Search Results
MT2012-11C: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.
Teresa Bekkala - tkivist1@fairview.org
MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a treatment guideline for allogeneic hemapoetic stem cell transplant (HSCT) in patients with primary immune deficiencies. Endpoints include time to engraftment, incidence of graft failure, incidence of chimerism at 100 days, 6 months, and 1 year; incidence of acute GVHD at 100 days and chronic GVHD at 6 months and 1 year; incidence of transplant related mortality at 6 months and incidence of disease free survival and overall survival at 6 months. All endpoints will be abstracted from routine data collected by the BMT Database.
Christen Ebens - ebens012@umn.edu
• up to 50 years old
• diagnosis of immunodeficiency or histiocytic disorder
• see link to clinicaltrials.gov for complete inclusion criteria
• pregnant or breastfeeding
• active, uncontrolled infection and/or HIV positive
• acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry
The primary objective for this observational study is to collect general and medical data from children, adolescents, and young adults who had pediatric onset rheumatic disease. This data will be used to evaluate the long-term safety and efficacy of therapeutic agents used to treat these diseases. This information will allow investigators to accurately report and follow changes in current medication use patterns and compare these to proposed standards and current treatment recommendations. The use of a single registry will allow for more analysis of the different therapeutic agents by allowing them to be compared to each other.
Colleen Correll - corr0250@umn.edu
• diagnosed with rheumatic disease prior to age 16 years for juvenile idiopathic arthritis (JIA)
• onset prior to age 19 years for all other rheumatic diseases
• younger than 21 years
Genetic Modifiers and Glycemic Variability in Turner Syndrome
This study is looking at glucose (blood sugar) patterns in participants with Turner Syndrome between the ages of 3-80 years old. This will be done by obtaining 2 hours frequent blood sampling by completing an Oral Glucose Tolerance Test and a Mixed Meal Tolerance Test. Participants will also wear a continuous glucose monitor for 2 weeks. Along with the OGTT and MMTT, participants are asked to provide a blood sample for DNA and RNA testing. Participant’s parents are asked to provide a saliva sample for DNA testing
Kyriakie Sarafoglou - saraf010@umn.edu
• Participants with a diagnosis of Turner syndrome by karyotype
• Ages 3 to 80
• Additional genetic diagnosis detected on karyotype, CMA, or FISH
• Prior diabetes diagnosis
• Current or recent (last 72 hours) use of systemic glucocorticoids
• Current use of hypoglycemic agents
• History of solid organ or bone marrow transplant
• Currently pregnant
• Non English-speaking
Ten Thousand Families Study
The purpose of this study is to study the evolution of early life risk factors that may lead to cancer and other conditions. This is a prospective cohort study of families who reside in Minnesota.
Clemen Wilcox - clemenw@umn.edu
• 1st Participant: 18+ living in MN
• Other family members: All ages and must live in MN, ND, SD, IA, or WI
• Participants ages 0-17 must have a parent consent to their participation and assist with study activities
• Unwilling or unable to provide DNA and blood sample
• Does not have at least 1 living family member in MN IA, ND, SD, or WI
MT2005-25 Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia
To determine the incidence of engraftment (defined as achieving donor derived neutrophil count >500/uL by day 42) in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.
Kim Nelson - knelso62@fairview.org
A Phase II, Multi-center, Open-Label Study to Assess Safety, Tolerability, Efficacy and Pharmacokinetics of R3R01 in Alport Syndrome Patients with Uncontrolled Proteinuria on ACE/ARB Inhibition and in Patients with Primary Steroid-Resistant Focal Segmental Glomerulosclerosis
The main purpose of this study is to check how safe the study drug is and how well your body handles taking it. The study will also check: • if the study drug works to improve your kidney function • whether the study drug has an impact on your daily life • the amount of the study drug in your blood over a period of time (called pharmacokinetics)
Amy Hanson - amhanson@umn.edu
• at least 12 years of age
• for people with Alport Syndrome: confirmed diagnosis by genetic testing and /or kidney biopsy
• for primary Focal Segmental Glomerulosclerosis (FSGS), (without any identifiable cause, and where the FSGS is confirmed by renal biopsy) or FSGS where there is documentation of a genetic mutation in a podocyte protein
• female patients, as well as, female partners of male patients who are of child-bearing potential must be willing to not become pregnant for the complete duration of the study (90 days after the last dose of study medication)
• males (including sterilized subjects) whose female partners have child-bearing potential, must agree to use male contraception (condoms) during the period from the time of signing the informed consent form (ICF) through 90 days after the last dose of study drug
• contact study staff for additional criteria
• uncontrolled diabetes mellitus as evidenced by an HbA1c greater or equal to 11%
• uncontrolled high blood pressure
• moderate or severe liver impairment
• BMI greater than 40
• women who are pregnant or breast feeding
• additional exclusion criteria apply (study staff will review)
A Phase 2, Open-Label, Basket Study of Atrasentan in Patients with Proteinuric Glomerular Diseases (AFFINITY)
The purpose of the research is to find out if atrasentan delays worsening of kidney function in IgAN, FSGS, and Alport Syndrome.
