Leukemia, Myeloid, AcutePhase 1NCT03110354
DS-3201b for Acute Myelogenous Leukemia (AML) or Acute Lymphocytic Leukemia (ALL)
A Phase 1 study testing DS-3201b in people with Leukemia, Myeloid, Acute and Leukemia, Lymphocytic, Acute, run by Daiichi Sankyo at 6 sites in United States.
Trial summary
This research study tests an investigational drug called DS-3201b. An investigational drug is a medication that is still being studied and has not yet been approved by the United States Food and Drug Administration (FDA). The FDA allows DS-3201b to be used only in research. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute
- What is being tested?
- DS-3201b
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 6 sitesUnited States
- Study dates
- 5 Apr 2017to 9 Mar 2021
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Has AML or ALL and failed any prior induction therapy regimen or have relapsed after prior therapy
- Has Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Has adequate renal and hepatic function
- Had at least 14 days for prior treatment to clear the body before initiation of DS-3201b administration (except for hydroxyurea that needs only 2 days for clearance)
- Able to provide written informed consent, comply with protocol visits and procedures, be able to take oral medication, and not have any active infection or comorbidity that would interfere with therapy.
- Agrees to use an adequate method of contraception during the study and until 3 months after the last treatment.
- Is willing to provide bone marrow biopsies and comply with protocol-defined evaluations
- Has a life expectancy of at least 3 months
Who cannot join
- Has presence of central nervous system (CNS) involvement of leukemia or a history of CNS leukemia
- Has a second concurrent active primary malignancy such as solid tumor or lymphoma under active treatment
- Has refractory nausea and vomiting, malabsorption, biliary shunt, significant bowel resection, graft versus- host disease (GVHD) significantly affecting gut motility or absorption, or any other condition that would preclude adequate absorption of DS- 3201b in the opinion of the treating physician and/or principal investigator (PI)
- Has an uncontrolled infection requiring intravenous antibiotics, antivirals, or antifungals, known human immunodeficiency virus infection, or tested positive for active hepatitis B or C infection
- Has a concomitant medical condition that would increase the risk of toxicity, in the opinion of the Investigator or Sponsor
- Has unresolved toxicities from previous anticancer therapy
Show all 14Show fewer
- Has received hematopoietic stem cell transplantation (HSCT) within 60 days of the first dose of DS-3201b
- Has received concomitant treatment with a strong inhibitor or inducer of cytochrome P450 (CYP)3A4/5 within 7 days of first receipt of DS-3201b
- Has consumed herbs/fruits that may have an influence on pharmacokinetics (PK) of DS-3201b from 3 days (14 days for St. John's wort) prior to the start of the study and throughout the entire study
- Had major surgery within 4 weeks before study drug treatment
- Has prolonged corrected QT interval by Fridericia's method (QTcF) at rest, where the mean QTcF interval is > 450 milliseconds (ms) based on triplicate electrocardiograms (ECGs)
- Is pregnant or breastfeeding
- Has substance abuse or medical, psychological, or social conditions that, in the opinion of the Investigator, may interfere with the subject's participation in the clinical study or evaluation of the clinical study results
- Has received prior treatment with enhancer of zeste homolog (EZH) inhibitor
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
University of Michigan
Ann Arbor, Michigan, 48109
Massachusetts General Hospital
Boston, Massachusetts, 02115
Duke University
Durham, North Carolina, 27710
MD Anderson Cancer Center
Houston, Texas, 77030
Vanderbilt University
Nashville, Tennessee, 37232
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Phase 1 Study of DS-3201b in Subjects With Acute Myelogenous Leukemia (AML) or Acute Lymphocytic Leukemia (ALL)
This research study tests an investigational drug called DS-3201b. An investigational drug is a medication that is still being studied and has not yet been approved by the United States Food and Drug Administration (FDA). The FDA allows DS-3201b to be used only in research. It is not known if DS-3201b will work or not.
This study consists of two parts. The first part (Part 1) is a dose escalation that will enroll subjects with AML or ALL that did not respond or no longer respond to previous standard therapy. The purpose of Part 1 of this research study is to determine the highest dose a patient can tolerate or recommended dose of DS-3201b that can be given to subjects with AML or ALL. Once the highest tolerable dose is determined, additional subjects will be enrolled at that dose into Part 2 of the study.
