Home
Search
Study Topics
Glossary
|
|
|
|
|
Sponsored by: |
Children's Hospital Medical Center, Cincinnati |
---|---|
Information provided by: | Children's Hospital Medical Center, Cincinnati |
ClinicalTrials.gov Identifier: | NCT00479115 |
The purpose of this research study is to determine whether an experimental drug called AMD3100 used in combination with another medication called G-CSF is safe and can help to increase the amount of blood stem cells (called CD34+ stem cells) found in the peripheral blood of patients with Fanconi anemia.
While AMD3100 has been used successfully in adult volunteers and cancer patients, it has not been used in children or patients with Fanconi anemia and in only a few children with cancer.
Fanconi anemia is a rare genetic disease. Most Fanconi anemia patients eventually develop bone marrow failure, a condition in which the bone marrow no longer produces red blood cells (to carry oxygen), white blood cells (to fight infection), and platelets (to help blood clot). The only successful treatment for patients with Fanconi anemia with bone marrow failure is bone marrow transplantation. However, this treatment has many risks and is not available to all patients with Fanconi anemia.
CD34+ cells include stem cells found in the bone marrow or peripheral blood which are capable of making the red blood cells, white blood cells, and platelets. CD34+ stem cells can be collected from bone marrow or peripheral blood and purified using an experimental device called the CliniMACS.
However, most Fanconi anemia patients do not have enough CD34+ stem cells in their bone marrow or peripheral blood to be collected using standard methods that work well in children and adults who don't have Fanconi anemia.
Condition | Intervention | Phase |
---|---|---|
Fanconi Anemia |
Drug: AMD3100 Device: AmCell CliniMACs |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Supportive Care, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study |
Estimated Enrollment: | 15 |
Study Start Date: | May 2007 |
Estimated Study Completion Date: | December 2008 |
Ages Eligible for Study: | 1 Year to 30 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Age:
First cohort - > 7 Second cohort - > 3 Third cohort - >1.
Exclusion Criteria:
Contact: Robin Mueller, RN | 1 800 344 2462 ext 3218 | robin.mueller@cchmc.org |
Contact: Susan Radtke, RN | 513 636 4961 | susan.radtke@cchmc.org |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | Recruiting |
Cincinnati, Ohio, United States, 45229 | |
Principal Investigator: Stella Davies, MD |
Principal Investigator: | Stella Davies, MD | Children's Hospital Medical Center, Cincinnati |
Study ID Numbers: | CCHMCEH004, R01 HL081499 |
Study First Received: | May 23, 2007 |
Last Updated: | January 2, 2008 |
ClinicalTrials.gov Identifier: | NCT00479115 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Metabolic Diseases Anti-HIV Agents Hematologic Diseases Aplastic Anemia JM 3100 Fanconi Anemia Anemia |
Antiviral Agents Fanconi's Anemia Genetic Diseases, Inborn Anti-Retroviral Agents Anemia, Aplastic Bone Marrow Diseases Metabolic Disorder |
Anti-Infective Agents Metabolic Diseases Anti-HIV Agents Hematologic Diseases JM 3100 Fanconi Anemia DNA Repair-Deficiency Disorders Anemia |
Antiviral Agents Pharmacologic Actions Anemia, Hypoplastic, Congenital Genetic Diseases, Inborn Anti-Retroviral Agents Therapeutic Uses Anemia, Aplastic Bone Marrow Diseases |