Search Results
The Lung Cell Study (TLC)
The purpose of our research study is to increase our understanding on the human lung immune response to infection. We aim to use this knowledge to develop novel approaches on the prevention and treatment of lung infections. To accomplish this, we plan to obtain cells from the airways and blood of healthy volunteers to perform immune experiments.
• healthy volunteers without systemic or respiratory disease
• non-smokers or former smokers who quit more than 6 months ago (including marijuana)
• ages 18-50
• preferred but not required: have lived in countries in Africa, Latino America or South East Asia
• preferred but not required: history of positive tuberculosis (TB) skin or blood test
• recent illness, bleeding disorders, or a history of heart, lung, or systemic disease
• weakened immune system or use of immunosuppressant medication
• use of inhaled substances in the past six months or history of heavy smoking
• allergy or prior adverse reaction to lidocaine, midazolam, or fentanyl
• pregnancy
A Parallel-Arm, Phase 3, Open-Label, Active-controlled, Multicenter (Global), Randomized Clinical Trial to Investigate the Efficacy and Safety of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Participants with Growth Failure or Short Stature due to Growth Hormone Sufficient Causes- Turner Syndrome, SHOX Deficiency, Small for Gestational Age, and Idiopathic Short Stature
This study is evaluating two growth hormone treatments in children with short stature due to Turner syndrome, SHOX deficiency, being born small for gestational age without catch-up growth, or idiopathic short stature. Researchers want to compare how well the two treatments support growth, monitor their safety, and better understand how they affect the body. Participation in the study will last approximately 52 weeks.
• 2 to 18 years of age
• diagnosed with Turner syndrome, SHOX deficiency, born small for gestational age without catch-up growth, or idiopathic short stature
• have not previously received growth hormone or growth hormone-promoting therapy
• see link to ClinicalTrials.gov for complete inclusion criteria
• receiving medications or have a medical condition that may affect growth
• uncontrolled diabetes, thyroid disease, or other significant medical conditions
• pregnant or breastfeeding
• see link to ClinicalTrials.gov for complete exclusion criteria
CATALINA-2: A Phase 2 Study Evaluating the Efficacy and Safety of TORL-1-23 in Women with Advanced Platinum-Resistant Epithelial Ovarian Cancer (Including Primary Peritoneal and Fallopian Tube Cancers) Expressing Claudin 6
This study is being conducted to determine the safest and most effective dose of TORL-1-23 in treating advanced platinum-resistant ovarian cancer.
• diagnosis of advanced (unresectable) or metastatic (has spread) high grade serous ovarian, primary peritoneal (i.e, of primary origin), or fallopian tube cancer
• positive for CLDN6 expression
• have platinum-resistant disease
• may not be able to do strenuous activity but able to walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• must agree to use a highly effective birth control method from the time of the first study drug treatment through 7 months after the last study drug treatment
• must agree to not breastfeed from the first dose of study treatment through 90 days after the last dose of study treatment
• see link to clinicaltrials.gov for complete inclusion criteria
• have not recovered from serious side effects of previous treatment
• progressive or symptomatic brain metastases
• history of significant heart disease
• history of another cancer within 3 years (exception of basal or squamous cell carcinoma of the skin)
• see link to clinicaltrials.gov for complete exclusion criteria
HM2025-16: Phase Ib/II Trial of Epcoritamab plus Ibrutinib in Patients with Relapsed/Refractory Aggressive B-cell Non-Hodgkin Lymphoma.
This phase Ib/II trial evaluates the safety, optimal dose, and efficacy of the combination of epcoritamab and ibrutinib in treating patients with aggressive B-cell non-Hodgkin lymphoma that has come back (relapsed) or responded to previous treatment (refractory).
