ADI-PEG 20 for Leukemia

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
MD Anderson Cancer Center, Houston, TX
Leukemia+3 More
ADI-PEG 20 - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

This study is evaluating whether a drug combination can improve the outcome of people with AML.

See full description

Eligible Conditions

  • Leukemia
  • Adult Acute Myeloid Leukemia

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether ADI-PEG 20 will improve 1 primary outcome and 4 secondary outcomes in patients with Leukemia. Measurement will happen over the course of 6 months.

2 years
Determine preliminary evidence of tumor activity, per the revised 2017 European LeukemiaNet criteria
Determine the anti-drug antibodies of ADI-PEG 20 in combination with venetoclax and azacitidine
Determine the peripheral blood arginine levels of ADI-PEG 20 in combination with venetoclax and azacitidine
Determine the peripheral blood citrulline levels of ADI-PEG 20 in combination with venetoclax and azacitidine
6 months
Determine the RP2D of ADI-PEG 20 in combination with venetoclax and azacitidine, per the number of subjects with treatment-related adverse events by current CTCAE

Trial Safety

Safety Estimate

1 of 3

Trial Design

2 Treatment Groups

Previously Treated AML
1 of 2
Untreated AML With High Risk Features
1 of 2
Experimental Treatment

This trial requires 60 total participants across 2 different treatment groups

This trial involves 2 different treatments. ADI-PEG 20 is the primary treatment being studied. Participants will be divided into 2 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.

Previously Treated AML
Drug
Previously treated AML based on the revised 2017 European LeukemiaNet (ELN) criteria with age at least 18 years, and having ≥10% blasts in bone marrow or peripheral blood
Untreated AML With High Risk Features
Drug
Untreated AML per ELN criteria with high risk features, or age ≥ 65 years and ineligible for intensive chemotherapy because of older than 75 years, cardiac disease or prior anthracycline use or high probability of treatment-related mortality
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Azacitidine
FDA approved

Trial Logistics

Trial Timeline

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

Closest Location

MD 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 Leukemia or one of the other 3 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:
Lead In and Cohort 1
Previously treated AML based on the revised 2017 European LeukemiaNet (ELN) criteria and having ≥10% blasts in bone marrow or peripheral blood
Age ≥18 years
Cohort 2
Untreated AML per ELN criteria with high risk features
Age ≥ 65 years and ineligible for intensive chemotherapy because of older than 75 years, cardiac disease or prior anthracycline use or high probability of treatment-related mortality
Life expectancy reasonably adequate for evaluating the treatment
White blood cell (WBC) count of 10 × 109/L or less. (Use of hydroxyurea to control WBC is allowed till 48 hours prior to protocol treatment)
Adequate renal function: Creatinine ≤ 1.5 x upper limit of normal (ULN) or creatinine clearance > 40 mL/minute (measured or calculated according to the Cockcroft-Gault formula)
Adequate liver function

Patient Q&A Section

What is leukemia?

"Leukemia is a disease affecting the hematopoietic system which is caused by a genetic abnormality resulting in the uncontrolled proliferation of abnormal cells. It is usually diagnosed in people who are 20-50 years old. A person will develop leukemia once they accumulate enough genetic abnormalities in their DNA. Symptoms may not appear until the disease is advanced. Once a person has leukemia there isn't a cure, instead there are treatment options available to stop the disease. People with leukemia need to be monitored regularly to ensure that the cancer hasn’t returned. This monitoring includes regular blood counts and bone marrow examinations. People with ALL often start getting treatments before they have symptoms, whereas those with chronic leukemia are more likely to get treatment after symptoms appear." - Anonymous Online Contributor

Unverified Answer

What is the average age someone gets leukemia?

"Most people get leukemia between ages 20 and 40, and about 1 in 10 adults has the disease. People who are older or younger than 20 or 40 years old routinely get it. Men are more than twice as likely to get leukemia as women. Most cases occur in whites, Hispanics, and blacks. Most cases of acute leukemia affect children under age 15; however, at least one third of all people who get leukemia are over age 70." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of adi-peg 20?

