Actively Recruiting
Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML
Led by Montefiore Medical Center · Updated on 2025-11-10
38
Participants Needed
2
Research Sites
303 weeks
Total Duration
On this page
Sponsors
M
Montefiore Medical Center
Lead Sponsor
M
M.D. Anderson Cancer Center
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a Phase 1 study investigating the safety and efficacy of Danvatirsen as a monotherapy followed by combination with Venetoclax in patients with relapsed/refractory myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). Funding Source: FDA OOPD
CONDITIONS
Official Title
Danvatirsen Monotherapy Followed by Combination With Venetoclax in Relapsed/Refractory MDS & AML
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older at consent
- Confirmed diagnosis of AML or MDS by WHO criteria
- Relapsed or refractory AML after all standard therapies with no approved options
- Intermediate, high, or very high risk MDS by IPSS-R, refractory or relapsed after at least 2 cycles of hypomethylating agents or rapid disease progression
- At least 3 months since allogeneic stem cell transplant with no active graft versus host disease
- White blood cell count under 25,000 cells/uL (can use hydroxyurea to reduce)
- Bone marrow biopsy within 28 days before study entry with tissue collected for analysis
- ECOG performance status 0 to 2
- Recovery to Grade 1 or baseline for prior treatment toxicities (except alopecia)
- Adequate liver function (ALT/AST ≤3x ULN or ≤5x ULN if leukemia-related; direct bilirubin ≤1.5x ULN or ≤3x ULN with Gilbert's syndrome or leukemia-related)
- Creatinine clearance ≥45 mL/min/1.73 m2
- Women and men of childbearing potential (ages 18-50) must use two forms of contraception during study
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia
- Low or very low risk MDS by IPSS-R after failure of first-line hypomethylating therapy
- Active uncontrolled infection (controlled infections with antibiotics allowed)
- Active central nervous system leukemia unless cleared by two consecutive lumbar punctures and no active symptoms
- Use of prohibited medications as listed in protocol
- Other anticancer treatments, major surgery, or investigational drugs within 14 days before study treatment
- Other cancers needing treatment within 6 months, except certain skin cancers or treated malignancies without active disease
- Pregnant or breastfeeding females
- Known current alcohol or drug abuse
- Significant cardiovascular disease within past 6 months
- Psychiatric conditions preventing informed consent
- Legal incapacity or limited legal capacity
- Any condition making participation unsafe or unsuitable
- Previous exposure to Danvatirsen
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Trial Site Locations
Total: 2 locations
1
Montefiore Medical Center
The Bronx, New York, United States, 10467
Actively Recruiting
2
M.D. Anderson Cancer Center, Department of Leukemia
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
A
Aditi Shastri, MBBS
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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