908 matching studies

Sponsor Condition of Interest
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: Jul 2015

open study

Prostate Cancer Genetic Risk Evaluation and Screening Study
Massachusetts General Hospital Prostatic Neoplasm Prostate Cancer BRCA2 Mutation BRCA1 Mutation ATM Gene Mutation
This study aims to define the natural history of men at high genetic risk for prostate cancer on the basis of specific germline genetic mutations or a positive family history. The hypothesis is that this targeted population of men are at high-risk of developing prostate cancer... expand

This study aims to define the natural history of men at high genetic risk for prostate cancer on the basis of specific germline genetic mutations or a positive family history. The hypothesis is that this targeted population of men are at high-risk of developing prostate cancer and will enable us to characterize the variable penetrance of specific mutations, potentially identify new genetic risk mutations, and develop improved techniques for early detection and diagnosis of prostate cancer.

Type: Observational [Patient Registry]

Start Date: Feb 2020

open study

Treatment of Milademetan Versus Trabectedin in Patient With Dedifferentiated Liposarcoma
Rain Therapeutics Inc. Dedifferentiated Liposarcoma
Randomized, multicenter, open-label, Phase 3 registration study designed to evaluate the safety and efficacy of milademetan compared to trabectedin in patients with unresectable (i.e., where resection is deemed to cause unacceptable morbidity or mortality) or metastatic DD... expand

Randomized, multicenter, open-label, Phase 3 registration study designed to evaluate the safety and efficacy of milademetan compared to trabectedin in patients with unresectable (i.e., where resection is deemed to cause unacceptable morbidity or mortality) or metastatic DD liposarcoma that progressed on 1 or more prior systemic therapies, including at least 1 anthracycline-based therapy.

Type: Interventional

Start Date: Jul 2021

open study

XTX101 Monotherapy and XTX101 and Pembrolizumab Combination Therapy in Patients With Advanced Solid Tumors
Xilio Development, Inc. Advanced Solid Tumor
This is a first-in-human, Phase 1/2, multicenter, open-label study designed to evaluate the safety and tolerability of XTX101 as monotherapy and XTX101 and pembrolizumab combination therapy in patients with advanced solid tumors. expand

This is a first-in-human, Phase 1/2, multicenter, open-label study designed to evaluate the safety and tolerability of XTX101 as monotherapy and XTX101 and pembrolizumab combination therapy in patients with advanced solid tumors.

Type: Interventional

Start Date: Sep 2021

open study

A Study Evaluating the Efficacy and Safety of Multiple Treatment Combinations in Participants With Breast...
Hoffmann-La Roche Inoperable, Locally Advanced or Metastatic, ER-positive Breast Cancer
This is a Phase Ib/II, open-label, multicenter, randomized umbrella study in participants with breast cancer. Cohort 1 will focus on participants with inoperable, locally advanced or metastatic, estrogen receptor (ER)-positive, HER2-negative breast cancer who had disease progression... expand

This is a Phase Ib/II, open-label, multicenter, randomized umbrella study in participants with breast cancer. Cohort 1 will focus on participants with inoperable, locally advanced or metastatic, estrogen receptor (ER)-positive, HER2-negative breast cancer who had disease progression during or following treatment with a cyclin-dependent kinase 4/6 inhibitor (CDK4/6i; e.g., palbociclib, ribociclib, abemaciclib) in the first- or second-line setting. Cohort 2 will focus on inoperable, locally advanced or metastatic, ER-positive, HER2-positive breast cancer with prior disease progression on trastuzumab-and-taxane- and ado-trastuzumab emtansine-based therapies. The study is designed with the flexibility to open new treatment arms as new treatments become available, close existing treatment arms that demonstrate minimal clinical activity or unacceptable toxicity, or modify the patient population. During Stage 1, participants in each cohort will be randomly assigned to treatment arms. Participants in the control or experimental arms who experience disease progression (as determined by the investigator according to RECIST v1.1) or unacceptable toxicity during Stage 1 will be given the option of receiving a different treatment combination during Stage 2, provided they meet eligibility criteria and a treatment arm is open for enrollment. No Stage 2 treatment is currently available.

