Marqibo for Leukemia

1
Effectiveness
1
Safety
All Children's Hospital, Saint Petersburg, FL
Leukemia+6 More
Marqibo - Drug
Eligibility
< 65
All Sexes
Eligible conditions
Leukemia

Study Summary

This study is evaluating whether a combination of chemotherapy drugs may help treat acute lymphoblastic leukemia.

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Eligible Conditions

  • Leukemia
  • Leukemia, Lymphocytic, Acute, L1
  • Acute Disease
  • Leukemia, Lymphoid
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • ALL, Childhood
  • Lymphoblastic Leukemia, Acute, Childhood
  • Acute Lymphoblastic Leukemia (ALL)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

This trial is evaluating whether Marqibo will improve 1 primary outcome and 1 secondary outcome in patients with Leukemia. Measurement will happen over the course of 5 weeks.

5 weeks
Number of Participants with Dose Limiting Toxicities as a Measure of Safety and Tolerability
Approx. 8 weeks
The response rate after treatment.

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

3 Treatment Groups

No Control Group
Cohort C: Marqibo and maintenance regimen

This trial requires 36 total participants across 3 different treatment groups

This trial involves 3 different treatments. Marqibo 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 and are in the first stage of evaluation with people.

Cohort C: Marqibo and maintenance regimen
Drug
Marqibo®: given by intravenous (IV) infusion on day 1 Dexamethasone orally twice daily on days 1-5 Methotrexate: given orally on days 1 and 8 Mercaptopurine: given orally daily on days 1-13
Cohort A: Marqibo and UK ALL R3 backbone
Drug
Marqibo®: given by intravenous (IV) infusion on days 1, 8, 15 and 22. Dexamethasone orally twice daily on days 1-5 and 15-19. Mitoxantrone: given by intravenous (IV) infusion on days 1 and 2. PEG-asparaginase: given as an injection into the muscle on says 3 and 17. Methotrexate IT: given intrathecally (used to treat the brain and spinal cord and is given using a needle inserted into the spinal canal) on days 1 and 8.
Cohort B: Marqibo and lower intensity UK ALL R3 backbone
Drug
Marqibo®: given by intravenous (IV) infusion on days 1, 8, 15 and 22. Dexamethasone orally twice daily on days 1-5 and 15-19. PEG-asparaginase: given as an injection into the muscle on days 3 and 17. Methotrexate IT: given intrathecally (used to treat the brain and spinal cord and is given using a needle inserted into the spinal canal) on days 1 and 8.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Vincristine
FDA approved

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: approx. 8 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly approx. 8 weeks for reporting.

Closest Location

All Children's Hospital - Saint Petersburg, FL

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. You must have received 2 prior treatments for Leukemia or one of the other 6 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:
Patients must have recovered from the acute toxic effects (≤ Grade 2 or baseline) of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study, unless otherwise specified show original
Age
-Patients must have a confirmed diagnosis of T-cell ALL show original
Diagnosis
Patients in cohort A must have a diagnosis of ALL or lymphoblastic lymphoma, and must have ≥ 5% blasts in the bone marrow (M2 or M3), with or without extramedullary disease. show original
Cohorts A, B, & C: Patients must have a diagnosis of acute lymphoblastic leukemia (ALL), lymphoblastic lymphoma, or mixed phenotypic acute leukemia with any level of detectable disease. show original
The performance level for patients is classified as Karnofsky > 50% forpatients aged 16 years or older and Lansky > 50% for patients aged 16 years or younger. show original
I have had prior therapy and it was helpful show original
People who have had a first remission and have then either relapsed or not responded to treatment may be candidates for this study. show original
Patients with Philadelphia chromosome t(9;22) positive disease must have received at least two prior tyrosine kinase inhibitors.

Patient Q&A Section

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

How many people get leukemia a year in the United States?

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Although the U.S. has become an advanced country for cancer treatment, there are many survivors of leukemia and lymphoma who have suffered long-term side effects as a result of the treatments.

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What are the signs of leukemia?

