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Sickle Cell Research at The Georgia Institute of Technology, Emory University School of Medicine, Department of Pediatrics, and Department of Medicine, Division of Hematology Oncology

RED CELL ADHESION - To study the red cell - endothelial adhesion interactions under flow conditions. This project is defining the mechanism of sickle red cell adhesion and possible pharmacological interventions. The P.I is Tim Wick Ph.D. at the Georgia Institute of Technology

PSYCHO SOCIAL INTERVENTION - This project is about the impact of educational and coping skills to improve patient’s compliance and control over their disease. P.I is Nadine Kaslow Ph.D

Randomized Pilot Study of Cognitive-Behavioral Intervention for Children and Adolescents with Sickle Cell Disease

Summary Last Modified: 02/98
Protocol IDs: EUC-32591
Protocol Type: supportive care
Sponsorship: National Heart, Lung and Blood Institute
Status: Approved - Not yet active
Age Range: 8 to 17
PROJECTED ACCRUAL:
200 patients will be entered over 4 years; at least 30 of these patients will be entered in the intervention portion of the study.
OBJECTIVES:
I. Examine relationships between type of sickle cell disease, severity of physical symptoms, and psychosocial difficulties in African-American children with sickle cell disease (SCD).
II. Examine adaptive functioning in daily living skills and communication.
III. Examine relationships between illness-related difficulties, psychosocial adjustment variables, and demographic variables mediating these relationships.
IV. Examine how body satisfaction and sexual adjustment relate to the nature of illness, stage of pubertal development, and demographic variables.
V. Examine how these patients handle stress, how these patterns are associated with pain, and how socioecologic factors influence the coping process.
VI. Examine combinations of risk and resistance factors predicting functioning and the associated demographic variables.
VII. Assess the efficacy of cognitive-behavioral family intervention.
VIII. Examine the relative contribution of SCD nature, poverty stressors, impaired family relationships, and insufficient support networks.
PROTOCOL ENTRY CRITERIA:
African-American children aged 8 to 17 with sickle cell disease, including:
Hemoglobin SS
Hemoglobin SC
Hemoglobin S-thalassemia
Primary caretaker required
No psychotic disorder using Diagnostic and Statistical Manual of Mental
Disorders IV
No moderate, severe, or profound mental retardation
PROTOCOL OUTLINE:
This is a randomized study.
All children undergo family assessment; those who meet high-risk referral criteria are randomly assigned to 1 of 2 interventions. One group receives interactive, age-appropriate intervention with a focus on disease education, family and peer relationships, cognitive strategies, and goal setting.
A control group receives standard treatment as usual with social service, pediatric consultation-liaison, and psychiatric outpatient clinic referrals as needed.
Patients are treated for 8 weeks with a follow-up at 6 months.
WARNING
The purpose of most clinical trials listed in this database is to test new treatments, or new
methods of diagnosing, screening for or preventing disease. Because all potentially harmful side
effects are not known before a trial is conducted, dose and schedule modifications may be
required for participants if they develop side effects from the treatment or test. The therapy or
test described in this clinical trial is intended for use by clinicians in carefully structured settings,
and may not prove to be more effective than standard treatment. A responsible investigator
associated with this clinical trial should be consulted before using this protocol.
PARTICIPATING ORGANIZATIONS/INVESTIGATORS
Nadine Kaslow, Chair, Ph: 404-616-4757
Emory University School of Medicine

NEURO PSYCHOLOGY STUDY - This project is to study the neuro-cognitive aspects of sickle cell disease with extensive psychological studies and MRI imaging. P.I. is Ron Brown, Ph.D.

BONE MARROW TRANSPLANT - This project is to identify potential bone marrow transplant candidates within the sickle cell population and study the safety and efficacy of transplants in severe disease. The P.I. is Andy Yeager, M.D.