Amy Hanson - amhanson@umn.edu
• Age 18 years and older for patients in the IgAN, FSGS, and Alport Syndrome cohorts
• age 18-70 years for patients in the DKD cohort
• receiving a maximally tolerated dose of RAS inhibitor therapy (ACEi or ARB) that has been stable for at least 12 weeks
• there are different requirements for each diagnosis category & study staff will review these
• current diagnosis of another cause of chronic kidney disease or another primary glomerulopathy
• history of kidney transplantation or other organ transplantation
• except for FSGS patients, use of systemic immunosuppressant medications, such as steroids, for more than 2 weeks in the past 3 months
• blood pressure above 150 mmHg systolic or 95 mmHg diastolic
• history of heart failure or a previous hospital admission for fluid overload.
• history of liver disease
• hemoglobin below 9 g/dL or blood transfusion for anemia within the past 3 months.
• cancer in the past 5 years (except nonmelanoma skin cancer and curatively treated cervical carcinoma in situ)
• women who are pregnant, breastfeeding, or intend become pregnant during the study
• recently received an investigational agent -clinically significant unstable or uncontrolled medical condition (study staff will review)
Defining Clinical Endpoints in LGMD (GRASP-01-001)
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.
Allison Johnston - joh21779@umn.edu
• 4 to 65 years of age
• diagnosis of Muscular Dystrophy with weakness in either a limb-girdle pattern, or in a arm or leg
• confirmed mutation in ANO5, CAPN3, DYSF, DNAJB6 or SGCA-G.
• bleeding disorder, platelet count less than 50,000, or currently taking an anticoagulant.
• women who are pregnant
• other illness that would interfere clinical trial (study staff will review)
MT2019-01: Adrenoleukodystrophy National Registry Study (ALD) and Biobank
In this protocol, we will enroll pediatric, adolescent and adult patients diagnosed with adrenoleukodystrophy (ALD). These patients will include probands diagnosed by newborn screening and their relatives subsequently diagnosed, as well other patients who are diagnosed with ALD due to other presenting signs and symptoms and subsequently were confirmed to have ALD. We will ask consenting subjects to provide a medical history (with verification via medical records), to participate in a semi-annual health survey and provide consent to collect biospecimens. The overarching goal of this work is to engage with families affected by ALD and to assemble a resource of clinical, medical, and biological data that will allow of to better understand the natural history of ALD, and how this is affected by newborn screening. The initial focus will be on patients within Minnesota, but participation will be open to any family interested in the study, as this will be web-based. This registry and biobank, together with other research conducted in tandem, will possibly provide information describing the natural history of ALD and outcomes with interventions. It is anticipated that the data collected will further our understanding of the natural history of the disease, basic biology of adrenoleukodystrophy, diagnosis and outcomes. Ultimately, this research may lead to new avenues for early diagnosis and development of safer and more effective therapies for ALD.
Rachael Eye - eye00002@umn.edu
• age 0 to 100
• patient or family member diagnosed with ALD (confirmed by positive VLCFA testing and/or genetic mutation
• patient or family member with known or presumed mutation with ALD based on pedigree or confirmed mutation in ABCD1 gene
• living in the United States and territories
• have undergone BMT or other cellular therapy
• not fluent in English who are unable to consent in-person
• people who are unable to read or write
National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC) - A Collaborative Initiative to Improve Care of Children with Complex Congenital Heart Disease (NPC-QIC)
This study is a prospective, non-randomized, observational multi-center study, utilizing a quality improvement methodology to facilitate systematic care coordination, interstage cardiovascular monitoring, and nutritional monitoring into every day practice. Utilizes a national registry to document the impact of these changes on various care processes and outcomes. The aim of this Phase II project intends to: 1) develop and support a robust national registry to gather clinical care process, outcome and developmental data on infants with HLHS between diagnosis and the first year of life, 2) engage pediatric cardiology and cardiac surgery programs in using the registry, and 3) use data from the registry to support and monitor the implementation of QI strategies to standardize and improve care for these infants.
