Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia
Public ClinicalTrials.gov record NCT00011505. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Investigation of G-CSF-Induced Stem Cell Mobilization Potential in Patients With Diamond-Blackfan Anemia
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study will provide information needed to develop more effective treatments for patients with Diamond-Blackfan anemia (DBA). Current treatments include steroids, such as prednisone, and blood transfusions. These treatments have potential long-term risk and side effects, including osteoporosis and impaired growth from steroids or iron overload from transfusions. In addition, as patients reach adulthood, they can develop acute leukemia or bone marrow failure. The only cure for DBA is bone marrow transplant, a procedure that itself carries serious risks and is an option for only about 25 percent of patients. DBA is caused by a mutation (error) in a gene that codes for producing red blood cells from stem cells (blood-forming cells produced by the bone marrow). In 5 to 10 years, gene transfer therapy may prove to be an effective treatment for DBA. Before this treatment can be considered, however, more information is needed about DBA patients and how their stem cells function. This study will examine: 1) whether stem cells of patients with DBA respond to G-CSF the same way those of healthy people do. (G-CSF is a drug that causes stem cells to move from the bone marrow to the blood stream, where they can be collected more easily and in larger numbers by a procedure called leukapheresis, described below. If G-CSF does not work well in DBA patients, other collection strategies will have to be explored); and 2) whether the genetic error in DBA can be corrected by gene transfer into patients' stem cells. Patients with Diamond-Blackfan anemia 4 years of age and older who weigh at least 27 pounds and who are dependent on red blood cell transfusions may be eligible for this study. Candidates will have a medical history taken and a physical examination and will be seen by the Clinical Center's Department of Medicine Transfusion for leukapheresis evaluation. They will have a bone marrow aspiration and biopsy to confirm the diagnosis of DBA. For these tests, the hip area is anesthetized and a needle is used to draw bone marrow from the hipbone. If needed, the procedure will be done under sedation. Patients will be given G-CSF by injection under the skin for up to 6 days. Blood and stem cell counts will be measured from a teaspoon of blood drawn each morning. On the morning of the fifth dose, the patient will undergo leukapheresis for collection of stem cells. For this procedure, a large catheter (with a diameter no larger than that of a straw) is placed in an arm vein to allow blood to flow into a cell separator machine. Most children and some adults do not have veins large and strong enough for this tubing, so a large intravenous line called a "central line" is placed into a large vein in the neck or groin. This is done under sedation and with a local anesthetic. While the patient lies on a bed or recliner, whole blood is collected through a catheter in one arm or the central line, the stem cells are separated out by spinning, then the red cells, platelets and plasma are returned through a second catheter in the other arm or a second opening in the central line. The procedure takes about 3 to 5 hours, during which the patient can watch television or videos and have family members at the bedside for company. When the procedure is completed, the patient's participation in the protocol ends. Some of the stem cells collected by leukapheresis will be used for research and some will be frozen and stored for possible future transplantation into the patient, if required.
Study identification
- NCT ID
- NCT00011505
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 15 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 31, 2001
- Primary completion
- Not listed
- Completion
- Mar 31, 2006
- Last update posted
- Mar 3, 2008
2001 – 2006
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| National Heart, Lung and Blood Institute (NHLBI) | Bethesda | Maryland | 20892 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00011505, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 3, 2008 · Synced Sep 12, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT00011505 live on ClinicalTrials.gov.