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Disease Category: Bone Density

Trial Information

Etanercept (Enbrel) in Children with Fanconi Anemia and Early Bone Marrow Failure, A Pilot Study

The Division of Hematology / Oncology and the Fanconi Anemia Comprehensive Care Center at Cincinnati Children's Hospital Medical Center would like to announce the opening of a new clinical trial for Fanconi anemia (FA) patients that show evidence of early bone marrow failure.

What is the purpose of this study?
The purpose of this research study is to evaluate the safety of the drug Etanercept (Enbrel) to determine if this drug can help in the treatment of early bone marrow failure in patients with Fanconi anemia.

Who can participate?
Patients must be at least four years of age and be diagnosed with Fanconi Anemia (FA). Evidence of early bone marrow failure must be present, as defined by two separate blood counts drawn one month apart which show at least one of the following: a platelet count of less than 100,000; a hemoglobin of less than 9, an absolute neutrophil count (ANC) of less than 1000. A negative TB (tuberculosis) skin test is required for study entry. The patient or caregiver must have the ability to administer injections given subcutaneously (just under the skin).

Why is this study being done?
Patients with FA are treated with blood products (transfusions), injections to stimulate white blood cell production, and/or androgen therapy once they reach advanced stages of bone marrow failure. Although these therapies lead to temporary improvement in the blood counts, they are associated with potential serious side effects. Currently, the only known potential cure for Fanconi anemia is a stem cell transplant, which is usually done at the late stages of bone marrow failure and is again associated with significant toxicity.

Studies show that patients with FA are very sensitive to and produce unusually high levels of a protein called tumor necrosis factor alpha (TNF-a) that causes bone marrow cells to die. We will study whether a drug called Etanercept that reduces levels of TNF-a will delay or prevent the progressive bone marrow failure associated with FA.

What is involved?
If accepted for enrollment in this study, a screening evaluation including a medical history, physical exam, blood tests, chest x-ray, and bone marrow aspirate and biopsy is required. Patients will receive weekly injections of the drug Etanercept for 6 months. A complete blood count is required weekly. A physical exam and bone marrow biopsy are repeated again at 3 months while on-study, at the end of the study, and every year for 2 years. Blood tests to check liver and kidney function are required at 3 months while on-study and at the end of the study. Health status and blood counts are monitored for an additional 6 months after receiving the study drug.

Some of the study visits will require coming to Cincinnati Children's. They are the screening evaluation, the three month, six month, and yearly clinic visits.

What are the benefits?
There may not be a direct medical benefit for you if you participate in this study. There is a chance that blood counts may improve without the need for other therapies.

What are the risks?
Some of the commonly known side effects from receiving Etanercept include redness, pain, and/or swelling at injection site, allergic reactions, and risk of serious infections. Patients enrolled on the study will be monitored very closely for these potential side effects.

Will I be paid for participating in this study?
No. All research costs and expenses related to this study, including the required screening blood tests and pre-study chest X-ray, and additional blood tests and bone marrow exams above those needed for routine clinical care during the study will be paid from research funds. The drug, Etanercept, will be provided at no cost.

Who should I contact for more information?

Robin Mueller, RN, Nurse Coordinator
Cincinnati Children's Hospital Medical Center/Cincinnati Children's Research Foundation
Fanconi Anemia Comprehensive Care Center
3333 Burnet Avenue, MLC 11013
Cincinnati, OH 45229-3039
Phone: 513-636-3218
EMail: robin.mueller@cchmc.org

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