Brittany Faanes - grego318@umn.edu
• up to 15 months old
• newborns diagnosed with HLHS or other univentricular condition
• intended to undergo Norwood procedure
A Pilot Study of a Parenting Intervention for Parents of Adolescents with Non-Suicidal Self-Injury
University of Minnesota researchers are conducting a research study to learn more about how an individually-delivered parenting program may help teens with self-harm. Eligible parents will be assigned by chance (like flipping a coin) to one of two conditions: (1) Healthy Emotions and Relationships with Teens – A Guide for Parents (HEART-P): a 12-session individually-delivered parenting program that teaches parents skills and strategies to help them respond to their adolescents’ emotions, or (2) wait list: parents will be offered the opportunity to receive HEART-P following their completion of the assessments during the study phase.
PTAD Lab - ptad@umn.edu
• Age 12-17 years
• at least 3 episodes of non suicidal self injury with at least one episode occurring in the past 12 weeks
• receiving mental health treatment that doesn't include individually delivered treatment for the parent. This can be at any mental health clinic
• at least one parent or caregiver who is willing to participate -for parents/caregivers: able to speak and write English
• history of a primary psychotic disorder
• neurodevelopmental disorder such as intellectual disability or autism
Targeting Family Meal Quality and Quantity to Reduce Childhood Obesity Using Ecological Momentary Intervention (EMI) and Video Feedback
The proposed study is an individual three-arm randomized controlled tiled aimed at utilizing state-of-the-art intervention methods to examine whether increasing the quality and the quantity of family meals reduces childhood obesity.
Marah Aqeel - aqeel002@umn.edu
• Child 5-10 years old
• Have a sibling who lives in the home with the child
• Live in the Metro area
• Speaks English or Spanish
RARE-OB-16: Rare CFTR Mutation Cell Collection Protocol (RARE) (RARE)
We are doing the RARE study to learn more about Cystic Fibrosis (CF). CF is caused by mutations in a gene that produces a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). In people with CF, the CFTR does not function correctly. Medications are being developed to help the CFTR function better, but those medications mostly benefit people with common CFTR mutations. There are more than 1,900 mutations of the CF gene. Some of these mutations are rare and found only in a few people. The goal of this research study is to collect specimens (blood, nasal cells, rectal cells) from people with rare CFTR mutations. Another purpose of this study is to create induced pluripotent stem cells or iPS cells. “Pluripotent” stem cells are cells that can be changed into almost any cell type of the body (such as lung or intestine). They can be kept alive and stored indefinitely. There are different kinds of pluripotent stem cells. Inducted pluripotent stem cells can be created from many different kinds of specimens (such as blood, nasal cells, rectal cells). This is different from embryonic stem cells, which can only be derived from embryos. The specimens collected during this study and iPS cells created from them will be stored for use in future research to learn more about CF and study the effect of new medications. This could identify new medications that may help people with rare CFTR mutations.
Dena Johnson - joh20459@umn.edu
• at least 12 years old
• documented diagnosis of Cystic Fibrosis (CF)
• willing to travel (if needed) to a regional study site for cell collection
• presence of a medical condition, abnormality, or laboratory value that would place the participant at risk (study staff will review)
Pediatric COVID-19: Does infection with the SARS-CoV-2 virus alter brain structure and function?
The goal of the proposed project is to investigate whether brain abnormalities are present in children to young adults after the recovery from coronavirus disease 2019 (COVID-19).
Monica Bondy - bondy023@umn.edu
• diagnosis of COVID-19 in the past
• experiencing long covid symptoms for at least 2 months
• 3 to 25 years old at the time of entry into the study
• active positive COVID-19 diagnosis (as confirmed by a medical provider &/or certified testing site) for at least 4 weeks prior to projected enrollment date
• surgically implanted pacemaker
• indwelling electronic device, including programmable shunts
• orthodontic braces, unless non-metallic
• implanted metal in the body other than titanium
• inability or unwillingness to complete an MRI
• pregnancy
• anyone not meeting standard MRI requirements according to CMMR protocol (ie presence of metal in body or implanted pacemaker) will be excluded from that portion of the study
Natural History Study for DNA Repair Disorders
A single-center, single-arm, non-interventional natural history study to evaluate the longitudinal clinical course, functional outcome measures, and candidate biomarkers for individuals with DNA repair disorders, including Cockayne syndrome (CS), xeroderma pigmentosum (XP), and trichothiodystrophy (TTD).
Erin Aguero - aguer008@umn.edu
• at least 6 months old
• diagnosis of Cockayne syndrome (CS), xeroderma pigmentosum (XP), or trichothiodystrophy (TTD), based on genetic testing and/or key clinical characteristics
• have one or more of the neurodevelopmental or neurological complications such as gross motor delay, language delay, altered muscle tone (study staff will review)
• family member of an individual with the above condition
• prior history of systemic gene or cell-based therapy
• participation in a clinical trial for treatment
State Representation in Early Psychosis (STEP)
In the first phase of the study, participants will be asked to complete two sets of appointments six months apart. During both sets of appointments, participants will be asked to complete interviews and questionnaires about their life experiences and mental health, and they will have an EEG and fMRI while completing computerized tasks. The second phase of the study is optional. In this phase, participants will test one of two forms of computerized cognitive training, or brain games. They will be asked to complete 10 hours of training over a 3-6 week period. After the training period is over, they will have two sets of follow up visits, one right after the training period and one five months later. At these appointments, participants will complete the same activities done in the first phase of this study, including the interviews, questionnaires, and imaging (fMRI and EEG) combined with computerized tasks. We are recruiting two groups of participants for this study. One group will include individuals who experience hallucinations, delusions, paranoia, or a psychosis disorder (i.e., schizophrenia), and the other group will be individuals who do not have a diagnosis or family history of schizophrenia, bipolar disorder, or autism spectrum disorder.
STEP Study - stepstudy@umn.edu
• able to speak and write English
• 15 to 40 years old
• diagnosis of schizophrenia, schizoaffective disorder, psychosis, bipolar disorder with psychosis, or major depressive disorder with psychosis, with psychotic symptoms starting in the past 5 years
• no hospitalizations and on stable doses of medications for the past one month or more
• For healthy volunteers without a mental health diagnosis: will match on age, sex, etc. to people enrolled in the study.
• currently pregnant
• history of neurological disorder
• previous head injury with loss of consciousness
• currently suicidal or has attempted suicide in the past 6 months
Cortical Inhibitory Biomarkers of Acute Suicidal States in Adolescents
Prospective study investigating brain activity associated with markers of suicidal behavior (SB) in adolescents.
Alanna Esquivel - esqui051@umn.edu
• ages 13-18
• current diagnosis of depression, without or without histoy of suicidal behavior
• must be able to communicate verbally and in writing in English
• must have reliable internet connection
•
• Active substance use in the past month
• Neurological disorders such as seizures, head injury
Study to Evaluate the Efficacy (Effect on Disease) and Safety of Finerenone on Morbidity (Events Indicating Disease Worsening) and Mortality (Death Rate) in Participants With Heart Failure and Left Ventricular Ejection Fraction (Proportion of Blood Expelled Per Heart Stroke) Greater or Equal to 40% (FINEARTS-HF) (FINEARTS-HF)
This study is a randomized, double-blind, parallel-group, placebo-controlled, multicenter, event-driven Phase 3 study with independently adjudicated clinical outcome assessments.
Michelle Pitt - henni032@umn.edu
• at least 40 years old
• diagnosis of heart failure with New York Heart Association(NYHA) class II-IV, or hospitalized primarily for heart failure
• on a diuretic medication for at least 30 days
• left ventricular ejection fraction (LVEF) of at least 40% measured any way in the last 12 months
• Myocardial infarction, coronary artery bypass (CABG), stroke or transient ischemic attack (TIA) in the last 90 days
• systolic blood pressure (SBP) 160 mmHg or greater if not on treatment with at least 3 blood pressure lowering medications or 180 mmHg or greater irrespective of treatments
• additional criteria apply (study staff will review)
ALX-HPP-501: An Observational,Longitudinal Prospective, Long-term Registry of Patients with Hypophosphatasia
This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.
Kyriakie Sarafoglou - saraf010@umn.edu
• confirmed diagnosis of HPP.
• documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
• able to read and speak English
• currently participating in an Alexion-sponsored clinical trial
Determinants of Renal Structural Responses to Enzyme Replacement Therapy (ERT) in Fabry Disease Study (LDN6702)
The effect of enzyme replacement therapy on how well your kidneys are responding to enzyme replacement therapy (ERT) is not clear from blood and urine tests alone, but may be more clear in comparisons of kidney biopsies performed before and some time after ERT has been initiated, and this is what we are focusing our study efforts on. The purpose of this study is to obtain your permission to allow us to study the kidney biopsy tissues (collected for medical reasons) after the regular routine studies have been completed. Through our special research measurements and additional study, we hope to be able to see and measure very specific changes in the kidney tissues from Fabry patients taking ERT. We also hope that through these studies of what happens within the kidney before and after starting ERT, we are able to reveal valuable information about the importance of factors like your age that you started ERT, the amount or dosage of ERT, and any differences seen between males and females.
