Shwachman SyndromePhase 2NCT00004787
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
A Phase 2 study testing filgrastim in people with Shwachman Syndrome and Fanconi's Anemia, run by National Center for Research Resources (NCRR).
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. Read more about this study
- Who is it for?
- Ages 0 years and older
- Conditions the study is for
- Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita
- What is being tested?
- filgrastim
- How many people
- 20 participants
- Where
- No sites listed yet
- Study dates
- Dec 1994to not given
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
About this study
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.
Read the 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.
- Being tested
- filgrastim
- Keywords
- Fanconi's anemia, Shwachman syndrome, aplastic anemia, dermatologic disorders, dyskeratosis congenita, hematologic disorders, rare disease, thrombocytopenia
- Medical subject headings
- Shwachman-Diamond Syndrome, Fanconi Anemia, Dyskeratosis Congenita, Thrombocytopenia, Anemia, Aplastic, Skin Diseases, Hematologic Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
filgrastimDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2000 (estimated)
- Study startDec 1994
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Shwachman SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- National Center for Research Resources (NCRR)
- Sponsor type
- US National Institutes of Health
- Collaborators
- James Whitcomb Riley Hospital for Children
- Investigators
- David A. Williams, Study chair, James Whitcomb Riley Hospital for Children
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004787
- Sponsor's protocol ID
- 199/11877
- Other IDs
- UTMB-416
- Status last verified
- Dec 2001
- First submitted
- 24 Feb 2000
- Last updated
- 24 Jun 2005
- Results posted
- No
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.
PubMed 8781414