Clinical Trial: Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Etanercept (Enbrel) in Children With Fanconi Anemia and Early Bone Marrow Failure: A Pilot Study

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

Detailed Summary:

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 bone marrow failure in 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-α) that causes bone marrow cells to die. We will study whether a drug called Etanercept that reduces levels of TNF-α will delay or prevent the progressive bone marrow failure associated with FA. Etanercept has been successfully used in children with arthritis.

Primary Objectives:

  1. To assess toxicity of Etanercept (Enbrel) in children with Fanconi Anemia (FA) and early marrow failure.
  2. To assess efficacy of Etanercept (Enbrel) in improving hematopoiesis (i.e. peripheral counts) in patients with FA.

Secondary Objectives:

1) Correlation of biological studies to measure the impact of Etanercept (Enbrel) on Tumor Necrosis Factor - α (TNF-α) production.

Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leuke
Sponsor: Children's Hospital Medical Center, Cincinnati

Current Primary Outcome:

  • To assess toxicity of Etanercept (Enbrel) in children with Fanconi Anemia (FA) and early marrow failure. [ Time Frame: 24 months ]
  • To assess efficacy of Etanercept (Enbrel) in improving hematopoiesis (i.e. peripheral counts) in patients with FA. [ Time Frame: 24 months ]


Original Primary Outcome: Same as current

Current Secondary Outcome: Correlation of biological studies to measure the impact of Etanercept (Enbrel) on Tumor Necrosis Factor - alpha (TNF-alpha) production. [ Time Frame: 24 months ]

Original Secondary Outcome: Same as current

Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: August 24, 2009
Date Started: October 2005
Date Completion:
Last Updated: March 7, 2012
Last Verified: March 2012