NCT00004787

Brief 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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 1994

Completed
5.2 years until next milestone

First Submitted

Initial submission to the registry

February 24, 2000

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 25, 2000

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2001

First QC Date

February 24, 2000

Last Update Submit

June 23, 2005

Conditions

Shwachman SyndromeFanconi's AnemiaDyskeratosis CongenitaThrombocytopenia

Keywords

Fanconi's anemiaShwachman syndromeaplastic anemiadermatologic disordersdyskeratosis congenitahematologic disordersrare diseasethrombocytopenia

Interventions

filgrastimDRUG

Eligibility Criteria

Age0 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Related 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.

    PMID: 8781414BACKGROUND

MeSH Terms

Conditions

Shwachman-Diamond SyndromeFanconi AnemiaDyskeratosis CongenitaThrombocytopeniaAnemia, AplasticSkin DiseasesHematologic DiseasesRare Diseases

Interventions

Filgrastim

Condition Hierarchy (Ancestors)

Exocrine Pancreatic InsufficiencyPancreatic DiseasesDigestive System DiseasesCongenital Bone Marrow Failure SyndromesBone Marrow Failure DisordersBone Marrow DiseasesHemic and Lymphatic DiseasesLipid Metabolism, Inborn ErrorsLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesLipomatosisAnemia, Hypoplastic, CongenitalAnemiaGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersSkin AbnormalitiesCongenital AbnormalitiesGenetic Diseases, X-LinkedSkin Diseases, GeneticSkin and Connective Tissue DiseasesBlood Platelet DisordersCytopeniaDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating FactorColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological Factors

Study Officials

  • David A. Williams

    James Whitcomb Riley Hospital for Children

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

February 24, 2000

First Posted

February 25, 2000

Study Start

December 1, 1994

Last Updated

June 24, 2005

Record last verified: 2001-12