Clinical trial NCT03821987: Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA
Official title: Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: NA
Conditions: Aplastic Anemia
Stem Cell Transplant Complications
Engraft Failure
Keywords: haploidentical transplantation
aplastic anemia
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2019-01-30
Enrollment: 55
Sex: ALL
Minimum age (years): 3
Maximum age (years): 55
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.

BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that we monitored BM or blood samples included at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.
Primary purpose: TREATMENT

Brief summary: The haplotype HSCT system including Bu(0.8mg/kg Q6hx2d)CTX(50mg/kgx4d)rATG(2.5mg/kgx4d) , established in Institute of Hematology of Peking University ,has been evaluated to be effective for acquired SAA.But some patients with high risk factors may not tolerate CTX 200mg/kg,alternative conditioning regimen including Bu/Fludarabine/dercreased CTX was studied in this trial.

Detailed description: Patients enrolled in this study would receive Bu (IV)0.8mg/kg Q6hx2d,Fludarabine 30mg/m2x5d ,CTX(cyclophosphamide) 25mg/kg/dx4d,rATG (thymoglobulin,Sang Stat,France) 2.5mg/kg/dx4d.

BM or Blood samples from patients were obtained to assess engraftment and chimerism after HSCT. The time point that investigators monitor BM or blood samples at 1 month,2 months, 3 months,6 months, 9 months and 1year,2years,3years 5 years after HSCT.

Eligibility criteria: A：inclusion criteria

1. Patients diagnosed as acquired severe aplastic anemia(SAA) /very vSAA
2. patients with age 3-55 years
3. patients have no matched sibling donor
4. Patients have no matched unrelated donor
5. patients have no severe infection
6. Patients have no severe organ dysfunction
7. patients have risk factors of potential intolerance to previous condition regimen including BuCy(200mg/kg)and ATG
8. Consent form signed

B. Exclusion criteria ：

1. patients with congenital SAA/vSAA
2. patients with age\< 3years or \>55 years
3. patients with matched sibling donor
4. patients with matched URD
5. patients with severe infection
6. patients with severe organ dysfunction
7. pregnancy women
8. no Consent form signed

Primary outcomes:
measure: 1 year cumulative incidence overall survival
timeFrame: 1 year post HSCT
Secondary outcomes:
measure: one month Transplantation related mortality
timeFrame: 1 month post HSCT
measure: Engraftment
timeFrame: 1 month post HSCT
measure: 0ne month regimen-related toxicity
timeFrame: 1 month post HSCT
measure: aGVHD
timeFrame: 100 days post HSCT
measure: failure-free survival
timeFrame: 1 year post HSCT
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Institute of Hematology,People's hospital Peking University
state: Beijing
Study officials:
affiliation: Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy
name: Xiaojun Huang, Prof.
role: PRINCIPAL_INVESTIGATOR
References: