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

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

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Ages Eligible for Study: | 3 Years to 55 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

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
Contact: Xiaojun Huang, Prof. | 861088326006 | lpxu_0415@sina.com | |
Contact: Lanping Xu | 861088326904 | lpxu_0415@sina.com |
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 |
Principal Investigator: | Xiaojun Huang, Prof. | Peking Universiy Institute of Hematology,People's Hospital Pekiking Universiyy |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
haploidentical transplantation aplastic anemia |
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 |