
Search Clinical Trials
Sponsor Condition of Interest |
---|
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed D1
National Human Genome Research Institute (NHGRI)
Genetic Disease
Without an explanation for severe and sometimes life-threatening symptoms, patients and
their families are left in a state of unknown. Many individuals find themselves being
passed from physician to physician, undergoing countless and often repetitive tests in
the hopes of finding answers and insig1 expand
Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases. Type: Observational Start Date: Sep 2015 |
Milk And Growth In Children (MAGIC) Born Very Preterm: A Randomized Trial
University of Alabama at Birmingham
Prematurity
The goal of this multi-center randomized, parallel group trial is to determine the effect
of human milk diets ranging between 180 and 200 mL/kg/day on the body composition
outcomes of moderately preterm infants born between 27 and 31 weeks of gestation. expand
The goal of this multi-center randomized, parallel group trial is to determine the effect of human milk diets ranging between 180 and 200 mL/kg/day on the body composition outcomes of moderately preterm infants born between 27 and 31 weeks of gestation. Type: Interventional Start Date: Feb 2025 |
Postoperative Pain Management Following Robotic Assisted Sacrocolpopexy
University of Alabama at Birmingham
Pelvic Organ Prolapse
Post Operative Pain
In the effort to reduce postoperative opioid use, there has been increasing interest in
developing multimodal pain regimens to better manage postoperative pain while minimizing
opioid use and their subsequent side effects that can be detrimental to the healing
process. Standard of care approaches t1 expand
In the effort to reduce postoperative opioid use, there has been increasing interest in developing multimodal pain regimens to better manage postoperative pain while minimizing opioid use and their subsequent side effects that can be detrimental to the healing process. Standard of care approaches to better manage postoperative pain include the Enhanced Recovery After Surgery (ERAS) protocol and the use of peripheral and truncal nerve blocks. Truncal nerve blocks are widely used as an additional modality to provide longer lasting postoperative analgesia and have been adopted as part of the standard of care. The goal of this clinical trial is to compare the effects of ERAS alone versus the quadratus lumborum (QL) nerve block on the postoperative pain experience for women with pelvic organ prolapse undergoing robotic assisted sacrocolpopexy. Subjects will be randomized to the ERAS protocol or the QL block. The main questions the study aims to answer are: 1) does the QL block decrease patient reported pain scores postoperatively; and 2) does the QL block decrease the amount of opioid pain medications in the immediate postoperative period? The primary outcome measure will be median patient reported pain score in the post-anesthesia care unit (PACU) following surgery. Type: Interventional Start Date: Jan 2025 |
A Proof-of-Concept Study to Assess the Efficacy, Safety and Tolerability of Itepekimab (Anti-IL-331
Sanofi
Bronchiectasis
ACT18018 is a multinational, randomized, double-blind, placebo-controlled,
parallel-group, Phase 2 study with 3 treatment groups. The purpose of this study is to
evaluate efficacy, safety and tolerability with 2 dosing regimens of itepekimab compared
with placebo in male and/or female participants1 expand
ACT18018 is a multinational, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 study with 3 treatment groups. The purpose of this study is to evaluate efficacy, safety and tolerability with 2 dosing regimens of itepekimab compared with placebo in male and/or female participants with NCFB aged 18 years of age up to 85 years of age (inclusive). Study details include: - The study duration (screening, 24-52-week treatment, 20-week safety follow-up) will be up to 47-77 weeks. - The treatment duration will be up to 24-52 weeks. - The follow-up duration will be 20 weeks. - Site/phone visits are at a monthly interval. Type: Interventional Start Date: Feb 2024 |
A Study to Evaluate Efficacy and Safety of Povorcitinib in Participants With Nonsegmental Vitiligo1
Incyte Corporation
NonSegmental Vitiligo
This study is being conducted to determine the efficacy and safety of povorcitinib in
participants with nonsegmental vitiligo. expand
This study is being conducted to determine the efficacy and safety of povorcitinib in participants with nonsegmental vitiligo. Type: Interventional Start Date: Nov 2023 |
Efficacy and Safety of Seralutinib in Adult Subjects With PAH (PROSERA)
GB002, Inc.
