Expanded Access Program for Venetoclax and Navitoclax in Children with Relapsed or Hard-to-Treat Acute Lymphocytic Leukemia or Lymphoblastic Lymphoma

Sponsor: Kathleen Ludwig

Expanded Access
Sponsor score: 40
Inactive

ClinicalTrials ID:NCT05215405

Provider View

This program provides access to Venetoclax and Navitoclax for children with acute lymphocytic leukemia (ALL) or lymphoblastic lymphoma (LL) who have tried all other standard treatments without success.

Patient Parameters

Program Overview

The goal of this program is to offer Venetoclax and Navitoclax to children with acute lymphocytic leukemia (ALL) or lymphoblastic lymphoma (LL) who have not responded to standard treatments. ALL is a cancer affecting immature white blood cells, while LL is a rare and aggressive form of non-Hodgkin lymphoma. Both conditions involve abnormal cells that replace healthy ones in the body. Although treatments for these cancers have improved, children who relapse often face poor outcomes. Venetoclax and Navitoclax, when combined with chemotherapy, have shown promise in treating these conditions, even in patients who have undergone previous treatments like stem cell transplants or CAR-T therapy.

Description

Acute lymphocytic leukemia (ALL) is a type of cancer that affects immature white blood cells, which are crucial for the immune system. In ALL, these cells do not mature properly and replace healthy cells in the bone marrow and other parts of the body. Lymphoblastic lymphoma (LL) is a rare and aggressive form of non-Hodgkin lymphoma, where abnormal cells are found in the lymph nodes or thymus gland. Both ALL and LL are treated similarly, but children who relapse after initial treatment often have poor outcomes.

Recent advances in treatment have shown that targeting specific pathways in cancer cells, such as with the drugs Venetoclax and Navitoclax, can be effective. These drugs, when used with chemotherapy, have been well-tolerated and have shown promising results in patients who have already undergone extensive treatment, including stem cell transplants or CAR-T therapy. Many patients achieved complete responses, with some showing no detectable cancer cells after treatment. Ongoing studies are looking into additional markers that might predict treatment success.

Eligibility Criteria

Inclusion Criteria

  • The child, parent, or guardian must agree to participate and sign a consent form.
  • The child must have relapsed or hard-to-treat acute lymphocytic leukemia (ALL) or lymphoblastic lymphoma (LL) and have tried all other available treatments.
  • The child must be 4 years or older.
  • The child must weigh at least 20 kg.
  • The child must have good liver function, with specific blood test results within acceptable limits.
  • The child must have normal kidney function or a creatinine clearance of at least 60 mL/min/1.73m2.
  • The child must be in good overall health, with a performance status score of at least 50.
  • Female participants who can become pregnant and their male partners must use birth control during treatment and for at least 30 days after the last dose.
  • Male participants must agree to use condoms during treatment.
  • Female participants must have a negative pregnancy test before starting treatment.

Exclusion Criteria

  • Children with cancer that has spread to the brain and requires radiation.
  • Children who are less than 100 days post-transplant, or more than 100 days post-transplant with active Graft-versus-host disease (GVHD), or are still on immunosuppressant therapy within 7 days before starting the study drugs.
  • Children who have taken certain medications, like strong CYP3A inhibitors or aspirin, within 7 days before starting the study drugs.
  • Children with active, uncontrolled infections.
  • Children who have not recovered from significant side effects of previous treatments.
  • Children with conditions that prevent them from taking oral medications.
  • Female participants who are pregnant or breastfeeding, and male participants planning to father a child or donate sperm during treatment or shortly after.

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