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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03198195
• Other study ID #: CIP-2015-08
• Status: Enrolling by invitation
• Phase: N/A
• First received: June 22, 2017
• Last updated: June 26, 2017
• Start date: March 10, 2015
• Est. completion date: July 10, 2020

Study information

• Verified date: June 2017
• Source: Capital Research Institute of Pediatrics
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

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

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 5
• Est. completion date: July 10, 2020
• Est. primary completion date: March 10, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Months to 10 Years

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

• without indication of Hematopoietic stem cell transplantation

Related Conditions & MeSH terms

• Syndrome
• Wiskott-Aldrich Syndrome

Intervention

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

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Capital Research Institute of Pediatrics

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate of aGvHD	after post transplant cyclophospamide	3month