"The most important side effect was headache (>50%). Other side effects were vomiting (3%), diarrhoea (2%), abdominal pain (1%), constipation (1%), nausea (1%) and prolonged bleeding (≤1%). Adi-peg 20 seems to be well tolerated and safe. Some of the side effects observed may be related to the drug itself, while others may be due to the concomitant use of other drugs." - Anonymous Online Contributor

Unverified Answer

Can leukemia be cured?

"Results from a recent clinical trial suggests that it is possible to cure leukemia if the patient's disease goes into remission before the patient is treated for their leukemia. However, in some leukemias even then, the cancer regrows. There are no cures available for any type of leukemia currently. Only treatments exist for patients who have received allopurinol and/or other drugs which can help reduce the growth and spread of the disease. Thanks to our doctors and scientists working hard to find cures for this devastating disease, many more people will survive their disease than would otherwise." - Anonymous Online Contributor

Unverified Answer

How serious can leukemia be?

"The seriousness of leukemia can vary from case to case. Patients must be informed about their prognosis before treatment begins. Many patients receive chemotherapy, which may lead to more serious side effects than it protects against serious illness. Those who are treated early may recover without any long term side effects. People with leukemia are generally at risk for developing life-threatening infections such as pneumonia and cytomegalovirus infection." - Anonymous Online Contributor

Unverified Answer

What are the signs of leukemia?

"Leukocyte count is an important parameter for diagnosis and prognosis in patients with leukemias; however, it is not sufficient to diagnose AML and ALL. Bone marrow examination for morphology and cytogenetics is essential for diagnosis of leukemias. In order to increase diagnostic accuracy of bone marrow examinations, PCR-based techniques should be developed for early detection and accurate classification of AML and ALL." - Anonymous Online Contributor

Unverified Answer

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

"There are approximately 1.5 million new cases of sporadic acute leukemias and 2.0 million cases of chronic leukemias every year in the U.S. About 40% of newly diagnosed cases of all types of acute leukemias are ALL, 30% are AML, and 20% are CLL/SLL. Approximately 80% of newly diagnosed cases of ALL are under age 45, 15% are >45, and 5% are aged ≥65 years. Differentiation between ALL and AML is important for clinical management and the choice of therapeutic regimen. AML is more common among males than females." - Anonymous Online Contributor

Unverified Answer

Is adi-peg 20 typically used in combination with any other treatments?

"Adi-peg 20 has been shown to be effective in inducing complete remissions in patients with relapsed or refractory MM. This finding suggests that it may have similar utility in combination with treatments like autologous stem cell transplantation, bortezomib and lenalidomide." - Anonymous Online Contributor

Unverified Answer

How does adi-peg 20 work?

"Results from a recent paper demonstrated excellent efficacy and tolerability of Adi-peg 20 compared to placebo in achieving complete tumor response in patients with metastatic breast cancer. Adi-peg 20 is an effective and well-tolerated option for the treatment of advanced or metastatic breast cancer." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving adi-peg 20?

"Recent findings indicate that the Lisdexamfetamine/PEG-20 combination has a favorable safety profile, with minimal side effects. Overall, 56% (18/32) of patients experienced improvements in their symptoms compared to baseline. None of these data support the use of PEG-20 alone as a novel treatment for ANL. However, further study into this drug pairing should be considered." - Anonymous Online Contributor

Unverified Answer

Has adi-peg 20 proven to be more effective than a placebo?

"We conclude that peg-adacomab 20 mg/m(2) given every three weeks for the first 12 doses plus once weekly thereafter was superior to a placebo as measured by PFS and OS in patients with refractory or relapsed AL amL who received the drug according to the standard of care. Adacomab 20 provided significant activity against peripheral blasts in some patients." - 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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