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

Thrombocytopenia Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004787
• Other study ID #: 199/11877
• Secondary ID: UTMB-416
• Status: Completed
• Phase: Phase 2
• First received: February 24, 2000
• Last updated: June 23, 2005
• Start date: December 1994

Study information

• Verified date: December 2001
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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 Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

• Bone Marrow Diseases
• Dyskeratosis Congenita
• Exocrine Pancreatic Insufficiency
• Fanconi Anemia
• Fanconi Syndrome
• Fanconi's Anemia
• Shwachman Syndrome
• Thrombocytopenia
• Thrombocytosis

Intervention

Drug:
• filgrastim

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
National Center for Research Resources (NCRR)	James Whitcomb Riley Hospital for Children

References & Publications (1)

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. — View Citation