CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 2 enrolled
Drug / intervention
Campath, Chemo and/or TBI Allo SCTdrug
Likely dose
Not stated in record
Key inclusion· 9
  • ✓Sickle cell anemia with severe complications despite hydroxyurea: ≥2 acute chest syndrome episodes since age 13, pulmonary hypertension (TRJ velocity >2.5 m/s), ≥2 painful crises/year requiring care, or history of cerebrovascular accident
  • ✓Thalassemia major with cardiac or hepatic sequelae: hepatic (≥50% liver enlargement or doubled bilirubin/AST/ALT/alkaline phosphatase) or cardiac (LV dysfunction on MUGA/echo)
  • ✓Severe aplastic anemia: cytopenia with ≥2 of 3: ANC <500/μL, platelets <20,000/μL, reticulocyte count <50,000/μL
  • ✓PNH with life-threatening complications: history of life-threatening thrombosis, cytopenia, transfusion dependence, or recurrent debilitating hemolytic crisis
Key exclusion· 3
  • ✕Pregnant or lactating women
  • ✕Major medical or psychiatric illness compromising protocol compliance
  • ✕Known history of allergy to murine protein

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

Search/NCT00004143
NCT00004143Phase 2Completed
CompareCT.gov

Allogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure Syndromes

David Rizzieri, MD·interventional·Posted Jan 27, 2003·Updated Dec 5, 2014

In Brief

A Phase 2 clinical trial evaluating Campath, Chemo and/or TBI Allo SCT for Sickle Cell Anemia and 3 related conditions. Completed, enrolled 2 participants across 2 sites.

Detailed Summary

RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood. Unfortunately, the conventional approach to allogeneic stem cell transplantation is a risky procedure. For some non-cancerous conditions, the risks of this procedure outweigh the potential benefits. This protocol is designed to test a new approach to allogeneic stem cell transplantation. It is hoped that this approach will be better suited for patients with non-cancerous blood and bone marrow disorders.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
19992000200120022003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedJan 27, 2003
Enrollment StartSep 1, 1999
Primary CompletionMay 1, 2008
Study CompletionJun 1, 2009
TodayJul 2, 2026
Enrollment to primary: 8.7 yearsPosted 23.4 years ago

Interventions

Campath, Chemo and/or TBI Allo SCTdrug

Allogeneic PBSC/marrow will be collected/harvested from the donor after granulocyte colony-stimulating factor (G-CSF) priming. The allogeneic PBSCs will be infused as per current institutional practice.