Tafasitamab (MOR00208) in Pediatric Patients With Relapsed or Refractory Acute B Lineage Leukemia

Prior treatment

• The following immunosuppressive drugs (≥ 1 week of administration):
  • steroids ≥ 1mg/kg body weight, cytostatics (except intrathecal/ intracerebroventricular application for CNS treatment)
• Application of other experimental therapy modalities in the last 4 weeks
• Subjects that do not agree to refrain from donating blood while on study drug
• Concurrent severe or uncontrolled medical disease which by assessment of the treating physician could compromise participation in the study

Other illness

• Severe infection (HIV, Chronic active viral hepatitis), tests have to be conducted at screening
• Significant psychiatric disabilities, uncontrolled seizure disorders or severe peripheral neuropathy/ leukoencephalopathy
• Signs of autoimmune disease (i.e. idiopathic thrombocytopenic purpura, autoimmune hemolytic anemia)
• History of hypersensitivity to the investigational medicinal product or to any drug with similar chemical structure or to any excipient present in the pharmaceutical form of the investigational medicinal product.

Lab values

• Ejection fraction \<25% on echocardiography
• Cystatin C-clearance \<40ml/min
• Liver function abnormalities with bilirubin \>4 mg/dL and elevation of transaminases higher than 400 U/L

Other

• Frank relapse (\>5% leukemic blasts)
• Philadelphia chromosome-positive (Ph+) ALL
• Acute GvHD III-IV or extensive chronic GvHD
• Women during pregnancy and lactation

• Age ≥ 3 years and \< 18 years at enrollment
• B-lineage (CD19 positive) ALL (B, pro-B, pre-B or c-ALL)
• Patients must have either
  • underwent a first allogeneic stem cell transplantation after relapse with one of the following very high-risk somatic molecular alterations:
  • KMT2A::AFF1 \[t(4;11) rearrangement
  • TP53 alteration (mutation/deletion)
  • low hypodiploidy (\<40 chromosomes, evident or masked)
  • TCF3-PBX1 \[t(1;19)\]
  • TCF3::HLF \[t(17;19)\] and irrespective of MRD after SCT or
  • underwent a first allogeneic stem cell transplantation or a CAR T-cell therapy with newly emerging or persistent MRD load posttransplant / post CAR T- cell-treatment or
  • have received stem cell transplantation without having reached a sufficient molecular remission prior to transplant (defined as MRD ≥10E-4) irrespective of MRD after SCT or
  • underwent a second or subsequent allogeneic stem cell transplantation irrespective of MRD after SCT
• Females of childbearing potential (FCBP1) must agree
  • to utilize two reliable forms of contraception simultaneously or practice complete abstinence from heterosexual contact for at least 3 months before starting study drug, while participating in the study (including dose interruptions), and for at least 3 months after study treatment discontinuation and must agree to regular pregnancy testing during this timeframe
  • to abstain from breastfeeding during study participation and 3 months after study drug discontinuation.
• Males must agree
  • to use a latex condom during any sexual contact with FCBP while participating in the study and for 3 months following discontinuation from this study, even if he has undergone a successful vasectomy
  • to refrain from donating semen or sperm during study participation and for 3 months after discontinuation from this study treatment.