Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

NCT03821987 | Unknown

• 1 other identifier: PUIH-SAA
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia; Stem Cell Transplant Complications; Engraft Failure

Keywords

haploidentical transplantation; aplastic anemia

Outcome Measures

Primary Outcomes (1)

• 1 year cumulative incidence overall survival

  Tne cumulative incidence of overall survival at 1 year post HSCT

  1 year post HSCT

Secondary Outcomes (5)

• one month Transplantation related mortality

  1 month post HSCT

• Engraftment

  1 month post HSCT

• 0ne month regimen-related toxicity

  1 month post HSCT

• aGVHD

  100 days post HSCT

• failure-free survival

  1 year post HSCT

Study Arms (1)

BFCA regimen

EXPERIMENTAL

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

Interventions

Fludarabine DRUG

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.

Also known as: Busulfan, Cyclophosphamide, Thymoglobulin

BFCA regimen

Eligibility Criteria

• Age: 3 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

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

Beijing, Beijing Municipality, 100044, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

fludarabine; Busulfan; Cyclophosphamide; thymoglobulin

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds

Study Officials

• Xiaojun Huang, Prof.

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaojun Huang, Prof.

CONTACT

861088326006; lpxu_0415@sina.com

Lanping Xu

CONTACT

861088326904; lpxu_0415@sina.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Institute Hematology,Peking University

Model Details: 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.

Study Record Dates

• First Submitted: January 23, 2019
• First Posted: January 30, 2019
• Study Start: December 17, 2018
• Primary Completion: December 30, 2021
• Study Completion: March 30, 2022
• Last Updated: March 10, 2020

Record last verified: 2020-03

Locations

CN (1)