US Study of UM171-Expanded CB in Patients With High Risk Leukemia/Myelodysplasia

Launched by EXCELLTHERA INC. · Sep 24, 2019

Keywords

ClinConnect Summary

This clinical trial is studying a new approach to help patients with high-risk blood cancers, like acute leukemia and myelodysplastic syndrome, who need a cord blood transplant. Cord blood transplants are often a good option for patients who don’t have a matching donor, but they can have challenges like low cell counts and longer recovery times. The trial is testing a treatment called UM171, which can help grow more stem cells from cord blood, potentially leading to a safer and more effective transplant. Previous studies showed that patients who received a UM171-expanded cord blood transplant had good recovery rates and low risks of severe complications.

To be eligible for this trial, patients must have specific types of high-risk blood cancers, such as acute myeloid leukemia or myelodysplastic syndrome, and need to have at least two cord blood units that are fairly well-matched to their tissue type. Participants will receive the UM171 treatment and will be monitored closely to see how well they respond to the transplant. Overall, this trial aims to improve outcomes for patients facing serious blood cancers, especially those who have not had success with other treatments. If you think you or a loved one might be eligible or have questions, it's important to discuss this with your healthcare provider.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• 1. High and very high-risk hematologic malignancy defined as:
• • 1. Acute Myeloid Leukemia (Primary induction failure, Chemorefractory relapse, Relapse after allogeneic or autologous transplant, High risk AML in CR1, ≥ CR2)
• • 2. Acute Lymphoid leukemia (Primary induction failure, High risk ALL in CR1, ≥ CR2, Chemorefractory relapse, Relapse after allogeneic or autologous transplant)
• • 3. Myelodysplastic syndrome (Relapse after allogeneic or autologous transplant, ≥10% blasts within 30 days of start of conditioning regimen, Poor and very poor cytogenetics abnormalities, CMML with HCT-specific CPSS score high or intermediate-2, Stable disease, Progressive disease while on azacitidine).
• • 4. Chronic myelogenous leukemia (Patients who progressed to blast crisis)
• • 2. Availability of 2 CBs ≥ 4/6 HLA match with pre-freeze CD34+ cell count ≥0.5 x 10E5/kg and TNC≥1.5 x 10E7/kg
• • 3. Karnofsky ≥70.
• • 4. LVE fraction ≥ 40% or fractional shortening \>22%
• • 5. FVC, FEV1 and DLCOc ≥ 50% of predicted
• • 6. Bilirubin \< 2 x ULN; AST and ALT ≤ 2.5 x ULN; alkaline phosphatase ≤ 5 x ULN.
• • 7. Creatinine \< 2.0 mg/dl.
• • 8. HCT-CI ≤3 if patients have ≥5% blasts in the bone marrow and HCT-CI ≤5 if 60-65 years old.
• Exclusion Criteria:
• • 1. Allogeneic myeloablative transplant within 6 months.
• • 2. Autologous hematopoietic stem cell transplant within 6 months.
• • 3. Active or recent invasive fungal infection.
• • 4. Presence of a malignancy other than the one for which the UCB transplant is being performed and the expected survival related to the malignancy is estimated to be less than 75% at 5 years.
• • 5. HIV positivity.
• • 6. Hepatitis B or C infection with measurable viral load.
• • 7. Liver cirrhosis.
• • 8. Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
• • 9. Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient condition or study outcome.
• • 10. Active central nervous system involvement.
• • 11. Chloroma \> 2 cm.