Clinical trials: Newborn
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Rare Diseases
NICUSeq: A Trial to Evaluate the Clinical Utility of Human Whole Genome Sequencing (WGS) Compared to Standard of Care in Acute Care Neonates and Infants
Prospective, multi-site, study to evaluate the clinical utility of cWGS in a proband. One group will receive cWGS and a clinical report approximately 15 days after blood samples are received, while the other group will continue…
- Trial name
- NICU-Seq (NCT03290469)
- Testing
- clinical whole genome sequencing (cWGS)
- Where
- 5 sites in United States
- Volunteers
- 355 people
- Sponsor
- Illumina, Inc.
Skin Laxity
Retrospective Evaluation of Combination Treatment With the Ulthera System
A retrospective study to evaluate the Ulthera® System for its potential interaction with toxins and fillers. Up to 500 subjects will be enrolled.
- Trial name
- NCT02444169
- Testing
- Ultherapy, Incobotulinumtoxin A, Radiesse
- Where
- 5 sites in United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
Gout, Arthritis, Gouty
Phase 3 Evaluation of Efficacy and Safety of AR882 in Gout Patients (AR882-302)
This study will assess the serum uric acid lowering effect and safety of AR882 in gout patients at two doses compared to placebo over 12 months
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Trial name
- NCT06439602
- Testing
- AR882 50 mg, AR882 75 mg
- Where
- 68 sites in United States, Australia and 3 more
- Volunteers
- 818 people
- Sponsor
- Arthrosi Therapeutics
Phenylketonuria (PKU)
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570).
- Trial name
- NCT03694353
- Testing
- Pegvaliase
- Where
- 17 sites in United States
- Volunteers
- 37 people
- Sponsor
- BioMarin Pharmaceutical
Infant Nutritional Physiological Phenomena, Infant Formula
Gastrointestinal Tolerability of a Partially Hydrolyzed Ready-to-feed Infant Formula in Healthy Newborns
Gastrointestinal tolerability of a partially hydrolyzed, whey-based, ready-to-feed infant formula in healthy newborns during the birth hospitalization period: a post-market study
- Trial name
- NCT05097924
- Testing
- NAN Supreme HypoAllergenic Starter Infant Formula in ready-to-feed format
- Where
- Saudi Arabia
- Volunteers
- 150 people
- Sponsor
- Société des Produits Nestlé (SPN)
Lax Skin
Treatment for Lifting Upper Arm Lax Skin
Open-label, non-randomized, prospective, multi-center, self-controlled clinical study with masked evaluation.
- Trial name
- NCT05358327
- Testing
- Sofwave
- Where
- 4 sites in United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Anti-Psychotic, Management of Manifestations of Psychotic Disorders
Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride
Cycle Pharmaceuticals Ltd. (Cycle) is developing an oral tablet formulation of Chlorpromazine Hydrochloride and intends to conduct bioequivalence trials to demonstrate its similarity to the RLD.
- Conditions
- Anti-Psychotic, Management of Manifestations of Psychotic Disorders, Treatment of Schizophrenia, Control Nausea and Vomiting
- Trial name
- NCT02943213
- Testing
- Chlorpromazine Hydrochloride
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Dermatitis, Atopic, Pruritus
The Efficacy of 3% Kanuka Oil Versus 1% Hydrocortisone Cream in Patients With Atopic Dermatitis
Atopic dermatitis is a common disease affecting millions worldwide. A common associated symptom in this population is pruritus. We seek to investigate the use of an natural, over-the-counter cosmeceutical to help this population
- Trial name
- NCT06488742
- Testing
- 3% kanuka oil
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nexgen Dermatologics, Inc.
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Atopic Dermatitis
A First-in-Human Study of CEE321 in Adult Subjects
Open label, non-randomized, uncontrolled, First-in-Human Phase 1 Study in Healthy Subjects and Subjects with Atopic Dermatitis
- Trial name
- NCT04612062
- Testing
- CEE321
- Where
- 3 sites in United States, Japan
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109…
- Trial name
- NCT02544451
- Testing
- LUM/IVA
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 246 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
Tyrosinemias, Alkaptonuria
Evaluation of TYR Sphere
For 28 days, 20 patients with tyrosinaemia will take TYR sphere as part of the usual dietary management of their condition. Patients will record their adherence to recommended intakes each day, gastrointestinal symptoms (if any)…
- Conditions
- Tyrosinemias, Alkaptonuria, Tyrosinemia, Type I, Tyrosinemia, Type II
- Trial name
- NCT04196959
- Testing
- TYR sphere
- Where
- 2 sites in United Kingdom
- Volunteers
- 18 people
- Sponsor
- Vitaflo International, Ltd
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
Atopic Dermatitis
Clinical Relevance of Scratching and Sleep in Atopic Dermatitis
Atopic Dermatitis (AD), otherwise known as (atopic) eczema, is a chronic relapsing inflammatory skin disease. For difficult-to-treat AD, treatment options are limited.
- Trial name
- NCT05467046
- Testing
- Smartwatch system
- Where
- Netherlands
- Volunteers
- 16 people
- Sponsor
- Philips Clinical & Medical Affairs Global
Hemophilia B
A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy
Severe haemophilia B (HB) is a bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing.
- Trial name
- NCT03641703
- Testing
- FLT180a
- Where
- 6 sites in United Kingdom
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- RADICHOL 1 (NCT00607373)
- Testing
- mipomersen
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 51 people
- Sponsor
- Kastle Therapeutics, LLC
Gout, Hyperuricemia
Mass Balance Study of [14C]LC350189 in Healthy Volunteers
This is a Phase 1, open-label, single-dose, mass balance study designed to evaluate the absorption, metabolism, and excretion of [14C] radiolabeled LC350189 after oral administration.
- Trial name
- NCT04070846
- Testing
- [14C] LC350189
- Where
- United States
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- LG Chem
Skin Alterations Associated With Atopic Dermatitis
Sertaconazole 2% Cream to Improve Symptoms Associated With Atopic Dermatitis
Investigation aimed to explore the efficacy of a topical Sertaconazole cream 2% in patients with skin alterations associated with atopic dermatitis
- Trial name
- NCT01792713
- Testing
- Sertaconazole 2% cream
- Where
- 2 sites in Germany
- Volunteers
- 70 people
- Sponsor
- Spirig Pharma Ltd.