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Acute Myeloid LeukemiaPhase 1NCT03113643

SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

A Phase 1 study testing Azacitidine, SL-401 and Venetoclax in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Dana-Farber Cancer Institute at 3 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

This research study is studying a drug as a possible treatment for diagnosis of AML, BPDCN and high-risk MDS. The interventions involved in this study are: SL-401 Azacitidine Venetoclax Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Azacitidine, SL-401 and Venetoclax
Could I receive a placebo?
No, everyone receives an active treatment
How many people
72 participants
Where
3 sitesUnited States
Study dates
26 Jun 2017to 31 May 2027
Phase
Phase 1
Are there results?
Not yet: the study is under way

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

  • Histologically confirmed diagnosis of acute myeloid leukemia (AML) [Cohort B] or myelodysplastic syndrome (MDS) [Cohort A] or BPDCN [Cohort C] per 2016 WHO criteria
  • CD123 / IL3RA expression on the subject's AML or MDS blasts or BPDCN cells determined locally within 3 months of first protocol treatment
  • Age >= 18 years with relapsed or refractory AML (hydroxyurea is not considered a prior treatment regimen) [Cohort B]
  • OR
  • Age >= 18 years with treatment-naïve AML who decline intensive induction chemotherapy or who are unfit due to co-morbidity or other factors (see APPENDIX A for unfitness definitions) (hydroxyurea is not considered a prior treatment regimen) [Cohort B]
  • OR
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  • Age >= 18 years with MDS and > 10% myeloblasts in the bone marrow [Cohort A]
  • OR
  • Age >= 18 years with relapsed or refractory BPDCN (hydroxyurea is not considered a prior treatment regimen) [Cohort C]
  • Adequate organ function as defined by:
  • Albumin > 3.2 g/dL (in the absence of receipt of intravenous albumin in the previous 72 hours) Serum creatinine < 1.5x ULN Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 2.5x ULN Total bilirubin < 1.5x ULN (if thought to be > 1.5x ULN due to Gilbert's disease or the patient's AML, must discuss with the PI) Creatine phosphokinase (CPK) < 2.5x ULN Left ventricular ejection fraction > institutional lower limit of normal by MUGA scan or echocardiogram within 30 days of first protocol treatment
  • [Cohorts B and C] WBC < 20,000 / uL on day of first therapy, cytoreduction may be achieved using hydroxyurea
  • Ability to understand and the willingness to sign a written informed consent document.
  • Able to adhere to study visit schedule and other protocol requirements including follow-up for survival assessment
  • Women of child-bearing potential must agree to use adequate contraception for the duration of study participation and for 2 months after completion of protocol treatment.
  • Men treated or enrolled on this protocol must also agree to use adequate contraception for the duration of study participation and 2 months after completion of protocol treatment.

Who cannot join

  • Prior treatment with venetoclax [Cohorts B or C], unless it was last taken >2 months before protocol therapy
  • Diagnosis of acute promyelocytic leukemia
  • Received treatment with chemotherapy, radiation, or biologic cancer therapy within 14 days of first protocol treatment, except for intrathecal chemotherapy. Prior and concurrent hydroxyurea is permitted.
  • Hematopoietic stem cell transplantation (HSCT) within 60 days of screening or active graft versus-host-disease
  • Active CNS involvement by AML or BPDCN. Screening lumbar puncture (LP) required for patients with BPDCN. If history of treated CNS involvement, must have had two consecutive negative LPs since last CNS involvement, which may include the screening LP
  • Known positive status for HIV infection; known active hepatitis B or hepatitis C infection
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  • Clinically significant cardiopulmonary disease including uncontrolled or NYHA class 3 or 4 congestive heart failure, uncontrolled angina, uncontrolled hypertension, uncontrolled arrhythmia, myocardial infarction or stroke within 6 months of first protocol treatment, or QTc > 480 ms
  • Patients with known active advanced malignant solid tumors are excluded (except for basal or squamous skin cancers, or carcinomas in situ). Patients with additional hematologic malignancies that require treatment are excluded.
  • Pregnant women are excluded from this study because there is an unknown but potential risk for adverse events in the developing fetus with SL-401, azacitidine, and venetoclax (negative urine or serum pregnancy test required within 14 days of Cycle 1, Day 1). Because nursing infants have unknown potential for adverse events secondary to treatment of the mother, breastfeeding should be discontinued if the mother is treated with SL-401, azacitidine, and venetoclax.
  • Patients with uncontrolled infection shall not be enrolled until infection is treated and brought under control. Patients with active infection are permitted to enroll provided that the infection is controlled
  • [Cohorts B and C] Patients with gastrointestinal (GI) tract disease causing the inability to take oral medication, malabsorption syndrome, a requirement for intravenous (IV) alimentation, prior surgical procedures affecting absorption, uncontrolled inflammatory GI disease (e.g., Crohn's disease, ulcerative colitis)
  • [Cohorts B and C] Patients on strong CYP3A inducers within 7 days of first dose of study treatment.

