BST-236 as a Single Agent in Adults With Relapsed or Refractory AML or HR-MDS

NCT04827719 | Completed Phase 2 DMC

• 1 other identifier: GFM-BST003
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 17

Brief Summary

To assess the efficacy, and safety of BST-236 in patients unfit for intensive chemotherapy with AML or HR MDS that failed or relapsed following first line therapy

Conditions

Myelodysplastic Syndromes; Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Overall hematological response

  Overall response rate

  2 months

Secondary Outcomes (1)

• response duration

  12 months

Study Arms (1)

BST-236

EXPERIMENTAL

Recurrent 6 days treatment courses with BST-236 (4.5 g/m2/d administered IV over 1 hour for 6 consecutive days)

Drug: BST-236

Interventions

BST-236 DRUG

Recurrent 6 days treatment courses with BST-236 (4.5 g/m2/d administered IV over 1 hour for 6 consecutive days)

BST-236

Eligibility Criteria

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

You may qualify if:

• Documented diagnosis of MDS, according to World Health Organization (WHO) classification (Appendix 1) and assessed as higher risk MDS, prior to first line hypomethylating agents (HMA) treatment, according to the Revised International Prognostic Scoring System (IPSS-R) (IPSS-R overall score ≥ 4.5) Or Diagnosed AML according to the 2016 revision to the WHO classification of myeloid neoplasms and acute leukemia: ≥20% blasts in peripheral blood or bone marrow
• Adult ≥18 years of age
• Failure/relapse following prior first-line AML or MDS treatment, defined as:
• For MDS:
• Relapse after initial complete or partial response or stable disease with hematologic improvement (HI), according to International Working Group (IWG) 2006 criteria following treatment with azacitidine or decitabine Or
• Failure to achieve complete or partial response or stable disease with hematologic improvement (HI) according to International Working Group (IWG) 2006 criteria after at least 4 cycles of azacitidine or decitabine, all within the last 1 year Or iii. MDS progression while on azacitidine or decitabine treatment irrespective of the number of cycles the patient has received
• For AML:
• Relapse after initial CR/CRi/CRh following treatment with: azacitidine, decitabine, Low-Dose Ara-C (cytarabine) \[LDAC\] (20 mg/m2/d), venetoclax+HMA, or venetoclax+LDAC Or
• Failure to achieve CR, CRh or CRi following at least 4 cycles of azacitidine or decitabine or 2 cycles of venetoclax+HMA or venetoclax+LDAC within the last 1 year.
• \- AML progression while on azacitidine, decitabine, LDAC, venetoclax+HMA, venetoclax+LDAC, irrespective of the number of cycles the patient has received.
• The participant is not able to receive an allogeneic bone marrow transplantation (BMT) at the time of study enrolment (BMT may be an option once the patient completed the study).
• Not eligible for intensive chemotherapy;
• Age ≥75 years Or
• Age ≥18 years with at least one of the following comorbidities:
• I. Significant heart or lung comorbidities, as reflected by at least one of the following:

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/L 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

• Groupe Francophone des Myelodysplasies: lead

Study Sites (17)

CHU NIMES Caremeau

Nîmes, Nîmes, 30029, France

Location

Centre Henri Becquerel

Rouen, Rouen, 76 038, France

Location

CHU Amiens

Amiens, 80054, France

Location

CHU d'Angers

Angers, 49100, France

Location

CHU Besançon - Hôpital Jean Minjoz

Besançon, 25030, France

Location

CHU de Bordeaux Haut-Lévèque

Bordeaux, 33604, France

Location

CHU de Grenoble

Grenoble, 38700, France

Location

CH Le Mans

Le Mans, 72037, France

Location

CHRU de Limoges - Hôpital Dupuytren

Limoges, 87042, France

Location

Institut Paoli Calmettes

Marseille, 13009, France

Location

CHU Nantes - Hôtel Dieu

Nantes, 44093, France

Location

CHU de Nice - Hôpital Archet 1

Nice, 06200, France

Location

Hôpital Saint Louis

Paris, 75010, France

Location

CHU de Poitiers

Poitiers, 86021, France

Location

Hôpital Pontchaillou

Rennes, 35033, France

Location

Institut de Cancérologie Lucien Neuwirth

Saint-Priest-en-Jarez, 42270, France

Location

CHU Toulouse - IUCT Oncopole

Toulouse, 31100, France

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes; Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Marie Anne HOSPITAL, MD

  Institut Paoli Calmettes, Marseille

  PRINCIPAL INVESTIGATOR

• PIERRE FENAUX, Pr

  Hôpital St Louis-Paris

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: March 18, 2021
• First Posted: April 1, 2021
• Study Start: May 1, 2021
• Primary Completion: January 13, 2025
• Study Completion: January 13, 2025
• Last Updated: May 18, 2025

Record last verified: 2025-05

Locations

FR (17)