Clinical trials: Congenital Deformity
Atopic Dermatitis
Effect of LEO 39652 Cream in Adults With Mild to Moderate Atopic Dermatitis (AD)
The aim of this trial is to assess the efficacy of LEO 39652 cream compared with LEO 39652 cream vehicle in adults with mild to moderate AD after 3 weeks of treatment.
- Trial name
- NCT02219633
- Testing
- LEO 39652 cream
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- LEO Pharma
Homozygous Familial Hypercholesterolemia
A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Clinical Study Evaluating the Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
This is a multicenter, randomized, double-blind, placebo-controlled phase III clinical study to assess the reduction of low-density lipoprotein cholesterol (LDL-C) by SHR-1918 in patients with homozygous familial…
- Trial name
- NCT06723652
- Testing
- SHR-1918
- Where
- China
- Volunteers
- 55 people
- Sponsor
- Beijing Suncadia Pharmaceuticals Co., Ltd
Primary Immune Deficiency Disorder
Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)
The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705).
- Trial name
- NCT01458171
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 23 people
- Sponsor
- CSL Behring
Hypercholesterolemia and Mixed Hyperlipidemia
A Study to Evaluate the Safety, Efficacy of SYH2053 in Chinese Participants With Non-familial Hypercholesterolemia and Mixed Hyperlipidemia
The purpose of this study is to compare the effectiveness, safety of SYH2053 in Participants with primary hypercholesterolemia or mixed dyslipidemia. A total of 156 Participants were actually enrolled
- Trial name
- NCT07421232
- Testing
- Inclisiran (Leqvio®) 300mg
- Where
- China
- Volunteers
- 156 people
- Sponsor
- CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
Prader-Willi Syndrome
Cannabidiol Oral Solution for the Treatment of Patients With Prader-Willi Syndrome
The primary objective of this study is to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior in patients with Prader-Willi Syndrome (PWS).
- Trial name
- NCT02844933
- Testing
- Cannabidiol
- Where
- 7 sites in United States
- Volunteers
- 7 people
- Sponsor
- Radius Pharmaceuticals, Inc.
Mild Cognitive Impairment, Dementia
COMPARATIVE EFFECTIVENESS OF MCI and DEMENTIA TREATMENTS IN A COMMUNITY-BASED DEMENTIA PRACTICE
This retrospective study is a more extensive, confirmatory analysis of the cognitive and functional outcomes initially seen in 2 groups of MCI/dementia patients in Springfield, MA and compares specialized dementia care and a…
- Conditions
- Mild Cognitive Impairment, Dementia, Hypoxia, Hyperhomocysteinemia
- Trial name
- NCT02860338
- Volunteers
- 900 people
- Sponsor
- Clionsky Neuro Systems Inc.
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENEREXT Study
This is an open-label, multi-centre study in subjects with a genetically confirmed mitochondrial deoxyribonucleic acid (DNA) transfer ribonucleic acid (tRNA)Leu(UUR) m.3243A>G mutation who completed study KH176-202.
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS)
- Trial name
- NCT04604548
- Testing
- Oral administration of 100 mg KH176 twice daily
- Where
- 4 sites in Denmark, Germany and 2 more
- Volunteers
- 11 people
- Sponsor
- Khondrion BV
RETT Syndrome With Proven MECP2 Mutation, Rett Syndrome
Efficacy and Safety of NTI164 in Children and Young Adults With Rett Syndrome
The FENRTT2 study will investigate the efficacy and safety of a medicinal cannabis plant extract with extremely low THC (delta-9-tetrahydrocannabinol), NTI164, on Rett syndrome (RTT) in a crossover design.
- Trial name
- TRANSCEND (NCT07257978)
- Testing
- NTI164
- Where
- Australia
- Volunteers
- 40 people
- Sponsor
- Fenix Innovation Group
Atopic Dermatitis
A Trial to Evaluate the Efficacy and Safety of Different Doses of LEO 138559 in Adults With Moderate-to-severe Atopic Dermatitis
The purpose of this trial is to test different doses of the trial medicine (LEO 138559) and see how well they work and how safe they are at treating moderate to severe atopic dermatitis in adults.
- Trial name
- NCT05923099
- Testing
- LEO 138559
- Where
- 73 sites in United States, Canada and 9 more
- Volunteers
- 262 people
- Sponsor
- LEO Pharma
Neurologic Disorder, Paralysis
SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis
The Synchron Motor Neuroprosthesis (MNP) is intended to be used in subjects with severe motor impairment, unresponsive to medical or rehabilitative therapy and a persistent functioning motor cortex.
