Clinical trials: Genetic Conditions
Gout, Hyperuricemia
A Study to Assess Efficay and Safety of LC350189 Different Doses in Gout Patients With Hyperuricemia
The aim of this 12-week randomized multicenter double-blind parallel group placebo-controlled dose finding study is to assess the efficacy and safety of three different doses of LC350189 in subjects with hyperuricemia and a…
- Trial name
- NCT03934099
- Testing
- LC350189 formulated capsule
- Where
- 34 sites in United States
- Volunteers
- 156 people
- Sponsor
- LG Chem
Atopic Dermatitis
A Dose Ranging Placebo-Controlled Double-Blind Study to Evaluate the Safety and Efficacy of Tezepelumab in Atopic Dermatitis
This phase 2b study is designed to evaluate the safety and efficacy of tezepelumab as a monotherapy and explore its efficacy as adjunct therapy in subjects with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT03809663
- Testing
- Tezepelumab
- Where
- 86 sites in United States, Australia and 13 more
- Volunteers
- 251 people
- Sponsor
- Amgen
Headache, Tumor
Dexmedetomidine and Propofol for Pediatric MRI Sedation
The purpose of this study is to compare the results of combining two anesthetic medications (dexmedetomidine and propofol) in low doses with a standard dose of a single drug that is commonly used to provide sedation/anesthesia…
- Conditions
- Headache, Tumor, Seizure Disorder, Neurofibromatoses
- Trial name
- NCT03513757
- Testing
- propofol, Dexmedetomidine, Glycopyrrolate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Medical College of Wisconsin
Sickle Cell Disease
Home-Based Intervention for Chronic Pain in Adults With Sickle Cell Disease
This project will evaluate AppliedVR's EaseVRx - a multi-modal, skills-based, 8-week, virtual reality, home intervention - in an exploratory randomized controlled trial for self-management of chronic pain among Black, young…
- Trial name
- NCT04906707
- Testing
- EaseVRx, Audio-only version of EaseVRx
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Emory University
Gout Attack
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Evaluation of RPH-104 Administered at Different Doses to Patients With Acute Gout Attack
The primary goal of the study was to evaluate the parameters of efficacy, pharmacokinetics, pharmacodynamics, safety and tolerability of a single dose of RPH-104 in adult patients with acute gout attack.
- Trial name
- NCT04067492
- Testing
- RPH - 104, Voltaren®
- Where
- 11 sites in Russia
- Volunteers
- 47 people
- Sponsor
- R-Pharm
Cystic Fibrosis
Multicenter Study of Patient-reported Gastrointestinal Symptoms in People With Cystic Fibrosis
This is a prospective, multicenter, observational study designed to collect gastrointestinal related data in patients with Cystic Fibrosis (CF).
- Trial name
- GALAXY (NCT03801993)
- Where
- 26 sites in United States
- Volunteers
- 402 people
- Sponsor
- Chris Goss
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Safety, Pharmacokinetics, Tolerability and Efficacy of Tolvaptan in Children and Adolescents With ADPKD (Autosomal Dominant Polycystic Kidney Disease)
The primary objective of the study is to assess the long term safety of treatment with tolvaptan in children and adolescents with autosomal dominant polycystic kidney disease (ADPKD).
- Trial name
- NCT02964273
- Testing
- Tolvaptan
- Where
- 20 sites in Belgium, Germany and 2 more
- Volunteers
- 91 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Fluid Loss, Symptom Complex, Triple
Saline vs. Lactated Ringers for Emergency Department IV Fluid Resuscitation
Administration of intravascular (IV) fluid is the most common emergency department (ED) procedure. IV fluids are integral to increasing effective blood volume and ensuring organ perfusion in patients with volume depletion and…
- Conditions
- Fluid Loss, Symptom Complex, Triple, Emergencies
- Trial name
- NCT03133767
- Testing
- Lactated Ringer Solution, Normal Saline 0.9% Infusion Solution Bag
- Where
- United States
- Volunteers
- 157 people
- Sponsor
- Nicholas M Mohr
Cystinosis
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
This was a long-term, open-label study of the safety, tolerability and effectiveness of RP103 in cystinosis patients who were naïve to any form of cysteamine treatment.
- Trial name
- NCT01744782
- Testing
- RP103
- Where
- 2 sites in United States, Brazil
- Volunteers
- 17 people
- Sponsor
- Amgen
Atopic Dermatitis
A Study to Evaluate the Safety and Efficacy of Ruxolitinib Phosphate Cream Applied Topically to Adults With Atopic Dermatitis
The purpose of this study is to establish the efficacy of each strength of ruxolitinib cream once daily (QD) or twice daily (BID) in participants with atopic dermatitis as compared with vehicle cream BID.
