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Completed

NCT Number: NCT01005576

Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia

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.

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Key information

Conditions

Severe Thalassemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Age range

1 year–16 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Regents of University of California- UCLA, Los Angeles, California, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan

Drug

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

Primary outcomes

  1. Primary Objective: Event-free Survival at 1 Year.

    Time frame: 1 year

Secondary outcomes

  1. Development of Graft Versus Host Disease (GVHD)

    Time frame: 2 years

  2. Median Time to ANC Engraftment

    Time frame: 100 days

  3. Median Time to Platelet Engraftment

    Time frame: 100 days

  4. Incidence of Disease Recurrence

    Time frame: 2 years

Sponsors and collaborators

Lead sponsor

Washington University School of Medicine

Other

Collaborators

  • Carelon Research
  • Pediatric Blood and Marrow Transplant Consortium

Registry information

Official study title

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

Acronym: URTH

Important dates

Study start
2010
Primary completion
2014
Study completion
2014
First posted
Nov 2, 2009
Registry last updated
Dec 13, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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