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

NCT00965666 | Completed Early Phase 1 DMC

• 1 other identifier: 05-07-24
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Fanconi Anemia

Keywords

Fanconi Anemia; Hemic and Lymphatic Diseases; Anti-Rheumatic Agents; Anemia, Hypoplastic, Congenital; DNA Repair-Deficiency Disorders; Congenital, Hereditary, and; Neonatal Diseases and Abnormalities

Outcome Measures

Primary Outcomes (2)

• To assess toxicity of Etanercept (Enbrel) in children with Fanconi Anemia (FA) and early marrow failure.

  24 months

• To assess efficacy of Etanercept (Enbrel) in improving hematopoiesis (i.e. peripheral counts) in patients with FA.

  24 months

Secondary Outcomes (1)

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

  24 months

Study Arms (1)

Open Label

EXPERIMENTAL

Drug: Etanercept

Interventions

Etanercept DRUG

All qualified subjects receive 24 weekly subcutaneous injections of Etanercept.

Also known as: Enbrel

Open Label

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have a diagnosis of FA proven by a DEB test conducted in the cytogenetics lab of Dr. Arleen Auerbach, Rockefeller University Hospital.
• 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
• 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)
• Sexually active patients of childbearing potential must agree to use medically acceptable form of contraception during screening and throughout the study
• Patients or designees must have the ability to self-inject investigational product or have a care giver at home who can administer subcutaneous injections
• 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
• Patients must have a negative TB skin test at entry into the study

You may not qualify if:

• Patients \< 4 yrs of age
• 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.
• 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"
• Patients on androgen therapy
• Patients who have received immunosuppressive agents within the last 3 months prior to enrollment
• 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
• Patients with active infections within 4 weeks before the screening/baseline visit
• Patients with untreated Lyme disease
• Patients with a recent or past history of fungal infection
• 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
• Patients with known hypersensitivity to Etanercept (Enbrel) or any of its components or who are known to have antibodies to Etanercept (Enbrel).
• 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.
• Patients with an available matched sibling donor and clinically indicated need for bone marrow transplant
• Patients with renal failure requiring dialysis
• Patients with a total bilirubin \>3 mg/dl and/or SGPT \>200 at time of enrollment

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Amgen: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

MeSH Terms

Conditions

Fanconi Anemia; Hemic and Lymphatic Diseases; Anemia, Hypoplastic, Congenital; DNA Repair-Deficiency Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Interventions

Etanercept

Condition Hierarchy (Ancestors)

Anemia, Aplastic; Anemia; Hematologic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin Fc Fragments; Immunoglobulin Fragments; Peptide Fragments; Peptides; Amino Acids, Peptides, and Proteins; Immunoglobulin Constant Regions; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Serum Globulins; Globulins; Receptors, Tumor Necrosis Factor; Receptors, Cytokine; Receptors, Immunologic; Receptors, Cell Surface; Membrane Proteins

Study Officials

• Stella M. Davies, MBBSPhd MRCP

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2009
• First Posted: August 25, 2009
• Study Start: October 1, 2005
• Primary Completion: October 1, 2010
• Study Completion: October 1, 2010
• Last Updated: September 14, 2020

Record last verified: 2012-03

Locations

US (1)