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Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

Primary Purpose

Aplastic Anemia, Stem Cell Transplant Complications, Engraft Failure

Status

Unknown status

Phase

Not Applicable

Locations

China

Study Type

Interventional

Intervention

Fludarabine

About this trial

This is an interventional treatment trial for Aplastic Anemia focused on measuring haploidentical transplantation, aplastic anemia

Eligibility Criteria

3 Years - 55 Years (Child, Adult)All SexesDoes not accept healthy volunteers

A：inclusion criteria

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

B. Exclusion criteria ：

patients with congenital SAA/vSAA
patients with age< 3years or >55 years
patients with matched sibling donor
patients with matched URD
patients with severe infection
patients with severe organ dysfunction
pregnancy women
no Consent form signed

Sites / Locations

Peking University Institute of Hematology,People's hospital Peking UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BFCA regimen

Arm Description

Detail: Patients enrolled in this study would receive Busulfan(B) (IV)0.8mg/kg Q6hx2d,Fludarabine(F) 30mg/m2x5d ,cyclophosphamide(C) 25mg/kg/dx4d,thymoglobulin (A :rATG ,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.

Outcomes

Primary Outcome Measures

1 year cumulative incidence overall survival

Tne cumulative incidence of overall survival at 1 year post HSCT

Secondary Outcome Measures

one month Transplantation related mortality

Tne cumulative incidence of transplantation related motality at 1 month post HSCT

Engraftment

Tne cumulative incidence of engraftment at 1 moths post HSCT

0ne month regimen-related toxicity

Tne cumulative incidence of regimen related toxicity at 1 month post HSCT

aGVHD

Tne cumulative incidence of acute GVHD at Day 100 post HSCT

failure-free survival

The cumulative incidence of failure-free survival at 1year post HSCT

Full Information

NCT ID

NCT03821987

First Posted

January 23, 2019

Last Updated

March 9, 2020

Sponsor

Peking University People's Hospital

1. Study Identification

Unique Protocol Identification Number

NCT03821987

Brief Title

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

2. Study Status

Record Verification Date

March 2020

Overall Recruitment Status

Unknown status

Study Start Date

December 17, 2018 (Actual)

Primary Completion Date

December 30, 2021 (Anticipated)

Study Completion Date

March 30, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Peking University People's Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Aplastic Anemia, Stem Cell Transplant Complications, Engraft Failure

Keywords

haploidentical transplantation, aplastic anemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

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.

Masking

None (Open Label)

Allocation

N/A

Enrollment

55 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

BFCA regimen

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Fludarabine

Other Intervention Name(s)

Busulfan, Cyclophosphamide, Thymoglobulin

Intervention 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.

Primary Outcome Measure Information:

Title

1 year cumulative incidence overall survival

Description

Tne cumulative incidence of overall survival at 1 year post HSCT

Time Frame

1 year post HSCT

Secondary Outcome Measure Information:

Title

one month Transplantation related mortality

Description

Tne cumulative incidence of transplantation related motality at 1 month post HSCT

Time Frame

1 month post HSCT

Title

Engraftment

Description

Tne cumulative incidence of engraftment at 1 moths post HSCT

Time Frame

1 month post HSCT

Title

0ne month regimen-related toxicity

Description

Tne cumulative incidence of regimen related toxicity at 1 month post HSCT

Time Frame

1 month post HSCT

Title

aGVHD

Description

Tne cumulative incidence of acute GVHD at Day 100 post HSCT

Time Frame

100 days post HSCT

Title

failure-free survival

Description

The cumulative incidence of failure-free survival at 1year post HSCT

Time Frame

1 year post HSCT

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

Eligibility Criteria

A：inclusion criteria Patients diagnosed as acquired severe aplastic anemia(SAA) /very vSAA patients with age 3-55 years patients have no matched sibling donor Patients have no matched unrelated donor patients have no severe infection Patients have no severe organ dysfunction patients have risk factors of potential intolerance to previous condition regimen including BuCy(200mg/kg)and ATG Consent form signed B. Exclusion criteria ： patients with congenital SAA/vSAA patients with age< 3years or >55 years patients with matched sibling donor patients with matched URD patients with severe infection patients with severe organ dysfunction pregnancy women no Consent form signed

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Xiaojun Huang, Prof.

Phone

861088326006

lpxu_0415@sina.com

First Name & Middle Initial & Last Name or Official Title & Degree

Lanping Xu

Phone

861088326904

lpxu_0415@sina.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Xiaojun Huang, Prof.

Organizational Affiliation

Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy

Official's Role

Principal Investigator

Facility Information:

Facility Name

Peking University Institute of Hematology,People's hospital Peking University

City

Beijing

State/Province

Beijing

ZIP/Postal Code

100044

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lanping Xu, Prof.

Phone

8613641028627

lpxu_0415@sina.com

First Name & Middle Initial & Last Name & Degree

Yanru Ma

Phone

8613641134402

lpxu_0415@vip.sina.com

12. IPD Sharing Statement

Learn more about this trial

Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

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