SNPMiner Trials by Shray Alag


SNPMiner SNPMiner Trials (Home Page)


Report for Clinical Trial NCT02992860

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

Single Agent JNJ-56022473 in MDS and AML Patients Failing Hypomethylating Agent Based Therapy

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.

NCT02992860 Myelodysplastic Syndrome (MDS) Acute Myeloid Leukemia (AML)
MeSH:Leukemia, Myeloid Leukemia, Myeloid, Acute Preleukemia Myelodysplastic Syndromes
HPO:Acute megakaryocytic leukemia Acute myeloid leukemia Myelodysplasia Myeloid leukemia

9 Interventions

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 ---


2 S239D

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 ---



HPO Nodes


HPO:
Protein Mutations 0
SNP 0