Fostamatinib for Leukemia

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
M D Anderson Cancer Center, Houston, TX
Leukemia+8 More
Fostamatinib - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This study is evaluating whether a drug called fostamatinib can help treat patients with lower-risk myelodysplastic syndromes or chronic myelomonocytic leukemia who have failed therapy with hypomethylating agents.

See full description

Eligible Conditions

  • Leukemia
  • Refractory Chronic Myelomonocytic Leukemia
  • Refractory Myelodysplastic Syndromes

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

This trial is evaluating whether Fostamatinib will improve 1 primary outcome in patients with Leukemia. Measurement will happen over the course of through study completion, an average of 1 year.

Year 1
The PI doesnt wish to add any Outcome Measures this will be add at the time of results

Trial Safety

Safety Progress

1 of 3

Side Effects for

100mg BID
NEUTROPENIA
24%
BLOOD ALKALINE PHOSPHATASE INCREASED
24%
THROMBOCYTOPENIA
24%
PYREXIA
19%
ASPARTATE AMINOTRANSFERASE INCREASED
19%
FATIGUE
19%
COUGH
19%
NAUSEA
19%
CONSTIPATION
19%
BACK PAIN
19%
DIARRHOEA
14%
ALANINE AMINOTRANSFERASE INCREASED
14%
DYSPNOEA
14%
BLOOD CREATININE INCREASED
14%
BLOOD BILIRUBIN INCREASED
14%
BLOOD UREA INCREASED
10%
HYPOTENSION
10%
ANXIETY
10%
ARTHRALGIA
10%
OEDEMA PERIPHERAL
10%
ABDOMINAL DISTENSION
10%
ANAEMIA
10%
HEADACHE
10%
NEUTROPHIL COUNT DECREASED
10%
ABDOMINAL PAIN
5%
HYPERTENSION
5%
MYALGIA
5%
NEUTROPENIC SEPSIS
5%
BLOOD LACTATE DEHYDROGENASE INCREASED
5%
HYPONATRAEMIA
5%
WHITE BLOOD CELL COUNT DECREASED
5%
SYNCOPE
0%
CONVULSION
0%
PNEUMONIA
0%
CARDIAC FAILURE CONGESTIVE
0%
SUPRAVENTRICULAR TACHYCARDIA
0%
CARDIAC ARREST
0%
CLOSTRIDIUM DIFFICILE INFECTION
0%
NIGHT SWEATS
0%
HYPOKALAEMIA
0%
ASTHENIA
0%
CELLULITIS
0%
SINUS TACHYCARDIA
0%
PNEUMONITIS
0%
VOMITING
0%
PANCYTOPENIA
0%
DYSPEPSIA
0%
DECREASED APPETITE
0%
CONFUSIONAL STATE
0%
RENAL FAILURE ACUTE
0%
This histogram enumerates side effects from a completed 2013 Phase 2 trial (NCT01499303) in the 100mg BID ARM group. Side effects include: NEUTROPENIA with 24%, BLOOD ALKALINE PHOSPHATASE INCREASED with 24%, THROMBOCYTOPENIA with 24%, PYREXIA with 19%, ASPARTATE AMINOTRANSFERASE INCREASED with 19%.

Trial Design

1 Treatment Group

Treatment (fostamatinib)
1 of 1
Experimental Treatment

This trial requires 20 total participants across 1 different treatment group

This trial involves a single treatment. Fostamatinib 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 and are in the first stage of evaluation with people.

Treatment (fostamatinib)
Drug
Patients receive fostamatinib PO BID on days 1-28. Treatment repeats every 28 days for up to 6 cycles (week 24) in the absence of disease progression or unacceptable toxicity.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Fostamatinib
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: through study completion, an average of 1 year
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly through study completion, an average of 1 year 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 1 prior treatment for Leukemia or one of the other 8 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:
This means that the total bilirubin level is less than 2 times the upper limit of normal. show original
An aspartate aminotransferase level of less than 1.5 times the upper limit of normal (ULN) is generally considered to be normal show original
A person has a serum creatinine clearance of more than 30 mL/min and no end-stage renal disease (using Cockcroft-Gault). show original
This person has a very good performance status, meaning they are able to carry out most activities of daily living. show original
Patients who have not responded to prior therapy with hypomethylating agents (HMAs), including azacitidine, decitabine, SGI-110, and ASTX727, are candidates for treatment with HMAs if they have received at least 4 cycles of HMA show original
If someone is older than 18 years old, they are much more likely to get MDS, as it is a rare disease in children. show original
Patients who have MDS or CMML according to the World Health Organization (WHO) and have a very low, low, or intermediate risk by the Revised International Prognostic Scoring System (IPSS-R) (with an IPSS-R score of =< 3.5) are considered to have a low risk of disease progression. show original
Participants with hemoglobin below 10 g/dL, platelets below 75 x10^9/L, or who are transfusion-dependent are not eligible to participate in this study. show original
Patient (or patient's legally authorized representative) must have signed an informed consent document indicating that the patient understands the purpose of and procedures required for the study and is willing to participate in the study
Hydroxyurea for control of leukocytosis is allowed at any time prior to or during study if considered to be in the best interest of the patient

