Clinical trials: Genetic Condition
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.
Urea Cycle Disorder
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
This is a randomized, controlled, open-label parallel arm study to assess the safety, tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to Sodium phenylbutyrate (NaPBA) in urea cycle disorder subjects…
- Trial name
- NCT03335488
- Testing
- RAVICTI, NaPBA
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 16 people
- Sponsor
- Amgen
Atopic Dermatitis, Healthy Volunteers
Safety Study of Tezepelumab (AMG 157) in Healthy Adults and Adults With Atopic Dermatitis
This study is a single dose escalation study of tezepelumab (AMG 157) in healthy adults (Part A) and adults with moderate to severe atopic dermatitis (Part B).
- Trial name
- NCT00757042
- Testing
- Tezepelumab
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Amgen
Gorlin Syndrome
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
- Trial name
- NCT06050122
- Testing
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Where
- 40 sites in United States, France and 5 more
- Volunteers
- 113 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Muscular Dystrophy, Duchenne
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety…
- Trial name
- NCT02369731
- Where
- 71 sites in Austria, Brazil and 14 more
- Volunteers
- 316 people
- Sponsor
- PTC Therapeutics
C1 Esterase Inhibitor Deficiency, Angio Edema
Biomarker for Hereditary AngioEdema Disease
International, multicenter, observational, longitudinal monitoring study to identify, validate and/or monitor Mass Spectrometry (MS)-based biomarker/s for Hereditary Angioedeme (HAE) disease and to test the clinical robustness…
- Conditions
- C1 Esterase Inhibitor Deficiency, Angio Edema, C4 Deficiency, HAE
- Trial name
- BioHAE (NCT03029728)
- Where
- 7 sites in Armenia, Georgia and 5 more
- Volunteers
- 42 people
- Sponsor
- CENTOGENE GmbH Rostock
X-linked Hypophosphatemia
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab…
- Trial name
- NCT03920072
- Testing
- Burosumab
- Where
- 10 sites in France, Ireland and 2 more
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Immune System and Related Disorders
Creation of a Prospective Cohort of Healthy and Sick Subjects and of a Collection of Associated Biological Resources, for the Study of the Immune System and of Its Genetic and Environmental Determinants.
CoSImmGEn is a protocol set up to respond to the current lack of healthy and sick population cohorts. Biological resources from these cohorts allow researchers to study the immune system and its genetic and environmental…
- Trial name
- CoSImmGEn (NCT03925272)
- Testing
- Collection of samples (blood, stool, etc.), Genetic determinants analysis, Sample obtained after surgery performed in the context of care
- Where
- 4 sites in France
- Volunteers
- 2,200 people, healthy volunteers welcome
- Sponsor
- Institut Pasteur