Study of SAR443579 Infusion for Adults and Children with Relapsed or Refractory Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia, HR-MDS, or BPDCN

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What is this study about?

This clinical trial is focused on studying the effects of a new treatment called in individuals with certain types of blood cancers. These include , , , and . The treatment, , is administered through an intravenous infusion, which means it is given directly into the bloodstream through a vein.

The purpose of the study is to find the most suitable dose of that can be safely given to patients and to evaluate its effectiveness in treating these blood cancers. The study is divided into two parts. In the first part, the focus is on determining the best dose for both adults and children. In the second part, the study aims to assess how well the treatment works at the recommended dose. Participants will receive the treatment and be monitored for any side effects and improvements in their condition.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how the treatment affects their cancer. The study will help researchers understand more about the potential benefits and risks of for treating these serious conditions. The information gathered from this trial could contribute to developing new treatment options for patients with these types of blood cancers.

1 joining the study

Upon joining the study, the participant will be informed about the trial’s purpose and procedures. The participant must provide informed consent to participate in the study.

2 initial assessment

An initial assessment will be conducted to confirm eligibility. This includes verifying the diagnosis of conditions such as relapsed or refractory acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL), high-risk myelodysplasia (HR-MDS), or blastic plasmacytoid dendritic cell neoplasm (BPDCN).

3 dose escalation phase

During the dose escalation phase, the participant will receive the medication SAR443579 through an intravenous infusion. The goal is to determine the maximum tolerated dose or maximum administered dose. The frequency and duration of administration will be determined by the study protocol.

4 monitoring for side effects

Participants will be closely monitored for any side effects or dose-limiting toxicities. This monitoring is crucial to ensure the safety and well-being of the participant.

5 dose expansion phase

In the dose expansion phase, the participant will receive the recommended dose for expansion. This phase aims to assess the anti-leukemic activity of SAR443579.

6 regular follow-up visits

Regular follow-up visits will be scheduled to monitor the participant’s response to the treatment and to check for any adverse effects. These visits are essential for tracking the progress and effectiveness of the treatment.

7 completion of the study

Upon completion of the study, a final assessment will be conducted to evaluate the overall outcomes and any long-term effects of the treatment.

Who Can Join the Study?

Participant must be at least 1 year old. Adults must be at least 12 years old, and children must be between 1 to 17 years old.
For the Escalation Part:
- Confirmed diagnosis of primary or secondary acute myeloid leukemia (AML), which is a type of blood cancer, except for specific subtypes like acute promyelocytic leukemia (APL) and juvenile myelomonocytic leukemia (JMML).
- Participants with AML must meet one of the following:
  - Primary Induction Failure (PIF) AML, meaning the disease did not respond to initial treatment attempts.
  - Early relapse (ER) AML, meaning the disease returned within 6 months after initial treatment.
  - Leukemia in first or higher relapse, meaning the disease has returned after being in remission.
  - For children aged 1 to 17, PIF is defined as the disease not responding after two cycles of treatment.
For adults only: Confirmed diagnosis of high-risk myelodysplastic syndrome (HR-MDS), a condition where the bone marrow does not produce enough healthy blood cells, with specific risk categories and no available effective therapy.
Confirmed diagnosis of B-cell acute lymphoblastic leukemia (B-ALL), a type of blood cancer, without disease outside the bone marrow and no available effective therapy.
For the Expansion Part (Adults only):
- Cohort A: Participants with AML that did not respond to initial treatment or relapsed within 6 months after remission.
- Cohort B: Participants with AML that relapsed more than 6 months after remission.
Body weight must be at least 10 kg.
For children and the escalation part only: Confirmed diagnosis of blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare type of blood cancer, with relapsed or refractory disease and no available effective therapy.
For children and the expansion part only: Cohort C: Participants with AML that have relapsed or have disease resistant or intolerant to available therapies.

Who Cannot Join the Study?

Patients with other types of cancer that are not part of the study.
Patients who have had a different treatment for their cancer recently.
Patients with serious infections that are not controlled.
Patients with significant heart problems.
Patients with severe liver or kidney issues.
Patients who are pregnant or breastfeeding.
Patients who are unable to follow the study procedures.
Patients with a history of allergic reactions to similar treatments.
Patients who have participated in another clinical trial recently.
Patients with certain blood disorders not related to the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Institut Gustave Roussy	Villejuif	France

Other Sites

Site Name	City	Country
Stichting Radboud University Medical Center	Nijmegen	The Netherlands
Prinses Maxima Centrum voor Kinderoncologie B.V.	Utrecht	The Netherlands
Hopital Beaujon	Clichy	France
Utivezjkzswq Mbljrvm Cfeoems Gsxxjlrsc	Groningen	The Netherlands
Apunjfqxg Udz	Amsterdam	The Netherlands
Adkckgxhwp Pykqpokm Htmpvyhl Dg Pnhjg	Paris	France
Ezrfkts Uouoimdnyagx Mtgvjyl Cswwfbt Rbodgaacp (gfkbhwr Mvy	Rotterdam	The Netherlands
Ildqsvli Pgrjygmpthcynto Cenjtx Ckknnp	Marseille	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	08.12.2021
The Netherlands	Not recruiting	08.12.2021

Trial locations

Investigated drugs:

SAR443579 is an investigational medication being studied for its potential to treat certain types of blood cancers. It is administered as a single agent through an intravenous infusion. The trial aims to find the most effective and safe dose for both adults and children with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), high-risk myelodysplastic syndrome (HR-MDS), or blastic plasmacytoid dendritic cell neoplasm (BPDCN). The study is divided into two parts: the first part focuses on determining the maximum tolerated dose, while the second part evaluates the medication’s effectiveness against leukemia at the recommended dose.

Investigated diseases:

Acute Lymphoblastic Leukemia – A type of cancer that affects the blood and bone marrow, characterized by the overproduction of immature white blood cells called lymphoblasts. It progresses rapidly, leading to a decrease in the number of healthy blood cells, which can cause symptoms like fatigue, frequent infections, and easy bruising or bleeding.

Acute Myeloid Leukemia – A cancer of the blood and bone marrow that results in the rapid growth of abnormal white blood cells. These cells accumulate in the bone marrow and interfere with the production of normal blood cells, leading to symptoms such as fatigue, fever, and an increased risk of infections.

Myelodysplastic Syndrome – A group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when the bone marrow does not produce enough healthy blood cells, leading to symptoms like fatigue, shortness of breath, and a higher risk of bleeding and infections.

Blastic Plasmacytoid Dendritic Cell Neoplasm – A rare and aggressive cancer that originates from a type of immune cell known as plasmacytoid dendritic cells. It often presents with skin lesions and can involve the bone marrow and blood, leading to symptoms such as skin rashes, fever, and swollen lymph nodes.

Trial ID:

2023-508357-58-00

Protocol code:

TCD17197

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study of SAR443579 Infusion for Adults and Children with Relapsed or Refractory Acute Myeloid Leukemia, B-Cell Acute Lymphoblastic Leukemia, HR-MDS, or BPDCN

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?