Clinical trials: Genetic Conditions
Huntington's Disease
A Open-label Food Effect Study With SEN0014196 in Subjects With Huntington Disease
The primary purpose of this study is to assess the effect of food upon the pharmacokinetics (PK) of SEN0014196 in subjects with Huntington disease (HD).
- Trial name
- NCT01485965
- Testing
- SEN0014196
- Where
- 7 sites in United States
- Volunteers
- 26 people
- Sponsor
- Siena Biotech S.p.A.
Entrapment Neuropathy
Study Evaluating Treatment of Forefoot Pain Related to Nerve Entrapment Using the Cryo-Touch III Device
A proof of concept study to evaluate the feasibility of safe and effective treatment through optimization of the Cryo-Touch III Device for temporary relief of pain.
- Trial name
- NCT01753778
- Testing
- Cryo-Touch III Device
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pacira Pharmaceuticals, Inc
Hyperuricemia, Gout
Safety, Tolerability and Pharmacokinetic/Pharmacodynamic Characteristics of LC350189
This will be a dose block-randomized, double-blind, active and placebo controlled, single and multiple dosing, dose-escalation study to evaluate tolerability, safety and pharmacokinetic / pharmacodynamic characteristics.
- Trial name
- NCT01361646
- Testing
- LC350189
- Where
- South Korea
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- LG Life Sciences
Respiratory Tract Diseases, Abnormal Mucus Secretions
A Clinical Trial to Compare the Efficacy and Safety of 1-week Treatment of Intravenous N-acetylcysteine (NAC) 600 mg Twice Daily, Ambroxol Hydrochloride 30 mg Twice Daily and Placebo as Expectorant Therapies in Adult Chinese Patients With Respiratory Tract Diseases and Abnormal Mucus Secretions
This is a phase 3, multicenter, randomized, rater- and patient-blind, placebo- and active-controlled, 3-arm parallel group clinical trial.
- Trial name
- NCT03843541
- Testing
- N-acetylcysteine (NAC) 600 mg, Ambroxol hydrochloride 30 mg
- Where
- 32 sites in China
- Volunteers
- 333 people
- Sponsor
- Zambon SpA
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Corneal Edema, Fuchs
Study of EO2002 in Subjects With Corneal Edema Secondary to Corneal Endothelial Dysfunction
This study will evaluate the safety and effectiveness of a single injection of EO2002 at one of two dose levels (150,000 or 500,000 magnetic human corneal endothelial cells [mHCECs]), compared with a placebo injection, in…
- Conditions
- Corneal Edema, Fuchs, Fuchs Dystrophy, Fuchs' Endothelial Dystrophy
- Trial name
- EMERALD (NCT07729137)
- Testing
- 150K cells, 500K cells
- Where
- 10 sites in United States
- Volunteers
- 121 people
- Sponsor
- Emmecell
Cystic Fibrosis, Chronic Pancreatitis
Study to Evaluate Symptoms of Exocrine Pancreatic Insufficiency in Adult Participants With Cystic Fibrosis or Chronic Pancreatitis Treated With Creon
Exocrine pancreatic insufficiency (EPI) is a condition that is caused by the inadequate pancreatic enzymes needed for normal digestion and is commonly associated with a wide range of chronic diseases, including cystic fibrosis…
- Trial name
- NCT05069597
- Testing
- CREON
- Where
- 21 sites in United States
- Volunteers
- 30 people
- Sponsor
- AbbVie
NALP3 Mutation
Safety, Efficacy, Pharmacokinetics, Pharmacodynamics of ACZ885 in Patients With NALP3 Mutations and Clinical Symptoms
This study will investigate the clinical efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of ACZ885, administered intravenously and subcutaneously to patients with NALP3 mutations whose clinical symptoms are…
- Trial name
- NCT00487708
- Testing
- canakinumab
- Where
- 10 sites in France, Germany and 3 more
- Volunteers
- 34 people
- Sponsor
- Novartis Pharmaceuticals
Pulmonary Disease, Chronic Obstructive
Repeat Dose Study in Male Healthy Volunteer Smokers
This is a 4 to 6 week study to look at the safety and PK profile of SB656933 in healthy male smokers
- Trial name
- NCT00615576
- Testing
- SB-656933
- Where
- United Kingdom
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Thalassemia Major
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as…
- Trial name
- NCT02049450
- Testing
- ruxolitinib
- Where
- 7 sites in Greece, Italy and 3 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
CLINICAL EFFICACY OF EMOLLIENTS IN ATOPIC DERMATITIS PATIENTS
his was a monocentric, intra-individual study, that was performed in at least 45 valid cases (50% children ≥3 YO; 50% adults). Study duration was 168 days with five (5) visits (D0, D14, D28, D84 and D168) to the research center.
