Clinical trials: Fetal Developmental Abnormality
Cystic Fibrosis (CF)
: TRANSITION: An Observational Study of Transition From Lumacaftor/Ivacaftor to Tezacaftor/Ivacaftor (Tez/Iva)
This study is a single center study of clinical and laboratory outcomes in patients ≥ 12 who transition from use of Orkambi to tez/iva.
- Trial name
- NCT03445793
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Jewish Health
Atopic Dermatitis (AD)
Study to Determine the Safety and Effectiveness of Dupilumab for Treatment of Atopic Dermatitis (AD)
The primary objective of the study was to assess the efficacy of Dupilumab, compared to placebo, in adult patients with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01979016
- Testing
- Dupilumab, Background treatment
- Where
- 4 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Regeneron Pharmaceuticals
Hereditary Angioedema
Open-Label Safety, PK, and Efficacy Trial of Sebetralstat (KVD900) in Pediatric Patients (Ages 2-11) With HAE Type I or II
KVD900-303 is an open-label, multicenter clinical trial in patients aged 2 to 11 years old with HAE Type I or II.
- Trial name
- KONFIDENT-KID (NCT06467084)
- Testing
- KVD900 150 mg, KVD900 300 mg, KVD900 600 mg
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Early Manifest Huntington Disease
A Dose Range Finding Study With Open-Label Extension to Evaluate the Safety of Oral LMI070/Branaplam in Early Manifest Huntington's Disease
This is the first study of branaplam in adults with Huntington's Disease (HD) to determine the correct dose required to lower mutant huntingtin protein (mHTT) levels in the cerebrospinal fluid (CSF) to a degree expected to be…
- Trial name
- VIBRANT-HD (NCT05111249)
- Testing
- Branaplam
- Where
- 12 sites in Canada, France and 3 more
- Volunteers
- 26 people
- Sponsor
- Novartis Pharmaceuticals
Degenerative Spinal Disease, Deformity of Spine
Fenestrated Screw Study
The primary objective of this post-market study is to demonstrate that Oswestry disability index (ODI) score improved significantly at 12 months post-operatively as compared to baseline for each indication (degenerative spinal…
- Trial name
- FNS (NCT03797144)
- Testing
- CD HORIZON® Fenestrated Screw System with Fenestrated Screw Cement
- Where
- 6 sites in Belgium, Finland and 3 more
- Volunteers
- 27 people
- Sponsor
- Medtronic Spinal and Biologics
Graves Ophthalmopathy
Glucocorticoids and Bone in Graves' Ophthalmopathy
Continuous use of systemic glucocorticoids decreases bone mineral density and increases fracture risk. Graves' orbitopathy is treated with weekly infusion of high-dose intravenous glucocorticoid.
- Trial name
- NCT03122847
- Testing
- Methylprednisolone
- Where
- 2 sites in Denmark
- Volunteers
- 39 people
- Sponsor
- Torben Harsløf
Homozygous Familial Hypercholesterolemia (HoFH)
A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
The purpose of the study is to establish the efficacy, safety and tolerability of rosuvastatin in children and adolescents with homozygous familial hypercholesterolemia.
- Trial name
- HYDRA (NCT02226198)
- Testing
- Rosuvastatin 20mg
- Where
- 9 sites in Belgium, Canada and 5 more
- Volunteers
- 20 people
- Sponsor
- AstraZeneca
Hereditary Angioedema, HAE
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in…
- Trial name
- APeX-J (NCT03873116)
- Testing
- BCX7353 capsules
- Where
- 10 sites in Japan
- Volunteers
- 19 people
- Sponsor
- BioCryst Pharmaceuticals
Atopic Dermatitis
Apremilast for Atopic Dermatitis - A Pilot Study in Adults
The purpose of this study is to obtain preliminary data regarding the safety and tolerability of apremilast in AD to support the design of larger controlled studies.
- Trial name
- NCT01393158
- Testing
- Apremilast
- Volunteers
- 16 people
- Sponsor
- Oregon Health and Science University
Erythropoietic Protoporphyria (EPP)
Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria
The purpose of this study is to investigate the efficacy and safety of MT-7117 on sunlight exposure duration without symptoms and tolerance in subjects with EPP.
