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

Phase 2

• Conditions: MDS; Relapse/Recurrence; AML, Adult; Refractory Acute Myeloid Leukemia
• Interventions: Drug: BST-236

• Registration Number: NCT04749355
• Lead Sponsor: BioSight Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-02-07
• Study First Submitted QC: 2021-02-07
• Study First Posted: 2021-02-11
• Study Start: 2021-11-14
• Status Verified: 2022-07
• Last Update Submitted: 2023-03-08
• Last Update Posted: 2023-03-09
• Primary Completion: 2023-12-14
• Study Completion: 2024-03-14

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

Not provided

Exclusion Criteria

1. MDS or AML evolving from a pre-existing myeloproliferative neoplasm (MPN)
2. MDS/MPN including chronic myelomonocytic leukemia (CMML), atypical chronic myeloid leukemia (CML), juvenile myelomonocytic leukemia (JMML) and unclassifiable MDS/MPN
3. Acute promyelocytic leukemia
4. Previous treatment for AML or MDS with drugs other than HMA or LDAC or combinations of venetoclax with either HMA or LDAC
5. Previous allogeneic hematopoietic stem cell transplantation (HSCT) or solid organ transplantation
6. 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
7. 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
8. Administration of HMA, LDAC, or venetoclax within 14 days prior to Study Day 1
9. Previous treatment with cytarabine at a dose higher than 20 mg/ m2/d
10. Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics or other treatment)
11. 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
12. 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)
13. 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
14. History of allergic reactions attributed to compounds of similar chemical composition as BST-236 and/or cytarabine
15. Life expectancy shorter than 3 months attributed to any known medical condition other than AML/MDS
16. In 12 leads ECG, corrected QT interval (QTc)>480msec or history of QT prolongation or Torsades de pointes

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Overall response rate (ORR)	To be assessed 5 months after the last patient was enrolled to the study	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
CR rate	To be assessed 5 months after the last patient was enrolled to the study	In AML patients -The proportion of patients who achieve a CR per the IWG 2006 Criteria

Trial Locations

Locations (4)

Seattle Cancer Care Alliance; 🇺🇸 Seattle, Washington, United States

Ochsner LSU Health Shreveport - Academic Medical Center; 🇺🇸 Shreveport, Louisiana, United States

University of Virginia Cancer Center; 🇺🇸 Charlottesville, Virginia, United States

The University of Texas MD Anderson Cancer center; 🇺🇸 Houston, Texas, United States