Unrelated umbilical cord blood transplant for EBV-associated lymphoproliferative diseases
A Single-arm, Two-center, Prospective Clinical Study of Unrelated Cord Blood Transplantation for EBV-associated Lymphoproliferative Disorders
This trial will try an unrelated umbilical cord blood transplant to treat people aged 14–80 with EBV-associated lymphoproliferative diseases, including chronic active EBV and EBV-related lymphoma.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 80 (estimated) |
| Ages | 14 Years to 80 Years |
| Sex | All |
| Sponsor | Fudan University Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 2 sites (Shanghai, Shanghai Municipality and 1 other locations) |
| Trial ID | NCT07381738 on ClinicalTrials.gov |
What this trial studies
This is a single-arm, two-center, open-label prospective Phase 2 trial in which all participants receive a standard-dose unrelated umbilical cord blood transplantation to treat EBV-associated lymphoproliferative diseases. The trial enrolls adolescents and adults with diagnoses such as chronic active EBV infection, EBV-associated hemophagocytic lymphohistiocytosis, or EBV-associated lymphoma and requires adequate performance status and expected survival. The primary focus is on safety and efficacy outcomes following cord blood transplantation in patients who may lack matched donors, with close monitoring of EBV-DNA levels, graft function, and transplant-related complications. The procedures and follow-up are conducted at two Fudan University hospitals in Shanghai.
Who should consider this trial
Good fit: Ideal candidates are people aged 14–80 with confirmed EBV-associated lymphoproliferative disease (including CAEBV, EBV-HLH, or EBV-associated lymphoma), ECOG 0–2 (or equivalent pediatric score), expected survival of at least three months, and who meet disease-specific entry criteria such as elevated EBV-DNA for CAEBV.
Not a fit: Patients with poor performance status (ECOG >2), expected survival under three months, severe uncontrolled organ failure or infections, or who cannot receive treatment at the Shanghai sites are unlikely to benefit from this protocol.
Why it matters
Potential benefit: If successful, unrelated cord blood transplantation could provide a curative option that reduces EBV viral load, controls abnormal lymphocyte growth, and improves survival for patients without matched donors.
How similar studies have performed: Allogeneic hematopoietic stem cell transplantation is an established curative approach for CAEBV and EBV-LPDs, and unrelated cord blood transplantation has been used successfully in other allo-HSCT settings, though its specific use for EBV-LPDs is less extensively studied.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria:General Inclusion Criteria: All subjects must meet all of the following criteria: Age range: 14 to 80 years old, inclusive. Diagnosed with an EBV-associated lymphoproliferative disease (including Chronic Active EBV Infection, EBV-associated Hemophagocytic Lymphohistiocytosis, and EBV-associated Lymphoma). Meet at least one of the following disease-specific criteria (see below). ECOG Performance Status score of 0-2 (or Lansky Play-Performance score ≥60 for children). Expected survival of ≥3 months. Signed informed consent form (for minors, consent must be provided by a legal guardian). Disease-Specific Inclusion Criteria: A. CAEBV Patients: Patients diagnosed with CAEBV according to the 2016 revised WHO classification criteria, who also meet all of the following: * Persistent or recurrent infectious mononucleosis-like symptoms for more than 3 months. * Elevated EBV-DNA load in peripheral blood or involved tissues (peripheral blood EBV-DNA \> 500 copies/ml). * Detection of EBV in T or NK cells (via EBER in situ hybridization or LMP1 immunohistochemistry). * Exclusion of other known immunodeficiency disorders, autoimmune diseases, or neoplastic diseases. B. EBV-HLH Patients: Meet ≥5 of the HLH-2004 diagnostic criteria: * Persistent or recurrent infectious mononucleosis-like symptoms for more than 3 months. * Fever ≥38.5°C. * Splenomegaly. * Cytopenias (affecting ≥2 of 3 lineages): * Hb \<90 g/L * PLT \<100 × 10⁹/L * ANC \<1.0 × 10⁹/L * Hypertriglyceridemia (\>3 mmol/L) and/or hypofibrinogenemia (\<1.5 g/L). * Serum ferritin \>500 μg/L. * sCD25 \>2400 U/mL. * Decreased or absent NK cell activity. * Evidence of hemophagocytosis in bone marrow, spleen, or lymph nodes. C. EBV-associated Lymphoma Patients: * Pathologically confirmed diagnosis according to WHO criteria. * EBV-positive (EBER+). * Failure of or relapse after first-line therapy. Exclusion Criteria:Patients meeting any of the following criteria will be excluded: History of another primary malignancy within 5 years prior to the first treatment. Uncorrected thyroid dysfunction. Grade II or higher cardiac disease according to the New York Heart Association (NYHA) classification. History of organ transplantation. Planned to receive other types of hematopoietic stem cell transplantation during the study period. Active infections, including: Active hepatitis B or hepatitis C. Positive serum HIV antigen or antibody. History of syphilis. Major surgery within 4 weeks prior to the first treatment, or anticipated need for major surgery during the study. Pregnant or breastfeeding women. History of severe psychiatric illness or substance abuse. High risk of complications, such as: Uncontrolled infection. Active major visceral hemorrhage. Known allergy to any component of the investigational drug(s), or a history of severe allergies. Patients deemed unable to comply with the study procedures and/or follow-up requirements. \-
Where this trial is running
Shanghai, Shanghai Municipality and 1 other locations
- Huadong hospital, Fudan university — Shanghai, Shanghai Municipality, China (Recruiting)
- Huashan Hospital, Fudan University — Shanghai, Shanghai Municipality, China (Recruiting)
Study contacts
- Principal investigator: Jiexian Ma — Huadong Hospital
- Study coordinator: Jiexian Ma
- Email: jiexianma@hotmail.com
- Phone: +86 13764520566
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.