Michael Mauer - mauer002@umn.edu
• diagnosed with Fabry disease and a clinical decision has been made to obtain a kidney biopsy, a GFR, and urinary albumin studies
• have previously completed clinical trials which included measures of renal function and renal biopsies
• serum creatinine more than 2.5 mg/dL
• known to have a renal disease other than Fabry
A person-centered employment preparation program for adolescents and young adults with autism spectrum disorder and their families
This study includes the development and evaluation of a person-centered employment preparation program for families of transition-aged youth with autism.
Rebekah Hudock - kale0040@umn.edu
• between the ages of 15-25
• have a medical diagnosis of Autism Spectrum Disorder (ASD) or educational eligibility for special education services under the category of autism
• verbally fluent (can speak in complete sentences and have basic conversation) and speak and understand English
• have access to WiFi and two devices with webcams
• at least one caregiver are able to commit to attending 8 90-minute virtual intervention sessions
• do not live in MN
• medical, behavioral, or mental health concerns that make it too difficult to participate in the study or that necessitates a higher level of care
DegenPRO: A multicenter prospective registry for the management of degenerative spine disorders
This is an observational registry database for adult patients diagnosed with degenerative spine disorders, which aims to add information to the understanding of the disease management of this spine diseases. By creating this registry, a more complete picture of degenerative spine disorders - including treatment practices - will be established, by collecting information about the health status of patients across several hospitals in several countries. Research of this kind will help future patients by providing doctors with information about degenerative spine disorders, and about patients' treatment outcomes. Data from this registry may be used to generate descriptive statistics on demographics, and clinical characteristics, including co-morbidities, treatment patterns and adverse outcomes (resulting from treatment or disease), as well as patients' quality of life measurements. Condition: Patients with a degenerative spine disorder Intervention/procedure investigated: No specific treatment required. Study design: Prospective case series
Christopher Martin - mart1865@umn.edu
Improving Diagnosis and Treatment in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
This is a multi-center, prospective, observational cohort registry study looking at kids and their relatives with Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT).
Brittany Faanes - grego318@umn.edu
• anyone diagnosed with CPVT before 19 years of age.
• significant medical history that isn't related to CPVT
Humanitarian Use Device: Medtronic DBS Therapy for Dystonia (HDE #H020007) (Dystonia IRB)
Humanitarian Use device application for DBS therapy for dystonia
Kelly Ryberg - rybe0010@umn.edu
• 7 to 99 years old
• diagnosed with chronic, intractable (drug refractory) primary dystonia determined by a neurologist or neurosurgeon
• patients who are at significant surgical risk as determined by the neurosurgeon and/or anesthesiologist
• patients who have not had an adequate trial of medical or non-surgical treatment
Focus in NeuroDevelopment (FIND) Network: A Statewide Network for Research in Neurodevelopment
The purpose of this research project is to develop a registry and database of families with neurodevelopmental disorders.
Amy Yang - yang3958@umn.edu
• All ages
• All individuals with disabilities and families within the neurodevelopmental disorder community
• Also looking for clinicians, educators, and professionals in the field
Synergistic Enteral Regimen for Treatment of the Gangliosidoses (SYNER-G) (Syner-G)
The Syner-G regimen research study seeks to evaluate the use of a combination of a medication called miglustat and a ketogenic diet for treatment of the gangliosidoses to learn if this combination will provide improved clinical outcomes compared to what we currently know about the natural course of the disease.
Jeanine Jarnes - utzx0002@umn.edu
• no more than 17 years old
• documented infantile or juvenile gangliosidosis disease
• severe kidney disease
• females who are pregnant or breast feeding
• females who are post puberty who are unwilling to use highly effective birth control
A Natural History Study of the Gangliosidoses
This study's primary aims are to define and characterize disease progression for the infantile and juvenile forms of the gangliosidoses, and the late-onset forms of gangliosidosis, including their heterogeneity; and to observe treatment outcomes for any treatments tried. The secondary aims of this study are to understand the neurological involvement in late-onset gangliosidosis; and to collect data on disease progression that can be used for creation of an objective disease stage and severity index.
Jeanine Jarnes - utzx0002@umn.edu
• documented gangliosidosis disease
• able to complete neuropsychological and neurobehavioral assessments
• Late-onset gangliosidosis subjects must be able to tolerate MRI of the head
• none