- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004787
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
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.
Study Overview
Status
Intervention / Treatment
Detailed Description
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.
Study Type
Enrollment
Phase
- Phase 2
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Collaborators
Investigators
- Study Chair: David A. Williams, James Whitcomb Riley Hospital for Children
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Metabolic Diseases
- Skin Diseases
- Kidney Diseases
- Urologic Diseases
- Disease
- Congenital Abnormalities
- Bone Marrow Diseases
- Hematologic Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Skin Diseases, Genetic
- DNA Repair-Deficiency Disorders
- Lipid Metabolism Disorders
- Blood Platelet Disorders
- Pancreatic Diseases
- Skin Abnormalities
- Anemia, Hypoplastic, Congenital
- Anemia, Aplastic
- Congenital Bone Marrow Failure Syndromes
- Bone Marrow Failure Disorders
- Renal Tubular Transport, Inborn Errors
- Lipomatosis
- Exocrine Pancreatic Insufficiency
- Syndrome
- Anemia
- Fanconi Syndrome
- Fanconi Anemia
- Thrombocytopenia
- Dyskeratosis Congenita
- Shwachman-Diamond Syndrome
- Physiological Effects of Drugs
- Immunologic Factors
- Adjuvants, Immunologic
- Lenograstim
Other Study ID Numbers
- 199/11877
- UTMB-416
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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