- Being tested
- DS-3201b
- Keywords
- AML, ALL, Enhancer of zeste homolog (EZH) inhibitor, DS-3201b
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DS-3201bDrug
DS-3201b is supplied as 25 and 100 mg capsules packaged in high-density polyethylene (HDPE) bottles.
| Study group | Role | What they receive |
|---|---|---|
| DS-3201b 100 mg | Experimental | Participants who received 100 mg DS-3201b administered orally to participants with AML or ALL. Receives: Drug: DS-3201b |
| DS-3201b 150 mg | Experimental | Participants who received 150 mg DS-3201b administered orally to participants with AML or ALL. Receives: Drug: DS-3201b |
| DS-3201b 250 mg | Experimental | Participants who received 250 mg DS-3201b administered orally to participants with AML or ALL. Receives: Drug: DS-3201b |
| DS-3201b 500 mg | Experimental | Participants who received 500 mg DS-3201b administered orally to participants with AML or ALL. Receives: Drug: DS-3201b |
| DS-3201b 700 mg | Experimental | Participants who received 700 mg DS-3201b administered orally to participants with AML or ALL. Receives: Drug: DS-3201b |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Any Grade Treatment-emergent Adverse Event Classified as Dose-limiting Toxicities (Dose Escalation)
Dose-limiting toxicity (DLT) is defined as a clinically significant non-hematologic treatment-emergent adverse event (TEAE) or abnormal clinical laboratory value that is clearly not related to disease progression, intercurrent illness, and occurring during the first cycle (28 days) on study that meets any of the following criteria: National Cancer Institute (NCI) - Common Terminology Criteria for Adverse Events (CTCAE), Version 4 Grade 3 aspartate aminotransferase (AST) (SGOT), alanine aminotransferase (ALT) (SGPT), or bilirubin for ≥7 days; NCI-CTCAE Grade 4 AST (SGOT) or ALT (SGPT) of any duration; All Grade 4 non-hematologic toxicities of any duration; Any Grade 5 toxicity, unless proven to be clearly and incontrovertibly related to disease progression or intercurrent illness will constitute a DLT; All other clinically significant, non-hematological NCI-CTCAE Grade 3/4 AEs. AEs were coded using the MedDRA dictionary, Version 23.0.
Measured: Baseline up to Day 28
Number of Participants Who Experienced Any Grade Treatment-emergent Adverse Event (Dose Escalation)
A treatment-emergent adverse event (TEAE) is defined as an adverse event (AE) that occurs, having been absent before the first dose of study drug or has worsened after initiating the study drug until 30 days after the last dose of the study drug. AEs were coded using the MedDRA dictionary, Version 23.0.
Measured: Baseline up to 30 days after last study dose, up to approximately 4 years
Secondary outcomes (4)
Pharmacokinetic Parameter Maximum (Peak) Observed Concentration (Cmax) of DS-3201b
Pharmacokinetic parameters were assessed using noncompartmental methods.
Measured: Cycle 1 Days 1 and 8 predose, 0.5, 1, 2, 4, 6, and 8 hours and Cycle 1 Day 15 postdose (each cycle is 28 days)
Pharmacokinetic Parameter Time to Maximum Observed Concentration (Tmax) of DS-3201b
Blood samples were collected for PK analysis. PK parameters were assessed using noncompartmental methods.
Measured: Cycle 1 Days 1 and 8 predose, 0.5, 1, 2, 4, 6, and 8 hours and Cycle 1 Day 15 postdose (each cycle is 28 days)
Pharmacokinetic Parameter Area Under Plasma Concentration-Time Curve of DS-3201b
Blood samples were collected for PK analysis. Area under the plasma concentration-time curve up to 24 hours (AUC24h) and area under the plasma concentration-time curve up to the last measurable concentration (AUClast) were assessed using noncompartmental methods.
Measured: Cycle 1 Days 1 and 8 predose, 0.5, 1, 2, 4, 6, and 8 hours and Cycle 1 Day 15 postdose (each cycle is 28 days)
Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough) of DS-3201b
Blood samples were collected for PK analysis. PK parameters were assessed using noncompartmental methods.