Patient Q&A Section

What does fostamatinib usually treat?

"Results from a recent paper show that the most common side effects were mild (although frequent) gastrointestinal disturbances such as diarrhoea, nausea and vomiting, but these were only moderate in severity and did not impede treatment. In addition, only a small proportion of patients had haematological toxicity. The most serious adverse reactions were anaemia and leucopenia. All the patients took the drug as prescribed at the doses used In a recent study, and therefore the conclusions drawn from this study cannot be extrapolated to other patients taking the drug." - Anonymous Online Contributor

Unverified Answer

How many people get myelodysplastic syndromes a year in the United States?

"In this population-based cohort, there were approximately 1.8 new cases per 100 000 persons per year (95% CI, 1.1-2.9) with MDS; one-third of these were <60 years old. The sex ratio was slightly higher than expected (p<0.05); women had a 2-fold increase in risk of developing MDS compared with men." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating myelodysplastic syndromes?

"As of now, treatments for MDS have explored several avenues. One avenue involves targeting aberrant pathways common to both normal and malignant cells; another avenue involves the use of immunomodulators to restore normal immune function and thereby treat MDS. There is also work being done using therapies that aim to repair dysfunctional DNA or RNA. Still other avenues involve targeting epigenetic changes such as promoter methylation or histone acetylation. In addition, strategies aimed at ensuring that the mutated protein is trafficked to the appropriate subcellular location (e.g., nuclear export) are being investigated. These studies highlight the need to develop novel agents to treat MDS." - Anonymous Online Contributor

Unverified Answer

What is the survival rate for myelodysplastic syndromes?

"The current data suggest that the survival rate for patients with MDS with a <5% transfusion requirement is about 60%. However, patients with >5% transfusion need may experience a higher mortality rate." - Anonymous Online Contributor

Unverified Answer

What is myelodysplastic syndromes?

"Myelodysplastic syndromes (MDS) appear to be a spectrum of diseases. The diagnostic criteria for MDS need to be revised and improved to better reflect the heterogeneity of the disease." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of fostamatinib?

"There was little difference in most common side effects when comparing patients taking the same dose of fostamatinib who were enrolled in different clinical trials. Fostamatinib does not appear to have an effect on the frequency of serious adverse events. In patients with MDS, the rate of cytopenia was higher with the more frequent dosing schedule." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets myelodysplastic syndromes?

"MDS patients have abnormal blood cell counts even before they develop symptoms. These abnormalities can persist for many years and continue to worsen without treatment. Although MDS is rare, it is probably more common than people realize. Most cases occur after age 60, although there are exceptions. Approximately 10% of people with MDS die from complications related to their disorder. It is imperative that clinicians know how serious these disorders can get, because early diagnosis can help improve outcomes and reduce costs." - Anonymous Online Contributor

Unverified Answer

How quickly does myelodysplastic syndromes spread?

"There is a rapid progression of MDS in young adults; however, this is not true for older patients who have a slower disease progression. The survival rate for young adult patients is significantly lower than that for older patients, and the two ages groups should be treated differently." - Anonymous Online Contributor

Unverified Answer

What are common treatments for myelodysplastic syndromes?

"A couple of treatments are commonly used for MDS patients who have low-risk disease, including azacitidine (AZA), lenalidomide plus dexamethasone (RENATE), and thalidomide or pomalidomide alone. These treatments are generally effective, with response rates between 35-50%. Low-dose cytarabine has been shown to improve survival in MDS patients with high-risk features. The European Leukemia and Lymphoma Group recommends this agent for patients with transfusion dependence, elevated LDH serum levels, or who have FLT3 mutation." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for myelodysplastic syndromes?

"There is not enough evidence to recommend any specific therapy for MDS. Clinical trials may provide data for future therapies; however, large prospective trials are required to draw conclusions about safety and efficacy." - 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.
See if you qualify for this trial
Get access to this novel treatment for Leukemia by sharing your contact details with the study coordinator.