Clinical trials: Genetic Syndrome
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Epilepsy, Lennox-Gastaut Syndrome
A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults
To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
- Trial name
- GWPCARE4 (NCT02224690)
- Testing
- GWP42003-P 20 mg/kg/day Dose
- Where
- 24 sites in United States, Netherlands and 1 more
- Volunteers
- 171 people
- Sponsor
- Jazz Pharmaceuticals
Dilated Cardiomyopathy, Heart Failure
PUFAs and Left Ventricular Function in Heart Failure
The purpose of this study is to test the hypothesis that n-3 PUFAs improve left ventricular systolic function in patients with stable chronic HF secondary to nonischemic dilated cardiomyopathy (NICM).
- Trial name
- CS-PUFA-02 (NCT01223703)
- Testing
- n-3 PUFAs
- Where
- Italy
- Volunteers
- 133 people
- Sponsor
- Università degli Studi di Brescia
Maroteaux-Lamy Syndrome
Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome
This is a single center study in which Naglazyme® will be given weekly for two years in patients with Maroteaux-Lamy syndrome, also known as mucopolysaccharide VI (MPS VI), who have previously been treated with an allogeneic…
- Trial name
- NCT02156674
- Testing
- Naglazyme®
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Ovarian Cancer, Breast Cancer
A Phase II Single Arm Pilot Study of the Chk1/2 Inhibitor (LY2606368) in BRCA1/2 Mutation Associated Breast or Ovarian Cancer, Triple Negative Breast Cancer, High Grade Serous Ovarian Cancer, and Metastatic Castrate-Resistant Prostate Cancer
Background: All cells go through cycles which allow them to divide. In normal cells, checkpoint kinase 1 (Chk1) and checkpoint kinase 2 (Chk2) (CHEK 2 (Chk1/2) stop cell division at various points to allow any damage to…
- Conditions
- Ovarian Cancer, Breast Cancer, Prostate Cancer
- Trial name
- NCT02203513
- Testing
- LY2606368
- Where
- United States
- Volunteers
- 111 people
- Sponsor
- National Cancer Institute (NCI)
Atopic Dermatitis, Eczema
A Trial of a Botanical Drug Containing East Indian Sandalwood Oil (EISO) For Treatment of Atopic Dermatitis
This trial will be a double-blind, randomized, placebo-controlled, safety, tolerability and efficacy trial of SAN007 (5% East Indian sandalwood oil in a cream formulation) treatment regimen when administered daily for up to 28…
- Trial name
- NCT02871479
- Testing
- SAN007 5% cream, SAN007 10% cream
- Where
- 5 sites in United States
- Volunteers
- 71 people
- Sponsor
- Santalis Pharmaceuticals, Inc.
Epilepsy; Seizure, Neuromuscular Diseases
North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2
The "North Carolina Clinical Genomic Evaluation by Next-gen Exome Sequencing, 2 (NCGENES 2)" study is part of a larger consortium project investigating the clinical utility, or net benefit of an intervention on patient and family…
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability
- Trial name
- NCGENES2 (NCT03548779)
- Testing
- Pre-visit prep, usual care + exome seq
- Where
- 3 sites in United States
- Volunteers
- 548 people
- Sponsor
- University of North Carolina, Chapel Hill
Von Willebrand Diseases, Postpartum Hemorrhage
Prevent Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial
This is a single-center randomized phase III clinical trial, the VWD-Woman Trial, in which 20 pregnant subjects with von Willebrand disease (VWD), defined as VWF ristocetin co-factor activity (VWF:RCo) <0.50 IU/ml (historic) and…
- Trial name
- NCT04344860
- Testing
- Recombinant Von Willebrand factor, Tranexamic Acid Injection [Cyklokapron]
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Nicoletta C Machin
Corneal Edema Secondary to Corneal Endothelial Dystrophy
Early Experience With Descemet's Stripping Automated Endothelial Keratoplasty
The purpose of this study is to report the early clinical outcomes in patients undergoing Descemet's Stripping Automated Endothelial Keratoplasty (DSAEK) for corneal edema secondary to corneal endothelial cell dysfunction, and to…
- Trial name
- DSAEK (NCT00744796)
- Testing
- DSAEK, Anterior Segment Optical Coherence Topography (OCT), Best Spectacle Corrected Visual Acuity (BSCVA)
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Moderate-to-Severe Atopic Dermatitis, Dermatitis, Dermatitis Atopic
Efficacy and Safety of Dupilumab in Participants ≥12 to <18 Years of Age, With Moderate-to-severe Atopic Dermatitis
The primary objective of the study was to demonstrate the efficacy of dupilumab as a monotherapy in participants ≥12 years to <18 years of age with moderate-to-severe atopic dermatitis (AD).
