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| Sponsor: | Children's Hospital Medical Center, Cincinnati |
|---|---|
| Information provided by: | Children's Hospital Medical Center, Cincinnati |
| ClinicalTrials.gov Identifier: | NCT00479115 |
Purpose
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: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Supportive Care |
| 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 2010 |
| Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
Show Detailed Description
Eligibility| 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:
Contacts and Locations| Contact: Robin Mueller, RN | 1 800 344 2462 ext 3218 | robin.mueller@cchmc.org |
| Contact: Stephanie Edwards, RN | 513 636 9292 | stephaniel.edwards@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 |
More Information
| Responsible Party: | Stella Davies, MD, Cincinnati Children's Hospital Medical Center |
| ClinicalTrials.gov Identifier: | NCT00479115 History of Changes |
| Other Study ID Numbers: | CCHMCEH004, R01 HL081499 |
| Study First Received: | May 23, 2007 |
| Last Updated: | January 13, 2010 |
| Health Authority: | United States: Food and Drug Administration |
|
Anemia Fanconi Anemia Fanconi Syndrome Hematologic Diseases Anemia, Hypoplastic, Congenital Anemia, Aplastic Bone Marrow Diseases Genetic Diseases, Inborn DNA Repair-Deficiency Disorders Metabolic Diseases Kidney Diseases |
Urologic Diseases Renal Tubular Transport, Inborn Errors Metabolism, Inborn Errors JM 3100 Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents Therapeutic Uses Pharmacologic Actions |