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Report for Clinical Trial NCT02992860
 Developed by Shray Alag, 2020-2021.
SNP Clinical Trial Gene 
      The outcome of HMA-refractory patients with MDS or AML is dismal with a median survival of 5
      months after failure, representing a significant unmet medical need due to the very limited
      treatment options. In this context, a specific targeting of the leukemic stem cell (LSC)
      seems a promising option to selectively combat the leukemic progenitor cells. In fact, CD123
      is overexpressed in AML and MDS progenitors making it an attractive target for
      immunotherapy-based approaches. JNJ-56022473 is a promising compound that has been engineered
      with regard to this strategy and the current phase II trial has the aim to evaluate the
      overall hematological response rate at 3 months in HMA refractory/relapsed AML and MDS
      patients.
    
Name: JNJ-56022473
Description: JNJ-56022473 will be supplied as a lyophilized product containing 100mg of active pharmaceutical ingredient (50 mg/mL after reconstitution with 2.0 mL sterile water for injection). The JNJ-56022473 dose administered will be dependent upon the subject's weight at baseline. The JNJ-56022473 dose should be adjusted in case the subject's weight changes by > 10%. JNJ- 56022473 will be administered in 250 mL IV infusion over approximately 180 minutes using an infusion pump.
 Type: Drug
Group Labels:  1  Treatment Arm  
 Name: Bone marrow analyses and CBC with differential
Description: Bone marrow analyses and CBC with differential will be performed by a central laboratory. If the marrow cannot be aspirated, a biopsy should be performed.
 Type: Procedure
Group Labels:  1  Treatment Arm  
 Name: Flow cytometry analyses
Description: Characterization of LSCs and blasts (including CD123 and CD38 expression) will be performed as study-related analyses in the context of this protocol by central flow cytometry using bone marrow samples.
 Type: Other
Group Labels:  1  Treatment Arm  
 Name: Central biobanking
Description: Central biobanking of study samples (bone marrow, peripheral blood as well as a buccal swab) will be carried out in Dresden.
 Type: Other
Group Labels:  1  Treatment Arm  
 Name: Histopathology analysis
Description: A bone marrow biopsy is taken whenever it seems necessary to the investigator.
 Type: Procedure
Group Labels:  1  Treatment Arm  
 Name: Cytogenetic analysis
Description: The cytogenetic analysis with banding analysis (optional FISH) have to be performed at local labs. Therefore 2 - 5 ml of bone marrow will be collected and analysed.
 Type: Genetic
Group Labels:  1  Treatment Arm  
 Name: Serum chemistry
Description: For serum chemistry 15 ml (2 x 7,5 ml) of peripheral blood have to be collected.
 Type: Procedure
Group Labels:  1  Treatment Arm  
 Name: Automated CBC
Description: For CBC a minimum of 3 ml of peripheral blood have to be collected.
 Type: Procedure
Group Labels:  1  Treatment Arm  
 Name: Pregnancy Test
Description: Serum or urine pregnancy testing β-HCG with a sensitivity of at least 25 mIU/mL is to be done not more than 3 days prior to initiation of JNJ-56022473 in female patients with childbearing potential. Furthermore, serum or urine pregnancy testing has to be done after end of treatment (EoT) visit.
 Type: Procedure
Group Labels:  1  Treatment Arm  
  Primary Outcomes 
 Description: Overall hematological response rate at 3 months (either CR, PR, marrow-CR, HI, SD)
 Measure: Overall hematological response rate
 Time: 3 months
    Secondary Outcomes 
 Description: Toxicity as measured by NCI CTCAE 4.03
 Measure: Toxicity
 Time: 3 or 12 months
 Measure: Overall survival
 Time: 1 year
 Measure: Progression-free-survival
 Time: 1 year
 Measure: Overall hematological response rate at 12 months
 Time: 1 year
 Description: measured by EORTC-QLQ30
 Measure: Quality of life EORTC-QLQ30
 Time: 9 or 15 months
 Measure: Time to treatment failure
 Time: 3 or 12 months
 Measure: Duration of response (best overall response)
 Time: 3 or 12 months
  
Purpose: Treatment
Allocation: N/A
Single Group Assignment 
There are 2 SNPs
SNPs
1 I332E 
To enhance the cytotoxicity of the first-generation antibody CSL360, the
      proprietary Xencor (Xmab®) technology was applied and two amino acid mutations (S239D and
      I332E) were introduced into the Fc region. --- S239D ---  --- I332E --- 
 To enhance the cytotoxicity of the first-generation antibody CSL360, the
      proprietary Xencor (Xmab®) technology was applied and two amino acid mutations (S239D and
      I332E) were introduced into the Fc region. --- S239D ---