Search Results
MT2012-11C: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
The primary purpose of this study is to record outcomes and patient characteristics in the Masonic Cancer Center and BMT databases for patients who are undergoing an allogeneic (donor) hematopoietic stem cell transplant. The data will be analyzed for transplant “milestones” such as time to blood count recovery (engraftment) and how patients are doing at 3 months and 6 months after the transplant. Participation in this study will not alter treatment or medical care. All information for this study will be collected from medical records.
• up to 55 years old
• diagnosis of any disease for which a second or greater hematopoietic stem cell transplant (HSCT) is needed
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• women who are pregnant or breastfeeding
• active, uncontrolled infection
• HIV positive
Use of Continuous Wave Doppler to assess Vascular Malformations in Pediatric Dermatology
The aim of our study is to look at blood flow in various tumors and irregularities located in blood vessels using a handheld continuous wave doppler. Correct and efficient diagnosis of vascular anomalies (outside of what is expected to happen in blood vessels) in pediatric patients will help determine a treatment plan. Blood flow in vascular anomalies has not been well described in the past.
• less than 21 years old
• have a vascular anomaly such as Arteriovenous malformations (AVM), Capillary malformations (CM), Venous malformations (VM), Lymphatic malformations (LM), Pyogenic granuloma (PG), Infantile hemangioma (IH), or Congenital hemangioma (CH)
• being treated at University of MN pediatric dermatology outpatient clinic or the multidisciplinary vascular anomalies clinic
• history of any prior surgical, radiologic, medications for treatment (including oral or topical beta blocking agents)
A Minimal-Risk, Multi-Center, Prospective, Clinical Trial to Evaluate the PrevisEA Device for Predicting Gastrointestinal Impairment
This device listens to and records abdominal sounds, which provides data that can help predict gastrointestinal impairment (GII). GII is a condition that is defined as the failure of oral re-feeding after abdominal surgery. This happens when any of the following events happen beyond 24-hours after abdominal surgery; vomiting, need to reverse the diet, or need to place a nasogastric (NG) tube.
• 18 to 90 years of age
• having an elective intestinal resection surgery (specific types, study staff will review)
• allergy to skin adhesive
• unable to have device applied to the skin on the abdomen
• evidence of infection before surgery, including a deep wound infection or urinary tract infection
• specific types of surgery (study staff will review)
A Randomized, Comparative Effectiveness Study of Staged Complete Revascularization with Percutaneous Coronary Intervention to Treat Coronary Artery Disease vs Medical Management Alone in Patients with Symptomatic Aortic Valve Stenosis undergoing Elective Transfemoral Transcatheter Aortic Valve Replacement: The COMPLETE TAVR Study (COMPLETE TAVR)
The purpose of this study is to find out, in patients with Coronary Artery Disease who had successful TAVR with a balloon expandable heart valve, whether restoring blood flow to all blocked or narrowed arteries via PCI using coronary stents (metal scaffold) is better than treating the coronary artery disease with medications only following TAVR. Participation in this study will last up to 5 years.
• at least 18 years old
• diagnosis of severe symptomatic aortic valve stenosis and coronary artery disease
• successful transfemoral transcatheter aortic valve replacement (TAVR) defined as the implantation of a single transcatheter aortic valve within the past 96 hours
• percutaneous coronary intervention (PCI) already completed less than 90 days before TAVR
• planned PCI or cardiac surgery
• additional significant heart or medical diagnosis (study team will review)
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.
• 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
Intermittent Pneumatic Compression with and without Exercise to Improve Functioning in Peripheral Artery Disease: The INTERCEDE TRIAL (INTERCEDE)
The purpose of this study is to establish whether the device, intermittent pneumatic compression (IPC), both with and without walking exercise, can improve the ability to walk and prevent decline in the ability to walk for people with PAD. Intermittent pneumatic compression consists of blood pressure cuffs that are wrapped around the lower legs (below the knees) that inflate three times per minute. These cuffs may improve blood flow to the lower legs and feet.