- Trial name
- NCT05783453
- Testing
- Cosmetic study
- Where
- Brazil
- Volunteers
- 63 people
- Sponsor
- Cosmetique Active International
Atopic Dermatitis, Atopic Dermatitis and Related Conditions
Efficacy and Safety of Blue Cap for the Treatment of Atopic Dermatitis in Children
To verify the efficacy and tolerability of Activated Piroctone Olamine (Blue Cap Foam, label volume 100 ml) by CATALYSIS, S. L. Madrid, applied in the management of all types of dermatitis (atopic, seborrheic, eczema) in patients…
- Conditions
- Atopic Dermatitis, Atopic Dermatitis and Related Conditions, Atopic Dermatitis Eczema, Atopic Dermatitis \(AD\)
- Trial name
- NCT06361992
- Testing
- Blue Cap Foam
- Where
- 3 sites in Serbia
- Volunteers
- 58 people
- Sponsor
- Catalysis SL
Atopic Dermatitis
Efficacy and Safety Study of the Use of Benralizumab for Patients With Moderate to Severe Atopic Dermatitis
The purpose of the study is to compare the efficacy and safety of benralizumab versus placebo and to compare benralizumab dosing regimens during extension period.
- Trial name
- NCT04605094
- Testing
- Benralizumab
- Where
- 51 sites in United States, Australia and 6 more
- Volunteers
- 194 people
- Sponsor
- AstraZeneca
Neoplasms, Breast
Pazopanib Plus Lapatinib Compared To Lapatinib Alone In Subjects With Advanced Or Metastatic Breast Cancer
This study is being conducted to compare the efficacy and safety of pazopanib in combination with lapatinib with that of lapatinib alone in subjects with locally advanced or metastatic breast cancer whose tumors overexpress the…
- Trial name
- NCT00347919
- Testing
- pazopanib (GW786034) 400 mg, lapatinib (GW572016) 1500 mg, lapatinib (GW572016) 1000 mg
- Where
- 84 sites in United States, Canada and 14 more
- Volunteers
- 189 people
- Sponsor
- GlaxoSmithKline
To Determine Bioequivalence Under Fed Conditions
Bioavailability Study of Tramadol/APAP Tablets Under Fed Conditions
To compare the relative bioavailability of Kali and Ortho-McNeil's products
- Trial name
- NCT00652821
- Testing
- Tramadol/ APAP, Ultracet
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Hepato-splenomegaly, Dysostosis Multiplex
Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)
Development of a new MS-based biomarker for the ear-ly and sensitive diagnosis of GM1/GM2 from blood
- Conditions
- Hepato-splenomegaly, Dysostosis Multiplex, Seizures, Mental Retardation
- Trial name
- BioGM1/BioGM2 (NCT02298647)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
22q.11.2 Deletion/Duplication
Non-Invasive Chromosomal Evaluation of 22q11.2
This study is being conducted to develop and evaluate a cell-free fetal DNA test (Harmony) for non-invasive prenatal detection of 22q11.2 chromosomal deletion or duplication.
- Trial name
- 22Q (NCT02541058)
- Where
- Belgium
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Cindy Cisneros
Arrhythmia, Cardiovascular Diseases
Diagnostic Investigation of Sudden Cardiac Event Risk
The overall objective of the DISCERN study is to develop and validate a genomic diagnostic assay to identify patients at increased risk for lethal ventricular arrhythmias and sudden cardiac death (SCD).
- Conditions
- Arrhythmia, Cardiovascular Diseases, Heart Failure, CVD
- Trial name
- DISCERN (NCT00500708)
- Where
- 8 sites in United States
- Volunteers
- 1,564 people
- Sponsor
- CardioDx
Xeroderma Pigmentosum
A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V
The CUV152 study will evaluate the safety of afamelanotide in XP-C and XP-V patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin.
- Trial name
- NCT05370235
- Testing
- Afamelanotide
- Where
- 2 sites in Belgium, Spain
- Volunteers
- 6 people
- Sponsor
- Clinuvel Europe Limited
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Observational Prospective Study on Patients Treated With Norditropin®
This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Adult Growth Hormone Deficiency, Genetic Disorder
- Trial name
- NCT00960128
- Testing
- somatropin
- Where
- 23 sites in Argentina, Belgium and 21 more
- Volunteers
- 21,249 people
- Sponsor
- Novo Nordisk A/S