Phase 2, Open-Label, Single Arm Study, With BST-236 in Adults With R/R AML or Higher-Risk MDS

NCT04749355 | Unknown Phase 2 DMC FDA Drug

• 1 other identifier: BST004
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 4

Brief Summary

An open label multi center study to assess the safety and efficacy of BST-236 as single agent in adult patients unfit for standard therapy with Acute Myeloid Leukemia (AML) or higher-risk (HR) Myelodysplastic Syndromes (MDS) who fail to respond or relapsed following first line therapy. Approximately 20 adult patients with relapsed and/or refractory AML and approximately 20 adult patients with relapsed and/or refractory HR MDS, will be enrolled into the study. Patients will be treated with 1-2 induction courses and 2-4 maintenance courses. All patients will be followed for 1 year in the study and additional 1 year post study follow-up.

Conditions

AML, Adult; MDS; Relapse/Recurrence; Refractory Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (2)

• CR rate

  In AML patients -The proportion of patients who achieve a CR per the IWG 2006 Criteria

  To be assessed 5 months after the last patient was enrolled to the study

• Overall response rate (ORR)

  In MDS patients -Overall response rate (ORR), defined as the proportion of patients who achieve a CR or PR per proposal for modification of the International Working Group (IWG) criteria for MDS, 2006

  To be assessed 5 months after the last patient was enrolled to the study

Study Arms (1)

BST-236

EXPERIMENTAL

BST-236 Intravenous, 4.5 g/m\^2/d or 2.3 g/m\^2/d, for 6 days

Drug: BST-236

Interventions

BST-236 DRUG

BST-236 is a conjugate of cytarabine and asparagine, provided as a sterile lyophilized powder for IV administration

Also known as: Aspacytarabine

BST-236

Eligibility Criteria

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

You may not qualify if:

• MDS or AML evolving from a pre-existing myeloproliferative neoplasm (MPN)
• MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN
• Acute promyelocytic leukemia
• Previous treatment for AML or MDS with drugs other than HMA or LDAC or combinations of venetoclax with either HMA or LDAC
• Previous allogeneic hematopoietic stem cell transplantation (HSCT) or solid organ transplantation
• Participation in a previous clinical trial involving use of an investigational drug within 30 days or at least 5 half-lives of tested drug (whichever is shorter) of study day 1
• Peripheral White Blood Cell (WBC) count \>30,000 /µL in the 48 hours prior to first BST-236 dose administration. Hydroxyurea administration or leukapheresis is permitted to meet this criterion
• Administration of HMA, LDAC, or venetoclax within 14 days prior to Study Day 1
• Previous treatment with cytarabine at a dose higher than 20 mg/ m2/d
• Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment)
• Any medical or surgical condition, presence of laboratory abnormalities or psychiatric illness that may preclude safe and complete study participation based on the Investigator's judgment
• Diagnosis of malignant disease (other than AML) within the previous 12 months (excluding basal cell carcinoma of the skin without complications, "in-situ" carcinoma, or other local malignancy excised or irradiated with a high probability of cure and not treated with systemic or topical chemotherapy)
• Surgical procedure, excluding central venous catheter placement or other minor procedures (e.g. skin biopsy) in the 14 days prior to first BST-236 dose administration
• History of allergic reactions attributed to compounds of similar chemical composition as BST-236 and/or cytarabine
• Life expectancy shorter than 3 months attributed to any known medical condition other than AML/MDS

Sponsors & Collaborators

• BioSight Ltd.: lead

Study Sites (4)

Ochsner LSU Health Shreveport - Academic Medical Center

Shreveport, Louisiana, 71104, United States

Location

The University of Texas MD Anderson Cancer center

Houston, Texas, 77030, United States

Location

University of Virginia Cancer Center

Charlottesville, Virginia, 22903, United States

Location

Seattle Cancer Care Alliance

Seattle, Washington, 98109, United States

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Recurrence

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, multi-center, single arm, single agent study

Study Record Dates

• First Submitted: February 7, 2021
• First Posted: February 11, 2021
• Study Start: November 14, 2021
• Primary Completion: December 14, 2023
• Study Completion: March 14, 2024
• Last Updated: March 9, 2023

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)