• diagnosis of Peripheral Artery Disease (PAD) based on specific criteria that study staff will review
• above or below-knee amputation
• wheelchair-bound
• currently have a foot ulcer on bottom of foot or ongoing infection of the toes, foot, or lower extremity
• walking is limited by a symptom other than PAD
• major surgery, revascularization, or orthopedic surgery in the past 3 months or planned in the next 12 months
• already exercising at a level consistent with exercise intervention. Current or recent participation in exercise rehabilitation (within the past three months)
• non English speaking
• BMI greater than 45kg/M2
• major medical illness including Parkinson's Disease, lung disease requiring oxygen, cancer requiring treatment in the previous two years, or life-threatening illness (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.
• 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)
Impact of Inflammatory Bowel Disease and IBD-related Surgery on Female Fertility: A Prospective Registry
This is a prospective questionnaire study that will examine the impact of IBD and its surgical treatments on fertility in women with IBD of reproductive age (18-45). Women with IBD who are attempting to get pregnant within the next year will answer questionnaires about their general, surgical and reproductive health, conception history and sexual partner information. This will help identify risk factors predictive of fertility outcomes and early fetal loss, determine time to conception, and assess efficacy of fertility services including in vitro fertilization.
• age 18-45 years
• diagnosis of inflammatory bowel disease (IBD)
• not currently pregnant or trying to get pregnant
• desire to become pregnant within the next 12 months (may become pregnant while in the study)
• prior pelvic surgery, including surgery of the cervix, ovary, uterus or fallopian tubes
• known female sterility
• known male infertility factors in partner
Defining Clinical Endpoints in LGMD (GRASP-01-001)
The purpose of this study to learn more about Limb Girdle Muscular Dystrophy by measuring how muscles change over a twelve-month period. Our clinical evaluator will test muscle strength and participants will be asked to complete a series of questionnaires to find out how Limb Girdle impacts daily activities. This information will help plan future studies and drug development for people with LGMD.
• 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)
Evaluate the perspectives of cancer survivor patients and caregivers on using an intrathecal drug delivery system as a continuum of pain management care using a qualitative study.
Targeted medication delivery near the spinal cord (intrathecal pump) may be offered for cancer pain treatment in carefully selected patients. Prior studies showed an improved functional status reduction in oral medications and their side effects. Cancer survivors receiving intrathecal pump treatment for pain are eligible to participate in the research and share their stories. After consenting, a interview (45 minutes by zoom) will be conducted before and after the treatment to improve our understanding of patient perceptions of pain treatment with an intrathecal pump.
• cancer survivors with pain for more than 3 months duration who may benefit from intrathecal pump treatment for pain
• people who are not eligible for treatment with an intrathecal pump
Phase II Multi-Institutional Study of Low-Dose (2Gy x 2) Palliative Radiotherapy in the Treatment of Symptomatic Bone metastases from Multiple Myeloma
There is no consensus on the radiation dose required to relieve the pain from bone lesions from multiple myeloma. Usually, patients receive between 10 and 12 radiation treatments to achieve pain relief. But a shorter course of radiotherapy may be more effective. This study will evaluate whether pain relief can be achieved using only 1 or 2 radiation treatments, which will be delivered to a painful bone lesion. Your study doctor will decide whether you receive 1 or 2 treatments. The total amount of radiation you will receive will be the same whether it is done in one or two treatment sessions.
