Inotuzumab ITCC-059 (ALL/B-cell leukemia/lymphoma)

Recruiting

Who can enter

• Children with 'very high risk' CD22-positive B-cell precursor acute lymphoblastic leukemia (BCP-ALL) in whom the disease has returned (relapsed) very quickly

• Age: 1-18 years

Goal

The safe dose of InO has been determined earlier in this study. The safety and efficacy of InO at this dose has been studied further in a larger group of children with relapsed/refractory ALL. In this part of the study the efficacy of InO will be examined in children with ‘very high risk’ BCP-ALL.

Background

Some children with B-cell precursor ALL or another B-cell malignancy have recurrent disease or do not respond to treatment. Only a minority of them can still be cured with intensive chemotherapy and a stem cell transplantation. This study with InO was designed to improve that.

The drug InO targets leukemia and lymphoma cells. It binds to a protein on the surface of the leukemia or lymphoma cell (called CD22), and then delivers a chemotherapeutic drug (calicheamicin) in the cell. CD22 only occurs on leukemia and lymphoma cells, and on some mature blood cells.

In Europe InO has already been approved (‘registered’) for the treatment of adults with relapsed/refractory ALL. In North America and Japan, it has also been registered as a drug for the treatment of children with relapsed/refractory BCP-ALL, based on the results of the first phase of this study.

In the first phase of this study the safe dose of InO was determined, and approximately 40 children were treated with the same InO dose. More than 80% of these children achieved complete remission. In 94% of the children who achieved remission no minimal residual disease was present. The latter is important because the results of a stem cell transplantation, which is usually required after achieving complete remission, are much better when there is no or very little residual disease detectable.

In the second phase of this study approximately 30 children were treated with a combination of InO and other chemotherapy. This did not result in a better response.

In the current part of the study the efficacy of InO will be examined in a specific group of patients.

This study is being performed by a collaboration of universities and pediatric hospitals throughout Europe. This European group is called ITCC.

In order to participate in a study please refer to your/your child’s doctor.

Last reviewed

March 23, 2026

The above information is intended as a brief summary only and may not reflect the most up-to-date information. For full details and the current status of a protocol, physicians can contact the Princess Máxima Center directly.

Summary of results of phase 1

Acute lymphoblastic leukemia (ALL) is the most predominant type of cancer in children. With the current standard treatment, consisting of intensive chemotherapy, the cure rate is more than 85-90%. However, approximately 10-15% of children with ALL relapse or do not respond to standard treatment. These children unfortunately have a poor prognosis. At the Princess Máxima Center researchers are therefore looking for new treatments.

The protein CD22 is present on the surface of leukemic cells (and healthy immune cells, so-called lymphocytes) in a vast majority of children with ALL. Inotuzumab ozogamicin (InO) is a novel drug that specifically binds to CD22 and then releases a cytotoxic agent (calicheamicin) that kills the leukemic cell. Although InO has been approved for the treatment of adults with ALL who do not or no longer respond to standard treatment, there is little experience in children.

Safe dose
In order to determine the safety and efficacy of InO in children, Dutch researchers, among whom dr. Erica Brivio and prof. dr. Michel Zwaan from the Princess Máxima Center, initiated and coordinated an international study. In phase 1 of this study the researchers determined which dose is tolerable and can be safely given to children. InO was well tolerated in children at the same dose as in adults (converted to body weight and height). At this dose InO also demonstrated anti-leukemic activity: after one course of treatment 85% of the patients responded, and no residual leukemic cells could be detected anymore (no minimal residual disease). Nine of these children went on to receive a bone marrow transplant or CAR T-cell therapy, improving their chance of a cure. These latter treatments could otherwise not have been given.

These are promising but preliminary results in a small number of patients (25 in total) with a relatively short follow-up time (eight months on average). The safe dose determined in phase 1 will be used in phase 2 of the study to further evaluate the anti-leukemic activity of InO in a larger number of children.

Would you like to read the scientific publication? Please look here:

Brivio E, et al. Blood. 2021 Mar 25;137(12):1582-1590. A Phase I study of inotuzumab ozogamicin in pediatric relapsed/refractory acute lymphoblastic leukemia (ITCC-059 study)