Stem Cell Transplant for Hemoglobinopathy

Status Completed

Clinical Trial 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.

Clinical Trial Description

Prior to transplantation, subjects will receive either:

Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI)

Or

Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte colony-stimulating factor (GSCF)

These drugs (and the radiation) are being given to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.

After stem cell transplantation, subjects will be given cyclosporine-A and mycophenolate (MMF)/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00176852
Study type	Interventional
Source	Masonic Cancer Center, University of Minnesota
Contact
Status	Completed
Phase	Phase 2/Phase 3
Start date	June 2002
Completion date	January 2020

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02227472` - Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Recruiting	`NCT06301893` - Uganda Sickle Surveillance Study (US-3)
Recruiting	`NCT04398628` - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed	`NCT02522104` - Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH)	Phase 4
Recruiting	`NCT04688411` - An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease	N/A
Terminated	`NCT03615924` - Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease	Phase 3
Not yet recruiting	`NCT06300723` - Clinical Study of BRL-101 in Severe SCD	N/A
Recruiting	`NCT03937817` - Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Completed	`NCT04134299` - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease	N/A
Completed	`NCT04917783` - Health Literacy - Neurocognitive Screening in Pediatric SCD	N/A
Completed	`NCT02580565` - Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
Recruiting	`NCT04754711` - Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition	N/A
Completed	`NCT04388241` - Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD	N/A
Recruiting	`NCT05431088` - A Phase 2/3 Study in Adult and Pediatric Participants With SCD	Phase 2/Phase 3
Completed	`NCT01158794` - Genes Influencing Iron Overload State
Recruiting	`NCT03027258` - Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome	N/A
Withdrawn	`NCT02960503` - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease	Phase 1/Phase 2
Completed	`NCT02620488` - A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease	N/A
Withdrawn	`NCT02630394` - A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease	Phase 1
Not yet recruiting	`NCT02525107` - Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid Supplements	Phase 3