AZD9829 for Blood Cancers

Recruiting at 23 trial locations

Overseen By AstraZeneca Clinical Study Information Center

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: AstraZeneca

Must not be taking: Immunosuppressants

Disqualifiers: CNS leukemia, Other malignancy, Infections, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

104 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AZD9829 for certain types of blood cancers. The goal is to determine the right dose and assess its safety and effectiveness, either alone or with other treatments. It targets individuals with blood cancers that test positive for CD123, such as acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (HR-MDS), who have not responded to other treatments. Participants should not have active central nervous system leukemia and should not have received similar treatments targeting CD123 before. This trial may suit those with no remaining treatment options. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that AZD9829 is likely to be safe for humans?

Research has shown that AZD9829 has been tested in lab studies on monkeys. These studies suggest potential for development in treating certain blood cancers, such as acute myeloid leukemia (AML). In these tests, AZD9829 produced promising results with only mild effects on healthy cells, indicating it might be well-tolerated in humans.

Although limited safety data from human trials exists, the treatment remains in early testing stages. Researchers are focused on understanding its safety and determining the right dose. Because AZD9829 is in these early stages, it is still under close study for any side effects in people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about AZD9829 because it targets CD123, a molecule commonly found on the surface of certain blood cancer cells like those in relapsed or refractory acute myeloid leukemia (R/R AML). Unlike traditional chemotherapy, which attacks rapidly dividing cells indiscriminately, AZD9829 is designed to specifically recognize and bind to CD123, potentially reducing damage to healthy cells and minimizing side effects. This targeted approach might offer a more effective and safer alternative to the current standard treatments, which often include chemotherapy and stem cell transplants. Additionally, AZD9829 may provide hope for patients with R/R AML who have few other options.

What evidence suggests that AZD9829 might be an effective treatment for blood cancers?

Research has shown that AZD9829 holds promise for treating blood cancers with the CD123 marker. In one study, AZD9829 reduced cancerous white blood cells by at least 50% in both blood and bone marrow samples from six out of seven models tested. This result indicates a significant reduction in cancer cells. Importantly, the drug specifically targets cancer cells, with minimal impact on healthy cells. The trial will explore AZD9829 in two separate treatment arms: one focusing on dose escalation and the other on dose optimization. These findings suggest that AZD9829 could effectively treat certain blood cancers, such as acute myeloid leukemia (AML). ^{5 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for individuals with blood cancers that express a marker called CD123. Specific details about who can join or reasons why someone might not be eligible are not provided.

Inclusion Criteria

My blood cancer tests positive for CD123.

My condition is relapsed or refractory AML.

My bone marrow has at least 5% cancer cells.

Exclusion Criteria

I have had cancer before, but it might not affect my eligibility.

I have no severe side effects from previous treatments.

I have leukemia that has spread to my brain.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Ascending dose level cohorts of AZD9829 in AML and MDS participants to determine safety and tolerability

4 weeks

Dose Optimization

Characterizing the safety, tolerability, PK/PD, and preliminary antitumor activity of AZD9829 in CD123+ R/R AML participants

Approximately 1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AZD9829

Trial Overview The study is testing AZD9829, given through the vein either alone or with other treatments, to see how it works in treating various blood cancers marked by CD123.

How Is the Trial Designed?

Group I: Module 1: Dose Optimization Experimental Treatment 1 Intervention

Characterizing the safety, tolerability, PK/PD, and preliminary antitumor activity of AZD9829 in CD123+ R/R AML participants, based on the data collected during dose escalation, dose optimization and backfill.

Group II: Module 1: Dose Escalation Experimental Treatment 1 Intervention

Ascending dose level cohorts of AZD9829 in AML and MDS participants.

Who Is Running the Clinical Trial?

AstraZeneca

Lead Sponsor

Trials

4,491

Recruited

290,540,000+

Sir Pascal Soriot

AstraZeneca

Chief Executive Officer since 2012

Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris

Dr. Cristian Massacesi

AstraZeneca

Chief Medical Officer since 2021

MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology

Pascal Soriot

AstraZeneca

Chief Executive Officer since 2012

Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris

Cristian Massacesi

AstraZeneca

Chief Medical Officer since 2021

MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology

Citations

1. library.ehaweb.org library.ehaweb.org/eha/2024/eha2024-congress/document?c_id=420584&cm_id=435417&type=document435417

azd9829 demonstrates combination benefit with hma and ... Our preclinical findings also offer justification for exploring combinations of AZD9829 with SoC drugs in the clinic to improve long-term AML patient outcomes.

2. researchgate.net researchgate.net/publication/376141249_First_Disclosure_of_AZD9829_a_TOP1i-ADC_Targeting_CD123_Promising_Preclinical_Activity_in_AML_Models_with_Minimal_Effect_on_Healthy_Progenitors

First Disclosure of AZD9829, a TOP1i-ADC Targeting CD123 AZD9829 achieved ≥50% leukemic blast reduction in blood (7/7 models) (Figure 2) and in bone marrow (6/7 models) at day 14 after the first dose.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12407849/

A bispecific antibody–drug conjugate targeting CD7 and ... Aberrant expression of CD7 in AML patients is linked to shorter overall survival and lack of response to standard of care therapy. CD33/CD7 co- ...

4. astrazeneca.com astrazeneca.com/content/dam/az/PDF/2023/fy/Full-year-and-Q4-2023-results-clinical-trials-appendix.pdf

Full-year-and-Q4-2023-results-clinical-trials-appendix.pdf AZD9829 (CD123 TOP1i ADC) acute myeloid leukaemia, myelodysplastic syndromes. COVID mRNA VLP vaccine# (vaccine). COVID-19. Additional ...

5. pharmaceutical-technology.com pharmaceutical-technology.com/data-insights/azd-9829-astrazeneca-relapsed-acute-myeloid-leukemia-likelihood-of-approval/

AZD-9829 by AstraZeneca for Relapsed Acute Myeloid ... According to GlobalData, Phase II drugs for Relapsed Acute Myeloid Leukemia have a 20% phase transition success rate (PTSR) indication benchmark ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S0006497123125560

First Disclosure of AZD9829, a TOP1i-ADC Targeting ... Safety studies in cynomolgus monkey support the clinical development of AZD9829, a promising therapeutic candidate for the treatment of AML ...

7. connect.careboxhealth.com connect.careboxhealth.com/en-US/trial/listing/446021

Study of AZD9829 in CD123+ Hematological Malignancies This is a modular, multicentre, open-label, Phase I/II, dose-setting study. AZD9829 will be administered intravenously as monotherapy or in combination in ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT06179511

Study of AZD9829 in CD123+ Hematological Malignancies Characterizing the safety, tolerability, PK/PD, and preliminary antitumor activity of AZD9829 in CD123+ R/R AML participants, based on the data collected during ...

9. astrazeneca.com astrazeneca.com/our-therapy-areas/oncology/medicines-portfolio-and-pipeline.html

Medicines: Oncology Portfolio and Pipeline AZD9829 acute myeloid leukemia, myelodysplastic syndromes. AZD9829 - Phase I ... data or safety data. Please refer to your approved national product ...