AZD6738 for Myelodysplastic Syndrome
Recruiting in Palo Alto (17 mi)
+3 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1
Recruiting
Sponsor: Massachusetts General Hospital
Must not be taking: CYP3A4 inhibitors, CYP3A4 inducers
Disqualifiers: Ataxia telangiectasia, Acute myeloid leukemia, HIV, Hepatitis B, others
No Placebo Group
Trial Summary
What is the purpose of this trial?This research study is studying a research drug called AZD6738 as a possible treatment for Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia .
Will I have to stop taking my current medications?
The trial requires a washout period (time without taking certain medications) of 5 half-lives for potent inhibitors or inducers of CYP3A4 before starting AZD6738, or three weeks for St. John's Wort. For other medications, the decision will be made on a case-by-case basis with the study doctor.
How is the drug AZD6738 (Ceralasertib) unique for treating myelodysplastic syndrome?
AZD6738 (Ceralasertib) is unique because it targets specific pathways involved in DNA damage response, which is different from other treatments that often focus on inhibiting blood vessel growth or targeting specific mutations. This novel approach may offer a new option for patients with myelodysplastic syndrome who have limited treatment choices.
12345Eligibility Criteria
Adults diagnosed with Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) who have not responded to, or cannot tolerate, certain chemotherapy treatments. They should be in a stable condition without acute leukemia and must not have other active cancers. Participants need functioning major organs and agree to use contraception.Inclusion Criteria
My MDS or CMML has returned after a stem cell transplant.
Your liver function tests should show AST and ALT levels that are not more than 2.5 times the upper limit of normal.
My MDS or CMML has returned after a stem cell transplant.
+24 more
Exclusion Criteria
Involvement in the planning and/or conduct of the study
My leukemia has spread to my brain or spinal cord.
I had a transplant less than 100 days ago and am not on strong immune-suppressing drugs.
+22 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive AZD6738 orally on a 28-day cycle to evaluate safety, tolerability, and effect on tumor growth
28 days
4 visits (in-person)
Follow-up
Participants are monitored for safety and effectiveness after treatment, including overall response rate and survival rates
2 years
Participant Groups
The trial is testing AZD6738, an investigational drug for treating MDS/CMML. It's designed for patients whose disease has progressed after standard therapies or those ineligible for stem cell transplantation. The study will assess the drug's effectiveness and safety.
1Treatment groups
Experimental Treatment
Group I: AZD6738Experimental Treatment1 Intervention
Patients will receive AZD6738 orally on a 28-day cycle
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
BIDMCBoston, MA
Massachusetts General Hospital Cancer CenterBoston, MA
Dana-Farber Cancer InstituteBoston, MA
Washington UniversitySaint Louis, MO
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Who Is Running the Clinical Trial?
Massachusetts General HospitalLead Sponsor
AstraZenecaIndustry Sponsor
References
A phase II study of AZD2171 (cediranib) in the treatment of patients with acute myeloid leukemia or high-risk myelodysplastic syndrome. [2021]Patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) not fit for intensive treatment need novel therapy options. Vascular endothelial growth factor (VEGF) receptor inhibition is one potential mechanism by which AML and MDS could be treated. The receptor tyrosine kinase inhibitor AZD2171 (cediranib) has activity against VEGF receptors KDR and FLT-1. This multicenter phase II study was designed to test cediranib's activity in patients with AML or high-risk MDS. The primary endpoint was confirmed disease response defined as a composite of complete remission, partial remission or hematologic improvement. The study enrolled 23 subjects in the AML cohort and 16 subjects in the MDS cohort. There were no confirmed responses in either group. Since the study met the stopping rule after the first stage of enrollment, the trial was closed to further accrual. Common adverse events in both cohorts included thrombocytopenia, neutropenia, anemia, fatigue, dyspnea, diarrhea, nausea and dehydration.
Targeting the Myeloid Lineages and the Immune Microenvironment in Myelodysplastic Syndromes: Novel and Evolving Therapeutic Strategies. [2022]To discuss the recent and emerging data for novel targeted therapies in myelodysplastic syndromes (MDS).
Positive First Trial of Enasidenib for AML. [2022]The first clinical trial of the investigational drug enasidenib, which targets IDH2 mutations, suggests that it is safe and may improve survival in patients with relapsed or refractory acute myeloid leukemia. It induced responses in 40.3% of patients, yielding a median overall survival of 9.3 months.
A phase II study of the oral VEGF receptor tyrosine kinase inhibitor vatalanib (PTK787/ZK222584) in myelodysplastic syndrome: Cancer and Leukemia Group B study 10105 (Alliance). [2021]Angiogenesis is implicated in the pathophysiology and progression of myelodysplastic syndromes (MDS). Vatalanib (PTK787/ZK222584; Novartis and Schering AG) inhibits receptor tyrosine kinases of vascular endothelial growth factor, platelet derived growth factor and c-Kit. We examined whether vatalanib induces hematological responses in MDS and/or delays progression to acute myeloid leukemia (AML) or death.
Enasidenib Approved for AML, but Best Uses Unclear. [2018]The FDA approved the targeted therapy enasidenib for patients with refractory or relapsed acute myeloid leukemia with mutant IDH2 The drug produces remissions in some patients and may reduce the need for blood transfusions, although researchers acknowledge that the FDA's approval came with less supporting evidence than usual.