Clinical trials: Newborn
Sickle Cell Disease
Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
- Trial name
- PROSPECT (NCT04930445)
- Testing
- Oxbryta® (voxelotor) 500mg Tablets
- Where
- 43 sites in United States
- Volunteers
- 265 people
- Sponsor
- Pfizer
Anemia, Aplastic, Red-Cell Aplasia, Pure
Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia
This study will test whether the immune-suppressing drug rituximab can increase blood counts and reduce the need for transfusions in patients with moderate aplastic anemia, pure red cell aplasia, or Diamond Blackfan anemia.
- Conditions
- Anemia, Aplastic, Red-Cell Aplasia, Pure, Anemia, Diamond-Blackfan
- Trial name
- NCT00229619
- Testing
- Rituximab
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Heart Defects, Congenital, Cardiopulmonary Bypass
Nesiritide Use Following Cardiac Surgery in Infants
The purpose of this study is to determine the effects of nesiritide on urine output and hemodynamics following cardiopulmonary bypass in infants. Safety and pharmacokinetic data will also be obtained.
- Trial name
- NCT00281671
- Testing
- nesiritide
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid…
- Conditions
- Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
- Trial name
- NCT01488097
- Testing
- sebelipase alfa
- Where
- 10 sites in United States, Canada and 3 more
- Volunteers
- 8 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pectus Excavatum
Magnetic Mini-Mover Procedure to Treat Pectus Excavatum
This is a medical research study. The study investigators have developed a method to gradually repair pectus excavatum (sunken chest) deformity by placing a magnet on the sternum (breastbone) and then applying an external…
- Trial name
- 3MP (NCT00466206)
- Testing
- Magnetic Mini-Mover Procedure (3MP), Magnatract (external magnet in a removable external brace), 3MP (Magnetic Mini-Mover Procedure)
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Evaluation of the Efficacy and Safety of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia and Statin Intolerant
The purpose of this study is to determine if bempedoic acid (ETC-1002) is effective and safe versus placebo in patients with elevated LDL cholesterol and who are statin-intolerant.
- Conditions
- Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, Statin Adverse Reaction
- Trial name
- CLEAR Serenity (NCT02988115)
- Testing
- bempedoic acid
- Where
- 67 sites in United States, Canada
- Volunteers
- 345 people
- Sponsor
- Esperion Therapeutics, Inc.
Sickle Cell Disease
A Study of IMR-687 in Subjects With Sickle Cell Disease
A Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
- Trial name
- NCT04474314
- Testing
- IMR-687
- Where
- 49 sites in United States, Ghana and 11 more
- Volunteers
- 115 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Atopic Dermatitis
Study of OPA-15406 Ointment in Patients With Atopic Dermatitis
The purpose of this study is to assess the efficacy and safety of OPA-15406 ointment in patients with atopic dermatitis.
- Trial name
- NCT02914548
- Testing
- OPA-15406
- Where
- 3 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
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
Sickle Cell Disease, Leg Ulcers
Resolution of Sickle Cell Leg Ulcers With Voxelotor
This study is a Phase 3, multicenter, randomized, placebo-controlled study to evaluate the efficacy of voxelotor and standard of care for the treatment of leg ulcers in participants with sickle cell disease.
- Trial name
- RESOLVE (NCT05561140)
- Testing
- Voxelotor Oral Tablet
- Where
- 23 sites in Brazil, Kenya and 1 more
- Volunteers
- 88 people
- Sponsor
- Pfizer
Respiratory Distress Syndrome, Newborn, Premature Birth
SURFAXIN® Treatment for Prevention of Bronchopulmonary Dysplasia (BPD) in Very Low Birth Weight (VLBW) Infants.
SURFAXIN® (lucinactant) treatment will be examined in very low birth weight infants to prevent development of chronic lung disease, commonly known as bronchopulmonary dysplasia (BPD), in premature infants who have required…
- Conditions
- Respiratory Distress Syndrome, Newborn, Premature Birth, Bronchopulmonary Dysplasia
- Trial name
- NCT00215540
- Testing
- Lucinactant 175 mg/kg, Lucinactant 90 mg/kg
- Where
- United States
- Volunteers
- 136 people
- Sponsor
- Windtree Therapeutics
Dilated Cardiomyopathy
Transcoronary Infusion of Cardiac Progenitor Cells in Pediatric Dilated Cardiomyopathy
A phase 1 prospective study to determine the procedural feasibility and safety and preliminary efficacy of intracoronary infusion of cardiosphere-derived cells (CDCs) in patients with dilated cardiomyopathy.
- Trial name
- TICAP-DCM (NCT03129568)
- Testing
- CDC infusion
- Where
- Japan
- Volunteers
- 5 people
- Sponsor
- Okayama University
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Norditropin NordiFlex® Device Compared to the Device Previously Used by Patients or Parents
This study is conducted in Europe. The aim of this study is to compare the easiness of use of Norditropin NordiFlex® device to the device previously used by patients or parents.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Foetal Growth Problem, Small for Gestational Age
- Trial name
- NCT01245374
- Testing
- Norditropin NordiFlex®
- Where
- France
- Volunteers
- 103 people
- Sponsor
- Novo Nordisk A/S
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
Cystic Fibrosis
Effects of Sildenafil on CFTR-dependent Ion Transport Activity
Dehydrated airway surfaces resulting from sodium hyperabsorption and lack of chloride secretion are critical to the pathology that leads to the morbidity and mortality from Cystic Fibrosis (CF) lung disease.
- Trial name
- NCT01132482
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Jewish Health
Cystic Fibrosis
Safety and Efficacy of Pioglitazone as an Anti-inflammatory for the Treatment of Cystic Fibrosis (CF) Lung Disease
Study Hypothesis: Pioglitazone may decrease inflammation in cystic fibrosis lung disease. Primary outcomes: Markers of inflammation (neutrophils, elastase, cytokines and bacteria)will be measured in induced sputum specimens…
- Trial name
- NCT00322868
- Testing
- pioglitazone
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- University Hospitals Cleveland Medical Center
Patent Ductus Arteriosus
Pocket Echocardiography System (PES) for Detection of PDA in Neonates
This is a single-center, feasibility study involving all neonates admitted to the neonatal intensive care unit with orders for a cardiac echo evaluation. Parents will be approached and provided a description of the study.
- Trial name
- NCT01790750
- Testing
- PES first, then FFES
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Florida
Familial Partial Lipodystrophy
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of fasting triglycerides in participants with familial partial lipodystrophy.
- Trial name
- NCT03514420
- Testing
- AKCEA-ANGPTL3-LRx
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Akcea Therapeutics
Atopic Dermatitis, Eczema
Steroid-reducing Effects of Crisaborole
Atopic dermatitis, or eczema, is a chronic skin condition affecting many children. Crisaborole is a non-steroid topical medication which is FDA approved for children aged 2 and older for eczema.
- Trial name
- NCT03832010
- Testing
- Crisaborole, Hydrocortisone Ointment, Triamcinolone ointment
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
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