may be beneficial in the treatment of myeloproliferative neoplasms (MPNs)." - Anonymous Online Contributor

Unverified Answer

Is gilteritinib typically used in combination with any other treatments?

"The most commonly used combination regimen consists of gilteritinib plus lenalidomide or pomalidomide. In patients treated with these combinations, the most commonly reported adverse events were neutropenia and fatigue." - Anonymous Online Contributor

Unverified Answer

What is gilteritinib?

"Gilteritinib (commonly known by its trade names Tykerb, Gilotrif, Sipuleucel-T) is an oral medication used to treat certain types of non-Hodgkin lymphoma (NHL). It binds to the BCR protein on B cells and inhibits the growth of these cells. In clinical trials, gilteritinib has shown efficacy against follicular NHL (FL), DLBCL, MCL, and hairy cell leukaemia (HCL) when used alone or in combination with rituximab, chlorambucil, cyclophosphamide, or bendamustine." - Anonymous Online Contributor

Unverified Answer

Has gilteritinib proven to be more effective than a placebo?

"Gilteritinib monotherapy was associated with significant improvement compared with placebo monotherapy for all primary endpoints evaluated In a recent study. The modest magnitude of improvements seen In a recent study suggests the need for larger studies to confirm these results." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets leukemia, myeloid, acute?

"The median age people get leukemia is 62 years old; however, this increases with gender (male: 63 years old vs female: 57 years old), ethnicity (white: 62 years old vs black: 60 years old), and ethnicity (Hispanic: 59 years old vs Asian: 58 years old)." - Anonymous Online Contributor

Unverified Answer

What does gilteritinib usually treat?

"Gilteritinib treats a variety of hematologic malignancies including acute myeloid leukemia and chronic myeloid leukemia. It also may be useful as a second line therapy for patients with relapsed or refractory mantle cell lymphoma. Gilteritinib is used at doses of 200mg once per day, (dosed according to body surface area) or 400mg twice daily. Patients who require more than one dose per day must take a break between doses of less than 24 hours. Additionally, the drug cannot be taken during pregnancy or breastfeeding due to potential harm to the fetus or child. Side effects include diarrhea, nausea, loss of appetite, rash, joint pain, vomiting, and constipation." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for leukemia, myeloid, acute?

"Clinical trials are needed for all patients with AML and MDS. Patients with MDS should be considered for clinical trials because they have an increased risk of death due to their disease as well as the development of complications." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating leukemia, myeloid, acute?

"While [many researchers have attempted to develop new treatments for leukemia, myeloid, acute] (https://www.nhlbi.nih.gov/news/chda/2016/04/07/leukemia-myeloid-acute/), none have fulfilled the goal recently. It is important to continue researching, as it will hopefully lead to a more effective way of treating leukemia, myeloid, acute.\n" - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving gilteritinib?

"No clinical studies have investigated gilteritinib in combination with other agents; however, the evidence suggests that gilteritinib may be effective when combined with other cytotoxic chemotherapy drugs such as bortezomib, lenalidomide, thalidomide, and pomalidomide. Therefore, it may be worth considering gilteritinib for patients with relapsed or refractory AML who receive more than one prior chemotherapeutic regimen. Additional studies are warranted." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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