High-dose gemcitabine, busulfan, and thiotepa with autologous stem cell transplant for primary central nervous system lymphoma.

A Study of High Dose Gemcitabine, Busulfan and Thiotepa With Autologous Stem Cell Transplantation for Primary or Relapse Central Nervous System Lymphomas

This trial tests if giving high-dose gemcitabine, busulfan, and thiotepa before an autologous stem cell transplant is safe and helps control primary CNS lymphoma in adults who are in remission or have a partial response.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 34 (estimated)
• Ages: 16 Years to 70 Years
• Sex: All
• Sponsor: Sichuan University (other)
• Locations: 1 site (Chengdu, Sichuan)
• Trial ID: NCT07014943 on ClinicalTrials.gov

What this trial studies

This single-arm, prospective phase 2 trial gives high-dose gemcitabine, busulfan, and thiotepa as a conditioning regimen prior to autologous stem cell transplantation in patients with primary or relapsed CNS lymphoma. Gemcitabine is administered IV on Days -9 and -3, busulfan IV on Days -9 to -7, thiotepa IV on Days -5 to -3, and peripheral stem cells are infused on Day 0. Patients are followed with clinic visits and imaging according to International Primary CNS Lymphoma Group recommendations to monitor safety and disease response. The study's main outcomes are the regimen's safety profile and measures of disease control after transplant.

Who should consider this trial

Good fit: Adults aged 18–70 with primary or relapsed CNS lymphoma who have achieved complete or partial response, have ECOG 0–2, adequate organ function, no systemic lymphoma, and are eligible for autologous stem cell transplantation.

Not a fit: Patients older than 70, those with uncontrolled systemic disease, poor organ or cardiac or pulmonary function, active viral infections (e.g., HIV or high‑burden hepatitis), or who are not in CR/PR or are ineligible for transplant may not benefit from this regimen.

Why it matters

Potential benefit: If successful, this regimen could provide a more effective conditioning approach that improves disease control and survival after autologous stem cell transplant for CNS lymphoma.

How similar studies have performed: Thiotepa‑ and busulfan‑based conditioning regimens have shown activity in CNS lymphoma, but adding high‑dose gemcitabine as part of a GemBuTT regimen is less well studied.

Eligibility criteria

Inclusion Criteria:

1. age between 18 and 70 years;
2. CNS status of complete remission (CR) or partial response (PR) as assessed by magnetic resonance imaging (MRI), positron emission tomography-computed tomography (PET/CT), or CSF analysis (if applicable);
3. Eastern Cooperative Oncology Group (ECOG) performance status of 0,1 or 2;
4. absence of systemic lymphoma in the neck, chest, abdomen, and pelvis as assessed by CT and bone marrow biopsy;
5. negative HIV and hepatitis virus infections (particularly hepatitis B or C, with HBV DNA ≥ 10,000 copies/mL);
6. left ventricular ejection fraction (LVEF) ≥50% and no uncontrolled arrythmias or symptomatic cardiac disease;
7. forced expiratory volume in one second (FEV1) ≥70%;
8. serum creatinine clearance ≥ 50 ml/min and/or serum creatinine ≤ 1.8 mg/dL;
9. serum bilirubin ≤ 2 times the upper limit of normal, serum glutamate oxaloacetate transaminase (SGOT) and/or serum glutamate pyruvate transaminase (SGPT) ≤ 3 times the upper limit of normal.

Exclusion Criteria:

1. relapse after stem cell transplantation;
2. other uncontrolled malignancies;
3. immunodeficiency;
4. active infection requiring parenteral antibiotics;
5. pregnant or lactation;
6. severe psychiatric or psychological conditions.

Where this trial is running

Chengdu, Sichuan

• West China Hospital of Sichuan University — Chengdu, Sichuan, China (RECRUITING)

Study contacts

• Study coordinator: Jie Ji, MD

Conditions: Primary Central Nervous System Lymphoma