Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
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Purpose
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 2 |
| Study Type: | Interventional |
| Study Design: | Primary Purpose: 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.
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Inherited bone marrow failure syndrome, including:
- Fanconi's anemia
- Dyskeratosis congenita
- Shwachman syndrome
- Amegakaryocytic thrombocytopenia
- Decreased megakaryocytes in infancy
- No thrombocytopenia with absent radius syndrome (TAR)
- No trisomy 13 or 18
- No clonal bone marrow karyotype
--Prior/Concurrent Therapy--
- At least 4 weeks since growth factors
- Concurrent therapy allowed if not altered for 30 days prior to entry through week 8
- No concurrent investigational drugs
--Patient Characteristics--
- Hematopoietic: ANC <1000
- No leukemia
- Other: No medical or psychiatric contraindication to protocol participation
- No pregnant or nursing women
Contacts and Locations More Information
Publications:
| ClinicalTrials.gov Identifier: | NCT00004787 History of Changes |
| Other Study ID Numbers: | 199/11877, UTMB-416 |
| Study First Received: | February 24, 2000 |
| Last Updated: | June 23, 2005 |
| Health Authority: | United States: Federal Government |
Keywords provided by Office of Rare Diseases (ORD):
|
Fanconi's anemia Shwachman syndrome aplastic anemia dermatologic disorders |
dyskeratosis congenita hematologic disorders rare disease thrombocytopenia |
Additional relevant MeSH terms:
|
Anemia Fanconi Anemia Fanconi Syndrome Thrombocytopenia Dyskeratosis Congenita Bone Marrow Diseases Lipomatosis Exocrine Pancreatic Insufficiency Hematologic Diseases Anemia, Hypoplastic, Congenital Anemia, Aplastic Genetic Diseases, Inborn DNA Repair-Deficiency Disorders Metabolic Diseases Kidney Diseases |
Urologic Diseases Renal Tubular Transport, Inborn Errors Metabolism, Inborn Errors Blood Platelet Disorders Skin Abnormalities Congenital Abnormalities Genetic Diseases, X-Linked Skin Diseases, Genetic Skin Diseases Lipid Metabolism Disorders Pancreatic Diseases Digestive System Diseases Lenograstim Adjuvants, Immunologic Immunologic Factors |
ClinicalTrials.gov processed this record on May 23, 2013