Clinical trials: Newborn
Anemia, Sickle Cell
Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Sickle cell anemia (SCA) is an inherited blood disorder that can cause organ damage. The BABY HUG study is evaluating the use of the medication hydroxyurea at preventing organ damage in children with SCA.
- Trial name
- NCT00890396
- Testing
- Hydroxyurea
- Where
- 14 sites in United States
- Volunteers
- 163 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchenne Muscular Dystrophy
Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT02858362
- Testing
- Ezutromid
- Where
- 16 sites in United States, United Kingdom
- Volunteers
- 43 people
- Sponsor
- Summit Therapeutics
Friedreich Ataxia
Efficacy of EGb761 in Patients Suffering From Friedreich Ataxia
The purpose of this protocol is to determine the efficacy of EGb 761 120 mg bid versus placebo in patients suffering from Friedreich Ataxia
- Trial name
- NCT00824512
- Testing
- EGb 761 120 mg
- Where
- France
- Volunteers
- 22 people
- Sponsor
- Ipsen
Osteogenesis Imperfecta
Post-market Observational Study on JTIN Telescopic Nail in Osteogenesis Imperfecta Pediatric Patients
This study has been planned as part of the Orthofix Srl post-market active surveillance plan for the collection of data on both the clinical performance and the safety profile of the JTIN.
- Trial name
- JTIN (NCT05612139)
- Testing
- JTIN implantation surgery
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Orthofix s.r.l.
Hemophilia A
A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors
This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older.
- Trial name
- HAVEN 3 (NCT02847637)
- Testing
- Emicizumab, Factor VIII (FVIII)
- Where
- 39 sites in United States, Australia and 12 more
- Volunteers
- 152 people
- Sponsor
- Hoffmann-La Roche
Dilated Cardiomyopathy
Coenzyme Q10 Supplementation in Children With Idiopathic Dilated Cardiomyopathy
This study aims to determine the effect of Coenzyme Q10 supplementation on conventional therapy of children with heart failure due to idiopathic dilated cardiomyopathy.
- Trial name
- NCT02115581
- Testing
- Coenzyme Q10
- Where
- Iran
- Volunteers
- 38 people
- Sponsor
- University of Tehran
HIV
A Study of the Safety, Tolerability, and Pharmacokinetics of Dolutegravir in Neonates Exposed to HIV-1
The purpose of this study was to evaluate the safety, tolerability, and pharmacokinetics (PK) of dolutegravir (DTG) in infants born to mothers living with HIV-1.
- Trial name
- NCT05406583
- Testing
- Dolutegravir 0.5 mg/kg liquid suspension (starts at 0-5 days of life), Dolutegravir 5 mg Dispersible Tablets (single doses), Dolutegravir 5 mg Dispersible Tablets (chronic dose)
- Where
- 15 sites in United States, South Africa and 1 more
- Volunteers
- 96 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Teamwork During Neonatal Resuscitation
Improving Teamwork for Neonatal Resuscitation
The Neonatal Resuscitation Program (NRP) is the curriculum used to teach providers how to care for newborns in the delivery room. Breakdowns in teamwork and communication contribute to NRP quality problems.
- Trial name
- NCT00651794
- Testing
- Teamwork training, Standard NRP curriculum, Skills practice with low-fidelity mannequin
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Dermatitis, Atopic
Safety and Efficacy of AN2728 Topical Ointment, 2% in Children, Adolescents, and Adults (Ages 2 Years and Older) With Atopic Dermatitis
The purpose of this study is to investigate the safety and efficacy of AN2728 Topical Ointment, 2% in children, adolescents, and adults (ages 2 years and older) with atopic dermatitis.
- Trial name
- NCT02118766
- Testing
- AN2728 Topical Ointment, 2%, Matching vehicle control
- Where
- United States
- Volunteers
- 763 people
- Sponsor
- Pfizer
Adolescent Behavior, Intellectual Disability
Promotion of Physical Activity in Adolescents With Intellectual and Developmental Disabilities
The objective of this study is to compare the effect of two strategies to increase MVPA in adolescents with intellectual and developmental disabilities (IDD): a single level intervention delivered to the adolescent only, and a…
- Conditions
- Adolescent Behavior, Intellectual Disability, Down Syndrome, Physical Activity
- Trial name
- NCT03684512
- Testing
- Group Exercise Sessions, Individual Support Sessions, Facebook Group
- Where
- United States
- Volunteers
- 116 people
- Sponsor
- University of Kansas
Persistent Pulmonary Hypertension, Respiratory Failure
Study of Sildenafil to Treat Newborns With Persistent Pulmonary Hypertension
The purpose of this study is to determine whether intravenous sildenafil reduces pulmonary artery pressure and improves oxygenation in near-term and term infants with persistent pulmonary hypertension.
