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

Sponsor

Beijing Friendship Hospital (Other)

Overall Status

Recruiting

CT.gov ID

NCT05854225

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.6

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
HLH	Drug: Thiotepa	N/A

Detailed Description

HLH is an immune disorder characterized by a potentially fatal cytokine storm induced by uncontrolled T lymphocyte and macrophage activation. 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. CNS involvement is one of the factors in the poor prognosis for 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Thiotepa Incorporating TBI/Cy Conditioning Regimen Followed by HSCT for EBV-HLH With Central Nervous System Involvement: a Prospective Single-arm Clinical Study

Actual Study Start Date :

Jan 1, 2022

Anticipated Primary Completion Date :

Jun 1, 2024

Anticipated Study Completion Date :

Jan 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Other: single-arm a prospective single-arm clinical study	Drug: Thiotepa 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. Other Names: TT

Arm

Intervention/Treatment

Other: single-arm

a prospective single-arm clinical study

Drug: Thiotepa

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.

Other Names:

Outcome Measures

Primary Outcome Measures

The clinical remission rate [24 weeks]
The number of cases with complete response (CR) and partial response (PR) after transplantation as a percentage of the total cases.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Beijing Friendship Hospital, Capital Medical University	Beijing	Beijing	China	100050

Sponsors and Collaborators

Beijing Friendship Hospital

Investigators

Principal Investigator: wang zhao, MD, Beijing Friendship Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Zhao Wang, Professor and Head of hematology, Beijing Friendship Hospital

ClinicalTrials.gov Identifier:

NCT05854225

Other Study ID Numbers:

2021-P2-250-01

First Posted:

May 11, 2023

Last Update Posted:

May 11, 2023

Last Verified:

May 1, 2023

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Thiotepa

Study Results

No Results Posted as of May 11, 2023