Pulmonary Arterial Hypertension
The primary objective of the study is to determine the effect of seralutinib on improving
exercise capacity in subjects with WHO Group 1 PAH who are FC II or III. The secondary
objective for this trial is to determine time to clinical worsening. expand
The primary objective of the study is to determine the effect of seralutinib on improving exercise capacity in subjects with WHO Group 1 PAH who are FC II or III. The secondary objective for this trial is to determine time to clinical worsening. Type: Interventional Start Date: Dec 2023 |
A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary1
Hoffmann-La Roche
Primary IgA Nephropathy
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of
sefaxersen (RO7434656), a novel Antisense Oligonucleotide (ASO) therapy in participants
with primary IgA nephropathy (IgAN) who are at high risk of progressive kidney disease
despite optimized supportive care. expand
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of sefaxersen (RO7434656), a novel Antisense Oligonucleotide (ASO) therapy in participants with primary IgA nephropathy (IgAN) who are at high risk of progressive kidney disease despite optimized supportive care. Type: Interventional Start Date: Aug 2023 |
The Impact of a Virtual Magic Trick Training Program
University of Alabama at Birmingham
Autism
Pervasive Developmental Disorder
The proposed project will attempt to confirm the benefits of a structured magic trick
training program (MTTP) experience in adolescents with autism. Benefits of participating
in a 6-week virtual MTTP will be evaluated using validated assessments to measure
social-emotional competencies. expand
The proposed project will attempt to confirm the benefits of a structured magic trick training program (MTTP) experience in adolescents with autism. Benefits of participating in a 6-week virtual MTTP will be evaluated using validated assessments to measure social-emotional competencies. Type: Interventional Start Date: Mar 2023 |
A Study of Aticaprant 10 Milligrams (mg) as Adjunctive Therapy in Adult Participants With MDD With1
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 had an inadequate respon1 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 |
A Study of Aticaprant in Adult and Elderly Participants With Major Depressive Disorder (MDD)
Janssen Research & Development, LLC
Depressive Disorder, Major
The purpose of this study is to assess the long-term safety and tolerability of
aticaprant administered as adjunctive therapy to a current antidepressant (selective
serotonin reuptake inhibitor [SSRI] or serotonin and norepinephrine reuptake inhibitor
[SNRI]) in all participants with major depressi1 expand
The purpose of this study is to assess the long-term safety and tolerability of aticaprant administered as adjunctive therapy to a current antidepressant (selective serotonin reuptake inhibitor [SSRI] or serotonin and norepinephrine reuptake inhibitor [SNRI]) in all participants with major depressive disorder (MDD). Type: Interventional Start Date: Sep 2022 |
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Satra1
Hoffmann-La Roche
NMDAR Autoimmune Encephalitis
LGI1 Autoimmune Encephalitis
The purpose of this study is to assess the efficacy, safety, PK, and PD of satralizumab
in participants with NMDAR and LGI1 encephalitis. expand
The purpose of this study is to assess the efficacy, safety, PK, and PD of satralizumab in participants with NMDAR and LGI1 encephalitis. Type: Interventional Start Date: Sep 2022 |
Curcumin, Resveratrol, and Stinging Nettle as Treatments for GWI
University of Alabama at Birmingham
Gulf War Illness
This project's objective is to identify effective treatments for Gulf War Illness (GWI).
The project tests three potential treatments: curcumin, stinging nettle, and resveratrol.
