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Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases...
National Human Genome Research Institute (NHGRI)
Genetic Disease
Background:
- Without an explanation for severe and sometimes life-threatening symptoms, patients and
their families are left in a state of unknown. The NIH helped create a network of medical
research centers, called the Undiagnosed Diseases Network (UDN), to provide care... expand
Background: - Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. The NIH helped create a network of medical research centers, called the Undiagnosed Diseases Network (UDN), to provide care and answers for these individuals. Objectives: - To improve diagnosis and care for people with undiagnosed diseases. Eligibility: - People with undiagnosed diseases, and their relatives. Design: - Participants will travel to one of the UDN medical centers for a 5-day clinical and research visit. - As part of the visit, UDN healthcare providers may ask participants to have: - Clinically indicated tests and procedures performed including: - A physical exam - Blood and urine tests - A review of health and family history - X-rays and body scans - Surveys - Photographs of the face and body - A special diet to see if the body can handle the food without having a reaction, like vomiting - Video or voice recordings - Other tests and procedures to help reach a diagnosis - Research tests and procedures performed including: - A skin biopsy. For this, a small piece of skin will be taken. - Surveys - Other tests and procedures for research that may not be related to a diagnosis or treatment. - Most participants will be asked to give samples for genetic testing. - Participants may be contacted after their visit to discuss test results. They may also be contacted in the future for interviews and surveys. - Relatives of participants may be asked to give samples for genetic testing. They may be asked to have parts of their visit recorded and to have additional tests. They may also be contacted in the future for interviews and surveys. - Clinical and research information collected will be stored in a database. - Information and samples collected will be shared with others for research purposes.... Type: Observational Start Date: Sep 2015 |
GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With...
Hoffmann-La Roche
Huntington Disease
This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with
placebo in participants with prodromal and early manifest Huntington's Disease
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This study will evaluate the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington's Disease Type: Interventional Start Date: Jan 2023 |
A Study of ANV419 Alone or in Combination With Approved Treatment in Patients With Cutaneous Melanoma...
Anaveon AG
Melanoma (Skin)
Cutaneous Melanoma
Adult Disease
Advanced Solid Tumor
Metastatic Melanoma
The purpose of this study is to evaluate the efficacy and safety of ANV419 monotherapy or the
combination of ANV419 with anti-PD1 antibody or with anti-CTLA4 antibody in adult
participants with advanced (unresectable or metastatic) cutaneous melanoma.
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The purpose of this study is to evaluate the efficacy and safety of ANV419 monotherapy or the combination of ANV419 with anti-PD1 antibody or with anti-CTLA4 antibody in adult participants with advanced (unresectable or metastatic) cutaneous melanoma. Type: Interventional Start Date: Dec 2022 |
Evaluation of BE1116 in Patients With Traumatic Injury and Acute Major Bleeding to Improve Survival (...
CSL Behring
Traumatic Injury
This is a prospective, multicenter, randomized, double-blind, placebo-controlled,
parallel-group, large simple trial to investigate the efficacy and safety of a single
intravenous (IV) infusion of BE1116 in subjects who have traumatic injury, with confirmed or
suspected acute... expand
This is a prospective, multicenter, randomized, double-blind, placebo-controlled, parallel-group, large simple trial to investigate the efficacy and safety of a single intravenous (IV) infusion of BE1116 in subjects who have traumatic injury, with confirmed or suspected acute major bleeding and / or predicted to receive a large volume blood product transfusion. Type: Interventional Start Date: Mar 2023 |
A Study of Aticaprant 10 Milligrams (mg) as Adjunctive Therapy in Adult Participants With MDD With Moderate-to-severe...
Janssen Research & Development, LLC
Depressive Disorder, Major
Anhedonia
The purpose of this study is to evaluate the efficacy of aticaprant compared with placebo as
adjunctive therapy to an antidepressant in improving depressive symptoms in adult
participants with major depressive disorder (MDD) with moderate to severe anhedonia (ANH+)
who have... expand
The purpose of this study is to evaluate the efficacy of aticaprant compared with placebo as adjunctive therapy to an antidepressant in improving depressive symptoms in adult participants with major depressive disorder (MDD) with moderate to severe anhedonia (ANH+) who have had an inadequate response to current antidepressant therapy with a selective serotonin reuptake inhibitor (SSRI) or serotonin-norepinephrine reuptake inhibitor (SNRI). Type: Interventional Start Date: Dec 2022 |
Study of Tecovirimat for Human Monkeypox Virus
National Institute of Allergy and Infectious Diseases (NIAID)
Monkeypox
A5418 is a randomized, placebo-controlled, double-blind study to establish the efficacy of
tecovirimat for the treatment of people with laboratory-confirmed or presumptive HMPXV
disease.