Phase II Pilot Study of HLA-Identical Allogeneic Bone Marrow
Transplantation Following Busulfan/Cyclophosphamide/Antithymocyte
Globulin for Children with Sickle Cell Disease
Summary Last Modified: 02/98
Protocol IDs: FHCRC-610.0
Protocol Type: treatment
Sponsorship: National Heart, Lung and Blood Institute
Status: Approved - Not yet active
Age Range: 15 and under
PROJECTED ACCRUAL:
30-60 patients will be entered over 5 years. The study will close if an unacceptable incidence of 100-day survival or graft failure occurs.
OBJECTIVES:
I. Define the role and risk-benefit ratio of HLA-identical allogeneic bone marrow transplantation in children with symptomatic organ impairment from sickle cell disease.
II. Evaluate the efficacy of sickle marrow ablation and donor marrow engraftment following a conditioning regimen of busulfan/cyclophosphamide/antithymocyte globulin.
III. Evaluate the reversibility of sickle cell vasculopathy and organ damage
following bone marrow transplantation in these patients.
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Sickle cell disease, i.e.:
Homozygous hemoglobin SS
Sickle beta-thalassemia
At least 1 of the following sickle cell complications: Stroke, hemorrhage, or neurologic defect lasting more than 24 hours Abnormal cerebral magnetic resonance imaging (MRI), cerebral arteriogram or MRI angiographic study, and impaired neuropsychologic testing Stage I/II sickle cell lung disease Moderate to severe proteinuria or glomerular filtration rate 30%-50% of
predicted normal for age
Bilateral proliferative retinopathy with major visual impairment in at least 1 eye
Acute chest syndrome with history of recurrent hospitalizations or prior exchange transfusion
Osteonecrosis of multiple joints
At least 2 episodes of debilitating pain per year for several years or priapism
Chronic transfusion therapy with development of at least 2 isoantibodies
No stage III/IV sickle cell lung disease
Donor criteria:
HLA-identical family member
Cells nonreactive in mixed lymphocyte culture
No sickle cell disease (sickle cell trait eligible)
No increased anesthetic risk due to pre-existing illness
Not HIV positive
No other medical, psychologic, or physiologic contraindication to donation
--Prior/Concurrent Therapy--
Not specified

--Patient Characteristics--
Age: 15 and under
Performance status: Karnofsky 70%-100%
Hematopoietic: Not specified
Hepatic: No active hepatitis No moderate to severe portal fibrosis
Renal: Glomerular filtration rate at least 30% of predicted for age
Other: No severe residual functional neurologic impairment Hemiplegia alone eligible
No demonstrated noncompliance with prior medical care
Not HIV positive
PROTOCOL OUTLINE:
Patients are treated with 4 doses of busulfan, 4 doses of cyclophosphamide, and 3 doses of antithymocyte globulin, followed by infusion of allogeneic bone marrow. Graft-versus-host disease prophylaxis is provided on protocol FHCRC-267.
WARNING
The purpose of most clinical trials listed in this database is to test new treatments, or new
methods of diagnosing, screening for or preventing disease. Because all potentially harmful side
effects are not known before a trial is conducted, dose and schedule modifications may be
required for participants if they develop side effects from the treatment or test. The therapy or
test described in this clinical trial is intended for use by clinicians in carefully structured settings,
and may not prove to be more effective than standard treatment. A responsible investigator
associated with this clinical trial should be consulted before using this protocol.
PARTICIPATING ORGANIZATIONS/INVESTIGATORS
Keith Sullivan, Chair, Ph: 206-667-4416
Fred Hutchinson Cancer Research Center

N3 FATTY ACIDS (Fish oil) AND COAGULATION - This project is to determine the role of clotting factors and platelets in the pathology of sickle cell disease. N-3 fatty acids will be administered in a double blind manner to determine the efficacy of this treatment to prevent pain episodes and sickle complications. The P.I. is James Eckman, M.D.

