This trial is active, not recruiting.

Condition severe thalassemia
Treatment transplant conditioning regimen of alemtuzumab, fludarabine, and melphalan
Phase phase 2
Sponsor Washington University School of Medicine
Collaborator New England Research Institutes
Start date January 2010
End date April 2014
Trial size 20 participants
Trial identifier NCT01005576, TCRN-NMD 0901


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose treatment
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
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

Primary Outcomes

Primary objective: To determine event-free survival at 1 year.
time frame: 1 year

Secondary Outcomes

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

Eligibility Criteria

Male or female participants from 1 year up to 16 years old.

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

Additional Information

Official title A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial)
Principal investigator Shalini Shenoy, MD
Trial information was received from ClinicalTrials.gov and was last updated in April 2014.
Information provided to ClinicalTrials.gov by Washington University School of Medicine.