Darbepoetin Alfa for Myelodysplastic Syndromes

Phase-Based Estimates
1
Effectiveness
1
Safety
Moffitt Cancer Center, Tampa, FL
Myelodysplastic Syndromes+2 More
Darbepoetin Alfa - Drug
Eligibility
18+
All Sexes
Eligible conditions
Myelodysplastic Syndromes

Study Summary

This study is evaluating whether a drug called canakinumab can be used to treat people with lower-risk myelodysplastic syndrome (MDS) who have failed prior treatment with an erythropoietin stimulating agent (ESA

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Eligible Conditions

  • Myelodysplastic Syndromes
  • Syndrome
  • Preleukemia

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Darbepoetin Alfa will improve 2 primary outcomes and 6 secondary outcomes in patients with Myelodysplastic Syndromes. Measurement will happen over the course of at 24 weeks per cohort.

Week 12
Phase 2: Rate of Hematologic Improvement-Erythroid (HI-E) response
Month 12
Phase 1b and Phase 2: Duration of Hematologic Improvement-Erythroid (HI-E) response
Up to 60 months
Phase 2: Duration of Response
Phase 2: Overall Response Rate (ORR)
Phase 2: Overall Survival (OS)
Phase 2: Progression Free Survival (PFS)
Week 24
Phase 1b and Phase 2: Degree in reduction of PRBC Transfusions
Day 28
Phase 1b: Maximum Tolerated Dose (MTD)

Trial Safety

Trial Design

3 Treatment Groups

No Control Group
Phase 2: Treatment at Maximum Tolerated Dose

This trial requires 41 total participants across 3 different treatment groups

This trial involves 3 different treatments. Darbepoetin Alfa is the primary treatment being studied. Participants will be divided into 3 treatment groups. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.

Phase 2: Treatment at Maximum Tolerated DosePatients will be treated with Darbepoetin alfa subcutaneously at a dose of 300 mg on days 1 and 15 of each cycle plus the maximum tolerated dose of Canakinumab.
Phase 1b: Dose Level 2Patients will be treated at dose level 2: Canakinumab 300 mg by subcutaneous injection on day 1 of each 28 day cycle. Darbepoetin alfa will be administered subcutaneously at a dose of 300mg on days 1 and 15 of each cycle.
Phase 1b: Dose Level 1Patients will be treated at dose level 1: Canakinumab 150 mg by subcutaneous injection on day 1 of each 28 day cycle. Darbepoetin alfa will be administered subcutaneously at a dose of 300mg on days 1 and 15 of each cycle.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Darbepoetin alfa
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 60 months
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 60 months for reporting.

Closest Location

Moffitt Cancer Center - Tampa, FL

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 6 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Patients must be transfusion dependent, defined as requirement for transfusion of at least 3 units of Packed Red Blood cells (PRBCs) 16 weeks for a Hgb<9.0g/dL or, in non-transfusion dependent patients (<3 units of PRBCs transfused in the preceding 16 weeks), must have a baseline Hgb of <9.0 g/dL at time of study enrollment
Women of child bearing potential must have negative urine or serum pregnancy test within 28 days prior to start of study drug.
Women of child bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence; tubal ligation, partner's vasectomy) prior to Cycle 1 Day 1 and for the duration of study participation.
Adequate organ function as defined by laboratory values per protocol
Documented diagnosis of MDS by World Health Organization (WHO) criteria, further meeting the following criteria according to disease risk classification
Eastern Cooperative Oncology Group (ECOG) Performance Status </=2.

Patient Q&A Section

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

Who should consider clinical trials for myelodysplastic syndromes?

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Clinical trials represent a useful tool to improve our knowledge and, ultimately, our therapeutic practice and, especially in this field, to provide individualized treatment to cancer patients in need.

Unverified Answer

What is myelodysplastic syndromes?

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MDS is a serious disease caused by the production and development of an abnormal number of blood cells by the bone marrow. A number of clinical subtypes occur, and they can be classified loosely based on their pathogenesis, cytogenetics, mutational abnormalities and clinical presentation. There is considerable ambiguity in the vocabulary to describe MDS. A large number of terms have been used in a wide variety of contexts. It is time for consensus and a definite terminology.

