GFH009 for Blood Cancer

Recruiting at 4 trial locations

Overseen By James Dean

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Genfleet Therapeutics (Shanghai) Inc.

Must be taking: Venetoclax

Must not be taking: CYP3A4 inhibitors, QT prolongers

Disqualifiers: Bulky disease, CNS metastases, cardiovascular disease, others

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

Compensation: Varies

Number of Visits: Unknown

160 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called GFH009 (also known as SLS009), which targets a protein involved in cancer growth, to assess its safety and effectiveness for people with specific blood cancers, such as AML (acute myeloid leukemia), lymphoma, and CLL/SLL (chronic lymphocytic leukemia/small lymphocytic lymphoma). The study divides participants into groups to test different doses of GFH009, either alone or with other drugs, for those whose cancer has returned or not responded to previous treatments. Suitable candidates have a confirmed diagnosis of relapsed or refractory blood cancer and have tried at least two other treatments without success. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

The trial requires you to stop taking medications that are strong CYP3A4 inhibitors or inducers at least 7 days before starting the study. Also, medications that prolong the QT interval should be stopped unless you're an AML patient taking azole antifungal medications.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that SLS009 (previously known as GFH009) is generally safe for individuals with certain blood cancers. In patients with lymphomas that have returned or are not responding to treatment, SLS009 proved effective and did not cause severe side effects. A similar study in patients with acute myeloid leukemia (AML) that had returned or was not responding found the treatment safe at different doses.

When combined with venetoclax and azacitidine, studies found SLS009 to be safe and effective for AML patients previously treated with venetoclax. This combination did not cause severe side effects and still helped combat the cancer. Overall, these findings suggest that SLS009, whether used alone or with other drugs, is well-tolerated in individuals with these specific blood cancers. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for blood cancers like r/r AML, which typically involve chemotherapy and targeted therapies such as venetoclax, GFH009 offers a unique approach. This investigational drug is more precisely targeted, working on specific pathways involved in cancer cell survival. Researchers are particularly excited about GFH009 because it uses a novel mechanism of action that could potentially overcome resistance seen with current treatments. Additionally, GFH009 is being tested in combination with existing drugs like venetoclax and azacitidine, which may enhance its effectiveness and offer new hope for patients who don't respond to standard therapies.

What evidence suggests that this trial's treatments could be effective for blood cancer?

Research shows that SLS009, also known as GFH009, may help treat certain blood cancers. In this trial, participants with relapsed or hard-to-treat acute myeloid leukemia (AML) will receive SLS009 combined with the drugs venetoclax and azacitidine. Previous studies have shown a response rate of 44% with this combination, more than twice the expected rate. For those with relapsed or hard-to-treat lymphoma, participants will receive SLS009 alone. Past research demonstrated an overall response rate of 14.7%, with some patients experiencing tumor shrinkage of up to 62%. These results suggest that SLS009 could be a promising option for patients facing these difficult conditions. ^{1 2 3 6 7}

Who Is on the Research Team?

Dragan Cicic, MD

Principal Investigator

SELLAS Life Sciences Group, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with certain blood cancers like AML, CLL/SLL, and lymphoma that have come back or didn't respond to treatment. They must have normal levels of bilirubin (unless they have Gilbert's syndrome), liver enzymes, amylase, lipase, and stable electrolytes. Women who can get pregnant and men with partners who can get pregnant must use two effective birth control methods during the study.

Inclusion Criteria

My liver enzymes are within the required limits.

Total bilirubin ≤ 1.5 × upper limit of normal (ULN) except for patients with Gilbert's syndrome, who are included if total bilirubin is < 3 × ULN or if direct bilirubin is < 1.5 × ULN

Amylase and lipase ≤1.5 × ULN

Exclusion Criteria

I am HIV positive.

Average QTcF ≥ 450 msec (males) or ≥ 470 msec (females) on screening ECG

Active hepatitis B or hepatitis C virus infection

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

Dose levels are escalated following the Bayesian optimal interval (BOIN) design in patients with relapsed/refractory AML and CLL/SLL or lymphoma

21 to 28 days

Dose Expansion

Assessment of safety, tolerability, and antitumor activity of SLS009 in combination with venetoclax and azacitidine in patients with relapsed/refractory AML across different cohorts

approximately 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 years

What Are the Treatments Tested in This Trial?