• diagnosis of B-cell non-Hodgkin lymphoma
• have relapsed or refractory aggressive B-cell lymphoma and received prior treatment with an anthracycline in combination with an anti-CD20 monoclonal antibody
• treatment with autologous stem cell transplant (ASCT) is allowed if ≥ 100 days
• capable of all selfcare and able to walk but unable to carry out any work activities; up and about more than 50% of waking hours
• women of child-bearing potential and men must agree to use adequate contraception 2 weeks before starting treatment, for the duration of study participation and for 12 months after completing treatment
• see link to clinicaltrials.gov for complete Inclusion criteria
• active central nervous system (CNS) involvement
• active uncontrolled infection
• current uncontrolled or symptomatic cardiovascular conditions
• liver cirrhosis with moderate to severe liver impairment
• significant lung disease
• treatment with coumadin/warfarin
• women who are pregnant or breastfeeding
• see link to clinicaltrials.gov for complete Exclusion criteria
An Open-Label Multiple-Dose, 52-Week Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of XYOSTED® for Testosterone Replacement in Male Adolescents (ages: 12 to <18 years) with Conditions Associated with Deficiency or Absence of Endogenous Testosterone Due to Primary or Secondary Hypogonadism (Congenital or Acquired)
This is a study to see if a new drug, named XYOSTED Injection (study drug) will help in the treatment of male adolescents ages 12 to less than 18 years old who have low or no testosterone due to a medical condition called Hypogonadism. Male Hypogonadism is a condition in which the body doesn’t produce enough of the hormone called testosterone that plays a key role in masculine growth and development during puberty. Participation in the study will last 52 weeks.
• 12 to < 18 years of age
• diagnosed with a deficiency or absence of testosterone due to hypogonadism
• if receiving testosterone treatment, must be on a stable dose for at least 12 weeks before starting the study
• body mass index (BMI)-for-age greater than the 5th percentile and weigh at least 40 kg (88 pounds)
• see link to clinicaltrials.gov for complete Inclusion criteria
• suspected or known constitutional growth delay in growth and puberty (CDGP)
• possible nutritional or gastrointestinal disorder that may impact growth
• allergy to foods or products containing sesame seeds or sesame oil
• history of suicidal behavior suicide attempts
• have a history of drug or alcohol abuse
• see link to clinicaltrials.gov for complete Exclusion criteria
Characterization of spleen motion and anatomy using imaging and sensors
This research is being performed to examine how the spleen moves during breathing in various body positions and breathing conditions. Physical measurements of the participant's body will be recorded (weight, height, and body dimensions) and then noninvasive recordings of the spleen and breathing patterns will be recorded. The spleen motion will be measured using standard abdominal ultrasound imaging, and breathing will be measured with accelerometers (small devices about the size of a quarter that measure the movement of the chest during breathing).
• at least 18 years old
• individuals who have had a splenectomy
• people with breathing difficulties and/or individuals for whom short breath holds and modification of breathing patterns is difficult or uncomfortable
• unable to maintain five body positions: sitting, sitting with a 45 degree recline, laying on back (supine), laying on right side, and laying face down (prone) comfortably and independently
• unable to speak and read English
A Randomized, Double-Blind, Multicenter, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Atumelnant in Adult Participants with Classic Congenital Adrenal Hyperplasia
The purpose of this research study is to evaluate the safety and effectiveness of a new investigational drug called atumelnant for adults with classic Congenital Adrenal Hyperplasia (CAH) caused by 21-hydroxylase deficiency (21-OHD). Researchers want to learn how well atumelnant helps control CAH symptoms, how the body processes the drug (pharmacokinetics or PK), and whether it produces the expected effects in the body (pharmacodynamics or PD). The study will also evaluate whether participants may be able to reduce their steroid (glucocorticoid) dose while taking atumelnant.
• diagnosis of classical congenital adrenal hyperplasia (CAH)
• on a stable dose of your current steroid (glucocorticoid) treatment
• see link to clinicaltrials.gov for complete inclusion criteria
• diagnosis of any other form of CAH
• are pregnant, breastfeeding, or unable/unwilling to use effective birth control during the study
• see link to clinicaltrials.gov for complete exclusion criteria
COG APEC14B1 The Project: Every Child Protocol: A Registry, Eligibility Screening, Biology and Outcome Study Additional Title: EVERYCHILD (APEC14B1) PCR - COG Foundation
This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.
• must be =< 25 years of age at time of original diagnosis, except for patients who are being screened specifically for eligibility onto a COG (or COG participating National Clinical Trials Network [NCTN]) therapeutic study, for which there is a higher upper age limit
• patients with a known or suspected neoplasm that occurs in the pediatric, adolescent or young adult populations
• enrollment must occur within 6 months of initial disease presentation OR within 6 months of refractory disease, disease progression, disease recurrence, second or secondary malignancy
• see link to clinicaltrials.gov for additional inclusion criteria
A pivotal Phase 3, multicenter, randomized, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with focal segmental glomerulosclerosis (FSGS) who are receiving an angiotensin II receptor blocker (ARB) (ACTION3)
A clinical research study for primary focal segmental glomerulosclerosis (FSGS), or genetic focal segmental glomerulosclerosis (FSGS), or focal segmental glomerulosclerosis (FSGS) of undetermined cause in pediatric (12-17 years) and adult patients. Eligible participants will be assigned to receive either DMX-200 (repagermanium) or placebo (50/50 chance) over a treatment period, with total participation up to 28 month, with potential for participation in an Open Label Extension study period. The main purpose of this study is to see if DMX-200 reduces proteinuria and slows the loss of kidney function in those with FSGS.
