DOROCUBICEL; ALLOGENEIC UMBILICAL CORD-DERIVED CD34- CELLS, NON-EXPANDED for Acute leukemia | Myelodysplastic syndrome

Inclusion criteria

• {"criterion_text":"- 18-70 years old"}
• {"criterion_text":"- Presence of a high and very high-risk hematologic malignancy"}
• {"criterion_text":"- Availability of 2 CBs ≥ 4/6 HLA match when DRB1 is performed at the allele level and A, B at antigen resolution (intermediate resolution) and ≥ 4/8 HLA match when A, B, C and DRB1 are performed at the allele level. An acceptable alternative would be a 3/6 or 5/8 as long as there is no double DRB1 mismatch."}
• {"criterion_text":"- Karnofsky ≥70."}
• {"criterion_text":"- Left ventricular ejection fraction ≥ 40% (within 3 months unless patient has received chemotherapy or radiation therapy to the thorax since the last cardiac evaluation) or fractional shortening >22%"}
• {"criterion_text":"- Forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1) and diffusing capacity corrected for hemoglobin (DLCOc) ≥ 50% of predicted"}
• {"criterion_text":"- Bilirubin < 2 x upper limit of normal (ULN) unless felt to be related to Gilbert’s disease or hemolysis; AST and ALT ≤ 2.5 x ULN; alkaline phosphatase ≤ 5 x ULN."}
• {"criterion_text":"- Adequate renal function defined as creatinine < 2.0 mg/dl (adults). All adults with a creatinine > 1.2 or a history of renal dysfunction must have estimated creatinine clearance > 50 ml/min."}
• {"criterion_text":"- Hematopoietic cell transplantation specific comorbidity index (HCT-CI) ≤3 if patients have ≥5% blasts in the bone marrow and HCT-CI ≤5 if 60-70 years old."}

Exclusion criteria

• {"criterion_text":"- Allogeneic myeloablative transplant within 6 months."}
• {"criterion_text":"- Liver cirrhosis."}
• {"criterion_text":"- Pregnancy, breastfeeding or unwillingness to use appropriate contraception."}
• {"criterion_text":"- Participation in a trial with an investigational agent within 30 days prior to entry in the study."}
• {"criterion_text":"- 3) Active or recent (prior 6 month) invasive fungal infection without ID consult and approval."}
• {"criterion_text":"- Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and tests."}
• {"criterion_text":"- Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient condition or study outcome."}
• {"criterion_text":"- Active central nervous system involvement."}
• {"criterion_text":"- Chloroma > 2 cm."}
• {"criterion_text":"- Autologous hematopoietic stem cell transplant within 6 months."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- HIV positivity."}
• {"criterion_text":"- Hepatitis B or C infection with measurable viral load."}

Primary endpoints

• {"endpoint_text":"- Evaluate relapse-free survival at 1- and 2-years post-transplant","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Kinetics of hematopoietic engraftment (Neutrophil engraftment and Platelet engraftment)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Transplant related mortality (TRM)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of acute and chronic GVHD","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of grade 3 or higher infectious complications","definition_or_measurement_approach":""}
• {"endpoint_text":"- Incidence of pre-engraftment and engraftment syndrome requiring therapy","definition_or_measurement_approach":""}

Netherlands

Earliest CTIS Part Ii Submission Date

05-11-2024

Latest Decision Or Authorization Date

29-11-2024

Processing Time Days

24

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Excellthera Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Canada