474 matching studies

Sponsor Condition of Interest
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
ACADIA Pharmaceuticals Inc. Rett Syndrome
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome expand

To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome

Type: Interventional

Start Date: Nov 2019

open study

Low-Dose Atropine for Treatment of Myopia
Jaeb Center for Health Research Myopia
Study Objectives The objectives for this randomized trial are: 1. To determine the efficacy of daily low-dose atropine (0.01%) for slowing myopia progression over a two-year treatment period in children aged 5 to less than 13 years (Primary Outcome On-Treatment).... expand

Study Objectives The objectives for this randomized trial are: 1. To determine the efficacy of daily low-dose atropine (0.01%) for slowing myopia progression over a two-year treatment period in children aged 5 to less than 13 years (Primary Outcome On-Treatment). 2. To determine the efficacy of atropine treatment on myopia progression 6 months following cessation of low-dose atropine treatment (Secondary Outcome Off-Treatment). Synopsis of Study Design The current study is designed as an efficacy study, making effort to maximize adherence to treatment group assignments. After a run-in phase during which all participants are treated with daily artificial tear eyedrops for 2-4 weeks (and glasses are updated if required) to assess their ability to adhere to daily eye drops, participants are randomly assigned to daily atropine or placebo for 24 months, followed by 6 months off treatment.

Type: Interventional

Start Date: Jun 2018

open study

Long-Term PF-06651600 for the Treatment of Alopecia Areata
Pfizer Alopecia Areata
This is a global Phase 3 study to evaluate the safety and effectiveness of an investigational study drug (called PF-06651600) in adults and adolescents (12 years and older) who have alopecia areata. Eligible patients from the prior studies B7931005 (NCT02974868) and B7981015... expand

This is a global Phase 3 study to evaluate the safety and effectiveness of an investigational study drug (called PF-06651600) in adults and adolescents (12 years and older) who have alopecia areata. Eligible patients from the prior studies B7931005 (NCT02974868) and B7981015 (NCT03732807) will have an opportunity to enroll as well as patients who have not previously participated in either of these studies. The study is open-label and all patients entering the study will receive active study drug.

Type: Interventional

Start Date: Jul 2019

open study

Exemestane in Treating Patients With Complex Atypical Hyperplasia of the Endometrium/Endometrial Intraepithelial...
National Cancer Institute (NCI) Atypical Hyperplasia Endometrial Intraepithelial Neoplasia FIGO Grade 1 Endometrial Endometrioid Adenocarcinoma FIGO Grade 2 Endometrial Endometrioid Adenocarcinoma
This pilot phase IIa trial studies how well exemestane works in treating patients with complex atypical hyperplasia of the endometrium/endometrial intraepithelial neoplasia or low grade endometrial cancer. Exemestane may stop the growth of tumor cells by blocking some of the... expand

This pilot phase IIa trial studies how well exemestane works in treating patients with complex atypical hyperplasia of the endometrium/endometrial intraepithelial neoplasia or low grade endometrial cancer. Exemestane may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Nov 2017

open study

Traditional Versus Early Aggressive Therapy for Multiple Sclerosis Trial
Johns Hopkins University Multiple Sclerosis, Relapsing-Remitting
FDA-approved multiple sclerosis (MS) disease-modifying therapies (DMTs) target the relapsing phase of MS but have minimal impact once the progressive phase has begun. It is unclear if, in the relapsing phase, there is an advantage of early aggressive therapy with respect to preventing... expand

FDA-approved multiple sclerosis (MS) disease-modifying therapies (DMTs) target the relapsing phase of MS but have minimal impact once the progressive phase has begun. It is unclear if, in the relapsing phase, there is an advantage of early aggressive therapy with respect to preventing long-term disability. The infectious risks and other complications associated with higher-efficacy treatments highlight the need to quantify their effectiveness in preventing disability. The TRaditional versus Early Aggressive Therapy for MS (TREAT-MS) trial is a pragmatic, randomized controlled trial that has two primary aims: 1) to evaluate, jointly and independently among patients deemed at higher risk vs. lower risk for disability accumulation, whether an "early aggressive" therapy approach, versus starting with a traditional, first-line therapy, influences the intermediate-term risk of disability, and 2) to evaluate if, among patients deemed at lower risk for disability who start on first-line MS therapies but experience breakthrough disease, those who switch to a higher-efficacy versus a new first-line therapy have different intermediate-term risk of disability.