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Andrew Lane, MD, PhD contact

857-215-1405

andrew_lane@dfci.harvard.edu

Veronica Zehnder contact

veronica_zehnder@dfci.harvard.edu

United States3 sites, 3 recruiting

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215

    Andrew Lane, MD, PhD, andrew_lane@dfci.harvard.edu

    Veronica Zehnder, veronica_zehnder@dfci.harvard.edu

    Recruiting

  • City of Hope

    Duarte, California, 91010

    Anthony Stein, MD, 626-359-8111, AStein@coh.org

    Anthony Stein, MD

    Recruiting

  • MD Anderson Cancer Center

    Houston, Texas, 77030

    Naveen Pemmaraju, MD, 713-792-4956, npemmaraju@mdanderson.org

    Naveen Pemmaraju, MD

    Recruiting

Other trials in United States

About this study

Official title: Phase 1 Study of SL-401 in Combination With Azacitidine and Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia (AML) and in Treatment-Naive Subjects With AML Not Eligible for Standard Induction and in Subjects With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or SL-401 in Combination With Azacitidine in Subjects With High-Risk Myelodysplastic Syndrome (MDS)

This research study is studying a drug as a possible treatment for diagnosis of AML, BPDCN and high-risk MDS.

The interventions involved in this study are:

  • SL-401
  • Azacitidine
  • Venetoclax
Read the detailed description

This research study is a Phase I clinical trial, which tests the safety of an investigational intervention and also tries to define the appropriate dose of the investigational intervention to use for further studies. "Investigational" means that the intervention is being studied.

The FDA (the U.S. Food and Drug Administration) has approved azacitidine and venetoclax as a treatment option for AML. However, the combination of these two drugs with SL-401 has not been FDA approved.

The combination of SL-401, azacitidine and venetoclax has not been FDA approved for BPDCN. However, SL-401 has been FDA approved for BPDCN.

The combination of SL-401 and azacitidine has not been FDA approved for BPDCN.

In this research study, the study drug SL-401 will be combined with the standard dose of azacitidine (for MDS patients) or azacitidine/venetoclax (for AML and BPDCN patients). The goal of this research study is to try and determine the safest, highest dose of study drug, SL-401, in combination with azacitidine or azacitidine/venetoclax that can be given to patients with AML, BPDCN or high-risk MDS. SL-401 works by stopping or slowing the growth of cancer stem cells, which are the undeveloped cells which can develop into cancer cells. The goals of this research study are to look at if this combination works to help treat your cancer and if there is any lasting effect of this combination. This study will also look at how the SL-401, in combination with azacitidine or azacitidine/venetoclax, affects certain proteins in your blood and bone marrow. SL-401 has been given to patients with AML, and MDS in the past, but this is the first time it will be given in combination with another drug.

Being tested
Azacitidine, SL-401, Venetoclax
Keywords
Acute Myeloid Leukemia, Myelodysplastic Syndrome, Blastic Plasmacytoid Dendritic Cell Neoplasm, BPDCN
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasm

What participants receive

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

  • AzacitidineDrug

    Also called Vidaza

    Chemotherapy

  • SL-401Drug

    SL-401 works by targeting leukemia cells (blasts), and also possibly by stopping or slowing the growth of cancer stem cells, which are the undeveloped cells which can develop into cancer cells.

  • VenetoclaxDrug

    Also called Venclyxto, Venclexta

    Venetoclax is a BH3-mimetic. Venetoclax blocks the anti-apoptotic B-cell lymphoma-2 (Bcl-2) protein, leading to programmed cell death of CLL cells. Overexpression of Bcl-2 in some lymphoid malignancies has sometimes shown to be linked with increased resistance to chemotherapy.