The project uses a decentralized clinical trial (DCT) design in which individuals can
participate from anywhere in the U1 expand
This project's objective is to identify effective treatments for Gulf War Illness (GWI). The project tests three potential treatments: curcumin, stinging nettle, and resveratrol. The project uses a decentralized clinical trial (DCT) design in which individuals can participate from anywhere in the United States. Recruitment efforts will be designed to obtain a geographically and demographically diverse study sample. Type: Interventional Start Date: May 2023 |
Azithromycin Treatment for Respiratory Syncytial Virus-induced Respiratory Failure in Children
University of Alabama at Birmingham
Respiratory Syncytial Virus Infections
The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM)
during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be
beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway. expand
The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM) during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway. Type: Interventional Start Date: Feb 2022 |
A Phase 1b/2 Study of Sonrotoclax (BGB-11417) as Monotherapy and in Various Combinations With Dexam1
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. expand
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 |
Isatuximab, Pomalidomide, Elotuzumab and Dexamethasone in Relapsed And/or Refractory Multiple Myelo1
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 with elotuzumab, po1 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 |
Study of Selinexor in Combination with Ruxolitinib in Myelofibrosis
Karyopharm Therapeutics Inc
Myelofibrosis
This is a global, multicenter, 2-part study to evaluate the efficacy and safety of
selinexor plus ruxolitinib in JAK inhibitor (JAKi) treatment-naïve myelofibrosis (MF)
participants. The study will be conducted in two phases: Phase 1 (open-label) and Phase 3
(double-blind). Phase 1 (enrollment comp1 expand
This is a global, multicenter, 2-part study to evaluate the efficacy and safety of selinexor plus ruxolitinib in JAK inhibitor (JAKi) treatment-naïve myelofibrosis (MF) participants. The study will be conducted in two phases: Phase 1 (open-label) and Phase 3 (double-blind). Phase 1 (enrollment completed) was an open-label evaluation of the safety and recommended Phase 2 dose (RP2D) of selinexor in combination with ruxolitinib and included a dose escalation using a standard 3+3 design (Phase 1a) and a dose expansion part (Phase 1b). Phase 3 (ongoing), double-blind, placebo-controlled part of the study comparing the efficacy and safety of combination therapy of selinexor + ruxolitinib with combination of placebo + ruxolitinib. Type: Interventional Start Date: Mar 2021 |
Long-term Safety, Tolerability and Effectiveness Study of Ofatumumab in Patients With Relapsing MS
Novartis Pharmaceuticals
Relapsing Multiple Sclerosis
The purpose of this study is to collect long-term safety, tolerability, effectiveness and
health outcomes data in eligible subjects who have participated in a Novartis ofatumumab
clinical MS study.
Vaccination sub-study The purpose of this research sub-study is to find out the effects
of ofatumuma1 expand
The purpose of this study is to collect long-term safety, tolerability, effectiveness and health outcomes data in eligible subjects who have participated in a Novartis ofatumumab clinical MS study. Vaccination sub-study The purpose of this research sub-study is to find out the effects of ofatumumab on the development of antibody responses to selected vaccines and keyhole limpet hemocyanin (KLH) neo-antigen in subjects with relapsing multiple sclerosis (RMS). COVID-19 sub-study: The purpose of this research sub-study is to explore the immune response following Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) vaccination in a subset of subjects on long-term ofatumumab 20 mg sc. Note: Novartis is not supplying the SARS-CoV-2 vaccine. Type: Interventional Start Date: Dec 2018 |
A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patien1
United Therapeutics
PAH
Pulmonary Hypertension
Pulmonary Arterial Hypertension
Hypertension
Connective Tissue Diseases
Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of
ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or
PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1
PAH. expand
Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1 PAH. Type: Interventional Start Date: Aug 2018 |
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients with Neurofi1
Healx Limited
Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study in 20 participants. The study
will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years
of age or older with progressive and/or symptomatic PN. expand
The trial will be an open label, single arm, phase 2 study in 20 participants. The study will assess the tolerability and efficacy of HLX-1502 in participants with NF1 16 years of age or older with progressive and/or symptomatic PN. Type: Interventional Start Date: Jan 2025 |
Study of Arlocabtagene Autoleucel (BMS-986393) a GPRC5D-directed CAR T Cell Therapy in Adult Partic1
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
Multiple Myeloma
The purpose of this study is to evaluate the effectiveness and safety of Arlocabtagene