- Trial name
- NCT03520036
- Testing
- MT-7117 low dose, MT-7117 high dose
- Where
- 9 sites in United States
- Volunteers
- 102 people
- Sponsor
- Tanabe Pharma America, Inc.
Atopic Dermatitis
Study of TER-101 Topical Ointment in Subjects With Atopic Dermatitis
This Phase 2 study will assess efficacy, safety, and tolerability of TER-101 ointment and vehicle twice daily for 28 days in adult and adolescent subjects with mild to moderate atopic dermatitis.
- Trial name
- NCT04753034
- Testing
- TER-101, Vehicle
- Where
- 12 sites in United States
- Volunteers
- 63 people
- Sponsor
- Teres Bio, Inc.
Trigeminal Nerve Injuries, Post-herpetic Neuralgia
Transcranial Magnetic Stimulation for Facial Pain
Complex oro-facial pain is a poorly diagnosed and poorly treated cause of significant suffering and disability. This study will explore the ability of fMRI to identify patients with complex oro-facial pain who respond to…
- Conditions
- Trigeminal Nerve Injuries, Post-herpetic Neuralgia, Facial Pain, Nervus Intermedius Neuralgia
- Trial name
- NCT02633306
- Testing
- transcranial magnetic stimulation
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Minnesota
Skin Laxity
Simulines Non-Inferiority Pivotal Study
Up to 260 subjects presenting with skin laxity on the face and neck will be randomized to one of two treatment groups. Subjects meeting all entrance criteria will receive study treatment, then complete two post treatment phone…
- Trial name
- NCT02736825
- Testing
- Ulthera System with standard transducer, Ulthera System with prototype 2 simulines transducer
- Where
- 8 sites in United States
- Volunteers
- 262 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
Ataxia, Cerebellar, Cerebellar Ataxia
Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia
Neurodegenerative cerebellar ataxias represent a group of disabling disorders for which we currently lack effective therapies. Cerebellar transcranial direct current stimulation (tDCS) is a non-invasive technique, which has been…
- Conditions
- Ataxia, Cerebellar, Cerebellar Ataxia, Spinocerebellar Ataxias, Ataxia, Spinocerebellar
- Trial name
- CStDCSAtaxia (NCT03120013)
- Testing
- Anodal cerebellar and cathodal spinal tDCS
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia
Congenital Pediatric Disorders
Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
The purpose of this study is to determine the safety and effectiveness of Umbilical Cord Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can decrease the incidence of GVHD.
- Trial name
- UCB (NCT00950846)
- Testing
- Busulfan, Cytoxan, Fludarabine
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Baylor College of Medicine
Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia
An Epidemiological Study to Assess Iron Overload Using MRI in Patients With Transfusional Siderosis (TIMES Study)
Iron, one of the most common elements in nature and the most abundant transition metal in the body, is readily capable of accepting and donating electrons.
- Trial name
- NCT01736540
- Testing
- MRI scan
- Where
- 13 sites in Australia
- Volunteers
- 243 people
- Sponsor
- Novartis Pharmaceuticals
Laryngomalacia, Gastro Esophageal Reflux
Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia
Laryngomalacia (LM) is the most common cause of stridor in infants. Symptoms of gastroesophageal reflux (GER) are often seen in the setting of LM; therefore, acid suppression therapy (AST) has been empirically used in the…
- Trial name
- NCT04614974
- Testing
- Famotidine, Speech Language Therapy
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Reema Padia
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00847379
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 173 people
- Sponsor
- PTC Therapeutics
Congenital Diaphragmatic Hernia
Airway Inflammation in Congenital Diaphragmatic Hernia Patients
We propose to examine Congenital Diaphragmatic Hernia (CDH) patients for evidence of airway inflammation as a first step to characterize the reported bronchodilator responsiveness.
- Trial name
- NCT02453750
- Testing
- Hypersaline, Bronchodilator Response
- Where
- Canada
- Volunteers
- 21 people
- Sponsor
- The Hospital for Sick Children
Recessive Dystrophic Epidermolysis Bullosa
A Pilot Study to Evaluate a Temporary Skin Substitute (Spincare® Matrix) for Wound Healing in RDEB Patients
Recessive dystrophic epidermolysis bullosa (RDEB) is a subtype of epidermolysis bullosa (EB), an inherited skin condition that presents with blistering skin.
- Trial name
- RDEB (NCT05944250)
- Testing
- Spincare matrix
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
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