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Danvatirsen for Relapsed/Refractory MDS & AML

This study is testing Danvatirsen, first by itself and then in combination with Venetoclax, for people with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) that has come back or not responded to previous treatments. Danvatirsen works by targeting a protein called STAT3, which is often overactive in some cancers. Venetoclax targets another protein called BCL-2. The study aims to see how safe and effective these treatments are. To join, you must be at least 18 years old and have a confirmed diagnosis of AML or MDS that has relapsed or is refractory to prior therapies. The main goal is to measure how many participants respond to the treatment, which will be checked for up to 14 weeks after starting treatment. This is a Phase 1 study, meaning it's an early look at a new treatment.

Study design
This is a Phase 1 study, which is an early-stage study to test safety and effectiveness. It plans to enroll 38 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
The main treatment response will be measured for up to 14 weeks after starting treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05986240

Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML

Recruiting
PHASE1Ages 18+InterventionalTreatment
Montefiore Medical Center
~38 participants
Updated 2025-11-10 on ClinicalTrials.gov
What's tested:DanvatirsenDanvatirsen + Venetoclax

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Response to Therapy as determined by Overall Response Rate
Measured over From 21 days after initiation of study treatment to within 14 days following treatment discontinuation; up to 14 weeks total

Conditions

AML/MDS
Acute Myeloid Leukemia
Myelodysplastic Syndromes

Explore related trials

Acute Myeloid LeukemiaMyelodysplastic Syndrome

Where it's being run

2 sites across 2 states
New York1
Texas1

Study leadership

  • Aditi Shastri, MBBS · PRINCIPAL_INVESTIGATOR · Montefiore Medical Center

Who to contact

Aditi Shastri, MBBS
[email protected]
718-920-4826
Email the study team

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Eligibility criteria

Inclusion

Subjects must be at least 18 years of age at the time of signing the Informed Consent Form (ICF); must voluntarily sign an ICF; and be able to meet all study requirements
Morphologically confirmed diagnosis of AML or MDS in accordance with World Health Organization (WHO) diagnostic criteria
Subjects with relapsed/refractory AML who are refractory or relapsed to all conventional therapy and do not have any FDA approved or standard therapeutic options \& subjects with intermediated/high/very high IPSS-R MDS who are refractory or relapsed to at least 2 cycles of hypomethylating agent based therapy (azacitidine / decitabine based) OR patients with rapid progression of disease regardless of number of cycles of therapy
At least 3 months from Allogenic stem cell transplantation and no clinical sign of active graft vs host disease (GVHD)
WBC must be \<25,000 cells/uL and may be reduced with hydroxyurea to reach this goal prior to study start. Hydroxyurea can be administered on trial with an increase in WBC counts at the discretion of the PI
A bone marrow biopsy must be performed within the screening window (day-28-day-1) and tissue collected for correlative analysis for entrance to this trial. Correlative sample collection is essential on this study
Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0-2
Recovery to ≤ Grade 1 or baseline for any toxicities considered to be due to prior systemic treatments, excluding alopecia
Must have adequate hepatic and renal function as follows:
Serum creatinine clearance ≥ 45 mL/min/1.73 m2 either measured or calculated using standard Cockroft-Gault formula
Subjects enrolled within childbearing ages of 18-50 years should use 2 forms of contraception while on study

Exclusion

Acute Promyelocytic Leukemia
Low or very low risk MDS by IPSS-R after failure/progression of first line therapy with hypomethylating agents
Active, uncontrolled infection. Patients with infection under active treatment and controlled with antibiotics are eligible. Use of prophylactic anti-microbials per institutional standards is allowed
Active documented central nervous system (CNS) leukemia. Patients with a known history of CNS leukemia will be eligible if they have at least two most recent consecutive LPs showing clearance of CNS disease and no active/progressive symptoms thought to be related to the CNS disease
Concurrent treatment with a non-permitted concomitant medication (as noted in protocol appendix)
Concurrent anticancer treatment, major surgery, or the use of any investigational drug within 14 days before the start of trial treatment
Other malignancy currently being treated or likely to need treatment in next 6 months with the exception of basal or squamous cell carcinoma of the skin or cervical carcinoma in situ, surgically removed malignancies or malignancies definitively treated with chemotherapy, XRT and/or surgery with no evidence of active malignancy or not anticipated to need treatment in next 6 months or malignancies on maintenance therapy (e.g. tamoxifen for breast cancer) will be allowed after discussion and approval by both MPIs
Pregnant or breastfeeding females
Known current alcohol or drug abuse
Clinically significant cardiovascular disease within the past 6 months (e.g. percutaneous intervention, coronary artery bypass graft, documented NYHA class III/IV cardiac heart failure, unstable angina or MI, poorly controlled atrial or ventricular arrhythmia) as determined by the investigator
Any psychiatric condition that would prohibit the understanding or rendering of informed consent
Legal incapacity or limited legal capacity to sign consent and/or participate in the trial
Any condition deemed by the investigator to make the patient a poor candidate for clinical trial and/or treatment with investigational agents
Previous exposure to the investigational agent (danvatirsen)

What this trial measures

  • Response to Therapy as determined by Overall Response RateFrom 21 days after initiation of study treatment to within 14 days following treatment discontinuation; up to 14 weeks total

    Overall Response Rate (ORR) will be used to assess clinical activity in AML and MDS participants. ORR will be measured as the percentage of participants demonstrating an objective overall response based upon a composite of remission related measures for AML and MDS participants as defined below. For AML participants, ORR will be defined as any confirmed Complete Remission (CR) + Complete Remission with Incomplete hematologic recovery (CRi) + Partial Remission (PR). or, AML = CR + CRi + PR For MDS participants, ORR will be defined as any confirmed Complete Remission (CR) + Complete Remission with Partial Hematologic Recovery (CRh) + Hematologic Response (HR). or, MDS = CR + CRh + HR Higher percentage is indicative of increased clinical activity of the danvatirsen monotherapy and danvatirsen + venetoclax combination therapies.