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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
Est. completion date
Est. primary completion date
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


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

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