Interventions

• GFH009

Trial Overview The trial tests GFH009 alone in patients with relapsed/refractory acute myeloid leukemia (AML) or lymphomas. It also tests GFH009 combined with venetoclax and azacitidine in AML patients resistant to venetoclax-based treatments. The goal is to see how safe it is and if it helps against these cancers.

How Is the Trial Designed?

Group I: Group 3 Cohort 5. 30 mg BIW in pts with r/rAML with other than ASXL1 mutations Experimental Treatment 3 Interventions

SLS009 (30 mg BIW) in combination with venetoclax and azacitidine in patients with r/r AML who have relapsed or are refractory to venetoclax-based regimens and with documented Defining somatic mutations, Cytogenetic abnormalities defining acute myeloid leukemia, myelodysplasia related, other than ASXL1 mutation per WHO 5th Edition classification.

Group II: Group 3 Cohort 4. 30 mg BIW in patients with r/r AML with ASXL1 mutation. Experimental Treatment 3 Interventions

SLS009 (30 mg BIW) in combination with venetoclax and azacitidine in patients with r/r AML who have relapsed or are refractory to venetoclax-based regimens and with documented ASXL1 mutation.

Group III: Group 3 Cohort 3. 30 mg BIW in patients with r/r AML. Experimental Treatment 3 Interventions

SLS009 (30 mg BIW) in combination with venetoclax and azacitidine in patients with r/r AML who have relapsed on or are refractory to venetoclax-based regimens. US study sites only. (Cohort completed).

Group IV: Group 3 Cohort 2. 60 mg QW in patients with r/r AML. Experimental Treatment 3 Interventions

SLS009 (60 mg QW) in combination with venetoclax and azacitidine in patients with r/r AML who have relapsed on or are refractory to venetoclax-based regimens. US study sites only. (Cohort completed).

Group V: Group 3 Cohort 1. 45 mg QW in patients with r/r AML Experimental Treatment 3 Interventions

SLS009 (45 mg QW) in combination with venetoclax and azacitidine in patients with r/r AML who have relapsed on or are refractory to venetoclax-based regimens. US study sites only. (Cohort completed)

Group VI: Group 2. Dose escalation in patients with r/r CLL/SLL or lymphoma Experimental Treatment 1 Intervention

In the dose escalation part, the dose levels will be escalated following the Bayesian optimal interval (BOIN) design. China and US study sites. (Completed).

Group VII: Group 1. Dose escalation in patients with r/r AML Experimental Treatment 1 Intervention

In the dose escalation part, the dose levels will be escalated following the Bayesian optimal interval (BOIN) design. China study sites only. (Completed).

Who Is Running the Clinical Trial?

Genfleet Therapeutics (Shanghai) Inc.

Lead Sponsor

Trials

8

Recruited

890+

Zhejiang Genfleet Therapeutics Co., Ltd.

Lead Sponsor

Trials

11

Recruited

900+

Sellas Life Sciences Group

Lead Sponsor

Trials

9

Recruited

770+

Published Research Related to This Trial

In a study of 56 AML patients, including children, a high-dose cytarabine consolidation followed by G-CSF led to a disease-free survival (DFS) rate of 61% and an overall survival rate of 62% at 30 months, indicating effective treatment outcomes.

The treatment resulted in faster recovery of neutrophils and platelets compared to traditional bone marrow rescue, with a median time of 13 days for neutrophil recovery and 32 days for platelet recovery, although there was an 11% rate of treatment-related deaths.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autologous peripheral blood progenitor cell transplantation mobilized with high-dose cytarabine in acute myeloid leukemia in first complete remission. Pavlovsky, S., Fernández, I., Milone, G., et al.[2019]

In a study of 18,245 older patients with non-Hodgkin lymphoma, the use of granulocyte colony-stimulating factors (G-CSFs) was associated with a modestly increased risk of developing myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML), particularly with the use of filgrastim.

Specifically, patients receiving 10 or more doses of filgrastim had a significantly higher risk of MDS/AML (HR 1.67), while pegfilgrastim did not show a similar risk increase, indicating that the type of G-CSF used may influence safety outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Myelodysplastic syndrome and acute myeloid leukemia after receipt of granulocyte colony-stimulating factors in older patients with non-Hodgkin lymphoma. Calip, GS., Moran, KM., Sweiss, KI., et al.[2023]

In a pilot study involving 56 patients with higher risk myelodysplastic syndrome (MDS), the G-HA regimen combining G-CSF with low-dose chemotherapy resulted in a complete remission rate of 61%.

