NCT00186810

Brief Summary

This protocol studied the effect of administration of a myeloablative pretransplant preparative regimen followed by an infusion of donor stem cells in children with severe sickle cell disease. The donor graft consisted of bone marrow or cord blood derived from a genetically matched sibling. The primary aim of the study was to evaluate how well the donated cells migrated to the bone marrow and begin producing healthy red blood cells, white blood cells and platelets (engrafted), how well the recipients immune system recovered, and assess any regimen related toxicities including a potentially life-threatening transplant related complication called graft-versus-host-disease or GVHD.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 1992

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 1992

Completed
12.8 years until next milestone

First Submitted

Initial submission to the registry

September 9, 2005

Completed
7 days until next milestone

First Posted

Study publicly available on registry

September 16, 2005

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2006

Completed
1.7 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2007

Completed
Last Updated

May 29, 2009

Status Verified

May 1, 2009

Enrollment Period

13.2 years

First QC Date

September 9, 2005

Last Update Submit

May 28, 2009

Conditions

Sickle Cell Disease

Keywords

AnemiaSickle Cell

Outcome Measures

Primary Outcomes (1)

  • To evaluate engraftment, GVHD, hematopoietic and immune reconstitution, and regimen-related mortality and morbidity in patients with severe sickle cell disease undergoing transplant using either HLA matched sibling bone marrow or cord blood grafts.

    March 2007

Study Arms (1)

1

OTHER
Drug: Busulfan, Cyclophosphamide, Horse ATGProcedure: Allogeneic stem cell transplant

Interventions

Busulfan, Cyclophosphamide, Horse ATGDRUG

Transplant recipients received a myeloablative conditioning regimen of cyclophosphamide, Anti-Thymocyte Globulin (horse), and Busulfan. Cyclosporine and methotrexate were administered for GVHD prophylaxis.

1
Allogeneic stem cell transplantPROCEDURE

Allogeneic stem cell transplant Matched sibling donor transplant Cord blood transplant

1

Eligibility Criteria

AgeUp to 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of severe' disease is denoted by one of the following:
  • Previous central nervous system vaso-occlusive episode with or without residual neurologic findings or
  • Frequent painful vaso-occlusive episodes with significant interference with normal life activities and which necessitates chronic transfusion therapy or
  • Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.

You may not qualify if:

  • Patient with SCD chronic lung disease greater than or equal to stage 3
  • Patient with severe renal dysfunction defined as creatinine clearance \< 40 ml/min/1.73m2.
  • Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction \< 25%.
  • Patient with HIV infection.
  • Pregnant or lactating.
  • Patient with unspecified chronic toxicity that in the opinion of the Principal Investigator is serious enough to detrimentally affect the patient's capacity to tolerate SCT.
  • Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Publications (1)

  • Dallas MH, Triplett B, Shook DR, Hartford C, Srinivasan A, Laver J, Ware R, Leung W. Long-term outcome and evaluation of organ function in pediatric patients undergoing haploidentical and matched related hematopoietic cell transplantation for sickle cell disease. Biol Blood Marrow Transplant. 2013 May;19(5):820-30. doi: 10.1016/j.bbmt.2013.02.010. Epub 2013 Feb 14.

    PMID: 23416852DERIVED

Related Links

MeSH Terms

Conditions

Anemia, Sickle CellAnemia

Interventions

BusulfanCyclophosphamideAntilymphocyte Serum

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedPhosphoramidesOrganophosphorus CompoundsImmune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex Mixtures

Study Officials

  • Gregory Hale, MD

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

September 9, 2005

First Posted

September 16, 2005

Study Start

December 1, 1992

Primary Completion

February 1, 2006

Study Completion

October 1, 2007

Last Updated

May 29, 2009

Record last verified: 2009-05

Locations

US(1)