Carilion Children's

Pediatric Oncology

Newly Diagnosed AML

A Phase III randomized trial for patients with de novo AML using Bortezomib and Sorafenib (IND#114480; NSC# 681239, NSC# 724772) for patients with high allelic ratio FLT3/ITD.
PROTOCOL ID
COG # AAML1031

Protocol Description

This study is for newly diagnosed patients with acute myeloid leukemia (AML). AML is a cancer of the bone marrow, the spongy tissue inside the bones of the body where blood cells are made.

Eligibility Criteria

• Patients must be less than 30 years of age at the time of study enrollment
• Be newly diagnosed with AML
• Agree to have bone marrow specimens sent to special COG labs prior to the start of treatment and at various times during treatment
• Patients with Down syndrome are not eligible

Primary Investigator: Mandy Atkinson, M.D.

Contact

Wendy McCarty, Clinical Research Coordinator
540-981-7376
wpmccarty@carilionclinic.org

Newly Diagnosed Ependymoma

A Phase III randomized trial of post-radiation chemotherapy in patients with newly diagnosed ependymoma ages 1 to 21 years.
PROTOCOL ID
COG # ACNS0831

Protocol Description

This study is for patients newly diagnosed with ependymoma. Ependymoma is a type of cancer that occurs in the brain. This slow-growing tumor begins in cells that line the fluid-filled spaces of the brain (called ventricles) or the fluid-filled space down the center of the spinal cord (the central canal). The current standard of treatment for newly diagnosed ependymoma includes surgery to remove as much of the tumor as possible, and radiation therapy.

This study wants to see if a short course of chemotherapy therapy can kill any remaining tumor cells for patients whose tumor was only partially removed by surgery. Treatment with chemotherapy after surgery is experimental. This study also wants to see if patients whose remaining tumor was killed by the short course of chemotherapy and/or removed by a second surgery and whose tumor was not in the upper part of the brain and does not have favorable-looking cells. See if treatment with chemotherapy after radiation therapy will work better than standard treatment to keep the tumor from coming back. Treatment with chemotherapy after radiation therapy is experimental.

Eligibility Criteria

• Patients must be greater than 12 months of age and less than 21 years of age at the time of study enrollment
• Patients must be newly diagnosed with intracranial ependymoma. Patients with classic ependymoma (WHO II) or anaplastic ependymoma (WHO III) are eligible, as are various subtypes described as clear cell, papillary, cellular or a combination of the above.
• Patients with evidence of metastatic disease by MRI or CSF cytology are NOT eligible

Primary Investigator: Mandy Atkinson, M.D.

Contact

Wendy McCarty, Clinical Research Coordinator
540-981-7376
wpmccarty@carilionclinic.org

Newly Diagnosed High-Risk B Precursor ALL

A Phase III randomized trial for newly diagnosed high-risk B-precursor acute lymphoblastic leukemia (ALL) testing clofarabine (IND # 73789, NSC # 606869) in the very high risk stratum.
PROTOCOL ID
COG # AALL1131

Protocol Description

This study is for newly diagnosed high-risk and very high-risk acute lymphoblastic leukemia (ALL) with or without Down Syndrome. Leukemia is cancer of blood cells. It develops in the bone marrow, which is the soft, spongy center of the bones that produces the three major blood cells.

Eligibility Criteria

Patients must be enrolled on AALL08B1 prior to enrollment on AALL1131.
White Blood Cell Count (WBC) Criteria

• Age 1-9.99 years: WBC ³ 50 000/_L
• Age 10-30.99 years: any WBC
• Age 1-30.99 years: any WBC with:
  • Testicular leukemia
  • CNS leukemia (CNS3)
  • Steroid pretreatment

Primary Investigator: Mandy Atkinson, M.D.

Contact

Wendy McCarty, Clinical Research Coordinator
540-981-7376
wpmccarty@carilionclinic.org

For additional information on Children's Oncology studies, please visit Children's Oncology Group.