• diagnosis of multiple myeloma
• painful bone metastasis that has been confirmed by a xray
• may have had any number of prior chemotherapy/immunotherapy regimens
• at least able to walk and do all selfcare but may be unable to carry out any work activities; up and about more than 50% of waking hours
• see link to clinicaltrials.gov for additional requirements
• received prior radiation therapy or prior palliative surgery to the bone lesion that is causing pain
• pathologic fracture or impending fracture at the area of the bone lesion causing pain
• women who are pregnant
• additional criteria apply (study staff will review)
Neuroplasticity in REM Sleep Behavior Disorder
REM sleep behavior disorder may predict the eventual symptom development of Parkinson’s disease, dementia with Lewy bodies, or multiple system atrophy. This occurs over years to decades and the sleep disorder may hide other typical symptoms and result in a delay in diagnosis. We are studying the changes in the brain over two years. We will do high field MRI’s (7T) and other tests of neurological function of people who have REM sleep disorder and people who don’t have this disorder (matched for age and sex).
• Diagnosis of isolated iRBD confirmed by sleep study
• Able to walk independently without the use of an assistive device (e.g., cane) for at least 50 meters.
• 21 to 75 years old
• Diagnosis of Dementia
• History of musculoskeletal disorders that significant affect movement of lower or upper limbs
• Other significant neurological disorders
• Anti-depressant associated RBD. Individuals will be excluded if their dream enactment emerged or clearly worsened after initiating an antidepressant medication.
• Meet criteria for overt Parkinson's disease, dementia with Lewy bodies, Multiple Systems Atrophy, Alzheimer's disease, or other neurodegenerative disorder, or other known cause of RBD (e.g., narcolepsy and drug induced RBD).
• Untreated sleep-disordered breathing
• Pregnant women
Fully Automated Motion-corrected MR Spectroscopy in Human Brain and Spinal Cord
The goal of this proposal is to develop fully automated, high performance, motion-corrected MRS sequences for the brain and spinal cord, that are also easy to share (no additional external hardware needed) with other institutions and easy to use.
• Participants who cannot have an MRI, as determined by the CMRR safety screening form (e.g. metal implant)
• Pregnancy
• Claustrophobia
• Inability or unwillingness to complete an MRI because of low cognitive function or behavioral dysregulation
• Diabetes that has been diagnosed within the past 3 months (diabetes is OK if it is stably controlled (per participant report of either HbA1c <7.0 or stable control for at least 3 months))
• Hearing loss sufficient to prevent communication via telephone
• Weight > 250 and BMI > 35.
• Uncontrolled high blood pressure (>170/100) or working with doctor to stabilize blood pressure
• Severe lung, liver, kidney or heart disease of other major organ failure.
• Head size > 23.25 inches
Neural and Behavioral Markers of the Temporal Dynamics of Language Planning and Phonological Working Memory Processes in Persons Who Stutter
This is a study of persons who do and do not stutter between ages 7 and 65 years. This study is trying to figure out how attention skills influence memory, language, and speech fluency skills.
• age 7 through 65 years
• remaining eligibility will be determined based on response to an intake survey
teleABLE: Adapting a Behavioral Activation-Based Intervention to Reduce Post-Stroke Sedentary Behavior Using Telehealth (Formative Phase) (teleABLE)
We are exploring ways to increase physical activity after stroke using a web-based rehabilitation program. The purpose of this study is to adapt the Activating Behavior for Lasting Engagement (ABLE) program using video visits so people with stroke can participate from home.
• experienced a stroke in the last 12 months
• currently spend at least 6 hours per day sitting
• access to a device for virtual video visits
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 being sponsored by the National Pediatric Cardiology Quality Improvement Collaborative (NPC-QIC). We want to learn more about improving the treatment and outcomes for patients who have a diagnosis of a single ventricle, such as Hypoplastic Left Heart Syndrome (HLHS) or similar heart defects.
• up to 15 months old
• newborns diagnosed with HLHS or other univentricular condition
• intended to undergo Norwood procedure
Composur, A Patient-centric, Phase IV, Open-label, Prospective, Real World US Study to Evaluate Vibegron on Patient Treatment Satisfaction, Quality of Life, and Healthcare Resource Utilization in Patients with Overactive Bladder
This study will evaluate treatment satisfaction, discontinuation, reasons for discontinuation, quality of life, healthcare resource utilization, and safety with vibegron for the treatment of OAB in the context of real-world clinical practice.
