Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

June 26, 2017 updated by: Yan Yue, Capital Research Institute of Pediatrics

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

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:

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

Study Overview

Status

Unknown

Conditions

Wiskott-Aldrich Syndrome

Intervention / Treatment

Procedure: cyclophosphamide

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

Enrollment (Actual)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 months to 10 years (CHILD)

Accepts Healthy Volunteers

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

Patient with Wiskott-Aldrich Syndrome :

life-threatening bleeding
strong demand of parents

Description

Inclusion Criteria:

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

Exclusion Criteria:

without indication of Hematopoietic stem cell transplantation

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of aGvHD Time Frame: 3month	after post transplant cyclophospamide	3month

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Capital Research Institute of Pediatrics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

March 10, 2015

Primary Completion (ACTUAL)

March 10, 2017

Study Completion (ANTICIPATED)

July 10, 2020

Study Registration Dates

First Submitted

June 22, 2017

First Submitted That Met QC Criteria

June 22, 2017

First Posted (ACTUAL)

June 26, 2017

Study Record Updates

Last Update Posted (ACTUAL)

June 27, 2017

Last Update Submitted That Met QC Criteria