Type: Interventional

Start Date: May 2018

open study

Safety Study of SEA-CD40 in Cancer Patients
Seattle Genetics, Inc. Cancer Carcinoma Carcinoma, Non-Small-Cell Lung Carcinoma, Squamous Cell Hematologic Malignancies
This study is being done to find out if SEA-CD40 is safe and effective when given alone, in combination with pembrolizumab, and in combination with pembrolizumab, gemcitabine, and nab-paclitaxel. The study will test increasing doses of SEA-CD40 given at least every 3 weeks to... expand

This study is being done to find out if SEA-CD40 is safe and effective when given alone, in combination with pembrolizumab, and in combination with pembrolizumab, gemcitabine, and nab-paclitaxel. The study will test increasing doses of SEA-CD40 given at least every 3 weeks to small groups of patients. The goal is to find the highest dose of SEA-CD40 that can be given to patients that does not cause unacceptable side effects. Different dose regimens will be evaluated. Different methods of administration may be evaluated. The pharmacokinetics, pharmacodynamic effects, biomarkers of response, and antitumor activity of SEA-CD40 will also be evaluated.

Type: Interventional

Start Date: Feb 2015

open study

Safety and Efficacy of SGN-LIV1A Plus Pembrolizumab for Patients With Locally-Advanced or Metastatic...
Seattle Genetics, Inc. Breast Neoplasms
This trial studies SGN-LIV1A in combination with pembrolizumab to find out what their side effects are and if they work for patients with triple-negative breast cancer that has spread to other parts of the body. Patients will be given both experimental drugs every 3 weeks. expand

This trial studies SGN-LIV1A in combination with pembrolizumab to find out what their side effects are and if they work for patients with triple-negative breast cancer that has spread to other parts of the body. Patients will be given both experimental drugs every 3 weeks.

Type: Interventional

Start Date: Feb 2018

open study

A Safety Study of SGN-LIV1A in Breast Cancer Patients
Seattle Genetics, Inc. Breast Cancer
This study will examine the safety and tolerability of SGN-LIV1A (ladiratuzumab vedotin) in patients with metastatic breast cancer. SGN-LIV1A will be given alone or in combination with trastuzumab. expand

This study will examine the safety and tolerability of SGN-LIV1A (ladiratuzumab vedotin) in patients with metastatic breast cancer. SGN-LIV1A will be given alone or in combination with trastuzumab.

Type: Interventional

Start Date: Oct 2013

open study

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 and... 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

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome
Rett Syndrome Research Trust Rett Syndrome
This 4 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Approximately 12 patients per cohort and... expand

This 4 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Approximately 12 patients per cohort and up to approximately 48 patients in total are anticipated to participate for approximately 8-10 weeks at approximately 7 US study centers.

Type: Interventional

Start Date: Mar 2019

open study

An Efficacy Study Comparing Ponatinib Versus Imatinib, Administered in Combination With Reduced-Intensity...
Millennium Pharmaceuticals, Inc. Lymphoblastic Leukemia, Acute, Adult Acute Lymphoid Leukemia Leukemia, Acute Lymphoblastic Leukemia, Lymphoblastic Ph1 Chromosome
The purpose of this study is to compare the efficacy of ponatinib versus imatinib, administered as first-line therapy in combination with reduced-intensity chemotherapy, in participants with newly diagnosed Ph+ ALL, as measured by the minimal residual disease (MRD)-negative complete... expand

The purpose of this study is to compare the efficacy of ponatinib versus imatinib, administered as first-line therapy in combination with reduced-intensity chemotherapy, in participants with newly diagnosed Ph+ ALL, as measured by the minimal residual disease (MRD)-negative complete remission (CR) at the end of induction.

Type: Interventional

Start Date: Aug 2018

open study

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma
National Cancer Institute (NCI) Cutaneous Neurofibroma Neurofibromatosis Type 1 Optic Nerve Glioma
This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. expand

This pilot phase II trial studies how well selumetinib works in treating patients with neurofibromatosis type 1 and cutaneous neurofibromas. Selumetinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Apr 2017

open study

Brentuximab Vedotin and Nivolumab With or Without Ipilimumab in Treating Patients With Relapsed or Refractory...
National Cancer Institute (NCI) Recurrent Classic Hodgkin Lymphoma Refractory Classic Hodgkin Lymphoma
This phase I/II trial studies the side effects and best dose of ipilimumab and nivolumab when given together with brentuximab vedotin, and how well they work in treating patients with Hodgkin lymphoma that has returned after a period of improvement or has not responded to previous... expand

This phase I/II trial studies the side effects and best dose of ipilimumab and nivolumab when given together with brentuximab vedotin, and how well they work in treating patients with Hodgkin lymphoma that has returned after a period of improvement or has not responded to previous treatment. Immunotherapy with monoclonal antibodies, such as ipilimumab and nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of cancer cells to grow and spread. Brentuximab vedotin is a combining monoclonal antibody with an anticancer drug that binds to a protein on the surface of lymphoma cells called cluster of differentiation (CD)30 and may kill the cells. It is not known whether giving brentuximab vedotin and nivolumab with or without ipilimumab may kill more cancer cells.