Newly Diagnosed Standard Risk B Precursor ALL

Treatment for patients with newly diagnosed standard risk B-precursor acute lymphoblastic leukemia (ALL).
PROTOCOL ID
COG # AALL0932

Protocol Description

This study is for patients with newly diagnosed standard risk B-precursor acute lymphoblastic leukemia (ALL)

Eligibility Criteria

•    Patients must have newly diagnosed NCI standard risk B-precursor ALL
•    Patients must be older than 365 days and younger than 10 years of age
•    Patients must be enrolled on AALL08B1 prior to enrollment on AALL0932
•    Patients must have an initial white blood count of less than 50,000
•    Patients with Down Syndrome are eligible

Primary Investigator: Mandy Atkinson, M.D.

Contact

Wendy McCarty, Clinical Research Coordinator
540-981-7376
wpmccarty@carilionclinic.org

Treatment of Children with All Stages of Hepatoblastoma

PROTOCOL ID
COG # AHEP0731

Protocol Description

This study is for patients with hepatoblastoma. Hepatoblastoma is a type of cancer that occurs in the liver. Researchers now know that there are some features of hepatoblastoma that may help predict how children will respond to therapy. Factors that affect how well a child with hepatoblastoma will do include:

• Whether the tumor can be completely removed by surgery
• Whether the tumor or cancer cells have spread to other parts of the body (metastasized)
• How quickly a protein in the blood called alpha-fetoprotein (AFP) is decreased during treatment for hepatoblastoma; AFP is often very high in certain cancers, such as hepatoblastoma
• The appearance of tumor cells under the microscope

Researchers know that children need different treatments based on the factors associated with the tumor. These factors determine the risk of the tumor coming back. This study will group children as having a very low-risk, low-risk, intermediate-risk or high-risk disease. This study will try to learn more about the factors that lead to a good or a poor chance of recovery (prognosis) for children. We want to develop effective treatments that will increase the overall success rate and decrease the side effects of the therapy.

Eligibility Criteria

• Patients must be ≤ 21 years of age at the time of diagnosis
• Patients may have had surgical resection of some or all sites of hepatoblastoma prior to enrollment
• Patients must be newly diagnosed with histologically-proven hepatoblastoma except for patients too ill to undergo a biopsy safely

Primary Investigator: Mandy Atkinson, M.D.

Contact

Wendy McCarty, Clinical Research Coordinator
540-981-7376
wpmccarty@carilionclinic.org

Pediatric Gastroenterology

Eosinophilic Esophagitis (SHIRE 301)

Oral budesonide suspension (OBS) in adolescent and adult subjects (11-55 years of age, inclusive) with eosinophilic esophagitis: A Phase III randomized, double-blind, placebo-controlled study.

Protocol Description

To evaluate the safety and efficacy of OBS treatment administered twice daily for 12 weeks in adolescents.

Eligibility Criteria

Ages 11-17, with histologic evidence of EoE with a peak eosinophil count of >15/HPF, from two of three levels of the esophagus.

Primary Investigator: Michael Hart, M.D.

Contact

Christy Florow, Clinical Research Coordinator
540-985-9723
cmflorow@carilionclinic.org

Functional Constipation (LIN-MD-62)

A multi-center, randomized, double-blind, placebo-controlled, parallel-group, safety and efficacy study of a range of Linaclotide doses administered orally to children, ages 6-17 years, who fulfill modified Rome III criteria for child/adolescent functional constipation (FC).

Protocol Description

Multi-center, randomized, double-blind, placebo-controlled, parallel-group, safety and efficacy study comparing one of three Linactolide doses with placebo in pediatric patients, ages 6 to 17, with a functional constipation diagnosis.

Eligibility Criteria

• Ages 6-17
• Able to read and understand the assessments in the e-diary
• Must meet Rome III criteria for child/adolescent functional constipation
• Must be compliant with e-diary

Primary Investigator: Michael Hart, M.D.

Contact

Christy Florow, Clinical Research Coordinator
540-985-9723
cmflorow@carilionclinic.org

Irritable Bowel Syndrome and Functional Constipation (LIN-MD-63)

A multi-center, randomized, double-blind, placebo-controlled, parallel-group, safety and efficacy study of a range of Linaclotide doses administered orally to children, ages 7-17 years, with irritable bowel syndrome with constipation (IBS-C).

Protocol Description

Multi-center, randomized, double-blind, placebo-controlled, parallel-group, safety and efficacy study comparing one of three Linactolide doses with placebo in pediatric patients, ages 7 to 17, with IBS and functional constipation diagnosis.

