Induction of Stable Chimerism for Sickle Cell Anemia

NCT00029393 | Completed Phase 2

• 2 other identifiers: 142U01HL068091
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

To investigate a modified hematopoeitic cell transplantation (HCT) procedure for sickle cell disease that significantly reduces the toxicity of HCT, yet retains its therapeutic benefit.

Conditions

Blood Disease; Hematopoietic Stem Cell Transplantation; Anemia, Sickle Cell

Interventions

Hematopoietic Stem Cell Transplantation PROCEDURE

Immunosuppression DRUG

Eligibility Criteria

• Age: Up to 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

No eligibility criteria

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI): lead

Related Publications (4)

• Walters MC, Patience M, Leisenring W, Rogers ZR, Aquino VM, Buchanan GR, Roberts IA, Yeager AM, Hsu L, Adamkiewicz T, Kurtzberg J, Vichinsky E, Storer B, Storb R, Sullivan KM; Multicenter Investigation of Bone Marrow Transplantation for Sickle Cell Disease. Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia. Biol Blood Marrow Transplant. 2001;7(12):665-73. doi: 10.1053/bbmt.2001.v7.pm11787529.

  PMID: 11787529 BACKGROUND

• Atkins RC, Walters MC. Haematopoietic cell transplantation in the treatment of sickle cell disease. Expert Opin Biol Ther. 2003 Dec;3(8):1215-24. doi: 10.1517/14712598.3.8.1215.

  PMID: 14640947 BACKGROUND

• Iannone R, Casella JF, Fuchs EJ, Chen AR, Jones RJ, Woolfrey A, Amylon M, Sullivan KM, Storb RF, Walters MC. Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant. 2003 Aug;9(8):519-28. doi: 10.1016/s1083-8791(03)00192-7.

  PMID: 12931121 BACKGROUND

• Horan JT, Liesveld JL, Fenton P, Blumberg N, Walters MC. Hematopoietic stem cell transplantation for multiply transfused patients with sickle cell disease and thalassemia after low-dose total body irradiation, fludarabine, and rabbit anti-thymocyte globulin. Bone Marrow Transplant. 2005 Jan;35(2):171-7. doi: 10.1038/sj.bmt.1704745.

  PMID: 15531901 BACKGROUND

MeSH Terms

Conditions

Hematologic Diseases; Anemia, Sickle Cell

Interventions

Hematopoietic Stem Cell Transplantation; Immunosuppression Therapy

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Therapeutics; Transplantation; Surgical Procedures, Operative; Immunotherapy; Immunomodulation; Immunologic Techniques; Investigative Techniques

Study Officials

• Mark Walters

  UCSF Benioff Children's Hospital Oakland

Study Design

• Study Type: interventional
• Phase: phase 2
• Masking: NONE
• Purpose: TREATMENT
• Sponsor Type: NIH

Study Record Dates

• First Submitted: January 10, 2002
• First Posted: January 11, 2002
• Study Start: August 1, 2001
• Primary Completion: July 1, 2007
• Study Completion: July 1, 2007
• Last Updated: July 29, 2016

Record last verified: 2008-01