Search Results

This study aims to reduce the radiotherapy (RT) dose necessary to successfully treat patients with intracranial germ cell tumors who are in a state of complete response (CR) following chemotherapy. In this study, a further reduction in whole ventricular irradiation (WVI) will be tested. The primary aim of the study is to determine whether 12 Gy of WVI, and 12 Gy tumor boost, would be successful. Event-free survival (EFS) in patients with central nervous system germinoma, who meet criteria for CR or continued complete response (CCR) following chemotherapy/second-look surgery, would be the primary measurement of success.

In this study, we want to find out more about secondhand smoke from shisha smoking in the home. We want to help families learn more about the risks of shisha smoke in the home and find ways to stop smoking at home.

The purpose of this research is to compare the effects of nivolumab with vusolimogene oderparepvec (VO) against standard of care treatment drug(s) currently available for patients with advanced melanoma. We expect that taking part in this research will last up to 60 months.

This is a global, multicenter, prospective, observational, longitudinal registry conducted to characterize the natural history and real-world clinical management of patients diagnosed with AHP. This protocol will not recommend the use of any specific treatments, visits, or procedures. No medication is provided as part of registry participation.

This research study is for participants who have a weakened immune system (are immunocompromised), have a lower lung infection and are currently using a machine or device to help them breathe. The study will look at whether the study drug, DAS181, works and how safe it is compared with a placebo in adults who have a weakened immune system (immunocompromised) and a parainfluenza virus (PIV) infection of the lower respiratory tract. A placebo looks the same as the study drug but does not contain any active ingredients.

A study to evaluate if the randomized addition of venetoclax to a chemotherapy backbone (fludarabine/cytarabine/gemtuzumab ozogamicin [GO]) improves survival of children/adolescents/young adults with acute myeloid leukemia (AML) in 1st relapse who are unable to receive additional anthracyclines, or in 2nd relapse.

The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.

This study is enrolling children with Turner syndrome, SHOX deficiency, or Noonan syndrome to evaluate the effect of 3 doses of a study drug, vosoritide, versus the standard of care human Growth Hormone (hGH). The study will look at growth over a 6 month period of time. The study will also look at how well the the study drug works (efficacy) and its safety at the therapeutic dose up until the child reaches their final adult height.

This phase III trial tests how well adding dinutuximab to induction chemotherapy along with standard of care surgery radiation and stem cell transplantation works for treating children with newly diagnosed high risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found in greater than normal amounts on some types of cancer cells. This helps cells of the immune system kill the cancer cells. Chemotherapy drugs such as cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, dexrazoxane, doxorubicin, temozolomide, irinotecan and isotretinoin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing or by stopping them from spreading. During induction, chemotherapy and surgery are used to kill and remove as much tumor as possible. During consolidation, very high doses of chemotherapy are given to kill any remaining cancer cells. This chemotherapy also destroys healthy bone marrow, where blood cells are made. A stem cell transplant is a procedure that helps the body make new healthy blood cells to replace the blood cells that may have been harmed by the cancer and/or chemotherapy. Radiation therapy is also given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of induction.

We are conducting a research study for children ages 6-17 with patchy Alopecia Areata (AA). The purpose of this research study is to learn more about the safety, tolerability and efficacy of an investigational drug called Baricitinib. This study will compare the investigational drug to a placebo (inactive substance) to see how well the investigational drug works.

This is a new type 1 diabetes onset study for ages 12-35 years old. We are looking at JAK inhibitor drugs to see if they can preserve beta cell function.

We want to study if lower doses of a chemotherapy drug called busulfan will help babies with SCID achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants.

The purpose of this study is to evaluate the long-term safety and ability of a transplant with gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. Participants must have received investigational gene therapy with bb1111 in a clinical study sponsored by bluebird bio. There is no additional treatment associated with this study as this is a long-term follow-up study.

This is a small, Phase 0, window of opportunity study to provide human experience to support our pre-clinical data and gain preliminary information regarding the safety and tolerability of mirdametinib and vorinostat when given in combination.

The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.

