Acute Myeloid Leukemia, Myelodysplastic SyndromePhase 1NCT04743115
A Phase I, Open-Label, Dose Escalation and Cohort Expansion Study of BS HH 002.SA in Patients With AML and MDS
A Phase 1 study testing BS HH 002.SA in people with Acute Myeloid Leukemia, Myelodysplastic Syndrome, run by Shanghai Bensen Pharmaceutical Co., Ltd..
Trial summary
Indication:Relapsed or refractory AML in patients for whom no established treatment options are available (this indication will heretofore be referred to as the protocol AML indication), or adult patients with MDS who are classified as high risk or very high risk according to the Revised International Prognosis… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, Myelodysplastic Syndrome
- What is being tested?
- BS HH 002.SA
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- No sites listed yet
- Study dates
- Feb 2021to Feb 2025
- Phase
- Phase 1
- 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
- Female and male patients ≥18 years of age
- Patients with one of the following conditions:
- Primary or secondary AML, pathologically confirmed according to World Health Organization (WHO) or IWG classification, who have relapsed or refractory disease and for whom no established treatment options are available, or who are not a candidate for current therapies
- High risk or very high risk MDS, as evaluated according to the IPSS R, who are resistant or intolerant to standard treatment and not candidates for transplantation
- Patients must be willing to participate in the study and have the ability to understand and adhere to study visit schedule and other protocol procedures. They should be able to comprehend and willing to sign an Informed Consent Form (ICF)
- Women of childbearing potential must have two negative pregnancy tests during Screening, the second within 24 hours prior to the first administration of study drug, and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration. For the purposes of this study, women with tubal ligations are considered to be of childbearing potential but women who are surgically sterile (hysterectomy) or post menopausal ≥2 years are not considered to be of childbearing potential Note: Post-menopausal is defined as aged more than 50 years and amenorrhea for at least 12 months following cessation of all exogenous hormonal treatment. Women with irreversible surgical sterilization by hysterectomy, bilateral oophorectomy, or bilateral salpingectomy, but not tubal ligation, are acceptable.
- Male subjects must be surgically sterile. If male subjects are able to father children, they must use a highly effective physician-approved birth control method from Screening, during the study, and for 90 days after the last dose of study drug. The study physician will inform the subject which birth control methods are acceptable. Vasectomy and sexual abstinence are the recommended birth control methods Note: Female partners of male subjects should not become pregnant during the course of the study as the effects of the study drug on an unborn child and on a breast-fed baby are not known. Male participants will not be allowed to take part in this study if their female partner is trying to become pregnant. If female partner does become pregnant while male partner is taking part in the study, male participants should immediately inform the study physician. The female partner will be asked to sign a Pregnant Partner Data Release Form.
Who cannot join
- Eastern Cooperative Oncology Group (ECOG) performance status 3 or greater
- Known hypersensitivity to study drug or its excipients
- Acute promyelocytic leukemia
- Administration of any antineoplastic therapy or anti-MDS treatment within at least 2 weeks or 5 half-lives (whichever is greater) of the first dose of BS HH 002.SA, with the exception of hydroxyurea for AML patients which should be discontinued 1 day prior to the first dose of BS HH 002.SA
- Participation in other clinical trials within at least 2 weeks of the first BS HH 002.SA dose
- Concomitantly receiving other investigational agents
Show all 26Show fewer
- Clinical evidence of active central nervous system leukemia
- History of diabetes mellitus and a hemoglobin A1c ≥7.0% as assessed at Screening
- Active and uncontrolled infection including but not limited to known infection with HIV, active hepatitis B, or hepatitis C. Patients with an infection receiving treatment with antibiotics may be entered into the trial if they are afebrile and hemodynamically stable for 96 hours prior to trial entry
- Major surgery within 2 weeks prior to trial entry
