Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

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Brief Title

Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

Official Title

Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome

Brief Summary

      A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate
      of GvHD.

      The details of the protocal followed with:

        1. Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.

        2. GvHD Prophylaxis:

      Rabbit antihuman thymocyte globulin 7.5 mg/kg post-transplant cyclophosphamide (CY) (50
      mg/kg.d on days +3 and +4) Cyclosporine or tacrolimus, mycophenolate mofetil, on days +5
    

Detailed Description

      Patients were enrolled in CIP-2015 Protocol at the Capital Institute of Pediatrics (Beijing).
      The conditioning regimen consisted of fludarabine (40 mg/m2) from days -6 to -3, and Busulfan
      was administered intravenously for 4 days, from days -5 to -2,using dose targeting based on
      therapeutic drug monitoring. Thymoglobulin (Sanofi, Cambridge, MA) 7.5 to 10 mg/kg (cumulative
      dose over 4 days) was administered over 4 days, from days -5 to -2. Bone marrow (BM) and PBSC
      were infused on day 0, followed by post-transplant CY (50 mg/kg/day, on days +3 and +4). To
      protect against hemorrhagic cystitis, MESNA (2-mercaptoethane sodium sulfonate) was
      administered at 150% of the CY dose. Post grafting immunosuppression with mycophenolate
      mofetil and tacrolimus commenced on day +5 and extended until days +28 and +84, respectively.
      Tacrolimus was tapered off by day +90 if there was no GVHD.
    


Study Type

Observational [Patient Registry]


Primary Outcome

Rate of aGvHD


Condition

Wiskott-Aldrich Syndrome

Intervention

cyclophosphamide


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Procedure

Estimated Enrollment

5

Start Date

March 10, 2015

Completion Date

July 10, 2020

Primary Completion Date

March 10, 2017

Eligibility Criteria

        Inclusion Criteria:

        -Patients diagnosed with Wiskott-Aldrich Syndrome with indication of Hematopoietic stem
        cell transplantation

        Exclusion Criteria:

          -  without indication of Hematopoietic stem cell transplantation
      

Gender

All

Ages

5 Months - 10 Years

Accepts Healthy Volunteers

No

Contacts

, , 



Administrative Informations


NCT ID

NCT03198195

Organization ID

CIP-2015-08


Responsible Party

Principal Investigator

Study Sponsor

Capital Research Institute of Pediatrics


Study Sponsor

, , 


Verification Date

June 2017