Type: Interventional

Start Date: Jun 2021

open study

A Study Comparing Two Types of Supportive Interventions for Caregivers of Patients With Cancer
Memorial Sloan Kettering Cancer Center Cancer Patients Family Caregivers
The purpose of this study is to compare two types of therapy for caregivers of cancer patients: Emotion Regulation Therapy for Cancer Caregivers (ERT-C) and Cognitive Behavioral Therapy (CBT-C). The researchers want to see if ERT-C is better than, the same as, or worse than... expand

The purpose of this study is to compare two types of therapy for caregivers of cancer patients: Emotion Regulation Therapy for Cancer Caregivers (ERT-C) and Cognitive Behavioral Therapy (CBT-C). The researchers want to see if ERT-C is better than, the same as, or worse than traditional CBT-C at improving caregiver distress. The researchers will look at how the two types of therapy affect caregivers' anxiety, depression, and quality of life. The researchers will also see how ERT-C and CBT-C affect hormone and stress levels in caregivers' saliva samples. In addition, this trial will enroll cancer patients in this study to see how their caregivers' participation in ERT-C or CBT may affect the patients' quality of life, stress, and use of healthcare services.

Type: Interventional

Start Date: Feb 2021

open study

Effects of Romosozumab on Bone Density in Women With Anorexia Nervosa
Karen Klahr Miller, MD Bone Density, Low Bone Loss Anorexia Nervosa Eating Disorders
This protocol is a randomized, double-blind, placebo-controlled clinical trial which aims to investigate the effect of romosozumab on BMD in women with anorexia nervosa. The investigators will also investigate the safety of romosozumab in women with anorexia nervosa. The investigators... expand

This protocol is a randomized, double-blind, placebo-controlled clinical trial which aims to investigate the effect of romosozumab on BMD in women with anorexia nervosa. The investigators will also investigate the safety of romosozumab in women with anorexia nervosa. The investigators hypothesize that 12 months of romosozumab administration will result in an increase in bone mineral density, increase in markers of bone formation and decrease in markers of bone resorption, and improvement in bone microarchitecture in osteopenic women with anorexia nervosa compared with placebo. The extension study will offer subjects 12-month administration of open-label alendronate (an oral bisphosphonate) 70 mg once weekly after the initial 12 month administration of romosozumab or placebo. The investigators hypothesize that 12 months of romosozumab followed by 12 months of open-label alendronate will result in a greater increase in BMD compared to 12 months of placebo followed by 12 months of open-label alendronate. Within the group of women who receive sequential therapy with 12 months of romosozumab followed by 12 months of alendronate, the investigators hypothesize that BMD will be maintained between 12 and 24 months while on alendronate.

Type: Interventional

Start Date: Sep 2021

open study

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
Pharnext SA Charcot-Marie-Tooth Disease
The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1A. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.... expand

The purpose of this study is to determine whether PXT3003 is effective and safe in the treatment of Charcot-Marie-Tooth disease - Type 1A. This double-blind study will assess in parallel groups 1 dose of PXT3003 compared to Placebo in CMT1A patients treated for 15 months.

Type: Interventional

Start Date: Mar 2021

open study

A Study of Pembrolizumab (MK-3475) in Combination With Belzutifan (MK-6482) and Lenvatinib (MK-7902),...
Merck Sharp & Dohme Corp. Carcinoma, Renal Cell
The goal of this study is to evaluate the efficacy and safety of pembrolizumab plus belzutifan plus lenvatinib or pembrolizumab/quavonlimab plus lenvatinib versus pembrolizumab plus lenvatinib as first-line treatment in participants with advanced clear cell renal cell carcinoma... expand

The goal of this study is to evaluate the efficacy and safety of pembrolizumab plus belzutifan plus lenvatinib or pembrolizumab/quavonlimab plus lenvatinib versus pembrolizumab plus lenvatinib as first-line treatment in participants with advanced clear cell renal cell carcinoma (ccRCC). The primary hypotheses are (1) pembrolizumab plus belzutifan plus lenvatinib is superior to pembrolizumab plus lenvatinib with respect to progression-free survival (PFS) and overall survival (OS), in advanced ccRCC participants and (2) pembrolizumab/quavonlimab plus lenvatinib is superior to pembrolizumab plus lenvatinib with respect to PFS and OS, in advanced ccRCC participants.

