CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 10 enrolled
Drug / intervention
Fludarabine monophosphate +4 moredrug
Likely dose
Busulfan 8 mg/kg total (Days -8 and -7); Fludarabine 175 mg/m² total (Days -6 through -3); Total lymphoid irradiation 500 cGy; Anti-thymocyte globulin (ATG) 15 mg/kg (Days -2 and -1); Stem cell transplant minimum 1.0 × 10⁹/kg nucleated cells/kg ideal body weight (goal 2.0 × 10⁹/kg)AI-extracted
Key inclusion· 3
  • ✓Age <35 years
  • ✓Diamond-Blackfan anemia with steroid resistance requiring >6 transfusions yearly despite steroid therapy, or evidence of developing aplasia/myelodysplasia
  • ✓Kostmann's neutropenia or Shwachman-Diamond syndrome with failed G-CSF therapy (unable to maintain ANC >750 cells/ml or recurrent infections despite G-CSF)
Key exclusion· 9
  • ✕Age >35 years
  • ✕Karnofsky score <70%
  • ✕Hepatic dysfunction: bilirubin >3.0, ALT >150, or active hepatitis
  • ✕Pulmonary function: FVC and FEV <70% or O2 saturation <94%

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

Search/NCT00176878
NCT00176878Phase 3Completed
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Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders

Masonic Cancer Center, University of Minnesota·interventional·Posted Sep 15, 2005·Updated Dec 28, 2017

In Brief

A Phase 3 clinical trial evaluating Stem cell transplant, Fludarabine monophosphate, and 3 other interventions for Diamond-Blackfan Anemia and 2 related conditions. Completed, enrolled 10 participants across 1 site.

Detailed Summary

The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDiamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
CountriesUnited States
Collaborators--

Timeline

Phase 3CompletedFinished
2000200120022003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedSep 15, 2005
Enrollment StartJun 1, 2000
Primary CompletionMar 1, 2009
TodayJul 2, 2026
Enrollment to primary: 8.8 yearsPosted 20.8 years ago

Interventions

Stem cell transplantprocedure

Stem cell transplant on Day 0 - healthy marrow from an unrelated individual. A minimum of 1.0 x 10\^9/kg nucleated cells/kg ideal body weight will be collected with a goal of 2.0 x 10\^9/kg.

Fludarabine monophosphatedrug

fludarabine 175 mg/m\^2 (total) on Days -6 through -3.

Total lymphoid irradiationprocedure

Dose 500 cGy radiation therapy to specific areas of the body

Busulfandrug

Busulfan 8 mg/kg (total) on Days - 8 and -7 (orally or through the catheter),

anti-thymocyte globulinbiological

anti-thymocyte globulin (ATG) 15 mg/kg on days -2 and -1 via catheter