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

This study has been completed.
Sponsor:
Collaborator:
James Whitcomb Riley Hospital for Children
Information provided by:
Office of Rare Diseases (ORD)
ClinicalTrials.gov Identifier:
NCT00004787
First received: February 24, 2000
Last updated: June 23, 2005
Last verified: December 2001
  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 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment

Resource links provided by NLM:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

Sponsors and Collaborators
James Whitcomb Riley Hospital for Children
Investigators
Study Chair: David A. Williams James Whitcomb Riley Hospital for Children
  More Information

Publications:
ClinicalTrials.gov Identifier: NCT00004787     History of Changes
Other Study ID Numbers: 199/11877, UTMB-416
Study First Received: February 24, 2000
Last Updated: June 23, 2005
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:
Dyskeratosis Congenita
Anemia
Bone Marrow Diseases
Exocrine Pancreatic Insufficiency
Fanconi Anemia
Fanconi Syndrome
Lipomatosis
Syndrome
Thrombocytopenia
Anemia, Aplastic
Anemia, Hypoplastic, Congenital
Blood Platelet Disorders
Congenital Abnormalities
Digestive System Diseases
Disease
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hematologic Diseases
Kidney Diseases
Lipid Metabolism Disorders
Metabolic Diseases
Metabolism, Inborn Errors
Pancreatic Diseases
Pathologic Processes
Renal Tubular Transport, Inborn Errors
Skin Abnormalities
Skin Diseases
Skin Diseases, Genetic
Urologic Diseases

ClinicalTrials.gov processed this record on October 21, 2014