Type: Interventional

Start Date: Apr 2021

open study

A Study to Evaluate Guselkumab for the Treatment of Participants With New-onset or Relapsing Giant Cell...
Janssen Research & Development, LLC Giant Cell Arteritis
The primary purpose of this study is to evaluate the efficacy of guselkumab compared to placebo, in combination with a 26-week glucocorticoid (GC) taper regimen, in adult participants with new-onset or relapsing giant cell arteritis (GCA). expand

The primary purpose of this study is to evaluate the efficacy of guselkumab compared to placebo, in combination with a 26-week glucocorticoid (GC) taper regimen, in adult participants with new-onset or relapsing giant cell arteritis (GCA).

Type: Interventional

Start Date: Dec 2020

open study

LUMINOS-102: PVSRIPO With or Without Immune Checkpoint Blockade in Patients With Advanced PD-1 Refractory...
Istari Oncology, Inc. Melanoma
A Phase 2 study to investigate the efficacy and safety of PVSRIPO alone or in combination with a programmed death receptor-1 (anti-PD-1) inhibitor. expand

A Phase 2 study to investigate the efficacy and safety of PVSRIPO alone or in combination with a programmed death receptor-1 (anti-PD-1) inhibitor.

Type: Interventional

Start Date: Nov 2020

open study

To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who...
Incyte Corporation Myelofibrosis Primary Myelofibrosis Post Essential Thrombocythemia Myelofibrosis Post Polycythemia Vera Myelofibrosis
The purpose of the study is to compare the efficacy and safety of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in participants with myelofibrosis who have suboptimal response while receiving ruxolitinib monotherapy. expand

The purpose of the study is to compare the efficacy and safety of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in participants with myelofibrosis who have suboptimal response while receiving ruxolitinib monotherapy.

Type: Interventional

Start Date: May 2021

open study

A Study to Evaluate the Efficacy, Safety and Pharmacokinetics of a Higher Dose of Ocrelizumab in Adults...
Hoffmann-La Roche Multiple Sclerosis
This is a randomized, double blind, controlled, parallel group, multicenter study to evaluate efficacy, safety and pharmacokinetics of a higher dose of ocrelizumab per intravenous (IV) infusion every 24 weeks in participants with PPMS, in comparison to the approved 600 mg dose... expand

This is a randomized, double blind, controlled, parallel group, multicenter study to evaluate efficacy, safety and pharmacokinetics of a higher dose of ocrelizumab per intravenous (IV) infusion every 24 weeks in participants with PPMS, in comparison to the approved 600 mg dose of ocrelizumab.

Type: Interventional

Start Date: Dec 2020

open study

Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)
Genfit Primary Biliary Cirrhosis
The main objective of the study is to evaluate the effect of daily oral administration of 80mg elafibranor on cholestasis (impairment of bile formation and/or bile accumulation) in patients with PBC and inadequate response or intolerance to Ursodeoxycholic Acid (UCDA) expand

The main objective of the study is to evaluate the effect of daily oral administration of 80mg elafibranor on cholestasis (impairment of bile formation and/or bile accumulation) in patients with PBC and inadequate response or intolerance to Ursodeoxycholic Acid (UCDA)

Type: Interventional

Start Date: Sep 2020

open study

ACTIV-3: Therapeutics for Inpatients With COVID-19
University of Minnesota Covid19
This study looks at the safety and effectiveness of different drugs in treating COVID-19 in people who have been hospitalized with the infection. Participants in the study will be treated with either a study drug plus current standard of care (SOC), or with placebo plus current... expand

This study looks at the safety and effectiveness of different drugs in treating COVID-19 in people who have been hospitalized with the infection. Participants in the study will be treated with either a study drug plus current standard of care (SOC), or with placebo plus current SOC.

Type: Interventional

Start Date: Aug 2020

open study

Evaluation of FDY-5301 in Major Trauma Patients in ICU
Faraday Pharmaceuticals, Inc. ICU Acquired Weakness
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of FDY-5301 compared to placebo in major trauma ICU patients at risk of intensive care unit acquired weakness (ICUAW) expand

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of FDY-5301 compared to placebo in major trauma ICU patients at risk of intensive care unit acquired weakness (ICUAW)

