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About
This research study is studying a new drug, uproleselan, to see if it is safe and effective in decreasing relapse after stem cell transplant and improving leukemia-free survival in pediatric patients with acute myeloid leukemia (AML).
The name of the study drugs involved in this study are:
Full description
This is a single arm, multi-center, phase 1/2 trial involving the use of the study drug, uproleselan, as part of the pre stem cell transplant conditioning regimen for pediatric patients with acute myeloid leukemia (AML). This study is looking to learn what dose of uproleselan should be given and the safety of uproleselan when combined with other drugs as part of the pre stem cell transplant conditioning regimen.
The U.S. Food and Drug Administration (FDA) has not approved uproleselan as a treatment for any disease. This is the first time that uproleselan will be given to children. Uproleselan is expected to treat acute myeloid leukemia (AML) by making AML cells sensitive to chemotherapy drugs that are part of standard of care pre-transplant conditioning regimen which could help make the transplant more effective..The standard of care conditioning regimen will include the drugs busulfan, clofarabine, and fludarabine. The standard of care drugs tacrolimus, and either methotrexate or mycophenolate mofetil will be used during the stem cell transplant course.
The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits.
Participants will receive study drug doses for 7 days before their stem cell transplant and will be followed for 2 years following their stem cell transplant.
It is expected that about 28 people will take part in this research study.
GlycoMimetics, Inc., a pharmaceutical company, is supporting this research study by providing the study drug (uproleselan) and funding for some of the laboratory tests.
On 7/26/24 the Sponsor-Investigator was notified GlycoMimetics, Inc. was terminating contracting for NCT05569512 following company restructuring. One subject was enrolled on the study prior to termination. The study did not proceed from Phase 1 to Phase 2.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Age ≥12 months and ≤ 39 years
Lansky/Karnofsky performance status ≥70% (see Appendix A)
Weight ≥10 kg
Acute myeloid leukemia that arises de novo or is secondary to:
Disease status: Multidimensional flow cytometry (MDF) to assess disease status for eligibility will be performed centrally by Hematologics.
This sample will be used for eligibility as well as correlative biomarkers. Please see section 9.2 for details regarding collection, processing, and shipping of the sample.
Graft and Donor Types:
Ability to understand and/or the willingness of their parent or legally authorized representative to sign a written informed consent document.
Exclusion criteria
Patients with neutropenia due to disease related marrow dysfunction (refractory disease, underlying myelodysplasia or an underlying marrow failure disorder) will be eligible regardless of the absolute neutrophil count. However, enrolling centers must provide clear evidence that the neutrophil count is not rising, that the patient does not have an inadequately controlled infection (see section 3.2.15), and that the patient is on broad anti-fungal prophylaxis. Given the serious risk associated with starting conditioning in patients with severe neutropenia, centers are encouraged to delay transplant if they have any reason to believe that the absolute neutrophil count may improve.
Estimated GFR of <60 mL/min/1.73 m2. Estimated GFR may be calculated using the CKD-EPI Creatinine Equation (2009) for patients ≥19 years or creatinine-based Bedside Schwartz equation (2009) for patients <19 years. It is recommended that estimates be determined using the calculators found on the National Kidney Foundation website. the (https://www.kidney.org/professionals/KDOQI/gfr_calculator). Any patient for whom these equations yields a GFR less than 90 mL/min/1.73 m2 should have radionucleotide testing. Measurement of 24-hour urine creatinine clearance is not an acceptable substitute for radionucleotide testing.
Cardiac ejection fraction <50% or shortening fraction <27%
Total bilirubin (with elevated direct bilirubin) or ALT >2 X ULN.
Pulmonary disease with FVC, FEV1 or DLCO (corrected for hemoglobin) <50 % predicted or requiring supplemental oxygen. Children who are developmentally unable to perform pulmonary function testing will be assessed solely on their need for supplemental oxygen
Active hepatitis B or C infection
Active, poorly controlled infections. In patients being treated for infection at the time of enrollment, source documentation of the results of all microbiologic, radiographic and pathology assessments performed for diagnosis and for evaluation of response to treatment will be required.
Patients with a known history of HIV are excluded, unless they meet all of the following conditions:
Patients who have received another investigational drug within 28 days or 5 half-lives (whichever is longer).
Primary purpose
Allocation
Interventional model
Masking
1 participants in 1 patient group
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Central trial contact
Mary Kate Czepiel; Malika Kapadia, MD
Data sourced from clinicaltrials.gov
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