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Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03198195
Recruitment Status : Enrolling by invitation
First Posted : June 26, 2017
Last Update Posted : June 27, 2017
Information provided by (Responsible Party):
Yan Yue, Capital Research Institute of Pediatrics

Brief 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

Condition or disease Intervention/treatment
Wiskott-Aldrich Syndrome 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.

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Study Type : Observational [Patient Registry]
Actual Enrollment : 5 participants
Observational Model: Other
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: Post-transplant Cyclophosphamide for HLA-haploidentical Transplantation in Wiskott-Aldrich Syndrome
Actual Study Start Date : March 10, 2015
Actual Primary Completion Date : March 10, 2017
Estimated Study Completion Date : July 10, 2020

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

Primary Outcome Measures :
  1. Rate of aGvHD [ Time Frame: 3month ]
    after post transplant cyclophospamide

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Months to 10 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population

Patient with Wiskott-Aldrich Syndrome :

  1. life-threatening bleeding
  2. strong demand of parents

Inclusion Criteria:

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

Exclusion Criteria:

  • without indication of Hematopoietic stem cell transplantation

Additional Information:

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Responsible Party: Yan Yue, MD,PhD, Capital Research Institute of Pediatrics Identifier: NCT03198195     History of Changes
Other Study ID Numbers: CIP-2015-08
First Posted: June 26, 2017    Key Record Dates
Last Update Posted: June 27, 2017
Last Verified: June 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Wiskott-Aldrich Syndrome
Pathologic Processes
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Hemorrhagic Disorders
Leukocyte Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Immunologic Deficiency Syndromes
Immune System Diseases
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists