Pediatrics

Below are current clinical trials.

Filter this list of studies by location, status and more.

1. Participation in a Research Registry for Immune Disorders

   Rochester, Minn.

   The purpose of this study is to build a National Registry of individuals with one of the group of primary immune deficiency diseases. A "Registry" is a list of basic information about people who have a certain disease or condition in common. These immune deficiency diseases are thought to be rare and include: Severe combined immunodeficiency (SCID), leukocyte adhesion deficiency (LAD), X-linked Agammaglobulinemia (XLA), common variable immune deficiency (CVID), DiGeorge syndrome (DGS), Hyper IgM syndrome (HIGM), Wiskott Aldrich syndrome (WAS) and chronic granulomatous disease (CGD). We would like to contribute data on a number of subjects with these relatively rare diseases to this National Registry Data Base. The information will be age, sex, race or ethnic group, immunologic lab tests that were used to diagnose the condition, what complications may have occurred since the condition started, lung disease, blood changes, etc. and the results of various treatments used. The goal is to discover basic outcome data, ethnic, racial characteristics, kind of complications and useful treatments. You will not be contacted by anyone unless you authorize it. If a new study about you (or your child’s) immune defect comes up, your doctor will be notified, who can then share this with you to find out if you are interested in participating or not. Alternatively you may elect to be contacted directly by the Registry to determine your interest in participation.

2. Exercise during Heart Cath in CHD Patients

   Rochester, Minn.

   The purpose of this study is to compare the difference in the ability to detect blood flow abnormalities between invasive assessments performed at rest versus exercise.

3. Fecal Microbiota Transplant National Registry

   Rochester, Minn.

   A national data registry of patients receiving fecal microbiota transplantation (FMT) or other gut-related-microbiota products designed to prospectively assess short and long-term safety and effectiveness.

4. 3D US Imaging of Acute Nerve Root Avulsions in Injuries of the Brachial Plexus

   Jacksonville, Fla.

   The purpose of this study is to address a knowledge gap. Early diagnosis of nerve root avulsion injuries following brachial plexus trauma remain a barrier to the early treatment of these complex injuries.  Traditionally, an observation period of 6 months following brachial plexus injuries has been utilized to allow for reinnervation.  Reinnervation is not possible in the setting of nerve root avulsion from the spinal cord and waiting for reinnervation in this setting unnecessarily delays treatment and potentially compromises outcome.  Having the ability to provide a real time evaluation of nerve root integrity would obviate the need of other more expensive and complex advanced imaging such as CT/myelography, currently considered the good standard.

5. A Study to Predict Response to Virotherapy and Immunotherapy by Using an Ex-Vivo Three-Dimensional Patient-Derived Organoid Model of Pediatric Urological Cancers

   Rochester, Minn.

   The purpose of this study is threefold:  the first aim is to use patient-derived fresh tumor tissue to create cell lines and 3D tumor models (i.e. organoids) that preserve the characteristics of the original tumor. The preservation of the original tumor's drug resistance/response profile will be a major focus of this aim. The second aim is to conduct high-throughput testing of various drugs (e.g., virotherapy, immunotherapy) on these cell lines and 3D tumor models. The completion of the second aim is an important step towards developing a platform that can help guide treatment decisions for future patients, based on the drug response observed in the cell lines and 3D tumor models. The third aim is to use pan-omics approaches (i.e., genomics, proteomics, metabolomics) to find markers of drug response based on the results of the high-throughput drug testing on the cell lines and 3D tumor models.

6. Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome

   Rochester, Minn.

   The purpose of this study is to compare the 1-year cumulative incidence of severe GVHD (from day of HCT) defined as Grade III-IV acute GVHD (aGVHD) and/or chronic GVHD (cGVHD) that requires systemic immunosuppression and to compare the disease free survival (DFS) (from time of randomization) in children and young adults (AYA) with acute myeloid leukemia (AML), acute lymphoid leukemia (ALL), and Myelodysplastic Syndrome (MDS) who are randomly assigned to haploHCT or to an 8/8 adult MUD HCT.

7. 24-Hydroxylase Deficiency and CYP24A1 Mutation Patient Registry

   Rochester, Minn.

   You are being asked to take part in this research registry because you or your family member is suspected to have a 24-hydroxylase deficiency.

8. A Study Randomized Trial to Evaluate Sequential vs Simultaneous Spectacles Plus Patching for Amblyopia in Children 3 to < 13 Years Old

   Rochester, Minn.

   The purpose of this study is to determine whether simultaneous treatment with spectacles and patching has an equivalent VA outcome compared with sequential treatment, first with spectacles alone, followed by patching (if needed), for previously untreated amblyopia in children 3 to < 13 years of age.

    

    

9. T Cell Response to COVID19 Vaccinated Individuals

   Rochester, Minn., Jacksonville, Fla.

   The first aim of this study is to verify that the TCR beta sequencing and curation platforms we will be using are able to identify patients with severe COVID-19 based on the TCR repertoires. This may be accomplished by testing and analyzing results between two cohorts: patients presenting acutely to MCF with mild COVID-19 and patients presenting with severe COVID-19. This will be accomplished by utilizing the the immunoSEQ Human T-cell Receptor Beta (hsTCB) Assay (Adaptive Biothechnologies) and the publicly available ImmuneCODE Open Access Database to confirm previously published data (PMID: 34253751). Both procedures will be performed by the company that created both platforms, Adaptive Biothechnologies.

   The second aim of this study is to sequence the TCR receptors of participating patients and identify TCR motifs that may indicate a predisposition to (or protection from) severe SARS-CoV-2, leading to possible risk stratification of such patients, and whether or not age, gender or ethnicity has any contribution to said risk. This may require that we examine all three cohorts listed in the Subject Information section.

   The third aim is to measure SARS-CoV-2 anti-spike and anti-nucleocapsid antibodies to distinguish patients that have not been exposed to SARS-CoV-2 to those that have been exposed.

10. Study to Analyze the Metabolic Environment in Preventing Atopic Dermatitis

    Rochester, Minn.

    The purpose of this study is to compare the assessment of the composition of the fecal, nasal,oral and skin microbiota in patients with AD (cases) as compared to age/sex and diet matched control children without atopic dermatitis, and to apply mass-spectrometry-based metabolomic approach to analyzing fecal, nasal, oral and skin samples from cases, in order to characterize their biochemical metabolic profiles by comparison with those of their controls.