- Trial name
- NCT03011892
- Testing
- Ruxolitinib 0.15% Cream QD, Ruxolitinib 0.5% Cream QD, Ruxolitinib 1.5% Cream QD
- Where
- 52 sites in United States, Canada
- Volunteers
- 307 people
- Sponsor
- Incyte Corporation
Spinocerebellar Ataxia
Transcranial Magnetic Stimulation in Spino-Cerebellar Ataxia
Spinocerebellar Ataxia (SCA) refers to a family of genetic diseases that cause progressive problems with gait and balance, as well as other debilitating symptoms.
- Trial name
- TMS (NCT01975909)
- Testing
- Transcranial Magnetic Stimulation
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Beth Israel Deaconess Medical Center
Adrenoleukodystrophy
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II.
- Trial name
- NCT00004418
- Testing
- glyceryl trierucate/glyceryl trioleate
- Where
- 2 sites in United States
- Volunteers
- 126 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Ataxia, Cerebellar, Cerebellar Ataxia
Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia
Neurodegenerative cerebellar ataxias represent a group of disabling disorders for which we currently lack effective therapies. Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been…
- Conditions
- Ataxia, Cerebellar, Cerebellar Ataxia, Spinocerebellar Ataxias, Ataxia, Spinocerebellar
- Trial name
- CStDCSAtaxia (NCT03120013)
- Testing
- Anodal cerebellar and cathodal spinal tDCS
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Emphysema, Alpha 1-proteinase Inhibitor Deficiency
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v).
- Trial name
- NCT00670007
- Testing
- Alpha1- proteinase inhibitor [human]
- Where
- 21 sites in Australia, Canada and 9 more
- Volunteers
- 140 people
- Sponsor
- CSL Behring
Pregnancy
Local Anesthesia and Pain Perception During an Amniocentesis
This study is been designed to answer the question of whether local anesthesia (1% lidocaine) decreases the perception of pain associated with amniocentesis in a randomized double blind placebo controlled manner.
- Trial name
- NCT00583011
- Testing
- Local Anesthesia
- Where
- United States
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- University of Oklahoma
Alpha-1 Antitrypsin Deficiency
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
The purpose of the study is to determine the safety and tolerability of escalating doses of ARC-AAT and to evaluate the pharmacokinetics of ARC-AAT and the effect of ARC-AAT on circulating levels of alpha-1 antitrypsin (AAT).
- Trial name
- NCT02363946
- Testing
- ARC-AAT Injection, Diphenhydramine
- Where
- 4 sites in Australia, Germany and 2 more
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Alpha1-antitrypsin Deficiency, Healthy Volunteers
GLASSIA Infusion Rate Study
The purpose of this study was to generate sufficient safety and tolerability information in support of an increase in the infusion rate of intravenous GLASSIA in the prescribing information from 0.04 to 0.2 mL/kg/min.
- Trial name
- NCT01651351
- Testing
- Alpha1-proteinase inhibitor
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Baxalta now part of Shire
Leukemia, Myelogenous, Chronic, Breakpoint Cluster Region-Abelson Proto-oncogene (BCR-ABL) Positive
A Multicenter Phase 3, Open-Label Study of Bosutinib Versus Imatinib in Adult Patients With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia
Phase 3, 2-arm, randomized, open label trial. Patients will be randomized to receive bosutinib or imatinib for the duration of the study.
- Trial name
- NCT02130557
- Testing
- Bosutinib, Imatinib
- Where
- 189 sites in United States, Australia and 24 more
- Volunteers
- 536 people
- Sponsor
- Pfizer
Gout
Allopurinol Versus Febuxostat in Subjects Completing the Phase 3 Trials C02-009 or C02-010
The purpose of this study is to determine the long-term safety of febuxostat, once daily (QD), compared to allopurinol in reducing serum urate levels in subjects with gout.
- Trial name
- EXCEL (NCT00175019)
- Testing
- Febuxostat, Allopurinol
- Volunteers
- 1,086 people
- Sponsor
- Takeda
Sickle Cell Disease
Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of African origin.
- Trial name
- NCT01987908
- Testing
- Aes-103
- Where
- United Kingdom
- Volunteers
- 35 people
- Sponsor
- Baxalta now part of Shire