AML, AdultPhase 2NCT04749355
Phase 2, Open-Label, Single Arm Study, With BST-236 in Adults With R/R AML or Higher-Risk MDS
A Phase 2 study testing BST-236 in people with AML, Adult and MDS, run by BioSight Ltd. at 4 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AML, Adult, MDS, Relapse/Recurrence
- 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
- 40 participants
- Where
- 4 sitesUnited States
- Study dates
- 14 Nov 2021to 14 Mar 2024
- 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
None listed.
Who cannot join
- 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
Show all 16Show fewer
- 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
- In 12 leads ECG, corrected QT interval (QTc)>480msec or history of QT prolongation or Torsades de pointes
Trial locations and contacts
4 sites in 1 country.
United States4 sites
University of Virginia Cancer Center
Charlottesville, Virginia, 22903
The University of Texas MD Anderson Cancer center
Houston, Texas, 77030
Seattle Cancer Care Alliance
Seattle, Washington, 98109
Ochsner LSU Health Shreveport - Academic Medical Center
Shreveport, Louisiana, 71104
About this study
Official title: A 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 AML or Higher-Risk Myelodysplastic Syndromes
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
- Being tested
- BST-236
- Medical subject headings
- Leukemia, Myeloid, Acute, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BST-236Drug
Also called Aspacytarabine
BST-236 is a conjugate of cytarabine and asparagine, provided as a sterile lyophilized powder for IV administration
| Study group | Role | What they receive |
|---|---|---|
| BST-236 | Experimental | BST-236 Intravenous, 4.5 g/m^2/d or 2.3 g/m^2/d, for 6 days Receives: Drug: BST-236 |
What is being measured
The primary outcome is the main question the study is designed to answer.
CR rate
In AML patients -The proportion of patients who achieve a CR per the IWG 2006 Criteria
Measured: 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
Measured: To be assessed 5 months after the last patient was enrolled to the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2021
- Study start14 Nov 2021
- Primary completion14 Dec 2023 (estimated)
- Study completion14 Mar 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, AdultStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Model description
- Open-label, multi-center, single arm, single agent study
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioSight Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04749355
- Sponsor's protocol ID
- BST004
- Status last verified
- Jul 2022
- First submitted
- 7 Feb 2021
- Last updated
- 9 Mar 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 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 AML or Higher-Risk Myelodysplastic Syndromes
- ClinicalTrials.gov ID
- NCT04749355
- Acronym / sponsor ID
- BST004
- Conditions
- AML, Adult, MDS, Relapse/Recurrence, Refractory Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- BioSight Ltd.Industry (pharma, biotech, devices)
- Study start
- 14 Nov 2021 (actual)
- Primary completion
- 14 Dec 2023 (estimated)
- Study completion
- 14 Mar 2024 (estimated)
- First posted
- 11 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label, multi-center, single arm, single agent study
| Arm | Type | Receives |
|---|---|---|
| BST-236 | Experimental | Drug: BST-236BST-236 Intravenous, 4.5 g/m^2/d or 2.3 g/m^2/d, for 6 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BST-236Also: AspacytarabineBST-236 is a conjugate of cytarabine and asparagine, provided as a sterile lyophilized powder for IV administration | Drug | BST-236 |
Primary outcome
CR rate
Time frame: 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
Overall response rate (ORR)
Time frame: 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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BST-236 in people with AML, Adult and MDS, run by BioSight Ltd. at 4 sites in United States.
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 0 inclusion and 16 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: status not confirmed, as of the sponsor's last update on 9 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. 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?
Results are not available in the source record.
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?
CR rate (time frame: To be assessed 5 months after the last patient was enrolled to the study). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Mar 2023.