• diagnosis of overactive bladder (OAB) with or without urgency urinary incontinence
• symptoms of OAB for at least 3 months
• specific previous treatments for OAB (study staff will review)
• neurologic conditions associated with OAB symptoms, e.g., multiple sclerosis
• women who are pregnant or breast feeding or planning to become pregnant
PEPN2011 - A Phase 1/2 Study of Tegavivint (IND#156033, NSC#826393) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors
This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating children, adolescents, and young adults with recurrent or refractory solid tumors, including lymphomas and desmoid tumors.
• 12 months to 30 years old
• patients with recurrent or refractory solid tumors including non-Hodgkin lymphoma and desmoid tumors are eligible
• patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• pregnant or breast-feeding women
• patients who are currently receiving other anti-cancer agents
• patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant
• patients with primary brain tumors
• patients who have received a solid organ transplant
HEALthy Brain and Child Development Study
We are doing a study to learn how children develop early in life. There will be about 300 parent-child pairs in this study in Minnesota and about 7,500 pairs total across the country. There will be study visits from pregnancy through the first 10 years of your child’s life.
• at least 18 years old
• currently pregnant
• speak English
EPI-MINN: Targeting Cognition and Motivation in Coordinated Specialty Care for Early Psychosis: A National Comparison Study
This is a study enrolling patients receiving care for early psychosis across the United States. Participants will be randomly assigned to one of two groups. The first group will use two mobile apps, computerized brain training and a motivational smart phone app, for a period of 12 weeks. The other group will participate in their regular clinical care. There will be 4 study timepoints: intake, post-training, 6-month follow up, and 12-month follow up. There is also an optional interview about experiences of loneliness.
• aged 15-40 inclusive
• enrolled in an early psychosis coordinated specialty care clinic (PI will determine this)
• in good physical health & stable psychiatric status
• fluent in spoken and written English
• have access to a smart phone (or other mobile device) and computer or tablet
• participated in significant cognitive training programs within the last three years
• neurological disorder that may interfere with participation
• substance abuse disorder that would interfere with participation
• risk of suicidal behavior
Oxidative Stress Markers in Heart Failure II
This research is being done to better understand why people develop a type of heart failure where the heart contracts normally but does not relax well. By comparing levels of chemicals in the blood that are secreted by the body in subjects with normal hearts and in subjects with abnormal relaxation, we hope to gain a better understanding of why heart failure occurs.
• Looking for both healthy and diastolic dysfunction participants who have had an echo in the past 6 months
• Age greater than or equal to 18 years
• Transthoracic echocardiogram within 1 year prior to enrollment containing tissue Doppler, mitral inflow velocities, left ventricular ejection fraction and left ventricular end?diastolic volume index data
• Able to provide written consent
• Healthy patients with an E/e? ratio < 15
• Patients with asymptomatic diastolic dysfunction with an E/e? ratio > 15
• Able to give a blood sample
• EF greater than or equal to 50%
• EF<50%
• Any regional wall motion defects, any valvular heart disease with greater than a mild stenosis or regurgitation, any congenital or other significant structural heart disease,
• Patients undergoing cancer treatment
• Patients with an anticipated life expectancy less than 18 months.
• Age < 75 years
• Previous hospital admission for acute heart failure
• History of NYHA Class II, III or IV functional status
• The need for loop diuretics specifically for heart failure at any time.
• History of congestive heart failure.
• History of coronary artery disease.
• History of myocardial infarction.
• Significant structural heart disease
• Evidence of infiltrative cardiac disease
• Atrial fibrillation (AF) within 6 weeks
• Rhythm other than sinus at enrollment
• Patient with a pacemaker
• Cardiogenic shock
• History of heart transplant or left ventricular assist device
• Hemodialysis or peritoneal dialysis
• Active infection including bacteremia
• Major trauma or surgery within 6 weeks
• Collagen vascular disease if on active treatment including steroids and other immunomodulating drugs
• Systemic steroid use within 6 week.