The purpose of this study is to learn if removing the donor T cells from the donor product using this new method will be a better way to reduce the risk of GVHD. The benefit of removing these cells with this new method is that they will prevent GVHD without requiring drugs to suppress the immune system. Potentially, the immune system will recover from the transplant faster, which in turn will also lessen the risk of severe infections. As well, the patient will not have the other common undesired side effects of these immunosuppressive drugs.

This study is for people who have Epidermolysis Bullosa (EB), Fanconi Anemia (FA) or a bone marrow failure disorder that puts them at a higher risk of developing a form of skin cancer called squamous cell carcinoma (SCC). To learn more about these disorders and their relationship to cancer, researchers are collecting skin and blood samples to study in the lab. Blood and skin donated to the will be used by researchers at the University of Minnesota in studying the causes, diagnosis, prevention, and treatment of these disorders. We expect that this study will take about two hours, or the amount of time it takes to check in for a clinic visit and collect the specimens.

The purpose of the study is to see if GC1130A, delivered directly to the central ventricle of the brain is safe and tolerable as a means of treating the neurologic disease in MPS 3A.

Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.

This trial is an open label, randomized, stratified 2-arm Australian-led multicenter phase 3 clinical trial undertaken in two stages. Participants (age >= 11 years and <= 45 years) with intermediate and poor-risk metastatic germ cell tumors will be randomized into either a “standard BEP” group or “accelerated BEP” group. Participants will be assigned to the two treatment arms in a 1:1 ratio and evaluated weekly, and then for 5 years after completing the study to assess the long-term effects of the chemotherapy. Bleomycin, Etoposide, Cisplatin (BEP) administered 3-weekly x 4 remains standard 1st line chemotherapy for intermediate- and poor-risk metastatic germ cell tumours (GCTs). BEP is accelerated by cycling Cisplatin and etoposide 2-weekly instead of 3-weekly. The aim of this study is to determine if accelerated BEP is superior to standard BEP as first-line chemotherapy for intermediate and poor risk metastatic GCTs.

This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients (≥1 year and < 22 years ) with CD22 positive B acute lymphoblastic leukemia that has come back or does not respond to treatment. Immunotoxins, such as inotuzumab ozogamicin, are antibodies linked to a toxic substance and may help find cancer cells that express CD22 and kill them without harming normal cells.

The study is being conducted in order to further understand how we perceive the world, and if and how this may be different in individuals on the autism spectrum. We hope to further understand sensory processing differences in autism.

In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.

The purpose of this study is to develop a questionnaire specifically designed for children and adolescents, which will help health care providers to better measure how bothersome symptoms of cGVHD are for children and adolescents living with cGVHD. Another purpose of this study is to design a caregiver companion questionnaire, to allow caregivers to measure how bothersome the symptoms of cGVHD are for their child/adolescent.

The purpose of this study is to help investigators learn more about what are the best methods for diagnosing and monitoring lung problems.

The purpose of this research to enhance our understanding of adrenoleukodystrophy ALD and study biospecimens such assaliva, blood, urine and stool to identify potential biomarkers for early identification of dise. We invite people who have or are at risk to have ALD, including females who are known or at risk carriers of the mutation for ALD, to help us learn more.

The purpose of this study is to learn whether the use of Pregnyl with the drug ruxolitinib is able to reduce the need for high dose steroids to treat severe acute Graft versus Host Disease (GVHD).

This research study is for creating a registry of all ages with conditions in endocrine and both health. Registries are used very often these days by doctors and scientists to collect information and use to perform research into rare conditions. This registry will be part of a global registry, called "GloBE-Reg" with the University of Glasgow (Scotland) and with the University of MInnesota.

The goal of this study is to learn more about how children (ages 5-8 years) describe their gender identities. We are also interested in learning more about the ways children think and develop. We will be giving children several tasks designed as games to assess flexibility in thinking, creativity, and the ability to take another person’s perspective.

This study will seek to learn about barriers to management of Type 2 diabetes for children ages 10-17. This study is recruiting children, and their parent/guardian, who are managing with oral medication or insulin.