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

This study has been completed.
Sponsor:
Collaborator:
Amgen
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT00965666
First received: August 24, 2009
Last updated: March 7, 2012
Last verified: March 2012
  Purpose

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.


Condition Intervention
Fanconi Anemia
Drug: Etanercept

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Health Services Research
Official Title: Etanercept (Enbrel) in Children With Fanconi Anemia and Early Bone Marrow Failure: A Pilot Study

Resource links provided by NLM:


Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • To assess toxicity of Etanercept (Enbrel) in children with Fanconi Anemia (FA) and early marrow failure. [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
  • To assess efficacy of Etanercept (Enbrel) in improving hematopoiesis (i.e. peripheral counts) in patients with FA. [ Time Frame: 24 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Correlation of biological studies to measure the impact of Etanercept (Enbrel) on Tumor Necrosis Factor - alpha (TNF-alpha) production. [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]

Enrollment: 3
Study Start Date: October 2005
Study Completion Date: October 2010
Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open Label Drug: Etanercept
All qualified subjects receive 24 weekly subcutaneous injections of Etanercept.
Other Name: Enbrel

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   4 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients must have a diagnosis of FA proven by a DEB test conducted in the cytogenetics lab of Dr. Arleen Auerbach, Rockefeller University Hospital.
  2. Patients must have evidence of early marrow failure i.e. reduction in at least one cell line on two separate occasions at least one month apart e.g. platelet count of < 100,000 per cubic millimeter, hemoglobin < 9 gm/dl and/or absolute neutrophil count (ANC) of < 1000
  3. Negative pregnancy test (conducted via serum β-HCG screen) - done before the first dose of study drug in all women (except those surgically sterile, at least 5 years postmenopausal, or under the age of 10 years)
  4. Sexually active patients of childbearing potential must agree to use medically acceptable form of contraception during screening and throughout the study
  5. Patients or designees must have the ability to self-inject investigational product or have a care giver at home who can administer subcutaneous injections
  6. Patients or designees must be able and willing to give written informed consent and comply with the requirements of the study protocol and must authorize release and use of protected health information
  7. Patients must have a negative TB skin test at entry into the study

Exclusion Criteria:

  1. Patients < 4 yrs of age
  2. Patients with advanced marrow failure i.e. transfusion dependent, will not be eligible as we anticipate that stem cell depletion will already be advanced at this stage.
  3. Patients currently enrolled in another investigational device or drug trial(s) (defined as a drug not approved by the FDA), or who have received other investigational agent(s) within 28 days of baseline visit with the exception of CCHMC IRB protocol # 03-9-11, "Thyroid Hormone in Children with Fanconi Anemia"
  4. Patients on androgen therapy
  5. Patients who have received immunosuppressive agents within the last 3 months prior to enrollment
  6. Patients who have any grade 3 or 4 adverse event or laboratory toxicity other than in Blood or Bone Marrow (as per the NCI CTC criteria) at the time of the screening visit or at any time during the study, that in the opinion of the Investigator would preclude participation in the study
  7. Patients with active infections within 4 weeks before the screening/baseline visit
  8. Patients with untreated Lyme disease
  9. Patients with a recent or past history of fungal infection
  10. Patients who have history of TB or TB exposure, chronic hepatitis B or hepatitis C, SLE, history of multiple sclerosis, transverse myelitis, optic neuritis or epilepsy
  11. Patients with known hypersensitivity to Etanercept (Enbrel) or any of its components or who are known to have antibodies to Etanercept (Enbrel).
  12. Patients who have received hematopoietic growth factor for greater than 3 consecutive days in the 6 months before study enrollment (i.e., erythropoietin, filgrastim, neupogen, sargramostin) for clinical purposes to improve bone marrow function. Patients receiving hematopoietic growth factor for stem cell mobilization and collection only are not excluded from this study.
  13. Patients with an available matched sibling donor and clinically indicated need for bone marrow transplant
  14. Patients with renal failure requiring dialysis
  15. Patients with a total bilirubin >3 mg/dl and/or SGPT >200 at time of enrollment
  16. Patients who are pregnant or breastfeeding or are a female at risk of pregnancy and are unable to practice safe sex during the length of the study
  17. Patients who are HIV positive
  18. Patients with severe co-morbidities (diabetes mellitus requiring insulin, CHF of any severity, MI, CVA or TIA within 3 months of screening visit, unstable angina pectoris, uncontrolled hypertension, oxygen-dependent severe pulmonary disease, history of cancer within 5 years (other than resected cutaneous basal or squamous cell carcinoma or in situ cervical cancer)
  19. Patients who have any mycobacterial disease or known history of any other immuno-suppressing disease
  20. Patients with a history of recent alcohol or substance abuse (< 1 year)
  21. Patients with a history of non-compliance with other therapies
  22. Patients who have any condition judged by the patient's physician to cause this clinical trial to be detrimental to the patient
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00965666

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Amgen
Investigators
Principal Investigator: Stella M. Davies, MBBSPhd MRCP Children's Hospital Medical Center, Cincinnati
  More Information

No publications provided

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT00965666     History of Changes
Other Study ID Numbers: 05-07-24
Study First Received: August 24, 2009
Last Updated: March 7, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fanconi Anemia
Hemic and Lymphatic Diseases
Anti-Rheumatic Agents
Anemia, Hypoplastic, Congenital
DNA Repair-Deficiency Disorders
Congenital, Hereditary, and
Neonatal Diseases and Abnormalities

Additional relevant MeSH terms:
Anemia
Fanconi Anemia
Fanconi Syndrome
Anemia, Aplastic
Anemia, Hypoplastic, Congenital
Bone Marrow Diseases
DNA Repair-Deficiency Disorders
Genetic Diseases, Inborn
Hematologic Diseases
Kidney Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Renal Tubular Transport, Inborn Errors
Urologic Diseases
TNFR-Fc fusion protein
Analgesics
Analgesics, Non-Narcotic
Anti-Inflammatory Agents
Anti-Inflammatory Agents, Non-Steroidal
Antirheumatic Agents
Central Nervous System Agents
Gastrointestinal Agents
Immunologic Factors
Immunosuppressive Agents
Peripheral Nervous System Agents
Pharmacologic Actions
Physiological Effects of Drugs
Sensory System Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on October 23, 2014