• 12-80 years old;
• Primary FSGS, genetic FSGS or FSGS of undetermined cause
• Receiving an ARB, or willing to take one for the study
• see link to clinicaltrials.gov for complete inclusion criteria
• Secondary FSGS
• Not previously treated with standard of care therapies (including steroids)
• Unable to swallow oral medication
• see clinical to clinicaltrials.gov for complete exclusion criteria
NRG-GU012: Randomized Phase II Stereotactic Ablative Radiation Therapy (SABR) for Metastatic Unresected Renal Cell Carcinoma (RCC) Receiving Immunotherapy (SAMURAI) (SAMURAI)
The primary purpose of this study is to find out if adding a highly focused form of radiation therapy (called SABR) to the main kidney tumor, along with immunotherapy, helps people with advanced kidney cancer live longer or do better than using immunotherapy by itself.
• diagnosis of renal cell cancer with positive lymph nodes that can't be removed or metastasis to another area
• not recommended for or refused immediate nephrectomy
• must agree to use a highly effective contraception while on study drug and for 6 months following the last dose of study drug
• see link to clinicaltrials.gov for complete inclusion criteria
• untreated or unstable brain metastases
• prior radiotherapy to the kidney
• systemic therapy for metastatic renal cell carcinoma (RCC) that was initiated > 90 days before starting the study
• women who are pregnant or breastfeeding -see link to clinicaltrials.gov for complete exclusion criteria
DCIS: RECAST Trial Ductal Carcinoma In Situ: Re-Evaluating Conditions for Active Surveillance Suitability as Treatment: a breast cancer prevention pilot study
The trial offers women with ductal cell carcinoma in situ (DCIS) 6 months of neoadjuvant exposure to endocrine therapy with the intent of determining their suitability for long-term active surveillance without surgery.
• diagnosis of HR+ DCIS (at least 50% ER or PR (from biopsy at diagnosis) with or without microinvasion
• may have received endocrine therapy
• see link to clinicaltrials.gov for complete Inclusion criteria
• women who are pregnant or breast feeding
• breast cancer is invasive
• unable to swallow tablets or capsules
• gastrointestinal conditions that would interfere with absorption of medication -- see link to clinical trials.gov for complete Exclusion criteria
Zymfentra (Infliximab-dyyb) REal World Cohort STudy (ZEST)
The goal of this study is to learn more about the safety and effectiveness of Zymfentra treatment for people with inflammatory bowel disease (IBD), Crohn’s disease, and ulcerative colitis. Researchers will collect information about symptoms, treatment response, and side effects over time to better understand how Zymfentra works for people with IBD.
• diagnosis of Crohn’s disease (CD), ulcerative colitis (UC), or inflammatory bowel disease unclassified (IBDU)
• starting or switching to Zymfentra treatment for inflammatory bowel disease
An open, parallel-group, randomized, early feasibility trial to evaluate the initial safety and performance of the INGA catheter in labor induction
The purpose of this study is to evaluate the safety and effectiveness of a new device used during labor induction. We will enroll pregnant adults who are at least 37 weeks pregnant and are scheduled to have labor induced using a balloon catheter. The study will evaluate whether the device can improve monitoring of the pregnant person and baby during labor.
• pregnant adults ages 18–56
• at least 37 weeks pregnant
• pregnant with one baby
• planning to have labor induced using a balloon catheter
• pregnant with more than one baby (twins, triplets, etc.)
• baby is not in a head-down position
• previous cesarean section or other surgery that left a scar on the uterus
• you or your baby need immediate delivery for medical reasons
A PHASE 2 STUDY OF ALISERTIB IN COMBINATION WITH ENDOCRINE THERAPY IN PATIENTS WITH HR+, HER2-NEGATIVE RECURRENT OR METASTATIC BREAST CANCER (ALISCA-Breast1)
The purpose of this study is to see if the study drug, called alisertib, in combination with an ‘endocrine therapy’ such as anastrozole, letrozole, exemestane, tamoxifen or fulvestrant can help people with HR+, HER2-negative recurrent or metastatic breast cancer. The study will also look at how well people tolerate treatment with alisertib in combination with one of the endocrine therapies that are commonly used in clinical practice.