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A5418 is a randomized, placebo-controlled, double-blind study to establish the efficacy of tecovirimat for the treatment of people with laboratory-confirmed or presumptive HMPXV disease. Type: Interventional Start Date: Sep 2022 |
Study to Evaluate EP547 in Subjects With Cholestatic Pruritus Due to Primary Biliary Cholangitis or Primary...
Escient Pharmaceuticals, Inc
Pruritus
This phase 2 trial will evaluate the effects of EP547 in subjects with cholestatic pruritus
due to Primary Biliary Cholangitis (PBC) or Primary Sclerosing Cholangitis (PSC)
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This phase 2 trial will evaluate the effects of EP547 in subjects with cholestatic pruritus due to Primary Biliary Cholangitis (PBC) or Primary Sclerosing Cholangitis (PSC) Type: Interventional Start Date: Oct 2022 |
A Study To Evaluate The Efficacy, Safety, Pharmacokinetics, And Pharmacodynamics Of Satralizumab In Patients...
Hoffmann-La Roche
NMDAR Autoimmune Encephalitis
LGI1 Autoimmune Encephalitis
The purpose of this study is to assess the efficacy, safety, pharmacokinetics, and
pharmacodynamics of satralizumab in participants with anti-N-methyl-D-aspartic acid receptor
(NMDAR) and anti-leucine-rich glioma-inactivated 1 (LGI1) encephalitis.
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The purpose of this study is to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in participants with anti-N-methyl-D-aspartic acid receptor (NMDAR) and anti-leucine-rich glioma-inactivated 1 (LGI1) encephalitis. Type: Interventional Start Date: Sep 2022 |
Add-on Reparixin in Adult Patients With ARDS
Dompé Farmaceutici S.p.A
Acute Respiratory Distress Syndrome, Adult
Study objectives
1. To characterize the efficacy of reparixin in ameliorating lung injury and systemic
inflammation and expediting clinical recovery and liberation from mechanical ventilation
in adult patients with moderate to severe ARDS (PaO2/FIO2 ratio ≤ 200).... expand
Study objectives 1. To characterize the efficacy of reparixin in ameliorating lung injury and systemic inflammation and expediting clinical recovery and liberation from mechanical ventilation in adult patients with moderate to severe ARDS (PaO2/FIO2 ratio ≤ 200). 2. To evaluate the safety of reparixin vs. placebo in patients enrolled in the study. Type: Interventional Start Date: Feb 2023 |
A Cross-sectional Study of Lipoprotein(a) Levels in Patients With Documented History of Atherosclerotic...
Amgen
Atherosclerotic Cardiovascular Disease
The purpose of this study is to characterize the distribution of lipoprotein(a) (Lp(a))
levels among participants with a history of ASCVD as defined by their medical history and is
2-fold:
- Evaluate the distribution of Lp(a) value in the overall participants with documented... expand
The purpose of this study is to characterize the distribution of lipoprotein(a) (Lp(a)) levels among participants with a history of ASCVD as defined by their medical history and is 2-fold: - Evaluate the distribution of Lp(a) value in the overall participants with documented history of ASCVD - Evaluate the distribution of Lp(a) value in participants with documented history of ASCVD by demographics and regions Type: Interventional Start Date: Apr 2022 |
Pediatric Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients (P-ICECAP)
University of Michigan
Cardiac Arrest, Out-Of-Hospital
Hypothermia, Induced
Hypoxia-Ischemia, Brain
This is a multicenter trial to establish the efficacy of cooling and the optimal duration of
induced hypothermia for neuroprotection in pediatric comatose survivors of cardiac arrest.
