AzTrials
AzTrials

Myelodysplastic SyndromesPhase 2NCT04827719

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

A Phase 2 study testing BST-236 in people with Myelodysplastic Syndromes and Acute Myeloid Leukemia, run by Groupe Francophone des Myelodysplasies at 17 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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 Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
BST-236
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
38 participants
Where
17 sitesFrance
Study dates
1 May 2021to 13 Jan 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Patients must meet all of the following inclusion criteria to be eligible for enrollment into the study:
  • 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
Show all 29Show fewer
  • 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.
  • Or
  • 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:
  • Significant heart or lung comorbidities, as reflected by at least one of the following:
  • Left ventricular ejection fraction (LVEF) ≤50%
  • Lung diffusing capacity for carbon monoxide (DLCO) ≤65% of expected
  • Forced expiratory volume in 1 second (FEV1) ≤65% of expected II. Other comorbidity that the Investigator judges as incompatible with intensive chemotherapy, which must be documented
  • Creatinine clearance (estimated by the Modification of Diet in Renal Disease (MDRD) equation or measured by 24 hours urine collection) ≥45 mL/min
  • Liver enzymes (aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≤2.5 times the upper limits of normal (ULN), unless attributed to leukemia (in AML patients)
  • Total bilirubin ≤3 XULN unless due to Gilbert disease
  • Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
  • Women of reproductive potential must have a negative serum pregnancy test within 48 hours prior to the first day of any BST-236 treatment course
  • Women of reproductive potential must use two forms of effective birth control methods starting from at least 1 month prior to BST-236 first dose and until 6 months following the last BST-236 administration day (acceptable methods of birth control in this study include: surgical sterilization, intrauterine devices, intrauterine hormone-releasing system, long-acting injectable contraceptives, or vasectomized partner (provided that partner is the sole sexual partner)
  • Male subjects must agree to refrain from unprotected sex unless vasectomized and from sperm donation from initial study drug administration until 6 months following the last dose of study drug
  • Subject must voluntarily sign and date an informed consent, approved by an Independent Ethics Committee (IEC)/Institutional Review Board (IRB), prior to the initiation of any screening or study-specific procedures
  • Patient must be able to adhere to the study visit schedule and other protocol requirements

Who cannot join

  • Patients with any one of the exclusion criteria listed below are not eligible for the study:
  • 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
Show all 18Show fewer
  • 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
  • Known infection with Hepatitis B Virus (HBV) Hepatitis C Virus (HCV) or Human Immunodeficiency Virus (HIV)
  • In 12 leads ECG, corrected QT interval (QTc)>480msec or history of QT prolongation or Torsades de pointes

Trial locations and contacts

17 sites in 1 country.

17 sites in 1 country

France17 sites

  • CHU Amiens

    Amiens, 80054

  • CHU d'Angers

    Angers, 49100

  • CHU Besançon - Hôpital Jean Minjoz

    Besançon, 25030

  • CHU de Bordeaux Haut-Lévèque

    Bordeaux, 33604

  • CHU de Grenoble

    Grenoble, 38700

  • CH Le Mans

    Le Mans, 72037

  • CHRU de Limoges - Hôpital Dupuytren

    Limoges, 87042

  • Institut Paoli Calmettes

    Marseille, 13009

  • CHU Nantes - Hôtel Dieu

    Nantes, 44093

  • CHU de Nice - Hôpital Archet 1

    Nice, 06200

  • CHU NIMES Caremeau

    Nîmes, Nîmes, 30029

  • Hôpital Saint Louis

    Paris, 75010

  • CHU de Poitiers

    Poitiers, 86021

  • Hôpital Pontchaillou

    Rennes, 35033

  • Centre Henri Becquerel

    Rouen, Rouen, 76 038

  • Institut de Cancérologie Lucien Neuwirth

    Saint-Priest-en-Jarez, 42270

  • CHU Toulouse - IUCT Oncopole

    Toulouse, 31100

Other trials in France

About this study

Official title: Phase 2, Open-Label, Single Arm, Multi-Center Study to Assess the Efficacy and Safety of BST-236 as a Single Agent in Adults Unfit for Intensive Chemotherapy With Relapsed or Refractory Acute Myeloid Leukemia or HR-MDS .

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

Read the detailed description

This is a prospective, phase 2, open-label, multi-center, single arm, single agent study, in unfit adult patients with AML or HR MDS who failed or relapsed following first-line therapy.