• diagnosis of adenocarcinoma of the breast that has reoccurred of spread to other areas of the body (metastatic)
• treatment with at least two prior lines of endocrine therapy in the recurrent or metastatic setting
• see link to clinicaltrials.gov for complete inclusion criteria
• treatment with chemotherapy in the recurrent or metastatic setting
• see link to clinicaltrials.gov for complete exclusion criteria
Cell free and circulating tumor DNA in the management of muscle invasive bladder cancer, a prospective cohort study.
The goal of this study is to identify the role of cell-free fragments of DNA (cfDNA) and circulating tumor DNA (ctDNA) found in blood and/or urine of patients with bladder cancer in determining whether some patients can avoid chemotherapy and or radical cystectomy. Large numbers of samples need to be analyzed. Participants will be asked to provide additional blood and urine samples; no treatment is provided in this study.
• people who may have invasive bladder cancer
• planning to have a transurethral resection of bladder tumor (TURBT)
• women who are pregnant
• any other active cancer
• at least able to do self care, up and walking & carry out light work or sedentary activity
Molecular MRI Biomarkers in Parkinson's Disease
This study uses an advanced MRI scan to better understand how the brain produces and uses energy in people with Parkinson's disease (PD). What we learn may improve our understanding of PD and help guide future treatments.
• 40-80 years of age
• diagnosed with Parkinson's disease (PD)
• willingness to undergo brain MRI
• diagnosed with dementia
• have a severe tremor
• have a neurological disorder or condition other than Parkinson's disease
• pregnant or planning to become pregnant during the study
• foreign objects or metal in the body that would prevent MRI participation
A PHASE 3, MULTICENTER, RANDOMIZED, OPEN-LABEL, STUDY EVALUATING THE EFFICACY AND SAFETY OF NANVURANLAT IN PATIENTS WITH PREVIOUSLY TREATED ADVANCED BILIARY TRACT CANCER (Beacon-BTC)
This study is for people with advanced biliary tract cancer (cancer of the bile ducts or gallbladder) that cannot be removed with surgery or has spread to other parts of the body. Participants must have previously received one standard chemotherapy treatment for advanced disease. Researchers are studying an investigational medication called nanvuranlat to see if it is safe and effective for treating advanced biliary tract cancer. The study will compare nanvuranlat with standard treatment to learn how well it works, monitor its safety, and better understand how it affects the body and the cancer.
• diagnosed with advanced or metastatic biliary tract cancer (including intrahepatic, extrahepatic, or gallbladder cancer) that cannot be removed with surgery
• previously received one platinum-based chemotherapy treatment for advanced disease
• able to carry out light daily activities
• see link to clinicaltrials.gov for complete Inclusion criteria
• received certain cancer treatments, radiation therapy, or major surgery too recently before starting the study
• active or untreated brain metastases or uncontrolled serious medical conditions
• pregnant or breastfeeding
• see link to clinicaltrials.gov for complete Exclusion criteria
A Window of Opportunity Trial of Mirdametinib plus Vorinostat for NF1 Associated, Malignant Peripheral Nerve Sheath Tumor; MPNST (Le-Na)
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.
• Known Neurofibromatosis type 1 (NF-1) syndrome based on current diagnostic criteria
• Diagnosis of suspected MPNST by PET or MRI imaging
• Confirmation of histone H3 lysine 27 trimethylation-negative MPSNT by immunohistochemistry
• Twelve years of age or older - Complete blood count (CBC), platelet, liver and kidney function within institutional normal limits performed within 14 days of 1st dose of study drug
• Must be able to swallow capsules
• Females of childbearing potential must use highly effective contraception (see inclusion criteria section) from the time of study enrollment through 6 months after the last dose of vorinostat and mirdametinib
• Males with partners of childbearing potential must use highly effective contraception from the time of study enrollment through 3 months after the last dose of vorinostat
• Provides voluntary written consent prior to any study related activities, with parental/guardian consent and assent for those 12 to 17 years of age at enrollment
• Pregnant or breastfeeding – females of childbearing potential must have a negative pregnancy test (serum and urine) within 7 days prior to the 1st dose of the study drugs
• Significant cardiac disease
• Ophthalmologic conditions
• Radiation therapy or chemotherapy in the past year
• Participants receiving systemic or ocular glucocorticoid therapy within 14 days prior to the first dose of study treatment
Circuit-Based Deep Brain Stimulation for Parkinson's disease; Udall Clinical Core
The goal of this study is to provide comprehensive longitudinal assessments of a cohort of PD patients before, during, and after DBS surgery, including neurological, neurophysiological, and neuropsychological data.