Type: Interventional

Start Date: Jan 2014

open study

Evaluation of VISITAG SURPOINT™ Module With External Processing Unit (EPU)
Biosense Webster, Inc. Paroxysmal Atrial Fibrillation
Prospective, non-randomized, post market clinical evaluation of the VISITAG SURPOINT™ Module with External Processing Unit (EPU) when used with STSF catheter and ST catheter compared to an historical control performance goal. A maximum of 330 subjects will be enrolled across... expand

Prospective, non-randomized, post market clinical evaluation of the VISITAG SURPOINT™ Module with External Processing Unit (EPU) when used with STSF catheter and ST catheter compared to an historical control performance goal. A maximum of 330 subjects will be enrolled across up to 45 sites. Two hundred eighty (280) enrolled subjects will be treated using the STSF catheter with EPU and 50 subjects will be treated using the ST catheter with EPU. Prior to enrollment, a few sites will be selected to only enroll subjects who will be treated with the ST catheter and the remaining sites will only enroll subjects who will be treated with the STSF catheter. Bayesian adaptive design will be used to assess early success at up to two interims: one after all subjects have completed the 3- month follow-up assessment, and a second to occur after all subjects have completed the 6 months follow-up visit.

Type: Interventional

Start Date: Sep 2018

open study

Testing the Effectiveness of Two Immunotherapy Drugs (Nivolumab and Ipilimumab) With One Anti-cancer...
National Cancer Institute (NCI) Bladder Adenocarcinoma Bladder Mixed Adenocarcinoma Bladder Small Cell Carcinoma Bladder Squamous Cell Carcinoma Chromophobe Renal Cell Carcinoma
This phase II trial studies how well cabozantinib works in combination with nivolumab and ipilimumab in treating patients with rare genitourinary (GU) tumors that have spread to other places in the body. Cabozantinib may stop the growth of tumor cells by blocking some of the... expand

This phase II trial studies how well cabozantinib works in combination with nivolumab and ipilimumab in treating patients with rare genitourinary (GU) tumors that have spread to other places in the body. Cabozantinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving cabozantinib, nivolumab, and ipilimumab may work better in treating patients with genitourinary tumors that have no treatment options compared to giving cabozantinib, nivolumab, or ipilimumab alone.

Type: Interventional

Start Date: Apr 2019

open study

Effect of TTFields (150 kHz) in Non-small Cell Lung Cancer (NSCLC) Patients With 1-10 Brain Metastases...
NovoCure Ltd. Brain Metastases From Non-small Cell Lung Cancer (NSCLC)
The study is a prospective, randomized controlled phase III trial, to test the efficacy, safety and neurocognitive outcomes of advanced NSCLC patients, following stereotactic radiosurgery (SRS) for 1-10 brain metastases, treated with NovoTTF-100M compared to supportive treatment... expand

The study is a prospective, randomized controlled phase III trial, to test the efficacy, safety and neurocognitive outcomes of advanced NSCLC patients, following stereotactic radiosurgery (SRS) for 1-10 brain metastases, treated with NovoTTF-100M compared to supportive treatment alone. The device is an experimental, portable, battery operated device for chronic administration of alternating electric fields (termed TTFields or TTF) to the region of the malignant tumor, by means of surface, insulated electrode arrays.

Type: Interventional

Start Date: Jul 2016

open study

Study to Evaluate the Safety & Tolerability of MRT5005 Administered by Nebulization in Adults With Cystic...
Translate Bio, Inc. Cystic Fibrosis
This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with CF. expand

This Phase 1/2, first-in-human study will evaluate the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization to the respiratory tract of adult subjects with CF.

Type: Interventional

Start Date: May 2018

open study

Food Assistance, Diabetes, and HIV
University of Alabama at Birmingham Diabetes HIV/AIDS FOOD SECURITY
Diabetes prevalence is increasing among people living with HIV (PLWH), yet blood glucose control is less successful in this population who are also often food insecure. Food assistance programs often provide nutrient-poor foods. This proposal asses the feasibility of monitoring... expand

Diabetes prevalence is increasing among people living with HIV (PLWH), yet blood glucose control is less successful in this population who are also often food insecure. Food assistance programs often provide nutrient-poor foods. This proposal asses the feasibility of monitoring diabetes-related health outcomes among food insecure PLWH who are receiving food boxes higher in dietary protein and fiber and lower in simple carbohydrates.