The study team hypothesizes that longer durations of cooling may improve either the
proportion... expand
This is a multicenter trial to establish the efficacy of cooling and the optimal duration of induced hypothermia for neuroprotection in pediatric comatose survivors of cardiac arrest. The study team hypothesizes that longer durations of cooling may improve either the proportion of children that attain a good neurobehavioral recovery or may result in better recovery among the proportion already categorized as having a good outcome. Type: Interventional Start Date: Aug 2022 |
A Study of TAK-062 in Treatment of Active Celiac Disease in Participants Attempting a Gluten-Free Diet
Takeda
Celiac Disease
The main aim is to see how TAK-062 works to reduce celiac-related symptoms and improve small
intestinal damage due to gluten exposure, in participants with celiac disease (CeD)
attempting to maintain a gluten-free diet (GFD) in treated participants versus placebo
controls.... expand
The main aim is to see how TAK-062 works to reduce celiac-related symptoms and improve small intestinal damage due to gluten exposure, in participants with celiac disease (CeD) attempting to maintain a gluten-free diet (GFD) in treated participants versus placebo controls. Type: Interventional Start Date: Jun 2022 |
Moderately Carbohydrate-restricted Diet to Treat NAFLD in Adolescents
University of Alabama at Birmingham
Non-Alcoholic Fatty Liver Disease
Obesity
This will be a 6-month randomized clinical trial with two arms: moderately
carbohydrate-restricted diet and a fat-restricted control diet. This 6-month study will have
2 phases: a 12-week controlled feeding phase and a 12-week "free living" phase. During the
controlled feeding... expand
This will be a 6-month randomized clinical trial with two arms: moderately carbohydrate-restricted diet and a fat-restricted control diet. This 6-month study will have 2 phases: a 12-week controlled feeding phase and a 12-week "free living" phase. During the controlled feeding phase, all food will be provided to the families of the participants for the entirety of the 12 weeks. Participants (n=80) will have been diagnosed with NAFLD based on the presence of current evidence of active disease, which will be determined by the ongoing presence of hepatic steatosis estimated by diffusely echogenic liver via ultrasound suggestive of fatty liver and a serum alanine aminotransferase (ALT) level of 45 U/L or greater. All participants will be children and adolescents age 10-17 yrs.; will have an HbA1c <7.0; and will be overweight or obese (BMI >85th percentile). It is anticipated that most participants will be sedentary. The investigators will inquire as to routine physical activity at screening. All participants will be asked to maintain their usual level of physical activity throughout the study. Physical activity will be monitored via a smart watch provided to each participant at the beginning of the study, and participants will be queried weekly by the study dietitian regarding changes in physical activity. Participants who use oral contraceptives will be asked to maintain consistent use of these preparations throughout the study. Hormone use will be examined as a potential covariate in statistical analyses. Type: Interventional Start Date: May 2022 |
A Study to Evaluate Tirzepatide (LY3298176) in Pediatric and Adolescent Participants With Type 2 Diabetes...
Eli Lilly and Company
Type2 Diabetes
Diabetes Mellitus
Diabetes Mellitus, Type 2
T2D
T2DM (Type 2 Diabetes Mellitus)
The purpose of this study is to learn more about the safety and efficacy of tirzepatide
compared to placebo in children or teenagers with type 2 diabetes taking metformin, or basal
insulin, or both.
The overall study will last about 60 weeks with up to 14 clinic visits and... expand
The purpose of this study is to learn more about the safety and efficacy of tirzepatide compared to placebo in children or teenagers with type 2 diabetes taking metformin, or basal insulin, or both. The overall study will last about 60 weeks with up to 14 clinic visits and 6 phone visits. Clinic visits will include blood sample collection, physical exam and questionnaire. Type: Interventional Start Date: Apr 2022 |
Hyperhydration in Children With Shiga Toxin-Producing E. Coli Infection
University of Calgary
Shiga Toxin-Producing Escherichia Coli (E. Coli) Infection
Hemolytic-Uremic Syndrome
The objective of this study is to determine if early high volume intravenous fluid
administration (hyperhydration) may be effective in mitigating or preventing complications of
shiga toxin-producing E. coli (STEC) infection in children and adolescents when compared with
traditional... expand
The objective of this study is to determine if early high volume intravenous fluid administration (hyperhydration) may be effective in mitigating or preventing complications of shiga toxin-producing E. coli (STEC) infection in children and adolescents when compared with traditional approaches (conservative fluid management). Type: Interventional Start Date: Sep 2022 |
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female...