- Trial name
- NCT01409031
- Testing
- Intravenous Sildenafil
- Where
- 7 sites in United States
- Volunteers
- 3 people
- Sponsor
- University of Colorado, Denver
Gout
Safety and Efficacy Study of PEG-uricase in the Treatment of Hyperuricemic Patients With Symptomatic Gout
These are two replicate studies to evaluate the safety and efficacy of PEG (polyethylene glycol)-uricase in controlling the uric acid level in symptomatic gout patients with high uric acid levels who are unable to take standard…
- Trial name
- NCT00325195
- Testing
- pegloticase
- Where
- 56 sites in United States, Canada and 1 more
- Volunteers
- 225 people
- Sponsor
- Savient Pharmaceuticals
Obsessive-Compulsive Disorder, Trichotillomania (Hair-Pulling Disorder)
Epidiolex in Obsessive Compulsive Disorder and Related Disorders
The primary objective of the proposed study is to evaluate the safety and efficacy of Epidiolex (cannabidiol) in adults with obsessive compulsive and related disorders (OCRDs).
- Conditions
- Obsessive-Compulsive Disorder, Trichotillomania (Hair-Pulling Disorder), Tourette Syndrome, Hoarding Disorder
- Trial name
- NCT04978428
- Testing
- Cannabidiol
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Chicago
Thrombosis
Heparin and the Reduction of Thrombosis (HART) Trial
Heparin is frequently used in central venous catheters (CVCs) in post-operative cardiac patients. It remains unclear if a heparin infusion, compared to a normal saline infusion, prevents thrombosis of CVCs after surgery.
- Trial name
- NCT00779558
- Testing
- Heparin sulfate infusion at 10 units/kg/hour
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Stanford University
Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Evaluation of Long-Term Efficacy of Bempedoic Acid (ETC-1002) in Patients With Hyperlipidemia at High Cardiovascular Risk
The purpose of this study is to see if bemedoic acid (ETC-1002) is effective versus placebo in patients with high cardiovascular risk and elevated LDL cholesterol not adequately controlled by their current therapy.
- Trial name
- CLEAR Wisdom (NCT02991118)
- Testing
- bempedoic acid
- Where
- 2 sites in United States
- Volunteers
- 779 people
- Sponsor
- Esperion Therapeutics, Inc.
Impetigo, Folliculitis
Twice Daily Altabax Application for the Treatment of Uncomplicated Soft Tissue Infection
The purpose of this study is to document the clinical and bacteriological efficacy of retapamulin in the treatment of subjects with bacterial infections, including impetigo, folliculitis, and minor soft tissue infections…
- Conditions
- Impetigo, Folliculitis, Secondarily Infected Eczema, Minor Soft Tissue Infections
- Trial name
- NCT01126268
- Testing
- Retapamulin (Altabax)
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- The University of Texas Health Science Center, Houston
Turner's Syndrome
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years.
- Trial name
- NCT01066052
- Testing
- r-hGH
- Where
- France
- Volunteers
- 115 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Erythropoietic Protoporphyria, X-linked Protoporphyria
Effect of Oral Cimetidine in the Protoporphyrias
Erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) result from genetic defects of heme biosynthesis that cause life-long, painful cutaneous sensitivity to light.
- Trial name
- NCT05020184
- Testing
- Cimetidine
- Where
- 3 sites in United States
- Volunteers
- 26 people
- Sponsor
- Amy K. Dickey, M.D.
Colic
Preliminary Efficacy, Safety and Pharmacokinetics Study of Nepadutant in Infant With Feeding Intolerance
The present pilot study is aimed to obtain preliminary data on the effect of three ascending oral dose levels of nepadutant on the relief of symptoms associated with feeding intolerance.
- Trial name
- NCT01532518
- Testing
- Nepadutant
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- Menarini Group
Fabry Disease, Lysosomal Storage Diseases
A Fabry Disease Gene Therapy Study
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.
- Trial name
- MARVEL1 (NCT04040049)
- Testing
- FLT190
- Where
- 13 sites in United States, Austria and 5 more
- Volunteers
- 3 people
- Sponsor
- Spur Therapeutics
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