Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

Aplastic Anemia Clinical Trial

Official title:

Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03821987
• Other study ID #: PUIH-SAA
• Status: Recruiting
• Phase: N/A
• Start date: December 17, 2018
• Est. completion date: March 30, 2022

Study information

• Verified date: March 2020
• Source: Peking University People's Hospital
• Contact: Xiaojun Huang, Prof.
• Phone: 861088326006
• Email: lpxu_0415[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 55
• Est. completion date: March 30, 2022
• Est. primary completion date: December 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 55 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Aplastic
• Aplastic Anemia
• Engraft Failure
• Stem Cell Transplant Complications

Intervention

Drug:
• Fludarabine
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.

Locations

Country	Name	City	State
China	Peking University Institute of Hematology,People's hospital Peking University	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	1 year cumulative incidence overall survival	Tne cumulative incidence of overall survival at 1 year post HSCT	1 year post HSCT
Secondary	one month Transplantation related mortality	Tne cumulative incidence of transplantation related motality at 1 month post HSCT	1 month post HSCT
Secondary	Engraftment	Tne cumulative incidence of engraftment at 1 moths post HSCT	1 month post HSCT
Secondary	0ne month regimen-related toxicity	Tne cumulative incidence of regimen related toxicity at 1 month post HSCT	1 month post HSCT
Secondary	aGVHD	Tne cumulative incidence of acute GVHD at Day 100 post HSCT	100 days post HSCT
Secondary	failure-free survival	The cumulative incidence of failure-free survival at 1year post HSCT	1 year post HSCT