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What causes myelodysplastic syndromes?

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A [previous] diagnosis of myelodysplastic syndrome (MDS) does not appear to be an important risk factor for the development of PMF/HM. In MDS, [acquired] cytogenetic abnormalities, increased numbers of circulating blasts and high risk cytogenetic abnormalities are potential risk factors for PMF. Other potential risk factors include smoking and radiotherapy; however, these factors do not appear to account for the higher risk for PMF after previous MDS.

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What are the signs of myelodysplastic syndromes?

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The symptoms of MDS usually begin before the age of 30 years. There is not a specific laboratory test to diagnose MDS. Other symptoms may resemble those of MDS. The mainstay of all three MDSs appears to be marrow blasts as a result of hematopoietic stem cell dysfunction. This may result in symptoms which are not caused by marrow failure, but involve a decreased capacity to produce blood cells. Other causes of abnormal blood cells include aplastic anemia, thalassemia, and sickle cell disease. MDS is an enigmatic disorder that cannot be diagnosed before its symptoms begin. A diagnosis must be made on the basis of history and clinical findings.

Unverified Answer

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

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There are approximately 4,000 deaths due to MDS in the US every year, excluding deaths on diagnosis due to the disease. The lifetime risk of acquiring MDS is 1/70.5 (2/3.45) and 1/70 (3/3.45) respectively in Caucasians (C) and African-Americans (AA). In AA, the lower risk may reflect the lower prevalence of MDS in the AA population (i.e., a greater genetic predisposition for the disease).

Unverified Answer

What are common treatments for myelodysplastic syndromes?

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The treatment of myelodysplastic syndromes often consists of induction chemotherapy, consolidation chemotherapy and maintenance therapy. The goal of these treatments is to improve long-term survival. Unfortunately most myelodysplastic syndromes respond poorly to induction chemotherapy, even in cases where marrow blasts are normal.

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Can myelodysplastic syndromes be cured?

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Overall survival in MDS is low. In the absence of curative treatment, survival is mainly affected by the presence or absence of transformation. Survival is higher if patients with MDS without transformation have long-term survival. We demonstrated the possibility to cure 5% to 7% of patients with MDS.

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What is the average age someone gets myelodysplastic syndromes?

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Most cases of MDS have been estimated to arise on average at the age of 40 - 49 years old. However, some studies found that the mean age of diagnosis is approximately 61 years old. The difference is likely due to age of onset instead of age of diagnosis. The prevalence of MDS increases with age by about 1.02% for every 10-year increase in age.

Unverified Answer

Have there been other clinical trials involving darbepoetin alfa?

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The most common indication for darbepoetin alfa therapy is prophylaxis for anemia associated with chemotherapy in patients with MDS, and other uses for the drug are still being evaluated. To date, the drug has not been shown to improve outcomes in the treatment of MDS. If darbepoetin alfa proves effective for prophylaxis of chemotherapy-related anemia, then it will likely be the only drug available for this indication. Because the use of darbepoetin alfa is new, there is no available clinical evidence on which to base risks and benefits assessments. There may be potential benefits of this drug.

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Has darbepoetin alfa proven to be more effective than a placebo?

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In a large, double-blind, randomized, placebo-controlled study of darbepoetin alfa in chronic IBD, there was neither significant difference in efficacy nor safety in clinical outcomes compared with a placebo; the results are inconclusive.

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How does darbepoetin alfa work?

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There are some unanswered questions related to efficacy of ESA in patients with MDS following the initiation of ESA therapy, particularly in regards to whether patients with myelodysplastic syndromes respond differently to Epo than those with normal karyotypes. Results from a recent paper demonstrates that the use of darbepoetin alfa is associated with a high rate of safety. Despite some concerns regarding its mechanism of action, darbepoetin alfa appears to have the requisite immunomodulatory properties to be efficacious for the treatment and prolongation of MDS.

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What are the common side effects of darbepoetin alfa?

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At least 1.5% of patients have one or more adverse events during therapy with darbepoetin alfa. Patients should monitor for any unusual signs/symptoms that change over time. In certain situations (e.g., chemotherapy), routine laboratory testing with darbepoetin alfa should be undertaken before initiating therapy for treatment of anemia.

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