Study groups in NCT03113643
Study groupRoleWhat they receive
SL-401+ AzacitidineExperimentalSL-401 will be administered every 4 weeks, on a 28 day cycle; SL-401 will be given intravenously; Azacitidine will be administered every 4 weeks, on a 28 day cycle; Azacitidine will be given intravenously or subcutaneously
Receives: Drug: Azacitidine; Drug: SL-401
SL-401+ Azacitidine + VenetoclaxExperimentalSL-401 will be administered every 4 weeks, on a 28 day cycle; SL-401 will be given intravenously; Azacitidine will be administered every 4 weeks, on a 28 day cycle; Azacitidine will be given intravenously or subcutaneously; Venetoclax will be administered for 21 days on a 28 day cycle; Venetoclax will be taken orally
Receives: Drug: Azacitidine; Drug: SL-401; Drug: Venetoclax

What is being measured

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

  1. Maximum Tolerated Dose

    To determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of SL-401 in combination with azacitidine or in combination with azacitidine and venetoclax in this patient population and evaluate the safety of this regimen

    Measured: 2 years

Secondary outcomes (5)

  1. Complete Response Rate

    To estimate the complete remission (CR) / CR with incomplete count recovery (CRi) rate within 6 cycles of combination therapy consisting of SL-401 administered with azacitidine or in combination with azacitidine and venetoclax in subjects with AML and high-risk MDS

    Measured: 2 years

  2. Time to response

    To estimate the time to response with SL-401 in combination with azacitidine or in combination with azacitidine and venetoclax

    Measured: 2 years

  3. Duration of remission

    To estimate the duration of remission with SL-401 in combination with azacitidine or in combination with azacitidine and venetoclax

    Measured: 2 years

  4. Progression Free Survival

    To estimate the 1 and 2-year progression free survival (PFS) with SL-401 in combination with azacitidine or in combination with azacitidine and venetoclax

    Measured: 1 and 2 years

  5. Overall Survival

    To estimate the overall survival (OS) with SL-401 in combination with azacitidine or in combination with azacitidine and venetoclax

    Measured: 2 years

Timeline

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

  1. First posted13 Apr 2017
  2. Study start26 Jun 2017
  3. Primary completion15 Sep 2021
  4. Study completion31 May 2027 (estimated)

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
72 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03113643
Sponsor's protocol ID
17-056
Status last verified
Jun 2026
First submitted
10 Apr 2017
Last updated
17 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Lane AA, Luskin MR, Keating J, LeBoeuf NR, Garcia JS, Shimony S, Leonard R, Goodpaster A, Nguyen TT, Galinsky I, Eberly-Puleo A, Daver N, Jain N, Garcia-Manero G, Kantarjian H, Konopleva MY, Stein A, Pemmaraju N. Tagraxofusp, azacitidine, and venetoclax for blastic plasmacytoid dendritic cell neoplasm. Blood. 2026 Oct 29;148(18):2229-2238. doi: 10.1182/blood.2026033906.

    PubMed 42413008

  • Lane AA, Garcia JS, Raulston EG, Garzon JL, Galinsky I, Baxter EW, Leonard R, DeAngelo DJ, Luskin MR, Reilly CR, Stahl M, Stone RM, Vedula RS, Wadleigh MM, Winer ES, Mughal T, Brooks C, Gupta IV, Stevenson KE, Neuberg DS, Ren S, Keating J, Konopleva M, Stein A, Pemmaraju N. Phase 1b trial of tagraxofusp in combination with azacitidine with or without venetoclax in acute myeloid leukemia. Blood Adv. 2024 Feb 13;8(3):591-602. doi: 10.1182/bloodadvances.2023011721.

    PubMed 38052038

  • Wang SY, Thomassen K, Kurch L, Opitz S, Franke GN, Bach E, Platzbecker U, Kayser S. Combination of Tagraxofusp and Azacitidine Is an Effective Option for Relapsed Blastic Plasmacytoid Dendritic Cell Neoplasm After Allogeneic Hematopoietic Stem-Cell Transplantation. Clin Lymphoma Myeloma Leuk. 2021 Jul;21(7):e579-e582. doi: 10.1016/j.clml.2021.02.008. Epub 2021 Mar 2. No abstract available.

    PubMed 33795208

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Azacitidine, SL-401 and Venetoclax in people with Acute Myeloid Leukemia and Myelodysplastic Syndrome, run by Dana-Farber Cancer Institute at 3 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 16 inclusion and 12 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: looking for participants now, as of the sponsor's last update on 17 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 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?

This study is still under way. Results are not available in the source record yet.

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

Contact Andrew Lane, MD, PhD (857-215-1405, andrew_lane@dfci.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Tolerated Dose (time frame: 2 years). 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 NCT03113643, which also lists 3 linked publications.

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 17 Jun 2026.

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