A Study of Dual-SIgnaling Protein 107 (DSP107) for Patients With Hematological Malignancies

NCT04937166 | Terminated Phase 1 FDA Drug

• 1 other identifier: DSP107_002
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 2

Brief Summary

This study will be divided into two parts, Parts A and B and will enroll patients with relapsed/refractory AML or MDS/chronic myelomonocytic leukemia (CMML) patients who have failed up to 2 prior therapeutic regimens. Part A is a dose escalation study to explore the safety, efficacy, pharmacokinetic (PK) and pharmacodynamic (PD) profile of DSP107 when administered in combination with azacitidine (AZA). Part B is a dose escalation study to explore the safety, efficacy, PK and PD profile of DSP107 when administered in combination with AZA and venetoclax (VEN).

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia

Outcome Measures

Primary Outcomes (3)

• Adverse Events (AEs)

  An AE is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

  Duration of the study, estimated to be 12 months

• Dose Limiting Toxicities (DLT)

  A DLT is defined as a clinically significant AE or laboratory abnormality that is related to DSP107 or the combination of DSP107 and AZA, but is unrelated to disease progression, intercurrent illness or concomitant medications

  At the end of Treatment Cycle 2 (within 2 months of treatment initiation)

• Response Rate (RR) including Complete Remission (CR) and Complete Remission with Incomplete Blood Count Recovery (CRi)

  Response rates will be determined by assessing peripheral blood and bone marrow samples.

  Within 6 months of treatment initiation

Secondary Outcomes (7)

• Overall Response Rate (ORR)

  Within 6 months of treatment initiation

• Morphologic Leukemia-Free (MLF) Rate

  Within 6 months of treatment initiation

• Minimal Residual Disease (MRD) Status

  Duration of the study, estimated to be 12 months

• 4-week Mortality Rate

  Within 4 weeks of treatment initiation

• DSP107 Serum Concentration

  Duration of the study, estimated to be 12 months

Study Arms (1)

DSP107 in combination with azacitidine or azacitidine plus venetoclax.

EXPERIMENTAL

DSP107 will be administered by intravenous infusion once weekly during each 28-day cycle to all patients in this study. Azacitidine (75 mg/m2/day) will be administered subcutaneously or intravenously for the first 7 days of every cycle. Patients enrolled in Part B only will also receive venetoclax. During Cycle 1, venetoclax will be dose escalated daily to the goal dose of 400 mg daily. Patients will receive 100 mg on Day 1, 200 mg on Day 2 and 400 mg on Day 3 and onwards.

Biological: DSP107; Drug: Azacitidine; Drug: Venetoclax

Interventions

DSP107 BIOLOGICAL

DSP107 (SIRPα - 4-1BBL) is a bi-functional, trimeric, fusion protein.

DSP107 in combination with azacitidine or azacitidine plus venetoclax.

Azacitidine DRUG

Azacitidine is an analog of the pyrimidine nucleoside cytidine.

Also known as: Vidaza

DSP107 in combination with azacitidine or azacitidine plus venetoclax.

Venetoclax DRUG

Venetoclax is a B-cell lymphoma (BCL)-2 inhibitor

Also known as: Venclexta, Venclyxto

DSP107 in combination with azacitidine or azacitidine plus venetoclax.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2
• White Blood Cell count \< 20 x 10\^9/L.
• Adequate organ function
• Relapsed/refractory AML or MDS/CMML patients who have failed up to 2 prior therapeutic regimens.

You may not qualify if:

• Acute Promyelocytic leukemia
• Symptomatic central nervous system (CNS) leukemia or patients with poorly controlled CNS leukemia
• Life-threatening (grade 4) immune-mediated adverse event related to prior immunotherapy
• Immune-mediated adverse reaction that required discontinuation of prior immunotherapy
• Past or current history of autoimmune disease or immune deficiency
• History of severe interstitial lung disease or severe pneumonitis or active pneumonitis
• Clinically significant and poorly compensated liver disease
• Prior organ allografts (such as renal transplant) requiring active immunosuppression
• Active graft versus host disease
• Treatment with systemic immunostimulatory within 4 weeks prior to initiation of study treatment
• Treatment with any CD47/SIRPα targeting agent or immune agonists
• Known allergy or hypersensitivity to any of the test compounds, materials or contraindication to test product
• Received live, attenuated vaccine within 4 weeks prior to first dose of study treatment
• Active Hepatitis B or C infection
• History or evidence of any other clinically unstable/uncontrolled disorder, condition, or disease

Sponsors & Collaborators

• Kahr Medical: lead

Study Sites (2)

City of Hope

Duarte, California, 91010, United States

Location

The University of Texas MD Anderson Cancer Center, Department of Leukemia

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes; Leukemia, Myelomonocytic, Chronic

Interventions

Azacitidine; venetoclax

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This study will involve sequential enrollment of approximately 36 patients accrued into six dose cohorts, each testing a different DSP107 dose level in combination with azacitidine (Part A) or DSP107 in combination with azacitidine and venetoclax (Part B).

Study Record Dates

• First Submitted: June 6, 2021
• First Posted: June 23, 2021
• Study Start: January 13, 2022
• Primary Completion: April 23, 2025
• Study Completion: October 24, 2025
• Last Updated: November 10, 2025

Record last verified: 2025-11

Locations

US (2)