SAR443579 for Leukemia

No longer recruiting at 34 trial locations

Overseen By Trial Transparency email recommended (Toll free for US & Canada)

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Sanofi

Must not be taking: Antiretrovirals, Corticosteroids

Disqualifiers: Autoimmune disease, Invasive malignancy, HIV, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests SAR443579, a new treatment for certain blood cancers, including various forms of leukemia. The goal is to determine the drug's safety, how the body processes it, and its effectiveness against these cancers. Participants must have been diagnosed with acute myeloid leukemia (AML) or other specific blood cancers that have not responded to previous treatments. The study involves testing different doses to find the most effective and safe level. 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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you cannot be on certain treatments like high-dose corticosteroids or other investigational drugs when starting the trial.

Is there any evidence suggesting that SAR443579 is likely to be safe for humans?

Research has shown that SAR443579 was safe in earlier studies. For patients with relapsed or hard-to-treat acute myeloid leukemia (AML), it provided clinical benefits and lasting improvements. Patients managed side effects at doses up to 6.0 mg per kilogram per infusion.

These results suggest that SAR443579 is generally well-tolerated, but monitoring for side effects remains important. This information comes from past research, and ongoing studies will continue to assess its safety and effectiveness. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard chemotherapy treatments for leukemia, SAR443579 is unique due to its specific targeting mechanism and administration method. This drug is delivered intravenously, allowing for precise dose escalation to determine the most effective and safe level for patients. Researchers are excited about SAR443579 because it has the potential to target leukemia cells more directly, possibly leading to fewer side effects and improved outcomes compared to traditional therapies. This innovative approach could offer new hope for patients who may not respond well to existing treatments.

What evidence suggests that SAR443579 might be an effective treatment for leukemia?

Research has shown that SAR443579, which participants in this trial will receive, may help treat blood cancers such as leukemia. In patients with relapsed or hard-to-treat acute myeloid leukemia (AML), SAR443579 demonstrated positive results and lasting effects. Early studies indicate that the treatment has manageable side effects, controlled up to a certain dose. The treatment aids the immune system in identifying and destroying cancer cells. Overall, initial findings suggest SAR443579 could effectively manage leukemia. ^{1 2 4 5 6}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

This trial is for children and adults with certain blood cancers like AML, B-ALL, HR-MDS, or BPDCN that have come back or didn't respond to treatment. Participants must be at least 12 years old, weigh over 40 kg, and not have any treatments left that could help them. They can't join if they've had specific prior therapies like CAR-T or anti-CD123 agents, active infections including HIV/AIDS or hepatitis B/C, severe heart issues, poor physical condition (ECOG >2), autoimmune diseases needing strong medication, other active cancers requiring treatment (except some skin cancers), are pregnant/breastfeeding or legally institutionalized.

Inclusion Criteria

My condition is HR-MDS with intermediate or higher risk.

My body weight is at least 10 kg.

I am between 1 and 17 years old.

Exclusion Criteria

I have a genetic condition that affects my bone marrow.

I will use growth factors to prevent blood cell issues during the early trial phase.

Pregnant and breast-feeding women

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

SAR443579 administered intravenously at escalating dose levels to determine the recommended dose for expansion

Up to 6 months

Dose Expansion

SAR443579 administered intravenously at the recommended dose and schedule determined from the dose escalation

Up to 12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 30 months

What Are the Treatments Tested in This Trial?

Interventions

• SAR443579

Trial Overview The study tests SAR443579 infusion's safety and effectiveness against various blood-related cancers in a Phase 1/Phase 2 trial. It involves gradually increasing the dose to find the safest amount that works best (dose escalation) and then giving it to more people at this dose level (dose expansion).

How Is the Trial Designed?

Group I: SAR443579 Experimental Treatment 1 Intervention

Dose Escalation: SAR443579 administered intravenously at escalating dose levels. Dose Expansion: SAR443579 administered intravenously at the recommended dose and schedule determined from the dose escalation.

SAR443579 is already approved in United States, European Union for the following indications:

Approved in United States as SAR443579 for:

• None approved; received FDA Fast Track Designation for the treatment of acute myeloid leukemia

Approved in European Union as SAR443579 for:

• None approved; currently in clinical trials

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Published Research Related to This Trial

In a study of 1023 children with standard-risk acute lymphoblastic leukemia (ALL), 13.6% were identified as having Philadelphia chromosome-like ALL (Ph-like ALL), which is associated with poorer event-free survival rates compared to non-Ph-like ALL (82.4% vs 90.7%).

