Subcutaneous blinatumomab for children with relapsed/refractory or MRD-positive B‑cell precursor acute lymphoblastic leukemia

A Phase 1b/2 Study to Investigate the Safety, Efficacy and Pharmacokinetics of Administration of Subcutaneous (SC) Blinatumomab in Pediatric Participants With Relapsed/Refractory (R/R) and Minimal Residual Disease Positive (MRD+) B-Cell Precursor Acute Lymphoblastic Leukemia (B-ALL)

This trial tests subcutaneous blinatumomab in children under 12 who have relapsed/refractory or minimal residual disease–positive B‑cell precursor ALL.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 104 (estimated)
• Ages: 28 Days to 4383 Days
• Sex: All
• Sponsor: Amgen (Industry-sponsored)
• Drugs / interventions: blinatumomab, CAR-T
• Locations: 4 sites (Philadelphia, Pennsylvania and 3 other locations)
• Trial ID: NCT07134088 on ClinicalTrials.gov

What this trial studies

This Phase 1/2 study gives subcutaneous blinatumomab to children aged 28 days to under 12 to measure safety and anti-leukemia activity. Phase 1 will focus on dose finding and tolerability, while Phase 2 will expand into cohorts of relapsed/refractory (≥5% blasts) and MRD+ (≥0.1% to <5% blasts) patients to measure response. Prior CD19-directed therapy is allowed if CD19 expression persists and treatment ended more than 4 weeks before enrollment, and patients with active CNS leukemia are excluded. The trial is sponsored by Amgen and runs at major pediatric cancer centers in the United States.

Who should consider this trial

Good fit: Ideal candidates are children 28 days to under 12 years old with relapsed/refractory or MRD‑positive B‑cell precursor ALL, Lansky performance status ≥50%, and retained CD19 expression when applicable.

Not a fit: Patients with active CNS leukemia, loss of CD19 expression, recent CD19‑directed therapy with CNS complications, or other exclusionary conditions are unlikely to benefit from this protocol.

Why it matters

Potential benefit: If successful, subcutaneous blinatumomab could provide an effective, less invasive treatment option that helps clear residual disease or control relapse in young children with B‑cell precursor ALL.

How similar studies have performed: Intravenous blinatumomab has shown efficacy in MRD+ and relapsed/refractory B‑ALL, but subcutaneous administration in young children is a newer approach with limited prior data.

Eligibility criteria

Inclusion Criteria:

* Age ≥28 days to \<12 years at the time of informed consent/assent.
* Lansky Performance Status (LPS) of ≥ 50%.
* For Phase 1b and Phase 2 cohort in participants with R/R B-ALL:

  * Participants with B-ALL relapsed after or refractory to any line of treatment including allogeneic hematopoietic stem cell transplant (HSCT).
  * Greater than or equal to 5% blasts in the bone marrow (BM) is considered as relapse in the BM.
* For Phase 2 cohort in participants with MRD+ B-ALL:

  * Participants with MRD+ B-ALL must have between ≥ 0.1% and \< 5% blasts in the BM.
* Prior CD19-directed therapy will be allowed (with demonstrated continued CD19+ expression) if treatment ended \>4 weeks prior to start of protocol therapy and no prior central nervous system (CNS) complications.
* Any Philadelphia chromosome-positive (Ph+) participant intolerant or refractory to prior tyrosine kinase inhibitors (TKIs) are eligible.

Exclusion Criteria:

* Active ALL in the CNS.
* History or presence of clinically relevant CNS pathology or event such as epilepsy, childhood seizure, paresis, aphasia, stroke, severe brain injuries, cerebellar disease, organic brain syndrome, psychosis, or severe (≥ grade 3) CNS events including immune effector cell-associated neurologic syndrome (ICANS) from prior CAR-T or other T-cell engager therapies.
* Isolated EM disease.
* Current autoimmune disease or history of autoimmune disease with potential CNS involvement.
* Patients with Down Syndrome are not eligible for this study.
* Active acute or chronic graft versus host disease requiring systemic treatment with immunosuppressive medication.
* Known infection with human immunodeficiency virus (HIV) or chronic infection with hepatitis B virus or hepatitis C virus.
* Presence of an acute or uncontrolled chronic infection, or any other concurrent disease or medical condition that could be worsened by the treatment or interfere with the participant's ability to comply with the study protocol.
* Allogeneic HSCT within 12 weeks before the start of blinatumomab.

Where this trial is running

Philadelphia, Pennsylvania and 3 other locations

• Childrens Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
• St Jude Childrens Research Hospital — Memphis, Tennessee, United States (Recruiting)
• Seattle Childrens Hospital — Seattle, Washington, United States (Recruiting)
• Kanagawa Childrens Medical Center — Yokohami-shi, Kanagawa, Japan (Recruiting)

Study contacts

• Study coordinator: Amgen Call Center

Conditions Relapsed/Refractory B-Cell Precursor Acute Lymphoblastic Leukemia; Minimal Residual Disease + B-Cell Acute Lymphoblastic Leukemia; Blinatumomab; AMG 103; Minimal Residual Disease Positive B-Cell Precursor Acute Lymphoblastic Leukemia; R/R B-ALL; MRD+ B-ALL