SpHincterotomy for Acute Recurrent Pancreatitis (SHARP) Trial (SHARP)
We are studying the effectiveness of a new procedure to treat people who have episodes of acute pancreatitis with pancreas divisum. Of the participants, half will receive the new procedure called endoscopic retrograde cholangiopancreatography (ERCP) with minor papilla endoscopic spincterotomy (miES) the other half with receive only ERCP. We will monitor outcomes for at least eighteen months.
• at least 18 years old
• two or more episodes of acute pancreatitis, with one occurring in the last 24 months
• there is no certain explanation for recurrent acute pancreatitis
• prior minor papilla therapy (endoscopic or surgical)
• other causes of pancreatitis (study staff will review)
• regular use of opioid medication for abdominal pain for the past three months
RANDOMIZED NON-INFERIORITY TRIAL COMPARING OVERALL SURVIVAL OF PATIENTS MONITORED WITH SERUM TUMOR MARKER DIRECTED DISEASE MONITORING (STMDDM) VERSUS USUAL CARE IN PATIENTS WITH METASTATIC HORMONE RECEPTOR POSITIVE HER-2 NEGATIVE BREAST CANCER
This study is looking at how well serum tumor markers work to monitor people who have hormone receptor positive Her2 negative breast cancer that has spread to other places in the body. We want to see if using the markers (from a blood sample) is as good as using scans to monitor disease.
• diagnosis of hormone receptor positive (estrogen receptor positive [ER+] and/or progesterone receptor positive [PR+]), HER-2 negative, metastatic (M1) breast cancer
• receiving or plan to receive first-line systemic treatment for metastatic disease
• no other prior malignancy is allowed except for adequately treated basal (or squamous cell) skin cancer, in situ cervical cancer or other cancer for which the patient has been disease free for five years
• known cirrhosis, untreated B12 deficiency, thalassemia, or sickle cell anemia
• known brain leptomeningeal metastases
• must not be pregnant
EA5163/S1709 INSIGNA: A Randomized, Phase III Study of Firstline Immunotherapy alone or in Combination with Chemotherapy in Induction/Maintenance or Postprogression in Advanced Nonsquamous Non Small Cell Lung Cancer (NSCLC) with Immunobiomarker SIGNature driven Analysis
We are studying the use of pembrolizumab to treat people who have stage IV non-squamous, non-small cell lung cancer. Pembrolizumab may help the body’s own immune system attack cancer so tumor cells cannot grow and spread. We are looking at when it is most effective to give the pembrolizumab and when to combine it with other anticancer drugs, pemetrexed and carboplatin.
• confirmed stage IV non-squamous non-small cell lung cancer (NSCLC)
• PD-L1 expression Tumor Proportion Score (TPS) >= 1% in tumor cells -
• Prior systemic chemotherapy or immunotherapy for advanced metastatic NSCLC
• EGFR mutations (except exon 20 insertion), BRAF mutations (V600) or ALK or ROS1 translocations
• significant gastrointestinal disorders with diarrhea as a major symptom
• history of auto-immune condition (including Guillain-Barre Syndrome or Multiple Sclerosis) requiring ongoing or intermittent systemic treatment in the past 2 years
• pregnant or breast-feeding
A Randomized Phase II Study Comparing Single-Agent Olaparib, Single Agent Cediranib, and the Combination of Cediranib/Olaparib in Women with Recurrent, Persistent or Metastatic Endometrial Cancer
This study uses different drug combinations to treat women who have endometrial cancer that has come back or has not responded to treatment. The drugs have different ways of stopping the growth of tumor cells and we are looking to see if different combinations are more effective.
