Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

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.

Study Overview

• Status: Unknown
• Conditions: Aplastic Anemia; Stem Cell Transplant Complications; Engraft Failure
• Intervention / Treatment: Drug: Fludarabine

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.

• Study Type: Interventional
• Enrollment (Anticipated): 55
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Xiaojun Huang, Prof.; Phone Number: 861088326006; Email: lpxu_0415@sina.com
• Study Contact Backup: Name: Lanping Xu; Phone Number: 861088326904; Email: lpxu_0415@sina.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100044
      • Recruiting
      • Peking University Institute of Hematology,People's hospital Peking University
      • Contact: Lanping Xu, Prof.; Phone Number: 8613641028627; Email: lpxu_0415@sina.com
      • Contact: Yanru Ma; Phone Number: 8613641134402; Email: lpxu_0415@vip.sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 55 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: BFCA regimen; 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.

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.; Other Names: Cyclophosphamide; Thymoglobulin; Busulfan

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
1 year cumulative incidence overall survival	Tne cumulative incidence of overall survival at 1 year post HSCT	1 year post HSCT

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Peking University People's Hospital
• Investigators: Principal Investigator: Xiaojun Huang, Prof., Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy

Study record dates

Study Major Dates

• Study Start (Actual): December 17, 2018
• Primary Completion (Anticipated): December 30, 2021
• Study Completion (Anticipated): March 30, 2022

Study Registration Dates

• First Submitted: January 23, 2019
• First Submitted That Met QC Criteria: January 26, 2019
• First Posted (Actual): January 30, 2019

Study Record Updates

• Last Update Posted (Actual): March 10, 2020
• Last Update Submitted That Met QC Criteria: March 9, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Keywords: aplastic anemia; haploidentical transplantation
• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Anemia; Bone Marrow Failure Disorders; Anemia, Aplastic; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antirheumatic Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Cyclophosphamide; Fludarabine; Busulfan; Thymoglobulin
• Other Study ID Numbers: PUIH-SAA

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No