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.

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Key information

Conditions

Shwachman Syndrome Anemia Anemia, Aplastic Anemia, Hypoplastic, Congenital Blood Platelet Disorders Bone Marrow Diseases Bone Marrow Failure Disorders Congenital Abnormalities Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cytopenia DNA Repair-Deficiency Disorders Digestive System Diseases Disease Attributes Dyskeratosis Congenita Exocrine Pancreatic Insufficiency Fanconi Anemia Fanconi's Anemia Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipomatosis Metabolic Diseases Nutritional and Metabolic Diseases Pancreatic Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Shwachman-Diamond Syndrome Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases Thrombocytopenia

Age range

0 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Filgrastim

Drug

Sponsors and collaborators

Lead sponsor

National Center for Research Resources (NCRR)

Nih

Collaborators

James Whitcomb Riley Hospital for Children

Registry information

Important dates

Study start: 1994
First posted: Feb 25, 2000
Registry last updated: Jun 24, 2005

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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