The treatment was well tolerated, with manageable nonhematologic toxicities and a low mortality rate of 4% within the first 4 weeks, indicating its potential as a safe option for patients unable to undergo hematopoietic cell transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effect of granulocyte colony-stimulating factor priming combined with low-dose cytarabine and homoharringtonine in higher risk myelodysplastic syndrome patients. Wang, FX., Zhang, WG., He, AL., et al.[2018]

Growth factors (GFs) have become essential in treating hematological cancers, particularly for managing bone marrow failure and cytopenias caused by the disease or its treatments, despite initial concerns about their potential to induce leukemia.

The American Society of Clinical Oncologists (ASCO) has provided guidelines to standardize the use of GFs, which are now applied in various clinical settings, including chemotherapy support, stem cell transplantation, and emerging therapies for hematological malignancies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Growth factors in haematological cancers. Osuji, N., Pettengell, R.[2005]

In a study involving 282 breast cancer patients undergoing dose-dense chemotherapy, primary prophylaxis with lipegfilgrastim significantly reduced the incidence of severe neutropenia (SN) to 33.3% and febrile neutropenia (FN) to just 1.1%.

The treatment was well-tolerated, with 99.3% of patients benefiting from lipegfilgrastim, and no fatal serious adverse events reported, indicating its safety and efficacy in a real-world clinical setting.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prophylaxis of Neutropenia with Lipegfilgrastim in Breast Cancer Patients with Dose-Dense Chemotherapy: Results of a Noninterventional Study on Therapeutic Routine in Germany (NADENS). Kiechle, M., Schem, C., Lüftner, D., et al.[2023]

Citations

1. ir.sellaslifesciences.com ir.sellaslifesciences.com/news/News-Details/2025/SELLAS-Announces-Positive-Overall-Survival-in-Cohort-3-from-the-Ongoing-Phase-2-Trial-of-SLS009-in-rr-AML/default.aspx

SELLAS Announces Positive Overall Survival in Cohort 3 ... The data reveal that relapsed or refractory to venetoclax-based regimens patients receiving 30 mg BIW achieved a mOS of 8.8 months, far surpassing the ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04588922

NCT04588922 | Study of SLS009 (Formerly GFH009) a ... This study is investigating the safety, tolerability, and antitumor activity of SLS009 in patients with hematologic malignancies in three groups (two dose ...

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/2877/531059/Phase-2a-Study-of-SLS009-a-Highly-Selective-CDK9

Phase 2a Study of SLS009, a Highly Selective CDK9 Inhibitor ... This Phase 2a trial was a dose ranging, open label, single arm, study of AZA/VEN + SLS009 in R/R AML after prior Ven treatment conducted across 5 US ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124056295

Phase 2a Study of SLS009, a Highly Selective CDK9 ... This Phase 2a trial was a dose ranging, open label, single arm, study of AZA/VEN + SLS009 in R/R AML after prior Ven treatment conducted across 5 US ...

5. onclive.com onclive.com/view/cdk9-inhibitor-sls009-displays-activity-in-relapsed-refractory-aml

CDK9 Inhibitor SLS009 Displays Activity in Relapsed ... Enrollment of patients in a phase 2 study evaluating SLS009 in relapsed/refractory AML has been completed and the agent demonstrated initial ...

6. ir.sellaslifesciences.com ir.sellaslifesciences.com/news/News-Details/2025/SELLAS-Meets-All-Primary-Endpoints-in-Phase-2-Trial-of-SLS009-in-rr-AML-and-Receives-FDA-Guidance-to-Advance-into-First-Line-Therapy-Study/default.aspx

SELLAS Meets All Primary Endpoints in Phase 2 Trial of ... The trial met all endpoints, demonstrating strong efficacy and favorable safety and tolerability with robust anti-tumor activity.

7. onclive.com onclive.com/view/sls009-plus-venetoclax-azacitidine-displays-activity-tolerability-in-relapsed-refractory-aml

SLS009 Plus Venetoclax/Azacitidine Displays Activity, ... The addition of SLS009 to the combination of venetoclax and azacitidine produced anti-leukemic effects and was safe in patients with relapsed/ ...