Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

Wiskott-Aldrich Syndrome Clinical Trial

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

Status Enrolling by invitation

Clinical Trial 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

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Syndrome
• Wiskott-Aldrich Syndrome

• NCT number: NCT03198195
• Study type: Observational [Patient Registry]
• Source: Capital Research Institute of Pediatrics
• Status: Enrolling by invitation
• Phase: N/A
• Start date: March 10, 2015
• Completion date: July 10, 2020