• age 21 years and older
• diagnosis of Parkinson's disease
• approved candidate for standard of care DBS surgery at the University of Minnesota
• diagnosis of dementia
• women who are pregnant
An interventional Phase 3, open-label, two-cohort study to investigate the efficacy and safety of fosmanogepix in adult patients with invasive mold infections caused by Aspergillus spp., Fusarium spp., Lomentospora prolificans, Mucorales fungi, or other multidrug resistant molds
The purpose of this study is to compare the effects of the study drug fosmanogepix with that of other currently approved treatments to find out if fosmanogepix is safe and effective in treating participants with invasive mold infections. Participants will be assigned to one of two groups depending on the study treatment already received for the current mold infection. Participation in this study will be for a maximum of 8 months.
• diagnosis of proven or probable Invasive mold infection
• see link to clinicaltrials.gov for complete Inclusion criteria
• need or anticipated need for hemodialysis, peritoneal dialysis, or hemofiltration
• moderate or severe liver disease
• known human immunodeficiency virus infection
• women who are pregnant or breastfeeding
• see link to clinicaltrials.gov for complete Exclusion criteria
HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug. We are doing this research to find out if different treatments have an effect on Amyotrophic Lateral Sclerosis (ALS). We also want to find out if these treatments are safe to take without causing too many side effects.
• 18 years or older
• diagnosed with sporadic or familial ALS
• weakness started no more than 36 months ago -able to to swallow pills and liquids
• unstable medical or mental health condition
• limitations on prior or current use of certain medications (study staff will review)
• women who are pregnant or breast feeding
The University of Minnesota Perinatal Health Repository
This research is being done to collect blood and placenta samples to better understand how pregnancy impacts the health of mother and child. The goal of this research is to better understand what causes some pregnancy complications and how this impacts the longer term health of mothers and children.
• pregnant women who are at least 18 years of age and their neonates/children
• seen in Obstetrics and Gynecology Clinics for pre-conception, prenatal or postpartum care
Studying causal inference, affordances, and planning by playing Tetris
Researchers are studying how the brain processes visual information and plans actions. Participants will play the popular arcade game Tetris and watch replays of their gameplay while researchers measure brain activity using a safe, non-invasive EEG. The results may help researchers better understand how the brain solves problems and plans future actions.
• age 18-36 years old
• right-handed
• normal (or corrected) vision
• normal hearing
• history of epilepsy
Use of CA125 and Complementary biomarkers for the early detection of Ovarian Cancer in Low Risk Women (ID01-022)
This study aims to evaluate the ROCA2 algorithm, which calculates a woman's risk of having ovarian cancer based on age and biomarkers, for the early detection of ovarian cancer in a low risk cohort of women.
• women age 50 to less than 75 years old
• postmenopausal, which means no period for one year or longer
• have at least one ovary
• cancer-free and have not received any chemotherapy or radiation therapy for at least 12 months, exceptions may be available
• have a health care provider who does yearly well-woman exam
• willing to have a biomarker blood test yearly
• willing to have a transvaginal ultrasound if indicated by screening test
• prior removal of both ovaries
• have an active non-ovarian cancer
• high risk for ovarian cancer due to known familial predisposition (known mutation in BRCA1 or BRCA2, family history of ovarian or breast cancer). Study staff will review
EX-VIVO TARGETED TREATMENT OF CANCEROUS EXTIRPATED PROSTATE TISSUE
This study focuses on testing new treatments on prostate tissue that has been removed during surgery. All testing is done in a lab setting to help develop and improve potential treatment options.
