Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsors and Collaborators: |
National Center for Research Resources (NCRR) James Whitcomb Riley Hospital for Children |
---|---|
Information provided by: | Office of Rare Diseases (ORD) |
ClinicalTrials.gov Identifier: | NCT00004787 |
OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure syndromes.
II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily G-CSF.
III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before and after G-CSF using flow cytometry and immunohistochemistry.
Condition | Intervention | Phase |
---|---|---|
Shwachman Syndrome Fanconi's Anemia Dyskeratosis Congenita Thrombocytopenia |
Drug: filgrastim |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment |
Estimated Enrollment: | 20 |
Study Start Date: | December 1994 |
PROTOCOL OUTLINE: Patients receive granulocyte colony-stimulating factor (G-CSF) subcutaneously every day for 8 weeks; nonresponders receive an increased dose for an additional 8 weeks. Patients who respond at week 8 or 16 are then tapered to a lower maintenance dose of G-CSF administered every other day through week 40. The dose is adjusted to maintain an absolute neutrophil count above 1500.
Patients are removed from study for failure to achieve a complete response by week 16, unacceptable nonhematologic toxicity, the identification of a clonal karyotype in marrow, or the onset of leukemia.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Inherited bone marrow failure syndrome, including:
--Prior/Concurrent Therapy--
--Patient Characteristics--
Study ID Numbers: | 199/11877, UTMB-416 |
Study First Received: | February 24, 2000 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00004787 History of Changes |
Health Authority: | United States: Federal Government |
Fanconi's anemia Shwachman syndrome aplastic anemia dermatologic disorders |
dyskeratosis congenita hematologic disorders rare disease thrombocytopenia |
Metabolic Diseases Skin Diseases Aplastic Anemia Hematologic Diseases Fanconi Anemia Blood Platelet Disorders Skin Abnormalities Anemia Rare Diseases Pancytopenia Inherited Bone Marrow Failure Syndromes Fanconi's Anemia |
Thrombocytopathy Thrombocytopenia Genetic Diseases, Inborn Dyskeratosis Congenita Genetic Diseases, X-Linked Anemia, Aplastic Congenital Abnormalities Bone Marrow Diseases Metabolic Disorder Skin Diseases, Genetic Shwachman-Diamond Syndrome |
Metabolic Diseases Disease Skin Diseases Hematologic Diseases Blood Platelet Disorders Fanconi Anemia DNA Repair-Deficiency Disorders Skin Abnormalities Anemia Pathologic Processes |
Thrombocytopenia Anemia, Hypoplastic, Congenital Genetic Diseases, Inborn Dyskeratosis Congenita Syndrome Genetic Diseases, X-Linked Anemia, Aplastic Congenital Abnormalities Bone Marrow Diseases Skin Diseases, Genetic |