The study found that 38.8% of Ph-like ALL cases had kinase-activating alterations, but these alterations were less frequent in standard-risk patients compared to high-risk patients, indicating a potential for targeted therapy with kinase inhibitors may be limited in this group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Genomic and outcome analyses of Ph-like ALL in NCI standard-risk patients: a report from the Children's Oncology Group. Roberts, KG., Reshmi, SC., Harvey, RC., et al.[2021]

A rare case of acute myeloid leukemia (AML) with a specific translocation (t (11;12) (p15;q13)) was identified, which did not respond well to standard treatments like all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), similar to previous cases.

The findings suggest that standard AML treatment regimens may be more effective for this type of leukemia, and hematopoietic stem cell transplantation could be a beneficial option, highlighting the need for further research to explore effective therapies for this rare condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Rare Morphology Resembling APL with t (11;12) (p15;q13) in Acute Myeloid Leukemia: Case Report and Literature Review. Hua, J., Bao, X., Xie, Y.[2020]

A patient with acute myeloid leukemia (AML) developed a BCR::ABL alteration after a stem cell transplant, highlighting the genomic complexity of this rare subtype of AML, which is challenging to differentiate from myeloid blast crisis CML.

Functional drug screening suggested that combining ABL1 inhibitors with RAS or PI3K pathway inhibitors could enhance treatment responses, emphasizing the potential for personalized combination therapies based on genomic profiling in managing this disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comprehensive molecular characterization of a rare case of Philadelphia chromosome-positive acute myeloid leukemia. Rosenberg, MW., Savage, SL., Eide, CA., et al.[2023]

In a study of 994 newly diagnosed acute myeloid leukemia (AML) patients, those with mutations in splicing factor genes (SFmut) had shorter relapse-free survival (RFS) and overall survival (OS) when treated with intensive therapy, but these differences were not significant when patients received venetoclax alongside this therapy.

The presence of SF mutations did not independently worsen outcomes in patients treated with lower-intensity therapy (LI), suggesting that the combination of venetoclax with either treatment type may improve prognostic models for AML.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Venetoclax abrogates the prognostic impact of splicing factor gene mutations in newly diagnosed acute myeloid leukemia. Senapati, J., Urrutia, S., Loghavi, S., et al.[2023]

In a study of 4 patients with variant Ph chromosome-positive leukemia, 3 had chronic myeloid leukemia (CML) and 1 had adult B acute lymphoblastic leukemia (B-ALL), with varying cytogenetic abnormalities and responses to treatment.

While all patients responded to treatment with imatinib, the prognosis for the patient with the e13a3 type BCR/ABL fusion gene was poor, leading to relapse and death, whereas the CML patients remained in molecular remission for up to 27 months.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical Characteristics and Prognosis of patients with Variant Philadelphia Chromosome Positive Leukemia]. Zhang, XY., Wu, SX., Guo, XZ., et al.[2018]

Citations

1. innate-pharma.com innate-pharma.com/media/all-press-releases/innate-pharma-shares-updated-results-sanofi-developed-blood-cancer-phase-1/2-sar443579/iph6101-trial

Innate Pharma shares updated results from the Sanofi ... SAR443579/IPH6101 continues to show clinical benefit and durable responses along with a favorable safety profile in patients with R/R AML.

2. ascopubs.org ascopubs.org/doi/10.1200/JCO.2023.41.16_suppl.7005

A first-in-human study of CD123 NK cell engager ... We herein report preliminary safety and efficacy data of SAR'579 from a phase 1/2 trial in patients with relapsed or refractory acute ...

3. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/2883.3/529436/Phase-1-2-Open-Label-Multi-Center-Study-Assessing

Phase 1/2, Open-Label, Multi-Center Study Assessing the ... Early clinical results in relapsed/refractory AML pts demonstrated that SAR'579 had a manageable toxicity profile up to 6.0 mg/kg/infusion, and complete ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06508489

NCT06508489 | A Study to Investigate Natural Killer Cell ... A study to investigate natural killer cell engager (SAR443579) in combination with azacitidine + venetoclax in adult participants with newly diagnosed acute ...

5. firstwordpharma.com firstwordpharma.com/story/5867347

Innate Pharma Shares Updated Results From the Sanofi ... The study, led by Sanofi, tests SAR'579 as a monotherapy for the treatment of blood cancers with high unmet needs, including relapsed or ...

6. innate-pharma.com innate-pharma.com/media/all-press-releases/innate-pharma-shares-efficacy-and-safety-phase-1/2-results-nk-cell-engager-sar443579/iph6101-developed-sanofi-ash-2023

Innate Pharma shares efficacy and safety Phase 1 /2 results of ... SAR443579 has FDA Fast Track Designation for the treatment of acute myeloid leukemia. As of July 5, 2023, 43 patients (42 R/R AML and 1 HR-MDS) ...