Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia (URTH)

This study is ongoing, but not recruiting participants.

Sponsor:

Collaborators:

New England Research Institutes

Pediatric Blood and Marrow Transplant Consortium

Information provided by (Responsible Party):

Washington University School of Medicine

ClinicalTrials.gov Identifier:

NCT01005576

First received: October 29, 2009

Last updated: January 17, 2013

Last verified: January 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

October 29, 2009

Last Updated Date

January 17, 2013

Start Date ^ICMJE

January 2010

Estimated Primary Completion Date

April 2014 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Primary objective: To determine event-free survival at 1 year. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01005576 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Secondary objectives: To determine the effect of hematopoietic cell transplant on clinical and laboratory manifestations of thalassemia and determining the incidence of transplant-related outcomes for 2 years. [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

Official Title ^ICMJE

A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)

Brief Summary

This study is being done to determine if blood cell transplants, with either bone marrow or cord blood from unrelated donors, are effective in children with severe thalassemia and if this treatment approach has acceptable risks and side effects.

This study includes a preparative regimen with Hydroxyurea, Alemtuzumab, Fludarabine, Thiotepa and Melphalan that provides intense host immunosuppression without myeloablation. The primary hypothesis is that this regimen will promote stable engraftment of unrelated donor hematopoietic cells, support normal erythropoiesis, and result in an event free survival of > 75% of children with thalassemia major.

Detailed Description

Not Provided

Study Type ^ICMJE

Interventional

Study Phase

Phase 2

Study Design ^ICMJE

Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Severe Thalassemia

Intervention ^ICMJE

Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

Days -50 to -21: Hydroxyurea 30mg/kg po Day -22: Alemtuzumab 3mg IV Day -21: Alemtuzumab 10mg IV Day -20: Alemtuzumab 15mg IV Day -19: Alemtuzumab 20mg IV Day -8: Fludarabine 30mg/m2 IV Day -7: Fludarabine 30mg/m2 IV Day -6: Fludarabine 30mg/m2 IV Day -5: Fludarabine 30mg/m2 IV Day -4: Fludarabine 30mg/m2 IV Day -4: Thiotepa 8mg/kg IV Day -3: Melphalan 140mg/m2 IV Day 0: Stem cell infusion

Study Arm (s)

Experimental: Conditioning regimen

Hydroxyurea days -50 to -21 Alemtuzumab days -21 to -19 Fludarabine days -8 to -4 Thiotepa day -4 Melphalan day -3 Stem cell infusion day 0

Intervention: Drug: Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Active, not recruiting

Estimated Enrollment ^ICMJE

Completion Date

Not Provided

Estimated Primary Completion Date

April 2014 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

Inclusion Criteria:

1-16.00 years old
Have transfusion dependent thalassemia major
Shall not have an HLA-matched family donor
Must have a suitably matched unrelated marrow donor or UCB product
Lansky score >/= 70
Adequate pulmonary, renal, liver, and other organ function as defined in protocol
Negative pregnancy test
Adequate total nucleated cell or CD34+ dose of product as defined in protocol
Iron chelation must be discontinued >/= 48 hours prior to conditioning regimen

Exclusion Criteria:

Pregnant or breastfeeding
HIV positive
Prior allogeneic marrow or stem cell transplantation

Gender

Both

Ages

1 Year to 16 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT Number ^ICMJE

NCT01005576

Other Study ID Numbers ^ICMJE

TCRN-NMD 0901

Has Data Monitoring Committee

Yes

Responsible Party

Washington University School of Medicine

Study Sponsor ^ICMJE

Washington University School of Medicine

Collaborators ^ICMJE

New England Research Institutes
Pediatric Blood and Marrow Transplant Consortium

Investigators ^ICMJE

Principal Investigator:

Shalini Shenoy, MD

Washington University Medical Center

Information Provided By

Washington University School of Medicine

Verification Date

January 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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