The study consists of three periods:

Screening: Up to 28 days Treatment: Recurrent 6 days treatment courses with BST-236 Follow-up: The period of time from last day of BST-236 treatment to completion of one year (365 days ±7 days) from the first day of BST-236 treatment

Post-study Follow-up: The period of time from last day of BST-236 treatment to completion of one year (365 days ±7 days) from the first day of BST-236 treatment

Follow-up: One-Year follow-up of survival, starting at the end of study visit

Treatment period: After signing the Informed Consent Form (ICF), meeting all eligibility criteria, and undergoing screening assessments for up to four weeks, eligible patients will receive the first induction course with BST-236, 4.5 g/m2/d administered IV over 1 hour for 6 consecutive days. The first day of the induction is defined as Study Day 1. Following the first treatment course:

  1. AML patients in complete remission (CR) or CR with incomplete count recovery (CRi) or CR with partial hematologic recovery (CRh), and HR MDS patients with CR will receive between 2 to 4 maintenance courses of 6-day treatment with BST-236, every 4-6 weeks. The BST-236 dose in these subsequent treatment courses will be 2.3g /m2/d or 4.5 g/m2/d. The reduced dose must be used in case of previous courses toxicity and is allowed in certain cases according to physician discretion following consultation with the study medical monitor. The interval between treatment courses may be extended to up to 12 weeks to allow resolution of toxicities per investigator discretion and in consultation with principal investigators.
  2. Patients with stable disease defined as failure to achieve at least PR, but with no evidence of disease progression or patients in partial response (PR) will receive a second 6-day course with BST-236, at a dose of 4.5 g/m2/d. Following which:
  3. AML patients in PR or CR, (including CR, CRh or CRi) and HR MDS patients in PR or CR will be treated with maintenance courses as detailed above Patients in stable disease or disease progression may receive alternative treatment outside of the protocol and will be followed for overall survival (OS) until the completion of 1 year of participation in the study (365 days±7 days from the first day of BST-236 treatment). Additional 1 year of post study follow-up will be implemented for these patients in order to follow up on their survival status and their current AML/MDS treatments.

Patients that have a disease progression while treated in the maintenance courses, will not receive any additional BST-236 treatment and will be followed until the completion of 1 year of participation in the study. These patients may also receive alternative treatment outside of the protocol, in such case, they will be followed in regular intervals for progression free survival, OS and development of new malignancies.

Follow-up period: All patients will be followed by periodic in person in-clinic visits, until the completion of 1 year of participation in the study (365 days±7 days from the first BST-236 treatment day).

End of Study (EOS) visits: will be performed on the last day of participation in the study for all patients.

Scheduled in-clinic visits to assess safety and efficacy outcomes will be conducted during the study periods as specified in the Schedule of Activities.

Unscheduled visits may be conducted at any time for safety reasons or for any other reason.

Post study follow-up: All patients will be followed for survival for an additional 1 year from the EOS

QT Evaluation with Pharmacokinetics (PK) sub-study: Up to 20 patients participating in the main study in pre-selected sites, will be asked to participate in a QT evaluation with PK sub-study to assess the effect of BST-236 on the cardiac electrophysiology. In this study, up to 14 blood samples for PK analysis will be taken from each patient during the first induction course and a continuous electrocardiogram (ECG) recording by a Holter monitor will be done in the first 24 hours of BST-236 administration. Patients participating in this study will be asked to sign an additional ICF for the collection of the PK blood samples and ECG recording.

Being tested
BST-236
Medical subject headings
Myelodysplastic Syndromes, Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BST-236Drug

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

Study groups in NCT04827719
Study groupRoleWhat they receive
BST-236ExperimentalRecurrent 6 days treatment courses with BST-236 (4.5 g/m2/d administered IV over 1 hour for 6 consecutive days)
Receives: Drug: BST-236

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Overall hematological response

    Overall response rate

    Measured: 2 months

Secondary outcomes (1)

  1. response duration

    response duration

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Apr 2021
  2. Study start1 May 2021
  3. Primary completion13 Jan 2025
  4. Study completion13 Jan 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
38 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04827719
Sponsor's protocol ID
GFM-BST003
Status last verified
May 2025
First submitted
18 Mar 2021
Last updated
11 Aug 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BST-236 in people with Myelodysplastic Syndromes and Acute Myeloid Leukemia, run by Groupe Francophone des Myelodysplasies at 17 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall hematological response (time frame: 2 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04827719.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Aug 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in