Measured: Cycle 1 Day 2 postdose (each cycle is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2017
- Study start5 Apr 2017
- Primary completion9 Mar 2021
- Study completion9 Mar 2021
Why it stopped: This study was terminated due to slow accrual.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Leukemia, Myeloid, AcuteStudies of the same condition, open and under way.
- More trials run by Daiichi SankyoThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Daiichi Sankyo
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Leader, Study director, Daiichi Sankyo
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03110354
- Sponsor's protocol ID
- DS3201-A-U102
- Status last verified
- Jan 2024
- First submitted
- 31 Mar 2017
- Last updated
- 31 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
Publications
Tachibana M, Poland B, Kakurai Y, Chen Y, Li C, Lau Y. Concentration-QTc Analysis of Valemetostat in Patients With Hematologic Malignancies. Clin Transl Sci. 2025 Nov;18(11):e70391. doi: 10.1111/cts.70391.
PubMed 41258689
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study of DS-3201b in Subjects With Acute Myelogenous Leukemia (AML) or Acute Lymphocytic Leukemia (ALL)
- ClinicalTrials.gov ID
- NCT03110354
- Acronym / sponsor ID
- DS3201-A-U102
- Keywords
- AML, ALL, Enhancer of zeste homolog (EZH) inhibitor, DS-3201b
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was terminated due to slow accrual.
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Daiichi SankyoIndustry (pharma, biotech, devices)
- Study start
- 5 Apr 2017 (actual)
- Primary completion
- 9 Mar 2021 (actual)
- Study completion
- 9 Mar 2021 (actual)
- First posted
- 12 Apr 2017 (actual)
- Results
- Posted 30 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study tests an investigational drug called DS-3201b. An investigational drug is a medication that is still being studied and has not yet been approved by the United States Food and Drug Administration (FDA). The FDA allows DS-3201b to be used only in research. It is not known if DS-3201b will work or not.
This study consists of two parts. The first part (Part 1) is a dose escalation that will enroll subjects with AML or ALL that did not respond or no longer respond to previous standard therapy. The purpose of Part 1 of this research study is to determine the highest dose a patient can tolerate or recommended dose of DS-3201b that can be given to subjects with AML or ALL. Once the highest tolerable dose is determined, additional subjects will be enrolled at that dose into Part 2 of the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| DS-3201b 100 mg | Experimental | Drug: DS-3201bParticipants who received 100 mg DS-3201b administered orally to participants with AML or ALL. |
| DS-3201b 150 mg | Experimental | Drug: DS-3201bParticipants who received 150 mg DS-3201b administered orally to participants with AML or ALL. |
| DS-3201b 250 mg | Experimental | Drug: DS-3201bParticipants who received 250 mg DS-3201b administered orally to participants with AML or ALL. |
| DS-3201b 500 mg | Experimental | Drug: DS-3201bParticipants who received 500 mg DS-3201b administered orally to participants with AML or ALL. |
| DS-3201b 700 mg | Experimental | Drug: DS-3201bParticipants who received 700 mg DS-3201b administered orally to participants with AML or ALL. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Has AML or ALL and failed any prior induction therapy regimen or have relapsed after prior therapy
- Has Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Has adequate renal and hepatic function
- Had at least 14 days for prior treatment to clear the body before initiation of DS-3201b administration (except for hydroxyurea that needs only 2 days for clearance)
- Able to provide written informed consent, comply with protocol visits and procedures, be able to take oral medication, and not have any active infection or comorbidity that would interfere with therapy.
