Comparing two conditioning regimens for pediatric leukemia treatment

A Prospective, Open-label, Randomized Controlled, Multicenter Clinical Study of Haploidentical Hematopoietic Stem Cell Transplantation Using a TBI or TMLI Conditioning Regimen for Pediatric Acute Lymphoblastic Leukemia

This study is testing which of two treatment plans, Total Body Irradiation or Total Marrow Irradiation, works better for kids with leukemia getting a stem cell transplant.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 276 (estimated)
• Ages: 1 Year to 17 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Zhengzhou University (other)
• Locations: 1 site (Zhengzhou, Henan)
• Trial ID: NCT06559345 on ClinicalTrials.gov

What this trial studies

This study compares the effects of two conditioning regimens, Total Body Irradiation (TBI) and Total Marrow, Central Nervous System and Lymphoid Irradiation (TMLI), on pediatric patients with acute lymphoblastic leukemia (ALL) undergoing haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT). The goal is to determine which regimen more effectively eliminates leukemia cells while minimizing damage to normal tissues and reducing transplant-related mortality. Participants will be randomly assigned to receive either TBI or TMLI, and their outcomes will be monitored to assess the effectiveness and safety of each approach.

Who should consider this trial

Good fit: Ideal candidates are children aged 1-17 years diagnosed with acute lymphoblastic leukemia in remission who have a suitable haploidentical donor.

Not a fit: Patients who are not in remission or do not have a haploidentical donor may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved survival rates and quality of life for pediatric patients with acute lymphoblastic leukemia.

How similar studies have performed: While both conditioning regimens are supported by literature, there is currently no definitive evidence favoring one over the other, making this approach novel.

Eligibility criteria

Inclusion Criteria:

1. Informed Consent: Participants or guardians must voluntarily sign a written informed consent form.
2. Age and Gender: Participants should be male or female, aged 1-17 years, inclusive.
3. Diagnosis: Participants must be diagnosed with acute lymphoblastic leukemia (ALL) according to World Health Organization (WHO) criteria, and the diagnosis must apply to pediatrics aged 1-17 years.
4. Remission Status: The participant's leukemia must be in hematologic remission (complete remission, CR) prior to transplantation.
5. Donor Availability: There must be a suitable haploidentical donor available, and the participant must consent to undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).
6. Karnofsky Performance Status: The participant must have a Karnofsky score of 70 or higher, indicating that they are capable of caring for themselves and carrying out normal activities. Additionally, they must not have significant organ dysfunction, defined by the following:

   * Cardiac Function: New York Heart Association (NYHA) classification of class II or lower.
   * Liver Function: Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels should be no more than 2.5 times the upper limit of normal. Bilirubin levels should be no more than 2 times the upper limit of normal.
   * Renal Function: Serum creatinine levels should be no more than 1.5 times the upper limit of normal, or the creatinine clearance rate should be at least 60 ml/min.
   * Pulmonary Function: Participants should not experience significant dyspnea, should not require oxygen therapy, should not have interstitial lung disease, and should not have any active pulmonary infections.

Exclusion Criteria:

To be eligible for inclusion in the study, participants must not meet any of the following criteria:

1. The patient has not achieved hematologic remission before transplantation.
2. The patient has chosen a non-haploidentical related donor.
3. The patient has severe cardiac, hepatic, renal, or pulmonary diseases that make them unable to tolerate the conditioning regimen.
4. The patient has an active or refractory infection, or other life-threatening complications.
5. The patient has a history of other malignant tumors, psychiatric disorders, or HIV infection.
6. The patients or guardians refuses to sign the informed consent form, is unwilling to comply with clinical follow-up required by the study, or does not consent to the use of their data to support future research, project presentations, and clinical practices.
7. The investigator deems the patient unsuitable for participation in the study for any other reason.

Where this trial is running

Zhengzhou, Henan

• The First Affiliated Hospital of Zhengzhou University — Zhengzhou, Henan, China (RECRUITING)

Study contacts

• Principal investigator: Xiangbo Wan, PhD. — The First Affiliated Hospital of Zhengzhou University
• Study coordinator: Zhilei Bian, PhD.

Conditions: Acute Lymphoblastic Leukemia, Pediatric