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)
Participants in this research study are receiving treatment with chemotherapy for Acute Myeloid Luekemia (AML), and therefore at risk of getting an invasive fungal infection. Invasive fungal infections are infections caused by organisms called fungi. The infection is described as invasive when the fungi enters the blood stream and can then spread to different organs in the body. Preventing invasive fungal infections due to chemotherapy is therefore very important for the study treatment success of people with AML.
In this study, researchers want to find out if using a newer antifungal drug called caspofungin (also called Cancidas) will be better than the current standard antifungal drug fluconazole at preventing invasive fungal infections when given to people with AML after receiving chemotherapy study treatment
Approximately 6 people will take part in this study conducted by investigators at the University of Iowa. Approximately 550 people will be enrolled worldwide.
| age group: | 0 to 18 years |
trial start date: | November 28, 2011 |
| Gender Preference: | none |
Trial End Date: | November 28, 2021 |
| Contact Info: | Julie de la Garza, 319-356-3749 |
| Keywords: | acute myeloid luekemia (AML) ; cancer ; caspofungin (Cancidas) ; chemotherapy ; fluconazole ; fungal infections ; IRB#201109830 ; pediatric
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Principal Investigator: Katherine D. Mathews, MD
In this project, researchers will examine the clinical presentation of muscular dystrophy caused by abnormal glycosylation of alpha-dystroglycan. Patients with dystroglycanopathies could have mutations in one of the following genes: FKRP, fukutin, POMT1, POMT2, POMGnT1 or LARGE. Symptoms range from congenital muscular dystrophy that can also involve the brain and eye, through an adult-onset limb girdle muscular dystrophy. The purpose of the study is to describe the early signs and symptoms of the dystroglycanopathies, and to gather information that will be required for future clinical trials. Knowledge gained from this study will improve the health care recommendations for people with dystroglycanopathies, and provide a baseline for further study.
The study involves a clinical evaluation at theUniversityofIowa. The evaluation includes muscle strength and motor ability testing, lung function testing, quality of life and activity assessment, and a review of past medical history. Portions of this evaluation will be repeated on a yearly basis. Financial assistance is available for travel toIowa City. Support is also available for genetic testing for people with a dystroglycanopathy diagnosis based on muscle or skin biopsy analysis.
| age group: | 0 to 80 years |
trial start date: | July 1, 2011 |
| Gender Preference: | none |
Trial End Date: | July 31, 2015 |
| Contact Info: | This study is sponsored by the National Institutes of Health and is being directed by Dr. Katherine Mathews at the Universityof Iowa. If you are interested in participating or would like additional information, please contact the study coordinator Carrie Stephan R.N. by phone at (319) 356-2673 or by email at carrie-stephan@uiowa.edu |
| Keywords: | Dystroglycanopathy ; Neurology ; Pediatric
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Principal Investigator:Ayman El-Sheikh, MD
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
AML is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone marrow. They may also invade body organs including the brain, testes, ovaries, or skin. These cancerous AML cells can sometimes form a solid tumor called a chloroma.
The main goals of this study are:
For subjects without high amounts of the FLT3 gene mutation,
1. To see if an investigational drug, called bortezomib, is tolerated when added to the standard AML treatment without causing too many serious side effects.
2. To compare the effects, good and/or bad, of adding bortezomib to the standard AML treatment to find out which is better. In this study, you will get either the standard treatment plus bortezomib or the standard treatment alone.
For subjects with high amounts of the FLT3 gene mutation,
3. To determine the dose of sorafenib that can be safely given with the standard AML treatment.
4. To compare the effects, good and/or bad, of adding sorafenib to standard AML treatment to find out which is better.
5. To determine how effective the combination of sorafenib and chemotherapy will be at killing cancer cells.
Approximately 5 people will take part in this study conducted by investigators at the University of Iowa. The total number of people enrolled on this study nationally is expected to be 1250.
| age group: | 0 to 29 years |
trial start date: | June 20, 2011 |
| Gender Preference: | none |
Trial End Date: | June 20, 2016 |
| Contact Info: | Julie de la Garza, (319) 356-3749 |
| Keywords: | acute myeloid leukemia (AML) ; bortezomib ; cancer ; children ; pediatric ; phase III ; sorafenib
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Principal Investigator: Katherine D. Mathews, MD
The purpose of this study is to identify the most accurate tools which can be used to follow the disease progression in Friedreich’s Ataxia and be able to measure changes over a short period of time. Researchers anticipate that this study will result in reliable measurement tools to be used in future drug treatment research studies. The study is coordinated by Children’s Hospital of Philadelphia and is being conducted at multiple sites across the United States. Reimbursement for travel costs is available.
The study involves a yearly physical examination by a neurologist and an exam by the clinic coordinator. As part of the study, participants will undergo a vision test, measurements of hand function and coordination, and health and quality of life questionnaires. Information from tests such as ECG’s and echocardiograms will be collected.
| age group: | 7 to 80 years |
trial start date: | June 1, 2011 |
| Gender Preference: | none |
Trial End Date: | May 31, 2013 |
| Contact Info: | Carrie Stephan R.N., Clinical Research Coordinator at (319) 356-2673 or email at carrie-stephan@uiowa.edu |
| Keywords: | Friedreich ; Neurology ; Pediatric
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