Etanercept (Enbrel) for Juvenile Myelomonocytic Leukemia

NCT00509600 | Terminated Phase 2 Results DMC

• 1 other identifier: 2004-0215
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

Primary Objectives: 1.1 Estimate rate of response and define acute toxicity to etanercept used in an up-front phase II window in newly diagnosed or relapsed JMML. 1.2 Determine if response to Tumor Necrosis Factor (TNF) blockade correlates with genetic basis of Juvenile Myelomonocytic Leukemia (JMML) \[mutations in NF1, Ras, SHP2\] or levels of TNFa. 1.3 Determine if TNF blockade by etanercept results in inhibition of free levels of TNFa and other cytokines by ELISA and bioassay and improves blood counts. 1.4 Estimate the two year event free survival and overall survival in JMML patients following etanercept and allogeneic hematopoietic stem cell transplantation.

Conditions

Leukemia

Keywords

Juvenile Myelomonocytic Leukemia; JMML; Leukemia; Etanercept; Enbrel

Outcome Measures

Primary Outcomes (1)

• Patient Response

  Response is defined as a WBC \< 15,000 and platelets \> 75,000 at 12 weeks; toxicity is defined as a grade 3 or worse infection or non-hematologic toxicity within the first 4 weeks.

  12 weeks

Study Arms (1)

Etanercept

EXPERIMENTAL

0.8 mg/kg subcutaneously weekly for 90 days

Drug: Etanercept

Interventions

Etanercept DRUG

0.8 mg/kg Subcutaneously Once A Week for 90 Days

Also known as: Enbrel

Etanercept

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• All children greater than 6 months of age and less than 18 years of age with newly-diagnosed previously untreated or previously diagnosed JMML, which has reoccurred after treatment with chemotherapy, stem cell transplantation, and/or cis-retinoic acid.
• A diagnosis of JMML is confirmed only if the following criteria for JMML are met: a) ALL of the following: Absence of t(9;22) or BCR-ABL by PCR or FISH; Absolute monocyte count \>1000 (1 X 109/µL); \<20% bone marrow blasts; b) At least 2 of the following: Elevated Hb F hemoglobin; Myeloid precursors in peripheral blood; WBC \>10,000 (10 X 109/µL); GM-CSF hypersensitivity in methylcellulose culture of bone marrow progenitors cells.
• Adequate hepatic function (bilirubin equal or less than 2.0 mg/dl; ALT equal or less than 3x normal)
• Adequate renal function (serum creatinine equal or less than 2 x normal)
• Performance Status: Have a Karnofsky score \>50.
• Written, informed consent according to institution guidelines.

You may not qualify if:

• Pregnant or lactating.
• Receiving any other chemotherapy. Patients must have been off chemotherapy for at least 2 weeks and must have recovered from acute toxicity of all previous therapy prior to enrollment.
• Febrile neutropenia at study entry.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• Amgen: collaborator

Study Sites (1)

UT MD . Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

• UT MD Anderson Cancer Center website

MeSH Terms

Conditions

Leukemia; Leukemia, Myelomonocytic, Juvenile

Interventions

Etanercept

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Myeloid; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow 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

Limitations and Caveats

Early termination due to low accrual. Rare disease with less than 100 cases diagnosed per year.

Results Point of Contact

• Title: Robert J. Wells, MD / Professor
• Organization: UT MD Anderson Cancer Center

713-792-6620

Study Officials

• Robert J. Wells, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 27, 2007
• First Posted: July 31, 2007
• Study Start: September 1, 2004
• Primary Completion: April 1, 2009
• Study Completion: April 1, 2009
• Last Updated: August 7, 2012
• Results First Posted: December 2, 2010

Record last verified: 2012-08

Locations

US (1)