Clinical Trials Logo

Clinical Trial Summary

The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.


Clinical Trial Description

CD3/CD19 depletion of mismatched donor grafts in the setting of reduced intensity, immune-ablative conditioning for patients with sickle cell disease and other transfusion-dependent anemias should sufficiently achieve engraftment while decreasing the incidence of treatment-related toxicities and achieving an acceptable incidence of graft versus host disease. Utilizing mismatched unrelated volunteer donors and haploidentical related donors will increase the number of patients able to undergo hematopoietic stem cell transplant (HSCT) for these diseases. Additionally, the institutional availability of virus-specific, donor-derived cytotoxic T lymphocytes should address complicated viral infections refractory to standard anti-viral therapy. The purpose is to: - To provide alternate donor transplantation from cryopreserved stem cell grafts that are fully characterized for safety and potency to patients with severe sickle cell disease, beta-thalassemia major, or Diamond-Blackfan anemia who do not have matched sibling donor, matched unrelated donor or cord blood donor options. - To utilize a reduced-intensity conditioning regimen to achieve minimal treatment-related morbidity and mortality while attaining sustained donor engraftment and donor chimerism >20% in order to rescue disease phenotype, specifically in SCD patients. - To utilize ex-vivo T-cell depletion methods to prevent graft-versus-host disease in the setting of mismatched donor transplantation. - To utilize additional donor cell products to ensure sufficient immune reconstitution in the immediate post-transplant period, to improve mixed chimerism or provide non-specific anti-viral activity in patients with virus reactivation in the post-transplant period. - To utilize calcineurin inhibitor-free regimen in an effort to minimize/prevent central nervous system toxicity ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03653338
Study type Interventional
Source University of Pittsburgh
Contact Paul Szabolcs, MD
Phone 412-692-6225
Email paul.szabolcs@chp.edu
Status Recruiting
Phase Phase 1/Phase 2
Start date August 2, 2018
Completion date August 1, 2026

See also
  Status Clinical Trial Phase
Completed NCT04134299 - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease N/A
Completed NCT04581356 - Voxelotor Sickle Cell Exercise Study Phase 4
Completed NCT02712346 - The Role of Endothelin-1 in Sickle Cell Disease Phase 1
Completed NCT01976416 - Novel Use Of Hydroxyurea in an African Region With Malaria Phase 3
Withdrawn NCT02162225 - Study of Beet Juice for Patients With Sickle Cell Anemia Phase 2
Completed NCT01137721 - State Of The Art Functional Imaging In Sickle Cell Disease
Withdrawn NCT00937144 - Endothelial Function in Patients With Sickle Cell Anemia Before and After Sildenafil Phase 4
Terminated NCT01350232 - Treatment of Sickle Cell Anemia With Stem Cell Transplant N/A
Completed NCT00512564 - Clinical and Laboratory Assessment of Iron Overload in Sickle Cell Anemia and Sickle Cell Thalassemia N/A
Completed NCT00512226 - Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia N/A
Completed NCT00004143 - Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes Phase 2
Completed NCT00004412 - Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers Phase 2
Withdrawn NCT01925001 - Phase 2 Study of MP4CO to Treat Vaso-occlusive Sickle Crisis Phase 2
Completed NCT01848691 - Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis N/A
Completed NCT01783990 - Pediatric Hydroxyurea Phase III Clinical Trial (BABY HUG) Follow-up Observational Study II Protocol
Completed NCT01000155 - Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease Phase 2
Completed NCT00874172 - Effectiveness of New Analgesic Strategy Compared to the Usal Antalgic Strategy N/A
Completed NCT00236093 - Extension Study of ACTIQ Treatment for Children and Adolescents With Breakthrough Pain Phase 2
Completed NCT00399074 - Sulfadoxine- Pyrimethamine Versus Weekly Chloroquine for Malaria Prevention in Children With Sickle Cell Anemia Phase 3
Completed NCT00004492 - Phase I/II Randomized Study of Hydroxyurea With or Without Clotrimazole in Patients With Sickle Cell Anemia Phase 1/Phase 2