- Conditions
- Neurologic Disorder, Paralysis, Paralysis; Stroke, Amyotrophic Lateral Sclerosis
- Trial name
- NCT05041114
- Testing
- Motor Neuroprosthesis
- Where
- 3 sites in Australia
- Sponsor
- Synchron Medical, Inc.
Gout, Hyperuricemia
Placebo Controlled, Multiple Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SAP-001 in Gout Patients
This is a Phase II, Multicenter, Randomized, Double-blind, Placebo controlled, Multiple Dose study to evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SAP-001 in Gout Patients with Hyperuricemia.
- Trial name
- NCT04040816
- Testing
- SAP-001
- Where
- 6 sites in United States
- Volunteers
- 40 people
- Sponsor
- Shanton Pharma Co., Ltd.
Tourette Syndrome
The Safety and Efficacy of Long-term Treatment of PINS Stimulator System for Tourette Syndrome
The purpose of this clinical study is to verify the long term effectiveness and safety of a bilateral deep brain stimulation (DBS) produced by Beijing PINS Medical Co., Ltd.
- Trial name
- NCT02253498
- Testing
- deep brain stimulation (DBS)
- Volunteers
- 20 people
- Sponsor
- Beijing Pins Medical Co., Ltd
Coarctation of the Aorta, Pulmonary Artery Stenosis
Growth Trial: Study of the Renata Minima Stent
The objective of the clinical investigation is to access clinical safety and effectiveness of the Minima Stent in neonates, infants, and young children requiring intervention for common congenital vascular stenosis (i.e.…
- Trial name
- NCT05086016
- Testing
- Catheterized Stenting
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Renata Medical
Pectus Excavatum Deformity
Treatment of Pectus Excavatum Deformity Using Macrolane Filler
This is a prospective, open, non-comparative and baseline-controlled study to evaluate efficacy and safety of Macrolane VRF20 treatment in 40 subjects with pectus excavatum deformity.
- Trial name
- NCT01750112
- Testing
- Macrolane VRF20
- Where
- 2 sites in France, Sweden
- Volunteers
- 23 people
- Sponsor
- Galderma R&D
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.
Atopic Dermatitis
A Study to Evaluate Long-Term Maintenance Treatment With Once Daily Crisaborole Ointment 2% in Pediatric and Adult Participants With Mild-to-Moderate Atopic Dermatitis
This study will evaluate crisaborole therapy once daily (QD) as a long-term topical maintenance therapy for the reduction of flare in responders to crisaborole twice daily (BID) treatment.
- Trial name
- NCT04040192
- Testing
- Crisaborole 2%, Vehicle
- Where
- 44 sites in United States, Australia and 4 more
- Volunteers
- 620 people
- Sponsor
- Pfizer
Arginase I Deficiency, Hyperargininemia
A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency
A Phase 1/2 Open-label Study in Patients with Arginase I Deficiency to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics of Intravenous AEB1102.
- Trial name
- NCT02488044
- Testing
- AEB1102
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 16 people
- Sponsor
- Aeglea Biotherapeutics
Thyroid Eye Disease
A Study of IBI311 in Subjects With Active Thyroid Eye Disease
This is a multicenter, randomized, double-masked, placebo-controlled Phase II/III study in subjects with active thyroid eye disease.
- Trial name
- NCT05795621
- Testing
- IBI311
- Where
- China
- Volunteers
- 115 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Primary Hypercholesterolemia or Mixed Hyperlipidemia
A Study to Investigate the Effectiveness and Safety of Bempedoic Acid in Patients With Primary Hypercholesterolemia or Mixed Dyslipidemia in Taiwan
Cardiovascular disease (CVD) is a chronic non-communicable disease among the most common causes of death worldwide. Substantial reductions in risks of CVD can be achieved through the management of modifiable risk factors…
- Trial name
- CLEAR Taiwan (NCT06925100)
- Testing
- Bempedoic acid 180 mg tablet
- Where
- 3 sites in Taiwan
- Volunteers
- 180 people
- Sponsor
- Daiichi Sankyo
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)
This is an open label, Phase 1/2 study of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase, catalytic subunit alpha (PIK3CA)-related Overgrowth…
- Trial name
- MOSAIC (NCT03094832)
- Testing
- Miransertib
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 50 people
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
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