• recurrent or persistent endometrial carcinoma, which is refractory to curative therapy or established treatments
• following histologic epithelial cell types are eligible: endometrioid adenocarcinoma, serous adenocarcinoma, undifferentiated carcinoma, mixed epithelial carcinoma, adenocarcinoma not otherwise specified (N.O.S.); NOTE: clear cell histology is excluded
• must have had one prior chemotherapeutic regimen for management of endometrial carcinoma
• Body weight > 30 kg
• able to swallow and retain oral medications and without gastrointestinal illnesses that would preclude absorption
• Prior enrollment into a clinical trial including cediranib or olaparib; Note: prior bevacizumab is not an exclusion criterion
• Pregnant women are excluded
A Randomized Phase III Trial of Adjuvant Therapy Comparing Doxorubicin Plus Cyclophosphamide Followed by Weekly Paclitaxel with or without Carboplatin for Node-Positive or High-Risk Node-Negative Triple-Negative Invasive Breast Cancer
We are studying the addition of a drug to the treatment for people who have triple-negative breast cancer. Drugs used in chemotherapy work in different ways to stop the growth of tumor cells. Some people will receive the current treatment and others will have the current treatment with carboplatin added. The results of the two treatments will be compared.
• breast tumor must have been determined to be estrogen receptor (ER)-and progesterone receptor (PgR)-negative
• tumor must have been determined to be human epidermal growth factor receptor 2 (HER2)-negative
• surgery (mastectomy (total, skin-sparing, or nipple-sparing) or lumpectomy) completed no more than 60 days from enrollment
• T4 tumors including inflammatory breast cancer
• clinical or radiologic evidence of metastatic disease
• previous history of invasive breast cancer or DCIS in the same breast
• Chemotherapy administered for the currently diagnosed breast cancer prior to randomization
Brain Connectivity Patterns in Chronic Temporomandibular Joint Disorders
We are studying brain mechanisms related to chronic temporomandibular disorder (TMD) pain. We are looking at brain structural and functional characteristics that can potentially explain why some people experience persistent pain in their jaws for months or years. We will compare this to information we get from people who do not experience TMD pain. We expect that this new knowledge will improve our understanding of this chronic pain condition and help us develop better treatments.
• Female participants only (biological sex)
• Age between 18-65 years
• Pain-free participants OR
• Chronic jaw pain ( > 3 months)
• Certain medical conditions (examples: heart disease, diabetes, autism, trigeminal neuralgia)
• Any MRI contraindications (examples: metal implants, claustrophobia)
• Being left-handed only
Assessments of sound perception and brain activation in response to paired sound and electrical stimulation of surface ear regions
We are investigating how paired non-invasive electrical stimulation of surface body regions and sound changes sound perception and tinnitus. Body stimulation regions include: external ear/behind the ear, shoulder, neck, forearm, hand, and upper arm. We aim to better understand the optimal conditions of this paired stimulation, which opens opportunities for applying this method to improving hearing loss or tinnitus. We are studying three groups of people: those with normal hearing, those with mild to moderate hearing loss, and those with tinnitus.
• normal hearing or hearing loss with or without tinnitus
• not users of Cochlear Implant or hearing aids
Choline Supplementation as a Neurodevelopmental Intervention in Fetal Alcohol Spectrum Disorders Study (CHOLINE4)
The main purpose of this study is to see whether choline can help cognitive development (thinking / memory) in children with Fetal Alcohol Spectrum Disorders (FASD). The study will compare choline with a placebo to see if taking choline is better than taking a placebo. The placebo in this study looks and tastes like choline but has no choline in it. Choline is a nutrient found in many foods and is grouped within the vitamin B complex.
• ages 2.5 years to 5 years old (<6 years of age)
• prenatal alcohol exposure
• history of a neurological condition (epilepsy, traumatic brain injury)
• history of a medical condition known to affect brain function
• other neurodevelopmental disorder (autism, Down syndrome)
• history of very low birthweight (<1500 grams)
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.
• 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)