Type: Interventional

Start Date: Sep 2021

open study

A Study of Selinexor in Combination With Standard of Care Therapy for Newly Diagnosed or Recurrent Glioblastoma
Karyopharm Therapeutics Inc Glioblastoma Multiforme
This is a Phase 1/2 study of selinexor in combination with standard of care (SoC) therapy for newly diagnosed glioblastoma (nGBM) or recurrent glioblastoma (rGBM). This study will be conducted in 2 phases: a Phase 1a dose finding study followed by Phase 1b (dose expansion)... expand

This is a Phase 1/2 study of selinexor in combination with standard of care (SoC) therapy for newly diagnosed glioblastoma (nGBM) or recurrent glioblastoma (rGBM). This study will be conducted in 2 phases: a Phase 1a dose finding study followed by Phase 1b (dose expansion) and a Phase 2 randomized efficacy exploration study and will independently evaluate 3 different combination regimens in 3 treatment arms in patients with nGBM (Arms A and B) or with rGBM (Arm C). - Arm A: evaluating the combination of selinexor with radiation therapy (S-RT) in nGBM participants with uMGMT - Arm B: evaluating the combination of selinexor with radiation therapy and temozolomide (TMZ) (S-TRT) in nGBM participants with methylated-O6-methylguanine-DNA-methyltransferase (mMGMT) - Arm C: evaluating the combination of selinexor with lomustine (or carmustine, if lomustine is not available) (S-L/C) in rGBM participants regardless of MGMT status - Arm D: evaluating the combination of selinexor with bevacizumab in rGBM participants regardless of MGMT status - Arm E: evaluating the combination of selinexor with tumor treating fields (TTField) in rGBM participants regardless of MGMT status

Type: Interventional

Start Date: Jun 2020

open study

Study Of Venetoclax Tablet With Intravenous or Subcutaneous Azacitidine to Assess Change in Disease Activity...
AbbVie Myelodysplastic Syndrome (MDS)
Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells. Some people with MDS have a risk of the disease progressing to acute... expand

Myelodysplastic Syndrome (MDS) is a group of disorders that gradually affect the ability of a person's bone marrow (semi-liquid tissue present in many bones like backbones) to produce normal blood cells. Some people with MDS have a risk of the disease progressing to acute myeloid leukemia (AML), and a risk of death from the disease itself. Symptoms of MDS include fatigue, shortness of breath, unusual paleness due to anemia (low red blood cell count), easy or unusual bruising, and red spots just beneath the skin caused by bleeding. The purpose of this study is to see how safe and effective venetoclax and azacitidine (AZA) combination are when compared to AZA and a placebo (contains no medicine), in participants with newly diagnosed higher-risk MDS. Venetoclax is an investigational drug being developed for the treatment of MDS. The study consists of two treatment arms - In one arm, participants will receive venetoclax and AZA. In another arm, participants will receive AZA and placebo. Adult participants with newly diagnosed higher-risk MDS will be enrolled. Around 500 participants will be enrolled in approximately 220 sites worldwide. Participants in one arm will receive oral doses of venetoclax tablet and intravenous (infusion in the vein) or subcutaneous (given under the skin) AZA solution. Participants in another arm will receive oral doses of placebo tablet and intravenous or subcutaneous AZA solution. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the course of the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood and bone marrow tests, checking for side effects, and completing questionnaires.

Type: Interventional

Start Date: Sep 2020

open study

Beating Heart Mitral Valve Repair With the HARPOON™ System
Edwards Lifesciences Mitral Regurgitation
The objective of the study is to evaluate the safety and effectiveness of the HARPOON™ System in the treatment of patients with severe degenerative mitral regurgitation (DMR). expand

The objective of the study is to evaluate the safety and effectiveness of the HARPOON™ System in the treatment of patients with severe degenerative mitral regurgitation (DMR).

Type: Interventional

Start Date: Dec 2020

open study

A Study to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects With Relapsed or Refractory...
Celgene Lymphoma, Non-Hodgkin
This is a global Phase 2, open-label, single-arm, multicohort, multicenter study to evaluate efficacy and safety of JCAR017 in adult subjects with r/r FL or MZL. The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical... expand

This is a global Phase 2, open-label, single-arm, multicohort, multicenter study to evaluate efficacy and safety of JCAR017 in adult subjects with r/r FL or MZL. The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This study is divided into three periods: - Pretreatment, which consists of screening assessments, leukapheresis and the Pretreatment evaluation; - Treatment, which starts with the administration of lymphodepleting (LD) chemotherapy and continues through JCAR017 administration at Day 1 with follow-up through Day 29; - Posttreatment, which includes follow-up assessments for disease status and safety for 2 years.