Eligibility Criteria

• Ages 7-17
• Able to read and understand the assessments in the e-diary
• Must meet Rome III criteria for child/adolescent IBS-C
• Must be compliant with e-diary

Primary Investigator: Michael Hart, M.D.

Contact

Christy Florow, Clinical Research Coordinator
540-985-9723
cmflorow@carilionclinic.org

Ulcerative Colitis (MESALAMINE)

A Phase III, multi-center, randomized, double-blind study to determine the safety and efficacy of MMX® Mesalamine/Mesalazine in pediatric subjects with mild to moderate ulcerative colitis, in both acute and maintenance phases.

Protocol Description

To evaluate the safety and tolerability of a low and high dose of Mesalamine in children and adolescents with mild to moderate ulcerative colitis.

Eligibility Criteria

• Ages 5-17
• Diagnosed with mild to moderate ulcerative colitis
• Able to swallow the study drug whole

Primary Investigator: Michael Hart, M.D.

Contact

Christy Florow, Clinical Research Coordinator
540-985-9723
cmflorow@carilionclinic.org

Bowel Cleansing Prep (BLI800)

A Safety and Efficacy Study of a Bowel Cleansing Preparation (BLI800) in Pediatric Subjects Undergoing Colonoscopy.

Protocol Description

To compare the safety, tolerance and efficacy of BLI800 to NuLytely as bowel preparation prior to colonoscopy in adolescent patients.

Eligibility Criteria

• Male or female subjects between the ages of 12-16 (inclusive)
• Undergoing colonoscopy for routinely accepted indications
• Negative pregnancy test as screening, if applicable

Primary Investigator: Michael Hart, M.D.

Contact

Christy Florow, Clinical Research Coordinator
540-985-9723
cmflorow@carilionclinic.org

Pediatric Pulmonology

Pneumonia Study for Adolescents (CEMPRA)

A Phase 2/3, randomized, open-label, multi-center study to determine the safety and efficacy of Solithromycin in adolescents (12-17 years of age, inclusive) and children (≥2 months to <12 years of age) with suspected or confirmed community-acquired bacterial pneumonia.

Protocol Description

This is a Phase 2/3, randomized, open-label, active control, multi-center study to assess the safety and efficacy of solithromycin in children and adolescents with CABP. After informed consent/assent is obtained, subjects will be randomized to receive solithromycin or a comparator drug, administered by mouth (PO) based on weight and age. Pharmacokinetic samples will be collected to assess solithromycin disposition in subjects with CABP. The duration of participation, from the signing of the informed consent form and assent (if applicable), will be up to 40 days.

Eligibility Criteria

• Written informed consent from parents or other legally acceptable representatives and informed assent from subject (if age appropriate according to local requirements)
• ≥2 months to 17 years of age, inclusive
• Requiring hospitalization, emergency room or urgent care visit
• Presence of CABP

AND

• Presence of at least 1 of the following:
  • Leukocytosis (≥12,000 white blood cells [WBC]/mm3)
  • Leukopenia (<5000 WBC/mm3)
  • ≥10% immature neutrophils (bands) regardless of total peripheral WBC
  • Elevated inflammatory markers (C-reactive protein or procalcitonin)
  • Oxygen saturation <97% on room air
  • Organism consistent with a typical respiratory pathogen identified from a blood culture or isolated from an appropriate respiratory culture/polymerase chain reaction (sputum in children old enough to produce an acceptable specimen; sample from the lower respiratory tract airways, if performed [e.g., bronchoalveolar lavage]; or pleural fluid culture)

Exclusion Criteria

• Confirmed or suspected respiratory tract infection attributable to sources other than community-acquired bacterial pathogens (e.g., ventilator-associated pneumonia; hospital-acquired pneumonia).
• Received >48 hours of potentially effective systemic antibacterial therapy for CABP immediately prior to randomization (exception: clinical or microbiological treatment failure or progression of signs or symptoms of CABP as determined by the investigator).
• Confirmed or suspected bacterial meningitis.
• Known active pulmonary tuberculosis.

Primary Investigator: Andre Muelenaer, M.D.

Contact

Kellie Hirt, Clinical Research Coordinator
540-985-9934
kehirt@carilionclinic.org