Type: Observational

Start Date: Oct 2018

open study

AXL Inhibitor BGB324 in Treating Participants With Recurrent Glioblastoma Undergoing Surgery
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Brain and Central Nervous System Tumors
This phase I trial studies how well AXL inhibitor BGB324 works in treating participants with glioblastoma that has come back who are undergoing surgery. AXL inhibitor BGB324 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. expand

This phase I trial studies how well AXL inhibitor BGB324 works in treating participants with glioblastoma that has come back who are undergoing surgery. AXL inhibitor BGB324 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Jan 2020

open study

A Study To Evaluate PF-06730512 In Adults With Primary Focal Segmental Glomerulosclerosis
Pfizer Focal Segmental Glomerulosclerosis (FSGS)
The purpose of this Phase 2 adaptive study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of PF-06730512 following multiple intravenous infusion in adult subjects with primary FSGS. expand

The purpose of this Phase 2 adaptive study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of PF-06730512 following multiple intravenous infusion in adult subjects with primary FSGS.

Type: Interventional

Start Date: Oct 2018

open study

Dabrafenib and Trametinib Followed by Ipilimumab and Nivolumab or Ipilimumab and Nivolumab Followed by...
National Cancer Institute (NCI) Metastatic Melanoma Recurrent Melanoma Stage III Cutaneous Melanoma AJCC v7 Stage IIIA Cutaneous Melanoma AJCC v7 Stage IIIB Cutaneous Melanoma AJCC v7
This randomized phase III trial studies how well initial treatment with ipilimumab and nivolumab followed by dabrafenib and trametinib works and compares it to initial treatment with dabrafenib and trametinib followed by ipilimumab and nivolumab in treating patients with stage... expand

This randomized phase III trial studies how well initial treatment with ipilimumab and nivolumab followed by dabrafenib and trametinib works and compares it to initial treatment with dabrafenib and trametinib followed by ipilimumab and nivolumab in treating patients with stage III-IV melanoma that contains a mutation known as BRAFV600 and cannot be removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as ipilimumab and nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Dabrafenib and trametinib may block tumor growth by targeting the BRAFV600 gene. It is not yet known whether treating patients with ipilimumab and nivolumab followed by dabrafenib and trametinib is more effective than treatment with dabrafenib and trametinib followed by ipilimumab and nivolumab.

Type: Interventional

Start Date: Jul 2015

open study

Pembrolizumab in Treating Patients With Stage III-IV High-Risk Melanoma Before and After Surgery
National Cancer Institute (NCI) Acral Lentiginous Melanoma Clinical Stage III Cutaneous Melanoma AJCC v8 Clinical Stage IV Cutaneous Melanoma AJCC v8 Mucosal Melanoma Pathologic Stage III Cutaneous Melanoma AJCC v8
This phase II trial studies how pembrolizumab works before and after surgery in treating patients with stage III-IV high-risk melanoma. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with... expand

This phase II trial studies how pembrolizumab works before and after surgery in treating patients with stage III-IV high-risk melanoma. Immunotherapy with monoclonal antibodies, such as pembrolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Giving pembrolizumab before and after surgery may work better in treating melanoma.

Type: Interventional

Start Date: Dec 2018

open study

A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic...
Vertex Pharmaceuticals Incorporated Cystic Fibrosis
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561. expand

The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Type: Interventional

Start Date: Apr 2019

open study

A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966)...
Mereo BioPharma Alpha 1-Antitrypsin Deficiency Emphysema COPD
The purpose of this study is to investigate the effect of alvelestat (an oral neutrophil elastase inhibitor) on blood and sputum biomarkers in patients with Pizz or null genotype alpha-1 anti-trypsin deficient lung disease. Change in a number of different blood and sputum biomarkers... expand

The purpose of this study is to investigate the effect of alvelestat (an oral neutrophil elastase inhibitor) on blood and sputum biomarkers in patients with Pizz or null genotype alpha-1 anti-trypsin deficient lung disease. Change in a number of different blood and sputum biomarkers related to lung damage, inflammation and elastase activity will be measured over a 12 week period. The effect on lung function and respiratory symptoms will also be measured.

Type: Interventional

Start Date: Oct 2018

open study

Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in Patients With Acromegaly Being Treated With Long-acting...
Ionis Pharmaceuticals, Inc. Acromegaly
The purpose is to assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in up to 60 Patients with Acromegaly expand

The purpose is to assess the Safety, Tolerability, and Efficacy of IONIS-GHR-LRx in up to 60 Patients with Acromegaly

Type: Interventional

Start Date: Sep 2018

open study