Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure S… (NCT00004787) | Clinical Trial Compass
CompletedPhase 2
Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes
20 participantsStarted 1994-12
Plain-language 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.
Who can participate
Age range
0 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this trial is completed, has the published data on using G-CSF for my specific condition — whether that's Shwachman Syndrome, Fanconi's Anemia, Dyskeratosis Congenita, or thrombocytopenia — shown any meaningful improvement in blood counts or bone marrow function that would be relevant to my case?
2Because this was a Phase II pilot study, the primary goal was likely to test safety and feasibility rather than prove effectiveness — so how confident can we be in the results, and what would it mean for my treatment decisions today?
3G-CSF stimulates the bone marrow to produce more white blood cells, but given that Fanconi's Anemia and some of these other conditions carry an elevated cancer risk, is there any concern that G-CSF could increase my risk of leukemia or other blood cancers?
4Are there currently open trials or approved treatments for my specific inherited bone marrow failure syndrome that build on what this completed study found, and would those be a better option to explore right now?
5How does the evidence from this trial compare to standard of care approaches like bone marrow transplant or other supportive treatments, and should we consider those alternatives before looking at therapies based on this research?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Trial details
NCT IDNCT00004787
SponsorNational Center for Research Resources (NCRR)