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Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies (SCALLOP)
This study is currently recruiting participants.
Verified by Baylor College of Medicine, December 2007
Sponsored by: Baylor College of Medicine
Information provided by: Baylor College of Medicine
ClinicalTrials.gov Identifier: NCT00578344
  Purpose

This is a nonrandomized study looking at organ function improvement after Bone Marrow Transplant (BMT) in Sickle Cell disease(SCD) patients. Specifically, to determine whether organ dysfunction (brain, heart, lung, kidney, liver, spleen, etc.) secondary to these severe hemoglobinopathies can be improved or reversed following allogeneic BMT.


Condition Intervention
Sickle Cell Disease
HEMOGLOBIN SS
HEMOGLOBIN SC
Hemoglobin Sb0/+
Procedure: ALLOGENEIC BMT/SCT Infusion
Drug: Busulfan
Biological: Campath 1H
Drug: Cyclophosphamide and Mesna

Genetics Home Reference related topics: beta thalassemia sickle cell disease
MedlinePlus related topics: Anemia Bone Marrow Transplantation Sickle Cell Anemia
Drug Information available for: Mesna Cyclophosphamide Alemtuzumab Campath Busulfan
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study
Official Title: Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia

Further study details as provided by Baylor College of Medicine:

Primary Outcome Measures:
  • Evaluate recovery of organ function in patients with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors and if they can be improved or reversed. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Evaluate the use of PET scan examination in assessing metabolic function of organs in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+ after undergoing allogeneic SCT/BMT from HLA genotype identical donors. [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Evaluate response to immunization after BMT in patients with SCD, hemoglobin SC, or hemoglobin Sb0/+. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: July 2005
Estimated Study Completion Date: July 2013
Estimated Primary Completion Date: July 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Procedure: ALLOGENEIC BMT/SCT Infusion
    on day 0.
    Drug: Busulfan
    starting day -9 / Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg
    Biological: Campath 1H
    Days -5 through day -2 Campath 1H dosed as per institutional guidelines
    Drug: Cyclophosphamide and Mesna
    Days -5 through day -2 Cyclophosphamide 50mg/kg+MESNA
Detailed Description:

Patients will receive a BMT as described below:

day -9 Busulfan 4.0 mg/kg/day IV divided into four doses daily for four days; total dose = 16 mg/kg

day -5 through day -2 Campath 1H dosed per institutional guidelines

day -5 through day -2 CTX 50mg/kg +MESNA

day 0 stem cell infusion

  Eligibility

Ages Eligible for Study:   up to 40 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with a related HLA genotype identical donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ and at least one of the following conditions:

    • Previous central nervous system vaso-occlusive episode with or without residual neurologic findings, or has an abnormal transcranial doppler exam without neurologic findings, or abnormal MRI/MRA of the brain with or without neurologic findings
    • Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy
    • Recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy
    • Severe anemia which prevents acceptable quality of life and necessitates chronic transfusion therapy
    • Any of the above symptoms in which the patient is not undergoing chronic transfusion therapy
    • The patient is undergoing chronic transfusion therapy for symptoms other than those listed and which significantly interferes with normal life activities
    • Failed hydroxyurea therapy
    • Indication of pulmonary hypertension on 2 separate echocardiogram examinations
    • Patients who plan to return to resource poor areas/countries.
  2. Between the ages of birth and 40 years.
  3. Women of childbearing potential must have a negative pregnancy test.

Exclusion Criteria:

  1. Patient with biopsy proven chronic active hepatitis or fibrosis with portal bridging.
  2. Patient with SCD chronic lung disease > stage 3 (see Appendix 1). 3. Patient with severe renal dysfunction defined as creatinine clearance < 40 ml/min/1.73M2.

4. Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25% or NYHA class III or IV.

5. Patient with HIV infection.

6. Patient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.

7. Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.

8 Pregnant/lactating women and those unwilling to use acceptable contraception will be excluded.

9. Patient or patient's guardian who have not signed an informed consent.

NOTE: Patients who would be excluded from the protocol strictly for laboratory abnormalities can be included at the investigator's discretion after approval by the CAGT Protocol Review Committee and the FDA reviewer.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00578344

Contacts
Contact: Kathryn Suet Wa Leung 832-822-4200 kleung@bcm.edu
Contact: Kathryn Suet Wa Leung, MD 832-822-4200 kleung@bcm.tmc.edu

Locations
United States, Texas
Texas Children's Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Kathryn Suet Wa Leung, MD     832-822-4200     kleung@bcm.tmc.edu    
Principal Investigator: Kathryn Suet Wa Leung, MD            
Methodist Hospital Recruiting
Houston, Texas, United States, 77030
Contact: George Carrum, MD     713-441-1450     gcarrum@bcm.edu    
Contact: Kathryn Suet Wa Leung, MD     832-822-4200     kleung@bcm.tmc.edu    
Principal Investigator: Kathryn Suet Wa Leung, MD            
Sponsors and Collaborators
Baylor College of Medicine
Investigators
Principal Investigator: Kathryn Suet Wa Leung, MD Baylor College of Medicine/Texas Children's Hospital
  More Information

Responsible Party: Baylor College of Medicine/Texas Children's Hospital ( Kathryn Suet Wa Leung, MD )
Study ID Numbers: 16447, SCALLOP
Study First Received: December 19, 2007
Last Updated: December 26, 2007
ClinicalTrials.gov Identifier: NCT00578344  
Health Authority: United States: Institutional Review Board

Keywords provided by Baylor College of Medicine:
Sickle Cell Disease
SCD
Hemoglobin SS
Hemoglobin SC
Hemoglobin Sb0/+
HLA genotype
Severe anemia
Transfusion therapy

Study placed in the following topic categories:
Hemoglobin SC disease
Hematologic Diseases
Hemoglobin SC Disease
Anemia
Anemia, Hemolytic
Cyclophosphamide
Thalassemia
Sickle cell anemia
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Busulfan
Alemtuzumab
Hemoglobinopathies
Hemoglobinopathy
Mesna
Anemia, Sickle Cell

Additional relevant MeSH terms:
Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Therapeutic Uses
Physiological Effects of Drugs
Myeloablative Agonists
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Alkylating Agents
Immunosuppressive Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009