- Receipt of an allogeneic or autologous stem cell transplant within 60 days of the first BS HH 002.SA dose
- Toxicities from a previous anti-cancer therapy that are NCI CTCAE Grade 2 or greater, except for alopecia and Grade 2 nausea
- Liver function tests above the following limits at Screening: total bilirubin >1.5 × upper limit of normal (ULN) unless related to Gilbert's syndrome or hemolysis, aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >2.5 × ULN, or for patients with liver involvement AST and/or ALT >5 × ULN
- Liver malignancy (including metastases) or chronic liver disease
- Previous drug-induced liver injury (DILI)
- Serum creatinine >1.5 × ULN and/or creatinine clearance (CrCl) <30 mL/min at Screening (calculation according to Cockcroft \& Gault formula or Modification of Diet in Renal Disease [MDRD] formula)
- International normalized ratio (INR) >1.5 × ULN, or, if on warfarin therapy, INR >2.5 × ULN
- Corrected QT interval (QTc) by Fridericia's method (QTcF) >450 msec or concomitant use of drugs known to prolong the QTc (antiemetic therapy can be allowed with approval from the Medical Monitor if the QTc is normal, and calcium, magnesium, and potassium levels are normal on Day 1)
- New York Heart Association class III or IV heart disease, uncontrolled hypertension, congestive heart failure, or other uncontrolled cardiac condition
- Left ventricular ejection fraction (LVEF) <45% as assessed by multiple-gated acquisition (MUGA) scan or echocardiogram
- Uncontrolled congestive heart failure, unstable angina pectoris
- Patients with concomitant solid tumors or lymphoma, for whom the Investigator has clinical suspicion of active disease at the time of enrolment. Patients with adequately treated early-stage squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or cervical intraepithelial neoplasia, or with castrate sensitive prostate cancer with stable elevated prostate-specific antigen under anti-androgenic therapy are eligible for this study
- Other significant disease that in the Investigator's opinion would exclude the patient from the participation in the study protocol
- Women who are or plan to become pregnant, or who are currently breastfeeding
- Active alcohol or drug abuse
- Legal incapacity or limited legal capacity.
About this study
Official title: A Phase I, Open-Label, Dose Escalation and Cohort Expansion Study of BS HH 002.SA in Patients With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)
Indication:Relapsed or refractory AML in patients for whom no established treatment options are available (this indication will heretofore be referred to as the protocol AML indication), or adult patients with MDS who are classified as high risk or very high risk according to the Revised International Prognosis Scoring System (IPSS-R).
Number of Investigators and Study Centers:Up to 5 Investigators in the US. Objectives:Dose Escalation Part
Read more
Primary Objective:
- To determine the maximum tolerated dose (MTD) of BS HH 002.SA administered subcutaneously once per day for 12 days of a 28-day cycle. Secondary Objectives:
- To provide an initial safety profile of single and multiple cycles of BS HH 002.SA.
- To assess the pharmacokinetic (PK) profile of BS HH 002.SA.
- To explore the anti-tumor activity of BS HH 002.SA in patients with the protocol AML indication or high-risk MDS.
- To explore cytogenetics of the malignant cells in relation to response to BS HH 002.SA.
Cohort Expansion Part
Primary Objectives:
- To evaluate safety and tolerability of BS HH 002.SA at MTD and/or lower dose level (DL) in selected cohorts of patients with the protocol AML indication or high-risk MDS.
- To evaluate preliminary anti-tumor activity of BS HH 002.SA at MTD and/or lower DL in selected cohorts of patients with the protocol AML indication or high-risk MDS. Secondary Objectives:
- To assess the PK profile of BS HH 002.SA.
- To explore cytogenetics of the malignant cells in relation to response to BS HH 002.SA.
Study Population:Adult patients with the protocol AML indication or high-risk MDS.
- Being tested
- BS HH 002.SA
- Keywords
- BS HH 002.SA
- Medical subject headings
- GATA2 Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BS HH 002.SADrug
The Dose Escalation Part is a 3+3 design dose escalation study to determine the MTD of BS HH 002.SA in up to approximately 30 patients with the protocol AML indication or high risk MDS. Cohorts of 3 patients will be sequentially assigned to receive BS HH 002.SA at escalating DLs following the standard 3+3 dose escalation design, with 3 to 6 patients at each DL.