• age 18 or older
• scheduled to undergo a radical prostatectomy
• able to understand and provide informed consent
AREN2231; Risk Adapted Treatment of Unilateral Favorable Histology Wilms Tumors (FHWT)
This study aims to improve the outcomes within cohorts of favorable-histology Wilms tumor (FHWT) patients, and maintain those outcomes despite therapy reduction. In this case, an improved outcome would be considered an improvement in event-free survival rates (EFS). Patients will be stratified to different treatment groups based upon age, response to treatment, and other factors. This trial will include six chemotherapy treatment regimens, and there will also be expansion and refinement of the cohort of patients who are treated with nephrectomy only. Exploratory aims will address aspects such as radiology, surgery, radiation oncology, pathology, and the biological aspects of FHWT.
• Age: Patients must be more than 30 years old
• Diagnosis: Patients with newly diagnosed Stage I-IV - Favorable Histology Wilms Tumor confirmed by central review and with a qualifying Initial Stratum Assignment received on APEC14B1-REN
• Patient with a diagnosis of Stage V Bilateral Wilms Tumor
• Patients who in the opinion of the investigator are not able to comply with the study procedures are not eligible -Patients with any uncontrolled, intercurrent illness including but not limited to symptomatic congestive heart failure
• Patients with Stage I FHWT with a known or suspected Wilms Tumor predisposition syndrome or condition (contralateral nephrogenic rests and/or unilateral multicentric tumors) are excluded from treatment on the mVLR (Nephrectomy Only) arm
A Randomized, Double-blinded, Multiregional Phase 3 Study of Ivonescimab Versus Pembrolizumab for the First-line Treatment of Metastatic Non-small Cell Lung Cancer in Patients Whose Tumors Demonstrate High PD-L1 Expression (HARMONi-7)
The purpose of this research is to measure the safety and effectiveness of ivonescimab compared to pembrolizumab. Ivonescimab is an antibody designed to block proteins that help cancer cells grow and spread, and by blocking these proteins may potentially slow cancer progression. Participants will have a 50% chance of being assigned to either the ivonescimab treatment group or the pembrolizumab treatment group.
• may be restricted in physically strenuous activity but able to walk and carry out work of a light or sedentary nature, e.g., light house work, office work
• diagnosis of metastatic (Stage IV) Non-Small Cell Lung Cancer (NSCLC)
• have not received any treatment
• see link to clinicaltrials.gov for complete Inclusion criteria
• small cell lung cancer
• known genetic alterations that have treatment options
• have received treatment for this cancer
• see link to clinicaltrials.gov for complete Exclusion criteria
MOMENTOUS study (iMpact Of an ecg ai ModEl oN The diagnosis Of pUlmonary hypertenSion) (MOMENTOUS)
The purpose of this study is to understand if a new tool will help improve early detection of pulmonary hypertension (PH) in patients with interstitial lung disease. Pulmonary hypertension is high blood pressure in the pulmonary artery (which carries blood from your heart to the lungs). We are testing a tool that looks at an electrocardiogram (ECG) to find patients who are identified as being high risk for having PH that is not yet diagnosed. Participation in this study will last approximately six months.
• diagnosis of interstitial lung disease
• diagnosis of pulmonary hypertension
• LV ejection fraction ≤ 40% on most recent echo or cardiac MRI
EFC17574: A Phase 3, single-arm, multicenter, multinational, open label, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged >= 12 years with severe hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX (ATLAS-NEO)
A study to test a medicine (fitusiran) injected under the skin for preventing bleeding episodes in male adolescent or adult participants with severe Hemophilia.
• 12 years or older
• diagnosis of severe congenital hemophilia A or B
• participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months
• co-existing bleeding disorders other than congenital hemophilia A or B
• current participation in immune tolerance induction therapy (ITI)
• prior treatment with gene therapy
• acute hepatitis, ie, hepatitis A, hepatitis E, acute or chronic hepatitis B infection
• additional exclusion criteria apply (study staff will review)
MT2022-27: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
People planning to undergo a bone marrow transplant and are at risk for developing problems with the lungs related to this planned therapy. The types and seriousness of lung problems that may develop after transplant can be very different and currently, we don’t exactly know what risk factors influence who develops them or how they may respond to therapy. Also, we do not know what the best test is to monitor lung function after transplant, especially in children and young adults. The purpose of this study is to help investigators learn more about lung problems after bone marrow transplant including what is the best method for diagnosing lung problems and following how well the lungs are working. In this study, clinical information, laboratory results and imaging findings will be collected from medical records to assist researchers in learning more about lung complications after bone marrow transplant.
• up to 24 years old
• undergoing allogeneic or autologous HSCT