Genzyme, a Sanofi Company
Fabry Disease
This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled
study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of
Fabry disease in adult participants with Fabry disease who are treatment-naïve or untreated... expand
This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in adult participants with Fabry disease who are treatment-naïve or untreated for at least 6 months. - Study visits will take place approximately every 3 months. - The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for up to an additional 12 months. Type: Interventional Start Date: Mar 2022 |
FT576 in Subjects With Multiple Myeloma
Fate Therapeutics
Multiple Myeloma
Myeloma
This is a Phase I dose-finding study of FT576 as monotherapy and in combination with the
monoclonal antibody daratumumab in multiple myeloma (MM). The study will consist of a
dose-escalation stage and an expansion stage.
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This is a Phase I dose-finding study of FT576 as monotherapy and in combination with the monoclonal antibody daratumumab in multiple myeloma (MM). The study will consist of a dose-escalation stage and an expansion stage. Type: Interventional Start Date: Nov 2021 |
Embolization of the Splenic Artery After Trauma
Andrew J. Gunn
High-grade Splenic Injuries
Our aim is to conduct a multi-center, Bayesian, randomized clinical trial to evaluate the
primary technical success of coils and vascular plugs for proximal splenic artery
embolization in the setting of high-grade splenic trauma. The investigator has previously
demonstrated... expand
Our aim is to conduct a multi-center, Bayesian, randomized clinical trial to evaluate the primary technical success of coils and vascular plugs for proximal splenic artery embolization in the setting of high-grade splenic trauma. The investigator has previously demonstrated the feasibility of such a study in a single center pilot trial. Type: Interventional Start Date: May 2022 |
A Multicenter Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Subjects With Idiopathic...
Horizon Therapeutics Ireland DAC
Idiopathic Pulmonary Fibrosis
HZNP-HZN-825-303 (HARBOR) comprises of 2 parts. Part 1 (Core Phase) is a randomized,
double-blind, placebo-controlled, repeat-dose, multicenter trial to evaluate the efficacy,
safety and tolerability of HZN-825 in participants with Idiopathic Pulmonary Fibrosis (IPF).
Part... expand
HZNP-HZN-825-303 (HARBOR) comprises of 2 parts. Part 1 (Core Phase) is a randomized, double-blind, placebo-controlled, repeat-dose, multicenter trial to evaluate the efficacy, safety and tolerability of HZN-825 in participants with Idiopathic Pulmonary Fibrosis (IPF). Part 2 (Extension Phase) is an optional, open-label, repeat-dose, multicenter extension of the Core Phase. The trial will include up to an 8-week Screening Period and a 52-week Double-blind Treatment Period in the Core Phase and 52 weeks of open-label HZN-825 treatment in the Extension Phase. During the Core Phase, participants will be screened within 8 weeks prior to the baseline (Day 1) Visit. Approximately 135 participants who meet the trial eligibility criteria will be randomly assigned in a 1:1:1 ratio on Day 1 to receive HZN-825 300 mg QD, HZN-825 300 mg BID or matching placebo orally for 52 weeks using the following 2 stratification factors: 1. Concomitant use of approved IPF therapy (i.e., nintedanib or pirfenidone): yes or no 2. Forced vital capacity (FVC) % predicted at Baseline: ≥70% or <70% Participants who complete the 52-week Double blind Treatment Period of the Core Phase of the trial will be invited to extend their participation in the 52-week Extension Phase of the trial. Type: Interventional Start Date: Aug 2021 |
Combination Therapy for the Treatment of Diffuse Midline Gliomas
University of California, San Francisco
Diffuse Intrinsic Pontine Glioma
Diffuse Midline Glioma, H3 K27M-Mutant
Recurrent Diffuse Intrinsic Pontine Glioma
Recurrent Diffuse Midline Glioma, H3 K27M-Mutant
Recurrent WHO Grade III Glioma
This phase II trial determines if the combination of ONC201 with different drugs,
panobinostat or paxalisib, is effective for treating patients with diffuse midline gliomas
(DMGs). Despite years of research, little to no progress has been made to improve outcomes
for patients... expand
This phase II trial determines if the combination of ONC201 with different drugs, panobinostat or paxalisib, is effective for treating patients with diffuse midline gliomas (DMGs). Despite years of research, little to no progress has been made to improve outcomes for patients with DMGs, and there are few treatment options. ONC201, panobinostat, and paxalisib are all enzyme inhibitors that may stop the growth of tumor cells by clocking some of the enzymes needed for cell growth. This phase II trial assesses different combinations of these drugs for the treatment of DMGs. Type: Interventional Start Date: Oct 2021 |
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Cogent Biosciences, Inc.