Cohort Expansion Part: Following determination of the MTD, the study will continue at the MTD and/or a lower DL
| Study group | Role | What they receive |
|---|---|---|
| the Dose Escalation Level | Experimental | In the first 3-patient cohort, the dose of BS HH 002.SA will be 0.2 mg/m2/day. Enrollment to the next higher dose cohort will be initiated only if none of the 3 patients exhibits a DLT in the first 28-day cycle. Dose escalation will proceed according to a modified Fibonacci scheme, eg, increments of 100%, 100%, 66%, and 33% and the dose of BS-HH-002.SA will be 0.4, 0.8, 1.3, 1.7 mg/m2/day.
The dose will be administered subcutaneously once daily on Days 1 through 6 and on Days 10 through 15 of a 28 day cycle. Receives: Drug: BS HH 002.SA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Escalation Part: the maximum tolerated dose (MTD) of BS HH 002.SA administered subcutaneously once per day for 12 days of a 28-day cycle.
Measured: the first 28 day cycle
Cohort Expansion Part: To evaluate safety and tolerability of BS HH 002.SA at MTD and/or lower dose level (DL) in selected cohorts of patients with the protocol AML indication or high-risk MDStreatment
Measured: maximum of 12 months
Cohort Expansion Part: To evaluate preliminary anti-tumor activity of BS HH 002.SA at MTD and/or lower DL in selected cohorts of patients with the protocol AML indication or high-risk MDS.
Measured: maximum of 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Feb 2021
- Study startFeb 2021 (estimated)
- Primary completionFeb 2025 (estimated)
- Study completionFeb 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia, Myelodysplastic SyndromeStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 66 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Bensen Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04743115
- Sponsor's protocol ID
- BS HH 002.SA
- Status last verified
- Feb 2021
- First submitted
- 4 Feb 2021
- Last updated
- 8 Feb 2021
- 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 8 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label, Dose Escalation and Cohort Expansion Study of BS HH 002.SA in Patients With Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS)
- ClinicalTrials.gov ID
- NCT04743115
- Acronym / sponsor ID
- BS HH 002.SA
- Keywords
- BS HH 002.SA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 66 participants (estimated)
- Lead sponsor
- Shanghai Bensen Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- Feb 2021 (estimated)
- Primary completion
- Feb 2025 (estimated)
- Study completion
- Feb 2025 (estimated)
- First posted
- 8 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.
Indication:Relapsed or refractory AML in patients for whom no established treatment options are available (this indication will heretofore be referred to as the protocol AML indication), or adult patients with MDS who are classified as high risk or very high risk according to the Revised International Prognosis Scoring System (IPSS-R).
Number of Investigators and Study Centers:Up to 5 Investigators in the US. Objectives:Dose Escalation Part
Primary Objective:
- To determine the maximum tolerated dose (MTD) of BS HH 002.SA administered subcutaneously once per day for 12 days of a 28-day cycle. Secondary Objectives:
- To provide an initial safety profile of single and multiple cycles of BS HH 002.SA.
- To assess the pharmacokinetic (PK) profile of BS HH 002.SA.
- To explore the anti-tumor activity of BS HH 002.SA in patients with the protocol AML indication or high-risk MDS.
- To explore cytogenetics of the malignant cells in relation to response to BS HH 002.SA.
Cohort Expansion Part
Primary Objectives:
- To evaluate safety and tolerability of BS HH 002.SA at MTD and/or lower dose level (DL) in selected cohorts of patients with the protocol AML indication or high-risk MDS.
- To evaluate preliminary anti-tumor activity of BS HH 002.SA at MTD and/or lower DL in selected cohorts of patients with the protocol AML indication or high-risk MDS. Secondary Objectives:
- To assess the PK profile of BS HH 002.SA.
- To explore cytogenetics of the malignant cells in relation to response to BS HH 002.SA.
