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Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA

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ClinicalTrials.gov Identifier: NCT03821987
Recruitment Status : Recruiting
First Posted : January 30, 2019
Last Update Posted : March 10, 2020
Sponsor:
Information provided by (Responsible Party):
Xiaojun Huang,MD, Peking University People's Hospital

Study Description
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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.

Condition or disease Intervention/treatment Phase
Aplastic Anemia Stem Cell Transplant Complications Engraft Failure Drug: Fludarabine Not Applicable

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 55 participants
Allocation: N/A
Intervention Model: Single Group Assignment
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.
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA
Actual Study Start Date : December 17, 2018
Estimated Primary Completion Date : December 30, 2021
Estimated Study Completion Date : March 30, 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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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:
  • Busulfan
  • Cyclophosphamide
  • Thymoglobulin


Outcome Measures
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Primary Outcome Measures :
  1. 1 year cumulative incidence overall survival [ Time Frame: 1 year post HSCT ]
    Tne cumulative incidence of overall survival at 1 year post HSCT


Secondary Outcome Measures :
  1. one month Transplantation related mortality [ Time Frame: 1 month post HSCT ]
    Tne cumulative incidence of transplantation related motality at 1 month post HSCT

  2. Engraftment [ Time Frame: 1 month post HSCT ]
    Tne cumulative incidence of engraftment at 1 moths post HSCT

  3. 0ne month regimen-related toxicity [ Time Frame: 1 month post HSCT ]
    Tne cumulative incidence of regimen related toxicity at 1 month post HSCT

  4. aGVHD [ Time Frame: 100 days post HSCT ]
    Tne cumulative incidence of acute GVHD at Day 100 post HSCT

  5. failure-free survival [ Time Frame: 1 year post HSCT ]
    The cumulative incidence of failure-free survival at 1year post HSCT


Eligibility Criteria
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Ages Eligible for Study:   3 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03821987


Contacts
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Contact: Xiaojun Huang, Prof. 861088326006 lpxu_0415@sina.com
Contact: Lanping Xu 861088326904 lpxu_0415@sina.com

Locations
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China, Beijing
Peking University Institute of Hematology,People's hospital Peking University Recruiting
Beijing, Beijing, China, 100044
Contact: Lanping Xu, Prof.    8613641028627    lpxu_0415@sina.com   
Contact: Yanru Ma    8613641134402    lpxu_0415@vip.sina.com   
Sponsors and Collaborators
Peking University People's Hospital
Investigators
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Principal Investigator: Xiaojun Huang, Prof. Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy
More Information
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Responsible Party: Xiaojun Huang,MD, Director of Institute Hematology,Peking University, Peking University People's Hospital
ClinicalTrials.gov Identifier: NCT03821987    
Other Study ID Numbers: PUIH-SAA
First Posted: January 30, 2019    Key Record Dates
Last Update Posted: March 10, 2020
Last Verified: March 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Xiaojun Huang,MD, Peking University People's Hospital:
haploidentical transplantation
aplastic anemia
Additional relevant MeSH terms:
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Anemia, Aplastic
Anemia
Hematologic Diseases
Bone Marrow Diseases
Cyclophosphamide
Busulfan
Fludarabine
Thymoglobulin
Immunosuppressive Agents
 Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists