Clinical trials: Congenital Disorder
Congenital Heart Disease in Children
BAX602 in Preventing the Adhesion Around Great Cardiac Vessels in Pediatric Patients With Congenital Heart Disease Undergoing Re-do Open Heart Surgery (Japan Trial)
Congenital heart disease occurs in about 1% of live births and can range from (1) naturally curable conditions that require no treatment to (2) conditions that require multiple immediate operations or refractory severe…
- Trial name
- NCT05647161
- Testing
- BAX602, No Intervention
- Where
- 6 sites in Japan
- Volunteers
- 34 people
- Sponsor
- Baxter Healthcare Corporation
Atopic Dermatitis (Eczema)
The Effects of a Botanical Moisturizer and an Unscented Soap on Eczema and Itch
The purpose of this study is to examine the effects of a botanical moisturizer and an unscented soap on eczema severity, itch, mood, and various skin measures (such as skin hydration) in those with mild to moderate eczema.
- Trial name
- NCT06804070
- Testing
- Botanical Moisturizer + Unscented Soap
- Where
- Poland
- Volunteers
- 34 people
- Sponsor
- Codex Labs Corporation
Atherosclerosis, Coronary Artery Disease
MARS - Monitored Atherosclerosis Regression Study
The purpose of this study is to determine whether significant alterations in serum lipoproteins as provided by the drug lovastatin can substantially reduce atherosclerosis progression or even induce regression.
- Trial name
- NCT00116870
- Testing
- lovastatin
- Where
- United States
- Volunteers
- 270 people
- Sponsor
- Merck Sharp & Dohme LLC
Arteriovenous Fistula
Study Comparing Lutonix AV Drug Coated Balloon vs Standard Balloon for Treatment of Dysfunctional AV Fistulae
This prospective, global, multicenter, randomized, controlled study is designed to evaluate the safety and effectiveness of the Lutonix 035 AV Drug Coated Balloon PTA Catheter compared to a standard PTA Catheter in treating…
- Trial name
- NCT02440022
- Testing
- Lutonix DCB, Standard Balloon Angioplasty Catheter, Percutaneous Transluminal Angiography
- Where
- 23 sites in United States
- Volunteers
- 285 people
- Sponsor
- C. R. Bard
Atopic Dermatitis
A Study in Male and Female Adult Patients With Atopic Dermatitis Treated With Dupilumab in Taiwan
Primary objective: To characterize the use patterns of phototherapy and immunosuppressants prior to dupilumab treatment for adult AD patients, who are eligible for dupilumab reimbursement in Taiwan (e.g., used regimens, reason…
- Trial name
- NCT05442645
- Where
- China
- Volunteers
- 80 people
- Sponsor
- Sanofi
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Phenylketonurias, PKU
Evaluation of PKU Sphere in Italy
20 participants with PKU will build-up their dietary intake of PKU sphere over 2-16 weeks, depending on their level of metabolic control whilst doing so.
- Trial name
- NCT04679467
- Testing
- PKU sphere
- Where
- 4 sites in Italy
- Volunteers
- 13 people
- Sponsor
- Vitaflo International, Ltd
Hemophilia B
A Gene Therapy Study for Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
- Trial name
- NCT02484092
- Testing
- SPK-9001
- Where
- 10 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Pfizer
Hypercholesterolemia, Familial
Study To Evaluate The Effect Of Torcetrapib/Atorvastatin In Patients With Genetic High Cholesterol Disorder
The Torcetrapib project was terminated on December 2, 2006 due to safety findings. To evaluate the efficacy and safety of the lipid drug Torcetrapib/atorvastatin in patients with genetically known disorder of extremely high…
- Trial name
- NCT00134511
- Testing
- Torcetrapib/atorvastatin
- Where
- 6 sites in United States, Canada and 1 more
- Volunteers
- 30 people
- Sponsor
- Pfizer
OTC Deficiency
Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency
The primary objective is to evaluate the efficacy of DTX301 on the improvement of ornithine transcarbamylase (OTC) function by maintaining safe plasma ammonia levels.
- Trial name
- NCT05345171
- Testing
- DTX301, Oral Corticosteroids, Sodium Acetate
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 37 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Atopic Dermatitis
Study to Investigate the Effect of Rocatinlimab (AMG 451) on the Pharmacokinetics of Multiple Cytochrome P450 (CYP450) Substrates in Participants With Moderate to Severe Atopic Dermatitis
The primary objective of the study is to evaluate the pharmacokinetics (PK) of multiple cytochrome P450 (CYP450) substrates alone and in combination with rocatinlimab in participants with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT05891119
- Testing
- Caffeine, Metoprolol, Midazolam
- Where
- 7 sites in United States
- Volunteers
- 21 people
- Sponsor
- Amgen
Muscular Dystrophies
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.
- Trial name
- DMD114044 (NCT01254019)
- Testing
- GSK2402968 6mg/kg/week
- Where
- 47 sites in Argentina, Belgium and 18 more
- Volunteers
- 186 people
- Sponsor
- GlaxoSmithKline
Pseudomonas Aeruginosa, Cystic Fibrosis
Open Label Extension to Bridging Study CTBM100C2303
This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who had completed their study participation in CTBM100C2303 (all visits) and who were proven infected with Pseudomonas…
- Trial name
- NCT00982930
- Testing
- Tobramycin inhalation powder
- Where
- 2 sites in Estonia, Russia
- Volunteers
- 55 people
- Sponsor
- Novartis Pharmaceuticals
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
- Trial name
- EXIST-2 (NCT00790400)
- Testing
- Everolimus (RAD001)
- Where
- 25 sites in United States, Canada and 9 more
- Volunteers
- 118 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
Safety and Explore the Efficacy of Multiple Doses of FURESTEM-AD Inj. for Moderate to Severe Chronic Atopic Dermatitis
A Phase I/IIa Clinical Trial to Evaluate the Safety and Explore the Efficacy of Multiple Doses of FURESTEM-AD inj. for Moderate to Severe Chronic Atopic Dermatitis
- Trial name
- NCT04725136
- Testing
- FURESTEM-AD inj
- Where
- 2 sites in South Korea
- Volunteers
- 96 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Cystic Fibrosis
Tobramycin Administered by eFlow Rapid Nebulizer: Pharmacokinetic Study
This study assesses the aerosol delivery characteristics (measured by nebulization time, serum and sputum tobramycin pharmacokinetic parameters) and safety of tobramycin administered for inhalation by PARI eFlow rapid electronic…
- Trial name
- NCT00420836
- Testing
- Tobramycin
- Volunteers
- 20 people
- Sponsor
- Novartis
Lax Skin
Evaluation of Facial and Neck Skin Laxity With SofWave Treatment in Adults Initiating Tirzepatide for Weight Loss
Open-label, randomized double-arm prospective, single-center, self-controlled clinical study.
- Trial name
- NCT07685834
- Testing
- SUPERB
- Where
- Canada
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Sofwave Medical LTD
Hereditary Tyrosinemia, Type I
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to compare the bioavailability of the Test Product, Nitisinone 10 mg Tablet, under fasting and fed conditions (food-effect).
- Trial name
- NCT02750332
- Testing
- Nitisinone
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Transfusion Dependent Thalassemia
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent…
- Trial name
- PRAISE (NCT03329404)
- Testing
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Where
- 9 sites in United States, Israel and 2 more
- Volunteers
- 9 people
- Sponsor
- Terumo BCTbio
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and…
- Trial name
- ESSENCE (NCT02500381)
- Testing
- SRP-4045, SRP-4053
- Where
- 76 sites in United States, Argentina and 22 more
- Volunteers
- 228 people
- Sponsor
- Sarepta Therapeutics, Inc.
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.