Thiotepa Incorporating TBI/Cy Conditioning Regimen for EBV-HLH With Central Nervous System Involvement

NCT05854225 | Unknown

• 1 other identifier: 2021-P2-250-01
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

EBV-HLH is the most common in virus-associated hemophagocytic syndrome (VAHS). There are no uniform clinical criteria for the diagnosis of CNS-HLH. And there is still a lack of international consensus on the treatment for CNS-HLH. Following allogeneic HSCT after HLH induction therapy is recommended for CNS-HLH. One of the major factors influencing the transplantation effect is conditioning. Therefore, we conduct a prospective clinical study to explore the efficacy and safety of thiotepa incorporating TBI/Cy conditioning regimen followed by allogeneic hematopoietic stem cell transplantation for EBV-HLH with central nervous system involvement.

Conditions

HLH

Outcome Measures

Primary Outcomes (1)

• The clinical remission rate

  The number of cases with complete response (CR) and partial response (PR) after transplantation as a percentage of the total cases.

  24 weeks

Study Arms (1)

single-arm

OTHER

a prospective single-arm clinical study

Drug: Thiotepa

Interventions

Thiotepa DRUG

Thiotepa is an alkylating agent that is routinely used in solid tumors, but it is able to cross the blood-brain barrier into the center.

Also known as: TT

single-arm

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Diagnosed as HLH according to HLH-04 diagnostic criteria.
• a significant increase of EBV-DNA copies/ml in peripheral blood or tissue or EBER(+) in tissue specimens or peripheral blood.
• Cerebrospinal fluid EBV-DNA ≥5×10\^2 copies/ml.
• Estimated survival time ≥ 1 month.
• Age 18\~60， gender is not limited.
• Total bilirubin ≤ 2 times the upper limit of normal; serum creatinine ≤ normal value before study entry.
• Serum HIV antigen or antibody negative.
• HCV antibody negative.
• HBsAg negative, HBcAb negative. If any of the above is positive, then HBV-DNA titer in peripheral blood is required less than 1×102 copies/ml before enrollment.
• Cardiac ultrasound LVEF≥50%.
• Women in the child-bearing period must not be pregnant as determined by a pregnancy test and must be willing to use effective contraception during the trial and for ≥ 12 months after the last dose. Pregnant and lactating women cannot participate. All male subjects accept contraception during the trial and for ≥6 months after the last dose.
• Signed informed consent.

You may not qualify if:

• Heart function above grade II (NYHA).
• Active bleeding of the internal organs(digestive tract, lung, brain, etc.).
• Uncontrolled infection (pulmonary infection, intestinal infection, etc.).
• Severe mental illness.
• Patients are unable to comply during the trial and/or follow-up phase.
• Participate in other clinical research at the same time.

Sponsors & Collaborators

• Beijing Friendship Hospital: lead

Study Sites (1)

Beijing Friendship Hospital, Capital Medical University

Beijing, Beijing Municipality, 100050, China

RECRUITING

MeSH Terms

Interventions

Thiotepa

Intervention Hierarchy (Ancestors)

Phosphoramides; Organophosphorus Compounds; Organic Chemicals; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• wang zhao, MD

  Beijing Friendship Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

wang zhao, MD

CONTACT

63138303; wangzhao@ccmu.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor and Head of hematology

Study Record Dates

• First Submitted: April 20, 2023
• First Posted: May 11, 2023
• Study Start: January 1, 2022
• Primary Completion: June 1, 2024
• Study Completion: January 1, 2025
• Last Updated: May 11, 2023

Record last verified: 2023-05

Locations

CN (1)