Clinical Research

Treatment to Prevent Invasive Fungal Infections in Children Undergoing Chemotherapy for Acute Myeloid Leukemia (AML)

Principal Investigator: Ayman El-Sheikh, MD

A Randomized Open-Label Trial of Caspofungin versus Fluconazole to Prevent Invasive Fungal Infections in Children Undergoing Chemotherapy for Acute Myeloid Leukemia (AML)

Invasive fungal infections are infections caused by organisms called fungi that enter the blood stream and can then spread to different organs in the body. Chemotherapy used to treat AML severely reduces the body’s natural infection fighting ability. Preventing invasive fungal infections due to chemotherapy is therefore very important for the study treatment success of people with AML.

This study is organized by Children’s Oncology Group (COG). The overall goal of this study is to compare the effects of 2 antifungal drugs: caspofungin and fluxonazole on people getting treatment for AML. On this study, you will either get caspofungin or fluconazole. You will not get both drugs.

Approximately 6 people will take part in this study conducted by investigators at the University of Iowa.  Approximately 550 people will be enrolled worldwide.

An Open-Label Safety Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy

Principal Investigator:Katherine D. Mathews, MD

Duchenne/Becker muscular dystrophy (DBMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of DBMD in approximately 10-15% of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is an open-label trial for patients with nonsense mutation dystrophinopathy who received ataluren in a prior PTC-sponsored study at a US clinical trial site. This trial will be conducted at sites in the US and will evaluate the long-term safety of ataluren, as determined by adverse events and laboratory abnormalities. Patients will receive study drug 3 times per day (at breakfast, lunch, and dinner). Study assessments will be performed at clinic visits during screening and every 12 weeks until the end of the study.

Treatment of Patients with Newly Diagnosed Standard Risk Lymphoblastic Leukemia (B-ALL) or localized B-Lineage Lymphoblastic Lymphoma (B-LLy)

Principal Investigator: Ayman El-Sheikh, MD

Treatment of Patients with Newly Diagnosed Standard Risk Lymphoblastic Leukemia (B-ALL) or localized B-Lineage Lymphoblastic Lymphoma (B-LLy)

Participants in this study have newly diagnosed B-lymphoblastic leukemia (B-ALL) or B-lymphoblastic lymphoma (B-LLy). Patients with Down Syndrome are eligible.

The purpose of this study is to :

1. To determine if a Maintenance regimen containing weekly oral methotrexate at 40 mg/m²/week will result in an improved disease free survival (DFS) compared to that containing weekly oral methotrexate at 20 mg/m²/week in the AR subset of patients with Standard Risk B-precursor ALL.

2. To determine whether a reduced-pulses Maintenance regimen with vincristine/dexamethasone pulses delivered every 12 weeks can be used without adversely impacting DFS as compared to pulses given every 4 weeks in the AR subset of patients with Standard Risk B-precursor ALL.

3. To confirm that patients in the LR subset of Standard Risk B-precursor ALL, based on clinical and cytogenetic features and minimal residual disease (MRD) criteria, can attain a 5-year DFS of at least 95% with either a P9904-based regimen that includes 6 courses of intermediate dose (1 g/m2 over 24 hours) methotrexate without alkylating agents or anthracyclines (Arm LR-M), or an outpatient based regimen identical to that of AR patients with reduced vincristine/dexamethasone pulses at 12 week intervals during Maintenance (Arm LR-C).

4. To provide standardized treatment and enhanced supportive care to children with SR DS-ALL in order to improve outcomes and facilitate further study of this biologically and clinically unique patient subgroup.

5. To improve understanding of the biology of localized B-LLy and DS b-LLy by obtaining biologic data, Including FISH for recurrent cytogenetic lesions on paraffin specimen, and banking tissue for future research.

6. To describe the 5-year EFS and overall survi

Rare and Cutaneous Non-Hodgkin Lymphoma Registry

Principal Investigator:Ayman El-Sheikh, MD

Participants in this study have been diagnosed with an uncommon (rare) type of Non-Hodgkin Lymphoma (NHL). The purpose of this study is to learn about lymphoma, how it presents, and whether it behaves differently from the same lymphoma in adults. Study involvement is minimal and includes the results from the biopsy or surgery submitted to the study doctor for research. There will be follow ups made once a year by the study doctor to see how the participant is doing.

A Randomized, Double-Blinded, Placebo-Controlled, Multi-Institutional, Phase II.5 Study of AZD0530, a Selective Src Kinase Inhibitor, In Patients with Recurrent Osteosarcoma Localized to the Lung (10)

Principal Investigator: Mohammed Milhem, MD

The participants in this study have been diagnosed with recurrent osteosarcoma in the lung and have received standard treatments for this type of cancer. The purpose of this research study is to see if participants will benefit from taking a drug called AZD0530. Approximately 5 people will take part in this study conducted by researchers at the University of Iowa. Study involvement will last for approximately 5 years.