Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

This study has been completed.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

Sponsor:	National Center for Research Resources (NCRR)
Collaborator:	James Whitcomb Riley Hospital for Children
Information provided by:	Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:	NCT00004787

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 II

Study Type:	Interventional
Study Design:	Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: Shwachman-Diamond syndrome Help Me Understand Genetics

MedlinePlus related topics: Anemia

Drug Information available for: Filgrastim Lenograstim Granulocyte colony-stimulating factor

U.S. FDA Resources

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

Estimated Enrollment:	20
Study Start Date:	December 1994

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.

Eligibility

Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004787

Sponsors and Collaborators

National Center for Research Resources (NCRR)

James Whitcomb Riley Hospital for Children

Investigators

Study Chair:

David A. Williams

James Whitcomb Riley Hospital for Children

More Information

Publications:

Rackoff WR, Orazi A, Robinson CA, Cooper RJ, Alter BP, Freedman MH, Harris RE, Williams DA. Prolonged administration of granulocyte colony-stimulating factor (filgrastim) to patients with Fanconi anemia: a pilot study. Blood. 1996 Sep 1;88(5):1588-93.

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

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:

Metabolic Diseases
Immunologic Factors
Skin Diseases
Hematologic Diseases
Lenograstim
Fanconi Anemia
Blood Platelet Disorders
Physiological Effects of Drugs
DNA Repair-Deficiency Disorders
Adjuvants, Immunologic
Anemia

Skin Abnormalities
Pharmacologic Actions
Thrombocytopenia
Anemia, Hypoplastic, Congenital
Genetic Diseases, Inborn
Dyskeratosis Congenita
Genetic Diseases, X-Linked
Anemia, Aplastic
Congenital Abnormalities
Bone Marrow Diseases
Skin Diseases, Genetic

ClinicalTrials.gov processed this record on March 18, 2010