Key exclusion criteria (14 listed)
- Has presence of central nervous system (CNS) involvement of leukemia or a history of CNS leukemia
- Has a second concurrent active primary malignancy such as solid tumor or lymphoma under active treatment
- Has refractory nausea and vomiting, malabsorption, biliary shunt, significant bowel resection, graft versus- host disease (GVHD) significantly affecting gut motility or absorption, or any other condition that would preclude adequate absorption of DS- 3201b in the opinion of the treating physician and/or principal investigator (PI)
- Has an uncontrolled infection requiring intravenous antibiotics, antivirals, or antifungals, known human immunodeficiency virus infection, or tested positive for active hepatitis B or C infection
- Has a concomitant medical condition that would increase the risk of toxicity, in the opinion of the Investigator or Sponsor
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 |
|---|---|---|
| DS-3201bDS-3201b is supplied as 25 and 100 mg capsules packaged in high-density polyethylene (HDPE) bottles. | Drug | DS-3201b 100 mg, DS-3201b 150 mg, DS-3201b 250 mg, DS-3201b 500 mg, DS-3201b 700 mg |
Primary outcome
Number of Participants With Any Grade Treatment-emergent Adverse Event Classified as Dose-limiting Toxicities (Dose Escalation)
Time frame: Baseline up to Day 28
Dose-limiting toxicity (DLT) is defined as a clinically significant non-hematologic treatment-emergent adverse event (TEAE) or abnormal clinical laboratory value that is clearly not related to disease progression, intercurrent illness, and occurring during the first cycle (28 days) on study that meets any of the following criteria: National Cancer Institute (NCI) - Common Terminology Criteria for Adverse Events (CTCAE), Version 4 Grade 3 aspartate aminotransferase (AST) (SGOT), alanine aminotransferase (ALT) (SGPT), or bilirubin for ≥7 days; NCI-CTCAE Grade 4 AST (SGOT) or ALT (SGPT) of any duration; All Grade 4 non-hematologic toxicities of any duration; Any Grade 5 toxicity, unless proven to be clearly and incontrovertibly related to disease progression or intercurrent illness will constitute a DLT; All other clinically significant, non-hematological NCI-CTCAE Grade 3/4 AEs. AEs were coded using the MedDRA dictionary, Version 23.0.
Number of Participants Who Experienced Any Grade Treatment-emergent Adverse Event (Dose Escalation)
Time frame: Baseline up to 30 days after last study dose, up to approximately 4 years
A treatment-emergent adverse event (TEAE) is defined as an adverse event (AE) that occurs, having been absent before the first dose of study drug or has worsened after initiating the study drug until 30 days after the last dose of the study drug. AEs were coded using the MedDRA dictionary, Version 23.0.
4 secondary outcomes
Pharmacokinetic Parameter Maximum (Peak) Observed Concentration (Cmax) of DS-3201b
Time frame: Cycle 1 Days 1 and 8 predose, 0.5, 1, 2, 4, 6, and 8 hours and Cycle 1 Day 15 postdose (each cycle is 28 days)
Pharmacokinetic Parameter Time to Maximum Observed Concentration (Tmax) of DS-3201b
Time frame: Cycle 1 Days 1 and 8 predose, 0.5, 1, 2, 4, 6, and 8 hours and Cycle 1 Day 15 postdose (each cycle is 28 days)
Pharmacokinetic Parameter Area Under Plasma Concentration-Time Curve of DS-3201b
Time frame: Cycle 1 Days 1 and 8 predose, 0.5, 1, 2, 4, 6, and 8 hours and Cycle 1 Day 15 postdose (each cycle is 28 days)
Pharmacokinetic Parameter Trough Plasma Concentration (Ctrough) of DS-3201b
Time frame: Cycle 1 Day 2 postdose (each cycle is 28 days)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41258689Tachibana M, Poland B, Kakurai Y, Chen Y, Li C, Lau Y. Concentration-QTc Analysis of Valemetostat in Patients With Hematologic Malignancies. Clin Transl Sci. 2025 Nov;18(11):e70391. doi: 10.1111/cts.70391.
- Individual participant dataSharing planned: Yes. De-identified individual participant data (IPD) and applicable supporting clinical trial documents may be available upon request at https://vivli.org/. In cases where clinical trial data and supporting documents are provided pursuant to our company policies and procedures, Daiichi Sankyo will continue to protect the privacy of our clinical trial participants. Details on data sharing criteria and the procedure for requesting access can be found at this web address: https://vivli.org/ourmember/daiichi-sankyo/
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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.
Global Clinical Leader study director
Daiichi Sankyo
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing DS-3201b in people with Leukemia, Myeloid, Acute and Leukemia, Lymphocytic, Acute, run by Daiichi Sankyo at 6 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 8 inclusion and 14 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: stopped early, as of the sponsor's last update on 31 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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 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?
Yes. Results are posted in the registry (first posted 30 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Any Grade Treatment-emergent Adverse Event Classified as Dose-limiting Toxicities (Dose Escalation) (time frame: Baseline up to Day 28). 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?
On the ClinicalTrials.gov record for NCT03110354, which also lists 1 linked publication.
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 31 Jan 2024.