|
|
Home
Search
Study Topics
Glossary
|
 |
|
|
|
|
|
|
|
|
|
|
|
|
||||||||||||||||||||||||||||||||||||
| Sponsor: | The Korean Society of Pediatric Hematology Oncology |
|---|---|
| Information provided by: | The Korean Society of Pediatric Hematology Oncology |
| ClinicalTrials.gov Identifier: | NCT00885833 |
Purpose
Wiskott-Aldrich syndrome (WAS) is a rare X-linked congenital immune-deficiency syndrome and hematopoietic stem cell transplantation (HSCT) has become a curative modality. But the transplant with the conventional conditioning resulted in high incidence of treatment related toxicities and non-myeloablative conditioning resulted in high incidence of engraftment failure. Recently, fludarabine based reduced toxicity myeloablative conditioning regimen was developed for adult myeloid malignancies with promising result of good engraftment and low treatment related toxicities. To increase the engraftment potential without serious complication, reduced toxicity myeloablative conditioning regimen composed of fludarabine, busulfan, and thymoglobulin is designed for Wiskott-Aldrich syndrome.
| Condition | Intervention | Phase |
|---|---|---|
|
Wiskott-Aldrich Syndrome |
Drug: Fludarabine, Busulfan, Thymoglobulin |
Phase I Phase II |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome |
| Estimated Enrollment: | 5 |
| Study Start Date: | February 2007 |
| Estimated Study Completion Date: | January 2012 |
| Estimated Primary Completion Date: | January 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Fludarabine |
Drug: Fludarabine, Busulfan, Thymoglobulin
fludarabine (40 mg/m2 once daily i.v. on days -8, -7, -6, -5, -4 & -3) busulfan (0.8 mg/kg every 6 hours i.v. on days -6, -5, -4, & -3) thymoglobulin (2.5 mg/kg once daily i.v. on days -8, -7, -6 for cord blood and on days -4, -3, -2 for bone marrow or mobilized peripheral blood)
|
Wiskott-Aldrich syndrome (WAS) is an rare X-linked congenital immune-deficiency syndrome characterized by the triad of recurrent infection, eczema and thrombocytopenia with small size of platelet (Puck JM, 2006). Clinical studies revealed high rate of autoimmune disorder and malignancy in WAS (Ochs HD, 2006). The identification of the molecular defect in 1994 (Derry JM, 1994) has broadened the clinical spectrum of the syndrome to include chronic or intermittent X-linked thrombocytopenia (XLT), a relatively mild form of WAS and X-lined neutropenia caused by an arrest of myelopoiesis (Ochs HD, 2006).
The incidence of WAS in Korea was very low and only 6 patients diagnosed between 2001 and 2005 (Kim JG, 2006).
Conventional treatments for WAS such as prophylactic antibiotics and immune globin for infection and platelet transfusion for bleeding were not so successful (Thrasher AJ, 2000). Bone marrow transplantation (BMT) from an HLA-matched related donor is an effective treatment (Filipovich AH, 2001) and patients without appropriate related donor could receive alternative stem cell source such as matched unrelated donor or cord blood. But the transplant with the alternative donor needed more intensive conditioning to overcome the hematologic and immunologic barrier with increased treatment related toxicity. Further progress depends in particular on the development of alternative preparative conditioning regimens which allow stable engraftment of donor precursor cells with minimal systemic toxic side effects (Friedrich W, 2004).
Recently, we reported successful unrelated bone marrow transplantation in a boy with WAS with reduced toxicity myeloablative conditioning regimen to increase the engraftment potential without serious complication (Kang, 2008), and extended to multicenter phase I/II pilot study with this reduced toxicity myeloablative conditioning regimen in the HSCT for WAS.
Eligibility| Ages Eligible for Study: | 1 Year to 25 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases:
Exclusion Criteria:
Contacts and Locations| Contact: Hyoung Jin Kang, Ph. D | 82 2 2072 3304 | kanghj@snu.ac.kr |
| Contact: Ji won Lee, M.D | 82 2 2072 0177 | agnesjw@hanmail.net |
| Korea, Republic of | |
| Seoul National University Hospital | Recruiting |
| Seoul, Chongno-gu, Korea, Republic of, 110-744 | |
| Contact: Hyoung Jin Kang, Ph. D 82 2 2072 3304 kanhhj@snu.ac.kr | |
| Contact: Ji Won Lee, M.D 82 2 2072 0177 agnesjw@hanmail.net | |
| Principal Investigator: Hyo Seop Ahn, Ph. D | |
| Principal Investigator: | Hyo Seop Ahn, Ph. D | The Korean Society of Pediatric Hematology Oncology |
More Information
| Responsible Party: | The Korean Society of Hematology, The Korean Society of Pediatric Hematology Oncology |
| ClinicalTrials.gov Identifier: | NCT00885833 History of Changes |
| Other Study ID Numbers: | KSPHO-S0701 |
| Study First Received: | April 21, 2009 |
| Last Updated: | February 23, 2011 |
| Health Authority: | Korea: Food and Drug Administration |
|
Wiskott-Aldrich syndrome HSCT |
|
Wiskott-Aldrich Syndrome Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases Hemorrhagic Disorders Lymphopenia Leukopenia Leukocyte Disorders Genetic Diseases, Inborn Genetic Diseases, X-Linked Immunologic Deficiency Syndromes Immune System Diseases Busulfan Fludarabine monophosphate |
Fludarabine Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses Myeloablative Agonists Antimetabolites, Antineoplastic Antimetabolites |
