Clinical Drug Experience Knowledgebase

Criteria

Inclusion Criteria:

Male or female patients, age ≥ 18 years

Documented definitive diagnosis of AML or BPDCN (according to standard of care testing) and CD123 positivity of ≥20 % of blasts. In the case of MRD+ AML, if there are insufficient blasts at screening for CD123 testing, the most recent available sample with sufficient blasts should be used.

- Relapsed or refractory AML/BPDCN

Eastern Cooperative Oncology Group (ECOG) of 0 to 1
Life expectancy of at least 2 months
Adequate renal and hepatic laboratory assessments
Adequate cardiac function
Permanent venous access existing (e.g. port-system) resp. acceptance of implantation of a device
Able to give written informed consent
Weight ≥ 45 kg
Negative pregnancy; routinely using a highly effective method of birth control

Exclusion Criteria:

Acute promyelocytic leukemia (t15;17)
Refractory disease under anti-leukemic treatment lasting longer than 6 months
Manifestation of AML or BPDCN in central nervous system
Bone marrow failure syndromes
Cardiac disease: i.e. heart failure (NYHA III or IV); unstable coronary artery disease, myocardial infarction or serious cardiac ventricular arrhythmias requiring anti-arrhythmic therapy within the last 6 months prior to study entry
Patients undergoing renal dialysis
Pulmonary disease with clinical relevant hypoxia
Parkinson, epilepsy and, stroke or presence or history of seizures, paresis, aphasia or intracranial hemorrhage
Presence of disseminated intravascular coagulation (DIC) or thromboembolism, or history of such within 3 months prior to start of treatment
Hemolytic anemia
Multiple sclerosis
Active infectious disease considered by investigator to be incompatible with protocol or being contraindications for lymphodepletion therapy
Presence of urotoxicity from previous chemo- or radiotherapy or urinary outflow obstruction
Allogeneic stem cell transplantation within last two months or Graft versus host disease (GvHD) requiring immunosuppressive therapy
Vaccination with live viruses less than 2 weeks prior lymphodepletion therapy
Major surgery within 28 days
Other malignancy requiring active therapy but adjuvant endocrine therapy is allowed
Treatment with any investigational drug substance or experimental therapy within 4 weeks or 5 half-lives (whatever is shorter) of the substance prior to the day of apheresis
Prior treatment with gene therapy products
Use of checkpoint inhibitors within 5 half-lives of the respective substance
Autoimmune diseases requiring systemic steroids or other systemic immunosuppressants
Pregnant or breastfeeding women
Psychologic disorders, drug and/or significant active alcohol abuse
Known history of human immunodeficiency virus (HIV) or active/chronic infection with hepatitis C virus (HCV) or hepatitis B virus (HBV)
Presence of autoantibodies against La/Sjögren syndrome (SS)-B or presence or history of autoimmune diseases
Known hypersensitivity to cellular component (UniCAR02-T) and/or targeting module (TM123) excipients or to compounds of the lymphodepletion therapy or tocilizumab or corticosteroids
Evidence suggesting that the patient is not likely to follow the study protocol
Incapability of understanding purpose and possible consequences of the trial
Patients who should not be included according to the opinion of the investigator