CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 22 enrolled
Drug / intervention
Busulfan, Fludarabine, ATG, TLI +4 moredrug
Likely dose
Busulfan 0.8 mg/kg/dose IV (Days -8 to -9 through -6/7); Fludarabine 35 mg/m2 IV (Days -6 through -2); ATG 30 mg/kg IV (Days -2 and -1); TBI/TLI 300 cGy; Cyclophosphamide 50 mg/kg IV (Days -5 through -2) or Campath-1H 0.2 mg/kg (Days -10 through -6) depending on regimenAI-extracted
Key inclusion· 13
  • ✓Sickle cell disease or thalassemia patients aged 0-50 years with an acceptable stem cell donor
  • ✓SCD patients with stroke, CNS hemorrhage, or neurologic event lasting >24 hours, or abnormal cerebral MRI/arteriogram/angiography with impaired neuropsychological testing
  • ✓SCD patients with acute chest syndrome and history of recurrent hospitalizations or exchange transfusions
  • ✓SCD patients with recurrent vaso-occlusive pain (≥3 episodes/year for ≥3 years) or recurrent priapism
Key exclusion· 8
  • ✕Karnofsky or Lansky performance score <70
  • ✕Acute hepatitis or moderate/severe portal fibrosis or cirrhosis on biopsy
  • ✕Stage III-IV lung disease
  • ✕GFR <30% predicted

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00176852
NCT00176852Phase 3Completed
CompareCT.gov

Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism

Masonic Cancer Center, University of Minnesota·interventional·Posted Sep 15, 2005·Updated Feb 27, 2020

In Brief

A Phase 3 clinical trial evaluating Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, and 3 other interventions for Sickle Cell Disease and 4 related conditions. Completed, enrolled 22 participants across 1 site.

Detailed Summary

This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease. Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
CountriesUnited States
CollaboratorsNational Marrow Donor Program

Timeline

Phase 3CompletedFinished
20022003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedSep 15, 2005
Enrollment StartJun 1, 2002
Primary CompletionMar 1, 2014
Study CompletionJan 1, 2020
TodayJul 2, 2026
Enrollment to primary: 11.8 yearsPosted 20.8 years ago

Interventions

Busulfan, Fludarabine, ATG, TLIdrug

Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy

Busulfan, Cyclophosphamide, ATG, GCSFdrug

Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.

Campath, Fludarabine, Cyclophosphamidedrug

Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.

Total Body Irradiationradiation

300 cGY Day -1

Stem cell infusionprocedure

Given Day 0