SALT: Alternative Donor Bone Marrow and Cord Blood Transplantation for High Risk Sickle Cell Disease

NCT00257543 | Terminated Early Phase 1 DMC

• 1 other identifier: 876-2003
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

We hope to gain valuable information about the safety, success of engraftment, and rates of complications using alternate donor transplantation for children with severe SCD. Crucial information will be also collected about late effects from alternate donor BMT sickle cell, providing valuable information to clinicians and families making decisions among interventions for children with severe sickle cell disease. If successful, alternate donor transplantation in this setting could pave the way to offering curative treatment to many more patients with severe SCD.

Conditions

Sickle Cell Disease

Keywords

sickle cell disease; bone marrow transplant

Outcome Measures

Primary Outcomes (1)

• To determine the frequency of donor engraftment after alternate donor transplantation in children with severe sickle cell disease.

  1 year after completion of study accrual

Study Arms (1)

single arm study

EXPERIMENTAL

this is a single arm study

Procedure: Alternative donor bone marrow and cord blood

Interventions

Alternative donor bone marrow and cord blood PROCEDURE

bone marrow transplant - alternative donors for bone marrow and cord blood transplants

single arm study

Eligibility Criteria

• Age: Up to 16 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• Hemoglobin SS, hemoglobin SC, or hemoglobin S0 thalassemia
• Donor available: Partially (5/6) HLA-matched relative (PMRD), matched (6/6) unrelated marrow donor or umbilical cord (5/6 or 6/6) of appropriate size (see 6.3.2) , using high-resolution HLA typing. Donor must not be homozygous for HgbS and must meet standard donor eligibility criteria of the Blood and Marrow Transplant Program.
• Severe SCD, defined by one of the following (modified Walters criteria):
• oPrevious (6 months prior) central nervous system event lasting longer than 24 hours, plus objective imaging evidence of CNS vasculopathy, with or without residual neurologic findings oFrequent (3 per year for 2 years) painful vaso-occlusive episodes (defined as episode lasting 4 hours and requiring hospitalization or outpatient treatment with parenteral narcotics) oRecurrent (3 in lifetime) acute chest syndrome events which have necessitated exchange transfusion or chronic transfusion therapy. Must have failed a good-faith trial of hydroxyurea (failure defined as a reduction of less than 50% in the incidence of vaso-occlusive events over a period of at least 18 months) or have demonstrated an inability to take the drug due to side effects.
• oAny combination of 3 acute chest syndrome episodes and vaso-occlusive pain episodes (defined as above) yearly for 3 years. Must have failed a good-faith trial of hydroxyurea (failure defined as a reduction of less than 50% in the incidence of vaso-occlusive events over a period of at least 18 months) or have demonstrated an inability to take the drug due to side effects.
• oStage I or II sickle lung disease oRed-cell alloimmunization (2 antibodies) on chronic transfusion therapy

You may not qualify if:

• oSuitable HLA-identical relative donor is available oBiopsy proven chronic active hepatitis, portal fibrosis, or cirrhosis, or serologic evidence of active hepatitis.
• oSCD chronic lung disease stage III (see Appendix) oSevere renal dysfunction defined as \<50% of predicted normal GFR for age. oSevere cardiac dysfunction defined as shortening fraction \< 25%. oSevere residual neurologic impairment other than hemiplegia alone, defined as full-scale IQ 70, quadriplegia or paraplegia, inability to ambulate, inability to communicate without assistive device, or any impairment resulting in decline of Lansky performance score to \<70%.
• oCNS event occurring within 6 months prior to transplant oKarnofsky or Lansky functional performance score \< 70% (see Appendix) oConfirmed HIV seropositivity. oPatient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.
• oPatient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.
• oHistory of lack of compliance with medical care that would jeopardize transplant course.

Sponsors & Collaborators

• Emory University: lead

Study Sites (1)

Children's Healthcare of Altanta

Atlanta, Georgia, 30322, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

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

Study Officials

• Ann Haight, M.D.

  Children's Healthcare of Atlanta/Emory University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: November 22, 2005
• First Posted: November 23, 2005
• Study Start: January 1, 2006
• Primary Completion: August 1, 2012
• Study Completion: August 1, 2012
• Last Updated: September 17, 2013

Record last verified: 2013-09

Locations

US (1)