Study Population:Adult patients with the protocol AML indication or high-risk MDS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| the Dose Escalation Level | Experimental | Drug: BS HH 002.SAIn the first 3-patient cohort, the dose of BS HH 002.SA will be 0.2 mg/m2/day. Enrollment to the next higher dose cohort will be initiated only if none of the 3 patients exhibits a DLT in the first 28-day cycle. Dose escalation will proceed according to a modified Fibonacci scheme, eg, increments of 100%, 100%, 66%, and 33% and the dose of BS-HH-002.SA will be 0.4, 0.8, 1.3, 1.7 mg/m2/day. The dose will be administered subcutaneously once daily on Days 1 through 6 and on Days 10 through 15 of a 28 day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Female and male patients ≥18 years of age
- Patients with one of the following conditions:
- Patients must be willing to participate in the study and have the ability to understand and adhere to study visit schedule and other protocol procedures. They should be able to comprehend and willing to sign an Informed Consent Form (ICF)
- Women of childbearing potential must have two negative pregnancy tests during Screening, the second within 24 hours prior to the first administration of study drug, and must agree to use highly effective physician-approved contraception from Screening to 90 days following the last study drug administration. For the purposes of this study, women with tubal ligations are considered to be of childbearing potential but women who are surgically sterile (hysterectomy) or post menopausal ≥2 years are not considered to be of childbearing potential Note: Post-menopausal is defined as aged more than 50 years and amenorrhea for at least 12 months following cessation of all exogenous hormonal treatment. Women with irreversible surgical sterilization by hysterectomy, bilateral oophorectomy, or bilateral salpingectomy, but not tubal ligation, are acceptable.
- Male subjects must be surgically sterile. If male subjects are able to father children, they must use a highly effective physician-approved birth control method from Screening, during the study, and for 90 days after the last dose of study drug. The study physician will inform the subject which birth control methods are acceptable. Vasectomy and sexual abstinence are the recommended birth control methods Note: Female partners of male subjects should not become pregnant during the course of the study as the effects of the study drug on an unborn child and on a breast-fed baby are not known. Male participants will not be allowed to take part in this study if their female partner is trying to become pregnant. If female partner does become pregnant while male partner is taking part in the study, male participants should immediately inform the study physician. The female partner will be asked to sign a Pregnant Partner Data Release Form.
Key exclusion criteria (26 listed)
- Eastern Cooperative Oncology Group (ECOG) performance status 3 or greater
- Known hypersensitivity to study drug or its excipients
- Acute promyelocytic leukemia
- Administration of any antineoplastic therapy or anti-MDS treatment within at least 2 weeks or 5 half-lives (whichever is greater) of the first dose of BS HH 002.SA, with the exception of hydroxyurea for AML patients which should be discontinued 1 day prior to the first dose of BS HH 002.SA
- Participation in other clinical trials within at least 2 weeks of the first BS HH 002.SA dose
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 |
|---|---|---|
| BS HH 002.SAThe Dose Escalation Part is a 3+3 design dose escalation study to determine the MTD of BS HH 002.SA in up to approximately 30 patients with the protocol AML indication or high risk MDS. Cohorts of 3 patients will be sequentially assigned to receive BS HH 002.SA at escalating DLs following the standard 3+3 dose escalation design, with 3 to 6 patients at each DL. Cohort Expansion Part: Following determination of the MTD, the study will continue at the MTD and/or a lower DL | Drug | the Dose Escalation Level |
Primary outcome
Dose Escalation Part: the maximum tolerated dose (MTD) of BS HH 002.SA administered subcutaneously once per day for 12 days of a 28-day cycle.
Time frame: the first 28 day cycle
Cohort Expansion Part: To evaluate safety and tolerability of BS HH 002.SA at MTD and/or lower dose level (DL) in selected cohorts of patients with the protocol AML indication or high-risk MDStreatment
Time frame: maximum of 12 months
Cohort Expansion Part: To evaluate preliminary anti-tumor activity of BS HH 002.SA at MTD and/or lower DL in selected cohorts of patients with the protocol AML indication or high-risk MDS.
Time frame: maximum of 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2021.
- 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.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BS HH 002.SA in people with Acute Myeloid Leukemia, Myelodysplastic Syndrome, run by Shanghai Bensen Pharmaceutical Co., Ltd..
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 7 inclusion and 26 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 8 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Dose Escalation Part: the maximum tolerated dose (MTD) of BS HH 002.SA administered subcutaneously once per day for 12 days of a 28-day cycle. (time frame: the first 28 day cycle). The study lists 3 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 8 Feb 2021.