Autoleucel (BMS-986393) in participants with relapsed or refractory multiple myeloma. expand
The purpose of this study is to evaluate the effectiveness and safety of Arlocabtagene Autoleucel (BMS-986393) in participants with relapsed or refractory multiple myeloma. Type: Interventional Start Date: Mar 2024 |
A Study to Evaluate the Safety, Tolerability, Efficacy, and Drug Levels of CC-97540 in Participants1
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
Multiple Sclerosis
Myasthenia Gravis
The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug
levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS),
Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG). expand
The purpose of this study is to evaluate the safety, tolerability, efficacy, and drug levels of CC-97540 in participants with Relapsing Forms of Multiple Sclerosis (RMS), Progressive Forms of Multiple Sclerosis (PMS) or Refractory Myasthenia Gravis (MG). Type: Interventional Start Date: Mar 2024 |
Effect of a DASH-Style Diet on Urinary Risk Factors for Kidney Stone Disease
University of Alabama at Birmingham
Kidney Stone
The true capacity for a healthy diet to improve urinary stone risk factors is not
well-defined. The objective of this study is to measure the effect of adopting a healthy
dietary pattern on kidney stone disease (KSD) risk. The working hypothesis is that a
Dietary Approaches to Stop Hypertension (DA1 expand
The true capacity for a healthy diet to improve urinary stone risk factors is not well-defined. The objective of this study is to measure the effect of adopting a healthy dietary pattern on kidney stone disease (KSD) risk. The working hypothesis is that a Dietary Approaches to Stop Hypertension (DASH)-style diet will improve 24-hour urine stone risk parameters. The approach to testing this hypothesis will be to randomize participants with KSD to a standardized DASH-style vs. Western-style diet for one week. The Bionutrition Unit of the Center for Clinical and Translational Science will provide all meals to participants. The rationale for this study is that by measuring the effect of a DASH-style diet on urinary stone risk parameters, a benchmark for future real-world, implementation studies will be established. Based on available evidence, this will be the first controlled diet study to assess the DASH dietary pattern for improving urinary stone risk parameters. Type: Interventional Start Date: Jan 2025 |
Efficacy and Safety Studies of Frexalimab (SAR441344) in Adults With Relapsing Forms of Multiple Sc1
Sanofi
Multiple Sclerosis
The purpose of each study is to independently measure the annualized relapse rate (ARR)
with administration of frexalimab compared to a daily oral dose of teriflunomide in male
and female participants with relapsing forms of multiple sclerosis (aged 18 to 55 years
at the time of enrollment). People1 expand
The purpose of each study is to independently measure the annualized relapse rate (ARR) with administration of frexalimab compared to a daily oral dose of teriflunomide in male and female participants with relapsing forms of multiple sclerosis (aged 18 to 55 years at the time of enrollment). People diagnosed with relapsing forms of multiple sclerosis are eligible for enrollment as long as they meet all the inclusion criteria and none of the exclusion criteria. Study details include: - This event-driven study will have variable duration of approximately 40 months for the first participant being randomized and approximately 20 months for the last participant randomized. - The study intervention duration will vary ranging from approximately 12 to 40 months. - The assessment of scheduled visits will include 1 common end of study [EOS] visit and 3 follow-up visits) with a visit frequency of every 4 weeks for the first 6 months and then every 3 months. Type: Interventional Start Date: Dec 2023 |
A Study to Evaluate the Safety, Pharmacokinetics, and Activity of RO7566802 as a Single Agent and i1
Genentech, Inc.
Locally Advanced Solid Tumors
Recurrent Solid Tumors
Metastatic Solid Tumors
This is a first-in-human Phase I, open-label, dose-escalation and expansion study
designed to evaluate the safety, tolerability, pharmacokinetics, immunogenicity,
pharmacodynamic, and preliminary anti-tumor activity of RO7566802 as a single agent and
in combination with atezolizumab in participants1 expand
This is a first-in-human Phase I, open-label, dose-escalation and expansion study designed to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, pharmacodynamic, and preliminary anti-tumor activity of RO7566802 as a single agent and in combination with atezolizumab in participants with locally advanced, recurrent, or metastatic incurable solid tumor malignancies. Participants will be enrolled in 2 stages: dose escalation and expansion. Type: Interventional Start Date: Nov 2023 |
A Study to Evaluate the Efficacy, Safety, and Tolerability of BMS-986278 in Participants With Progr1
Bristol-Myers Squibb
Progressive Pulmonary Fibrosis
The purpose of this study is to evaluate the efficacy, safety, and tolerability of
BMS-986278 in Participants with Progressive Pulmonary Fibrosis. expand
The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986278 in Participants with Progressive Pulmonary Fibrosis. Type: Interventional Start Date: Oct 2023 |
- Previous
- Next