Trial record 15 of 22 for:    "Pure red cell aplasia"

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

This study has been completed.
Sponsor:
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00005893
First received: June 2, 2000
Last updated: June 23, 2005
Last verified: July 2004
  Purpose

OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders.

II. Determine the quality of life, absence of adverse effects (e.g., graft versus host disease and B cell lymphoproliferative disease), and completeness of recovery of their underlying condition in these patients with this treatment regimen.


Condition Intervention
Neutropenia
Sickle Cell Anemia
Thalassemia Major
Red-Cell Aplasia, Pure
Drug: anti-thymocyte globulin
Drug: busulfan
Drug: cyclophosphamide
Procedure: Bone marrow transplantation

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Office of Rare Diseases (ORD):

Study Start Date: June 2000
Detailed Description:

PROTOCOL OUTLINE: Patients receive IV or oral busulfan every 6 hours on days -9 to -6; cyclophosphamide IV on days -5 to -2; anti-thymocyte globulin IV on days -4 to -2; and allogeneic bone marrow transplantation (BMT) on day 0.

Patients with class 3 thalassemia (liver edge greater than 2 cm below costal margin, a history of inconsistent chelation, and portal fibrosis) receive a less intensive conditioning regimen consisting of oral busulfan every 6 hours on days -7 to -4; anti-thymocyte globulin IV on days -5 to -1 and days 1-5; cyclophosphamide IV on days -3 to -2; and allogeneic BMT on day 0.

Patients are followed at day 28, and then at 3, 6, 12, and 24 months.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Severe hemoglobinopathy including, but not limited to the following:

Homozygous beta thalassemia Other beta chain mutation as demonstrated by hemoglobin electrophoresis Sickle cell anemia (age 16 to 50 years) Evidence of one or more the following prior complications: Stage I-II sickle cell lung disease Sickle cell nephropathy (moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal for age) Acute chest syndrome requiring blood transfusions More than 3 debilitating pain episodes per year for 3 years lasting more than 4 hours each Any combination of episodes of acute chest syndrome and painful episodes adding up to 3 episodes per year for 3 consecutive years Requirement for chronic transfusions with alloimmunization (more than 2 antibodies) Sickle cell anemia (age under 16 years) Evidence of one or more the following prior complications: Prior stroke or hemorrhage Any neurologic event lasting more than 24 hours Abnormal cerebral MRI and cerebral arteriogram MRI angiographic impaired neuropsychologic testing Stage I-II sickle cell lung disease Sickle cell nephropathy (moderate to severe proteinuria or glomerular filtration rate of 30-50% predicted normal for age) Significant visual impairment in at least one eye with bilateral proliferative retinopathy Acute chest syndrome with history of recurrent hospitalizations or exchange transfusions Osteonecrosis of multiple joints with destructive changes More than 3 debilitating pain episodes per year or priapism Requirement for chronic transfusions with alloimmunization

OR

Bone marrow failure syndrome unresponsive to therapy, including but not limited to the following: Congenital pure red cell aplasia (Diamond Blackfan anemia) Confirmed by bone marrow aspirate More than 6 transfusions per year despite steroid therapy Kostmann's neutropenia Confirmed by bone marrow aspirate Unable to maintain absolute neutrophil count greater than 750/mm3 or recurrent life threatening infections or more than 4 hospitalizations per year despite therapy with filgrastim (G-CSF) No myelodysplasia or aplastic anemia

Must have a related donor with at least a 5 out of 6 HLA antigen match

--Patient Characteristics--

Age: Sickle cell anemia patients 0 to 50; All other patients under 18

Performance status: Karnofsky 70-100%

Hepatic: Bilirubin no greater than 3.0 mg/dL with a direct fraction no greater than 2.0 mg/dL ALT no greater than 150 U/L No moderate or severe portal fibrosis No active hepatitis

Renal: Glomerular filtration rate at least 30% predicted (except with sickle cell anemia) No renal dysfunction

Cardiovascular: Left ejection fraction at least 50% No cardiac compromise

Other: No severe, stable neurologic impairment HIV negative

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00005893

Locations
United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Fairview University Medical Center
Investigators
Study Chair: Paul J. Orchard Fairview University Medical Center
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00005893     History of Changes
Other Study ID Numbers: 199/15101, UMN-MT-1994-18, UMN-MT-9418
Study First Received: June 2, 2000
Last Updated: June 23, 2005
Health Authority: Unspecified

Keywords provided by Office of Rare Diseases (ORD):
congenital pure red cell aplasia
pure red cell aplasia
chronic congenital neutropenia
chronic neutropenia
disease-related problem/condition
genetic diseases and dysmorphic syndromes
hematologic disorders
neutropenia
rare disease
sickle cell anemia
thalassemia major

Additional relevant MeSH terms:
Anemia, Sickle Cell
Beta-Thalassemia
Hematologic Diseases
Neutropenia
Red-Cell Aplasia, Pure
Thalassemia
Agranulocytosis
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hemoglobinopathies
Leukocyte Disorders
Leukopenia
Antilymphocyte Serum
Cyclophosphamide
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Antirheumatic Agents
Immunologic Factors
Immunosuppressive Agents
Molecular Mechanisms of Pharmacological Action
Myeloablative Agonists
Pharmacologic Actions
Physiological Effects of Drugs
Therapeutic Uses

ClinicalTrials.gov processed this record on October 29, 2014