Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

Overview

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

Full Title of Study: “Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome”

Study Type

  • Study Type: Observational [Patient Registry]
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: March 10, 2017

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.

Interventions

  • Procedure: cyclophosphamide
    • on days +3,+4,using cyclophosphamide 50mg/kg

Clinical Trial Outcome Measures

Primary Measures

  • Rate of aGvHD
    • Time Frame: 3month
    • after post transplant cyclophospamide

Participating in This Clinical Trial

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 Eligibility: All

Minimum Age: 5 Months

Maximum Age: 10 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Capital Research Institute of Pediatrics
  • Provider of Information About this Clinical Study
    • Principal Investigator: Yan Yue, MD,PhD – Capital Research Institute of Pediatrics

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