Patient case: 36-year-old female with Brugada syndrome; genetic feature: SCN5A

Clinical trial: Fractionated Gemtuzumab Ozogamicin in Treating Measurable Residual Disease in Participants With Acute Myeloid Leukemia

Summary: This phase II trial studies how well fractionated gemtuzumab ozogamicin works in treating measurable residual disease in participants with acute myeloid leukemia. Antibody-drug conjugates, such as gemtuzumab ozogamicin, may block cancer growth in different ways by targeting certain cells.

Detailed description: Participants receive gemtuzumab ozogamicin intravenously (IV) on days 1, 4, 7. Treatment continues for 35 days in the absence of disease progression or unacceptable toxicity. Non-responders without significant adverse events during the first course and responders, may receive a second course of gemtuzumab ozogamicin within 60 days after course 1.
  After completion of study treatment, participants are followed up for 6 months.

Eligibility: Inclusion Criteria:
  -  Prior diagnosis of AML based on 2016 World Health Organization criteria.
  -  Patients must have MRD-level disease only and otherwise meet criteria for complete response (CR) or complete remission with incomplete hematologic recovery (CRi) per the 2017 European Leukemia Net response criteria (< 5% blasts in the marrow without a requirement for peripheral blood count recovery). MRD must be measurable by multiparameter flow cytometry (MPFC) and/or polymerase chain reaction (PCR)-based molecular markers and/or karyotypic markers (e.g., classical cytogenetics or fluorescence in situ hybridization). MRD status will be centrally confirmed by the UW/FHCRC clinical laboratory in order to standardize response assessment following administration of study therapy.
  -  Patients must have received at least 1 cycle of standard induction chemotherapy prior to enrollment on the study. However, adult patients (>= 18 years of age) are eligible for participation at any time point in treatment (after induction, during or after consolidation, pre-transplant, or post-transplant). Pediatric patients (2-18 years of age) must have MRD positivity during/after consolidation or post-transplant.
  -  Eastern Cooperative Oncology Group (ECOG) performance status =< 3 (for adults) or Lansky performance status >= 40 (for children).
  -  Patient's AML blasts must have CD33 expression.
  -  For adults (>= 18 years of age): Serum creatinine =< 2.0 mg/dL.
  -  For adults (>= 18 years of age): Total bilirubin =< 2 x institutional upper limit of normal for age (unless known history of Gilbert's disease).
  -  For adults (>= 18 years of age): Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 2.5 x institutional upper limit of normal for age (unless thought to be related to resolving infectious complications).
  -  For children (< 18 years of age): Glomerular filtration rate (GFR) in ml/min (age 2: 63-175; age 3-12: 89-165; females 13 and older: 75-115; males 13 and older: 85-125).
  -  For children (< 18 years of age): Total bilirubin =< 2 x institutional upper limit of normal for age (unless known history of Gilbert's disease).
  -  For children (< 18 years of age): AST and ALT < 2.5 x institutional upper limit of normal for age (unless thought to be related to resolving infectious complications).
  -  Ability of patient or representative to provide written informed consent.
  -  Females of childbearing potential must have a negative pregnancy test prior to receiving GO.
  Exclusion Criteria:
  -  Subjects who have had chemotherapy or radiation therapy within 14 days prior to entering the study.
  -  Subjects may not be receiving other investigational agents.
  -  Uncontrolled or concurrent illness including, but not limited to, uncontrolled infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.

Relevance to patient case: not relevant