Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

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:

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

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): 5

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 months to 10 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Patient with Wiskott-Aldrich Syndrome :

1. life-threatening bleeding
2. 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

How is the study designed?

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Capital Research Institute of Pediatrics

Study record dates

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: June 26, 2017
• Last Verified: June 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immunologic Deficiency Syndromes; Immune System Diseases; Disease; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Leukopenia; Leukocyte Disorders; Primary Immunodeficiency Diseases; Lymphopenia; Syndrome; Wiskott-Aldrich Syndrome; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antirheumatic Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Cyclophosphamide
• Other Study ID Numbers: CIP-2015-08

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No