Phase II/III Study of Dietary n-3 Fatty Acids for the Prevention of Thrombotic Pain Episodes in Sickle Cell Anemia
Summary Last Modified: 02/97
Protocol IDs: EUSM-56491
Protocol Type: supportive care
Sponsorship: National Heart, Lung and Blood Institute
Status: Active
Age Range: 18 and over
PROJECTED ACCRUAL:
20-25 patients will be entered in the phase II study and 50-75 patients in the phase III study. A study duration of 5 years is anticipated.
OBJECTIVES:
I. Evaluate in a phase II trial the extent of and temporal relationships among the following: n-3 fatty acids (n-3FAs) and thrombus-promoting characteristics of sickle erythrocytes; thrombin-generating properties and origins of circulating microparticles; flow cytometric expression of activation epitopes for platelets and monocytes; the blood markers for thrombus formation in vivo, including beta-thromboglobulin, platelet factor 4, fibrinopeptide A, thrombin:antithrombin complex, and D-dimer; endothelial-derived plasma gelatinase and endothelial-derived collagenase; and the frequency of treatment and course of pain episodes in patients with sickle cell anemia.
II. Assess whether dietary n-3FAs at 0.25 g/kg per day modify these relationships.
III. Characterize the pathogenetic thrombus-promoting mechanisms underlying sickle pain crises and the capacity of dietary n-3FAs to interrupt these processes.
IV. Determine the parameters that will be useful surrogate measures of treatment effect.
V. Compare in a phase III trial the following: erythrocyte n-3FA membrane properties; concentration, origin, and reactivity of circulating microparticles; flow cytometric expression of platelet and leukocyte activation epitopes; blood markers of thrombosis; plasma levels of endothelial-injury enzymes; and the frequency and severity of pain episodes in symptomatic sickle cell patients randomly assigned to dietary n-3FAs or placebo.
VI. Investigate the relationship between the prevention of thrombus formation and clinical episodes of pain.
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Sickle cell anemia with pain crises requiring parenteral analgesia 3 to 24 times in the year prior to entry
--Prior/Concurrent Therapy--Not specified
--Patient Characteristics--
Age:18 and over
Performance status: Not specified
Life expectancy: At least 2 years
Hematopoietic: Platelets at least 100,000
Hepatic: Bleeding time no greater than 15 minutes
No local or systemic hemostatic defect
No significant hepatic disease
Renal: No significant renal disease
Other:No history of gastrointestinal intolerance
No other medical contraindication to protocol therapy
Adequate contraception required of fertile women
PROTOCOL OUTLINE:
This protocol involves 2 studies: patients are nonrandomly assigned to n-3 fatty acids (n-3FAs) in the phase II study; additional patients are randomly assigned to n-3FAs versus olive oil placebo in the phase III study.
In phase II, patients are treated with daily oral n-3FAs for 1 year.
In phase III, additional patients are treated with daily oral n-3FAs or placebo for 1 year.
Concurrent conventional sickle cell management is allowed; oral anticoagulation is prohibited.
WARNING
The purpose of most clinical trials listed in this database is to test new treatments, or new
methods of diagnosing, screening for or preventing disease. Because all potentially harmful side
effects are not known before a trial is conducted, dose and schedule modifications may be
required for participants if they develop side effects from the treatment or test. The therapy or
test described in this clinical trial is intended for use by clinicians in carefully structured settings,
and may not prove to be more effective than standard treatment. A responsible investigator
associated with this clinical trial should be consulted before using this protocol.
PARTICIPATING ORGANIZATIONS/INVESTIGATORS
James R. Eckman, Chair, Ph: 404-616-5982
Emory University School of Medicine

RENAL STUDY - This project is to define the progression and pathophysiology of sickle cell disease on the kidneys. The study will define the best methods of early detection and test possible interventions. The P.I. is Antonio Guasch, M.D.

CHEST SYNDROME - This study is to identify the pathophysiology of chest syndrome and to identify possible therapeutic interventions. The P.I. is Clinton Lawrence, M.D.

DNA HAPLOTYPING - This project is to identify the beta haplotype and any alpha gene deletions in the entire sickle cell population. These results will be correlated with other projects to see is haplotype has any prognostic value. The P.I. is Skip Elsas, M.D.

MULTIMEDIA EDUCATION - This project is to develop new methods of professional and patient education using the latest in computer and video technology. The contact is Allan Platt, PA-C.

STROKE PREVENTION - STOP STUDY - This project is to identify sickle cell children at risk for stokes by using cranial Doppler flow studies. Children with high risk findings will undergo MRI and MRA evaluations and be randomized to a chronic transfusion program. The role of chronic transfusions in stroke prevention is under evaluation. Eldrida Carter is the Research Coordinator

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Copyright 1997 Sickle Cell Information Center
Last modified: March 28, 1998