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Certain signs, such as rash, lymph node enlargement, low white blood cell count, or high platelet count, as well as headaches (hemorrhages in the head) are suggestive of leukemia. Some signs point to specific diagnoses, such as fever (fever is rare in leukemia), but most commonly points to leukemia. The most common form is B cell leukemia at about 20%. All forms can show skin pigment changes. The signs of leukemia may change on the course of the disease.\n

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What is leukemia?

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Leukemia is a group of blood disorders caused by abnormal proliferation of a group of blood-forming or immaturing blood cells. The most common type is the myelogenous leukemia. The disease is usually fatal. It is estimated that in 2008 there were over 100,000 cases of newly diagnosed acute myeloid leukemia in the US.

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Can leukemia be cured?

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While leukemia can be cured, with appropriate treatment it can result in an extremely prolonged remission that may be followed by prolonged periods of disease-free remission. However, it remains to be seen whether these patients can achieve a full cure.

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What are common treatments for leukemia?

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Leukemia is often treated with induction chemotherapy and then with consolidation chemotherapy; additionally, some patients may be eligible for allogeneic bone marrow transplantation (BMT) with or without ongoing chemotherapy. Radiation therapy may be used to treat localized leukemia. In addition, many patients with leukemia require long-term immunoglobulin replacement.

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What causes leukemia?

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In patients with leukemia, a significant portion of cases occur spontaneously without a known cause. The most common types of leukemia include chronic myelogenous, acute myelogenous and acute lymphocytic.

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Does marqibo improve quality of life for those with leukemia?

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Marqibo can improve QOL among persons with [chronic pain](https://www.withpower.com/clinical-trials/chronic-pain) and/or cancer. In addition to providing improved pain-related and other QOL, marqibo usage reduced anxiety. Marqibo should be considered in the care of people with chronic health conditions and cancer.

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What is the average age someone gets leukemia?

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Approximately every three seconds, a baby is born with leukemia. Typically, those who are newborns are the most commonly diagnosed at 3 to 5 years. On average, one leukemia death is seen each day in the United States, and approximately 70,000 people are diagnosed in the U.S. each year during their lifetime.

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What is the latest research for leukemia?

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A recent study focused interest on the epigenetic factors which contribute to malignancy. A study conducted by a group of researchers from the Department of Cancer Medicine, The Royal Marsden Hospital in London analyzed the epigenetic profiles of blood cells of leukemia patients and healthy individuals. These authors found that epigenetic aberrant changes of microRNAs, DNA methylation and histone modifications participate in leukemogenesis or progression of leukemia cells and may offer a new approach for diagnosis and prevention of leukemia.

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How does marqibo work?

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The study shows the use of marqibo to be safe, a low-cost and convenient way to treat and prevent leukemias and lymphomas in most cases. Data from a recent study of the treatment were also encouraging for the patients.

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How quickly does leukemia spread?

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Most patients diagnosed with ALL or AML can expect a complete remission with standard-of-care therapy. There are, however, people who are misdiagnosed, ie, where they are misdiagnosed with other conditions such as acute bacterial infection, sarcoidosis, or tuberculosis. If misdiagnosed they may not receive appropriate treatment, and can become resistant to standard-of-care treatment. These patients must be identified as early as possible in order to ensure that they receive the most effective treatment available. Patients with ALL and AML who require aggressive therapy (courses of a higher dose, longer treatment duration, or inpatient therapy) have higher levels of infection and bleeding.

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Who should consider clinical trials for leukemia?

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There are many factors that make patients more likely to participate in a clinical trial for both acute and chronic leukemias, including having the disease that is being investigated, previous treatment, and previous participation in trial. Patients receiving more intensive chemotherapy and blood-transfusions tended to have lower participation rates over all. Clinical trial results provide patients with information that could change their treatment decisions, but there are significant risks. Many patients and families cannot be adequately informed to participate in these studies or are not even aware of the study. Thus they are left to their own devices. When considering clinical trials, patients and families should be informed of the risks and benefits of participation before initiating treatment.

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