Clinical trials: Abnormalities
Hereditary Angioedema (HAE)
A Survey of Lanadelumab in Participants With Hereditary Angioedema
This study is a survey in Japan of Lanadelumab used to treat people with hereditary angioedema (HAE). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics…
- Trial name
- NCT05397431
- Testing
- Lanadelumab
- Where
- Japan
- Volunteers
- 155 people
- Sponsor
- Takeda
Cystic Fibrosis
A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
This study will evaluate the efficacy of ivacaftor treatment in subjects with CF 6 years of age and older who have a 3849 + 10KB C→T or D1152H CFTR mutation.
- Trial name
- NCT03068312
- Testing
- Ivacaftor
- Where
- Israel
- Volunteers
- 38 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hallux Valgus Deformity
A Phase 1 Study of Intraoperative Administration of CA-008 for the Correction of Hallux Valgus Deformity
This is a single-center, randomized, double-blind, placebo-controlled, single ascending dose, sequential-group Phase 1 study. The study will be conducted utilizing a cohort design, with sequential groups of 8 subjects.
- Trial name
- NCT03307837
- Testing
- CA-008
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Concentric Analgesics
Skin Laxity, Skin Lines
Improved Skin Anti-aging Outcomes Associated With Collagen Fortified Consumption In 30 to 50-Year-old Women
The goal of this clinical trial is to study the efficacy of collagen supplement on the moisture content and elasticity. 70 participants of 30-50 years old women will be enrolled from two study sites and randomly assigned to use…
- Trial name
- NCT05670977
- Testing
- Study product plus collagen supplement, Study product without collagen supplement
- Where
- 2 sites in China
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- Guangdong HuaPeptides Biotechnology Co., Ltd.
Infantile Colic
Preliminary Efficacy and Safety Study of Oral Nepadutant in Infant With Colic Not Responding to Conventional Treatment
This phase IIa study is designed as a multi-centre, single country, randomised, double-blind, placebo controlled study in three parallel groups, with the aim to evaluate the efficacy and safety of Nepadutant given at two oral…
- Trial name
- nocry-a (NCT01309009)
- Testing
- Nepadutant oral solution
- Sponsor
- Menarini Group
Infertility
Preimplantation Genetic Diagnosis for the Indication of Advanced Reproductive Age
The objective of this study is to demonstrate that Preimplantation Genetic diagnosis will significantly reduce spontaneous abortions and increase ongoing pregnancy rates in patients of advanced maternal age (37-42) undergoing…
- Trial name
- NCT00646893
- Testing
- Preimplantation Genetic Diagnosis (PGD)
- Where
- 5 sites in United States, Spain
- Volunteers
- 1,200 people
- Sponsor
- Reprogenetics
Becker Muscular Dystrophy
A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight <50 kg) daily…
- Trial name
- NCT05166109
- Testing
- Vamorolone
- Where
- 2 sites in United States, Italy
- Volunteers
- 46 people
- Sponsor
- ReveraGen BioPharma, Inc.
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the…
- Trial name
- NCT02709109
- Testing
- VX-371 + HS, Hypertonic saline, Orkambi
- Where
- 40 sites in United States, France and 2 more
- Volunteers
- 147 people
- Sponsor
- Parion Sciences
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Polycystic Kidney Disease 1 (PKD1) Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease
The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.
- Trial name
- NCT06747572
- Where
- 45 sites in United States, Belgium and 7 more
- Volunteers
- 401 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Neuromuscular Diseases, Cerebral Palsy
Rehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral Palsy
The purpose of this multicentric study is to assess the rehabilitative effect derived from the use of the ATLAS exoskeleton in children with neuromuscular diseases or cerebral palsy and with a level of less than 3 or less in the…
- Conditions
- Neuromuscular Diseases, Cerebral Palsy, SMA II
- Trial name
- NCT04813601
- Testing
- ATLAS 2030
- Where
- Spain
- Volunteers
- 10 people
- Sponsor
- MarsiBionics
Multicystic Dysplastic Kidney, Polycystic Kidney Disease
Amnioinfusion for Fetal Renal Failure
The goal of this clinical trial is to learn if serial amnioinfusions can improve the chances of survival for fetuses with severe kidney problems that cause low amniotic fluid (anhydramnios).
- Conditions
- Multicystic Dysplastic Kidney, Polycystic Kidney Disease, Fetal Renal Anomaly, Anhydramnios
- Trial name
- NCT06728228
- Testing
- Amnioinfusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Fetal Care Center, PLLC
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
Evaluation of an Adapted Formula on Atopic Dermatitis.
The aim of this study is to show the efficiency of a new infant formula containing fiber on the management of moderate to severe atopic dermatitis.
- Trial name
- NCT05318300
- Testing
- tested formula with fiber, Control
- Where
- 3 sites in Belgium, France
- Volunteers
- 126 people
- Sponsor
- United Pharmaceuticals
Heart Septal Defect, Heart Septal Defects, Atrial
Safety and Efficacy Study of reSept ASD Occluder for Treating Secundum ASD
Evaluation of the safety and efficacy of the reSept ASD Occluder to treat patients with clinically significant secundum atrial septal defect
- Conditions
- Heart Septal Defect, Heart Septal Defects, Atrial, Heart Defects, Congenital, Cardiovascular Abnormalities
- Trial name
- ASCENT ASD (NCT04591392)
- Testing
- reSept ASD Occluder
- Where
- 32 sites in United States, Canada and 2 more
- Volunteers
- 250 people
- Sponsor
- atHeart Medical
Muscular Dystrophies
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
- Trial name
- NCT01480245
- Testing
- GSK2402968
- Where
- 59 sites in Argentina, Australia and 22 more
- Volunteers
- 233 people
- Sponsor
- GlaxoSmithKline
Hemophilia A or B With Inhibitors
A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male
A Phase 1, randomized, single-blind, placebo-controlled, single dose, dose-escalation study to assess the safety, pharmacokinetic and pharmacodynamic profile of subcutaneous administration of a long-acting recombinant factor VIIa…
- Trial name
- NCT02919800
- Testing
- MOD-5014
- Where
- Israel
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- OPKO Health, Inc.
Fabry Disease, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Wrinkle, Lax Skin
A Prospective Controlled Study to Evaluate the Safety and Effectiveness of Thermage® FLX Radiofrequency Treatment
A prospective, 180-day, randomized, multicenter, independent blinded evaluators, controlled study of treatment with the Thermage FLX System
- Conditions
- Wrinkle, Lax Skin, Lines Skin
- Trial name
- NCT07187297
- Testing
- Thermage FLX
- Where
- 7 sites in United States
- Volunteers
- 164 people, healthy volunteers welcome
- Sponsor
- Bausch Health Americas, Inc.