Advanced Systemic Mastocytosis (AdvSM)
SM With an Associated Hematologic Neoplasm (SM-AHN)
Mast Cell Leukemia (MCL)
Aggressive Systemic Mastocytosis (ASM)
This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of
patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM
(ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).
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This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). Type: Interventional Start Date: Nov 2021 |
A Phase 1b/2 Study of BGB-11417in Monotherapy and in Various Combinations With Dexamethasone and Carfilzomib...
BeiGene
Relapsed/Refractory Multiple Myeloma
Study consists of two parts, a part 1 dose escalation and a part 2 cohort expansion in
combination with dexamethasone and carfilzomib intravenously across two cohorts with a
monotherapy component as well.
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Study consists of two parts, a part 1 dose escalation and a part 2 cohort expansion in combination with dexamethasone and carfilzomib intravenously across two cohorts with a monotherapy component as well. Type: Interventional Start Date: Sep 2021 |
Study of Pembrolizumab (MK-3475) Plus Chemotherapy Versus Placebo Plus Chemotherapy for HR+/HER2- Locally...
Merck Sharp & Dohme LLC
Breast Neoplasms
The safety and efficacy of pembrolizumab plus the investigator's choice of chemotherapy will
be assessed compared to placebo plus the investigator's choice of chemotherapy in the
treatment of chemotherapy-candidate hormone receptor-positive, human epidermal growth factor
receptor... expand
The safety and efficacy of pembrolizumab plus the investigator's choice of chemotherapy will be assessed compared to placebo plus the investigator's choice of chemotherapy in the treatment of chemotherapy-candidate hormone receptor-positive, human epidermal growth factor receptor 2-negative (HR+/HER2-) locally recurrent inoperable or metastatic breast cancer. The primary hypotheses are that the combination of pembrolizumab and chemotherapy is superior to placebo and chemotherapy in regards to Progression-Free Survival (PFS) or overall survival (OS) in participants with programmed cell death-ligand 1 (PD-L1) combined positive score (CPS) ≥1 and ≥10. Type: Interventional Start Date: Jun 2021 |
Isatuximab, Pomalidomide, Elotuzumab and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma
Medical College of Wisconsin
Multiple Myeloma
This is a multicenter, open-label phase II study in subjects with relapsed and/or refractory
multiple myeloma with at least two prior lines of therapy. The main study consists of three
phases: a 28-day screening phase, treatment phase that consists of 28-day cycles of
isatuximab... expand
This is a multicenter, open-label phase II study in subjects with relapsed and/or refractory multiple myeloma with at least two prior lines of therapy. The main study consists of three phases: a 28-day screening phase, treatment phase that consists of 28-day cycles of isatuximab with elotuzumab, pomalidomide, and dexamethasone and a follow-up phase. Type: Interventional Start Date: Jan 2022 |
Evaluating the Implementation and Impact of Navigator-delivered ePRO System
University of Alabama at Birmingham
Cancer
1) Evaluate implementation of navigator-delivered Home ePRO for all cancer patients across
multiple practice sites; 2) examine the barriers, facilitators, and implementation strategies
used in implementing navigator-delivered Home ePRO; and 3) assess the impact of Home ePRO on... expand
1) Evaluate implementation of navigator-delivered Home ePRO for all cancer patients across multiple practice sites; 2) examine the barriers, facilitators, and implementation strategies used in implementing navigator-delivered Home ePRO; and 3) assess the impact of Home ePRO on clinical and utilization outcomes. Type: Interventional Start Date: May 2021 |
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