Type: Interventional

Start Date: Jul 2020

open study

Influence of Cooling Duration on Efficacy in Cardiac Arrest Patients
University of Michigan Cardiac Arrest, Out-Of-Hospital Hypothermia, Induced Hypoxia-Ischemia, Brain
A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in... expand

A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.

Type: Interventional

Start Date: May 2020

open study

A Study of Belzutifan (MK-6482) Versus Everolimus in Participants With Advanced Renal Cell Carcinoma...
Merck Sharp & Dohme Corp. Carcinoma, Renal Cell
The primary objective of this study is to compare belzutifan to everolimus with respect to progression-free survival (PFS) per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) as assessed by Blinded Independent Central Review (BICR) and to compare everolimus... expand

The primary objective of this study is to compare belzutifan to everolimus with respect to progression-free survival (PFS) per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) as assessed by Blinded Independent Central Review (BICR) and to compare everolimus with respect to overall survival (OS). The hypothesis is that belzutifan is superior to everolimus with respect to PFS and OS.

Type: Interventional

Start Date: Feb 2020

open study

A Study Evaluating the Efficacy and Safety of Inavolisib + Palbociclib + Fulvestrant vs Placebo + Palbociclib...
Hoffmann-La Roche Breast Cancer
This study will evaluate the efficacy, safety, and pharmacokinetics of inavolisib in combination with palbociclib and fulvestrant compared with placebo plus palbociclib and fulvestrant in patients with PIK3CA-mutant, hormone receptor (HR)-positive, HER2-negative locally advanced... expand

This study will evaluate the efficacy, safety, and pharmacokinetics of inavolisib in combination with palbociclib and fulvestrant compared with placebo plus palbociclib and fulvestrant in patients with PIK3CA-mutant, hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer whose disease progressed during treatment or within 12 months of completing adjuvant endocrine therapy and who have not received prior systemic therapy for metastatic disease.

Type: Interventional

Start Date: Jan 2020

open study

KEYMAKER-U01 Umbrella Master Study: Studies of Investigational Agents With Either Pembrolizumab (MK-3475)...
Merck Sharp & Dohme Corp. Carcinoma, Non-Small-Cell Lung
This study is referred to as the "umbrella master protocol" for pembrolizumab (MK-3475) in the treatment of non-small cell lung cancer (NSCLC). This pembrolizumab NSCLC umbrella master protocol uses a platform design and consists of this master screening study and three substudies.... expand

This study is referred to as the "umbrella master protocol" for pembrolizumab (MK-3475) in the treatment of non-small cell lung cancer (NSCLC). This pembrolizumab NSCLC umbrella master protocol uses a platform design and consists of this master screening study and three substudies. Each substudy will enroll a different population of NSCLC participants. The goal of this umbrella master protocol is to screen potential participants with NSCLC for enrollment into 1 of 3 substudies. Participants must first enroll in this pembrolizumab master protocol study and undergo screening for NSCLC that will be used to assign them to participation in 1 of 3 pembrolizumab substudies.

Type: Observational

Start Date: Dec 2019

open study

KEYMAKER-U01 Substudy 3: Efficacy and Safety Study of Pembrolizumab (MK-3475) When Used With Investigational...
Merck Sharp & Dohme Corp. Carcinoma, Non-Small-Cell Lung
The purpose of this study is to assess the efficacy and safety of pembrolizumab (MK-3475) in combination with MK-5890 or MK-4830 in participants with advanced squamous or non-squamous NSCLC that have been previously treated with anti-PD-L1 therapy. This study is one of three... expand

The purpose of this study is to assess the efficacy and safety of pembrolizumab (MK-3475) in combination with MK-5890 or MK-4830 in participants with advanced squamous or non-squamous NSCLC that have been previously treated with anti-PD-L1 therapy. This study is one of three pembrolizumab substudies being conducted under one pembrolizumab umbrella master protocol (MK-3475-U01/KEYMAKER-U01). Enrollment in the pembrolizumab+MK-5890 arm has been completed with Amendment 4.

Type: Interventional

Start Date: Jan 2020

open study