- Conditions
- Moderate-to-Severe Atopic Dermatitis, Dermatitis, Dermatitis Atopic, Eczema, Skin Diseases, Skin, Diseases Genetic, Genetic
- Trial name
- NCT03054428
- Testing
- Dupilumab
- Where
- 45 sites in United States, Canada
- Volunteers
- 251 people
- Sponsor
- Regeneron Pharmaceuticals
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Dermatitis, Atopic
Prevention of Asthma With Levocetirizine 18 Month Treatment in Infants (12 - 24 Months) Suffering From Eczema (Atopic Dermatitis) and Sensitized to Grass Pollen and/or House Dust Mite (HDM)
The Early Prevention of Asthma in Atopic Children (EPAAC™). 24 months study to evaluate the efficacy and safety of levocetirizine (LCTZ) in preventing the onset of asthma in 12 to 24 months old children.
- Trial name
- NCT00152464
- Testing
- Levocetirizine
- Volunteers
- 514 people
- Sponsor
- UCB Pharma SA
Atopic Dermatitis, Atopic Disorders
A Community-based Assessment of Skin Care, Allergies, and Eczema
Atopic dermatitis (AD) affects over 9 million children in the U.S. and often heralds the development of asthma, food allergy, skin infections and neurodevelopmental disorders.
- Conditions
- Atopic Dermatitis, Atopic Disorders, Eczema, Atopic Eczema
- Trial name
- CASCADE (NCT03409367)
- Testing
- Participant choice of over-the-counter emollients: Vaseline, Vanicream, CeraVe Healing Ointment, CeraVe cream, Cetaphil cream
- Where
- 4 sites in United States
- Volunteers
- 1,260 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Diamond Blackfan Anemia
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.
- Trial name
- ACE-011-DBA (NCT01464164)
- Testing
- Sotatercept, Sotatercept with prednisone boost
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Northwell Health
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
Study to Assess an Enteric Microgranule Formulation of Adrulipase in Patients With Cystic Fibrosis
Some cystic fibrosis patients are unable to digest food and absorb nutrition appropriately as they have a condition known as exocrine pancreatic insufficiency (EPI).
- Trial name
- SPAN (NCT05719311)
- Testing
- adrulipase
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Entero Therapeutics
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Atopic Dermatitis, Atopic Dermatitis Eczema
Immunogenetic Profiling of Dupilumab for the Treatment of Atopic Dermatitis
This is a single-arm, open-label study to examine the effect of dupilumab on the immunologic and genetic environment within atopic dermatitis skin lesions.
- Conditions
- Atopic Dermatitis, Atopic Dermatitis Eczema, Eczema, Atopic Dermatitis and Related Conditions
- Trial name
- NCT03293030
- Testing
- Dupilumab
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- University of California, San Francisco
Retinitis Pigmentosa
Feasibility Study of a Chronic Retinal Stimulator in Retinitis Pigmentosa
The objective of this study is to evaluate the safety and efficacy of the retinal stimulation system by evaluating the data after chronic implantation.
- Trial name
- (A16) (NCT00279500)
- Testing
- Argus 16 Retinal Stimulation System
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Second Sight Medical Products
Amyloidosis, Familial, Transthyretin Amyloidosis
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver…
- Trial name
- NCT03862807
- Testing
- Patisiran
- Where
- 9 sites in France, Germany and 5 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
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