Clinical trials: Congenital Anomalies
Sickle Cell Disease
A Stratified Sickle Event Randomized Trial (ASSERT)
The purpose of this study is to compare the effects of ICA-17043 to placebo with or without hydroxyurea (an oral drug used for treatment of sickle cell disease) in patients with sickle cell disease who have had 2 or more acute…
- Trial name
- ASSERT (NCT00102791)
- Testing
- ICA-17043
- Where
- 49 sites in United States, Brazil and 4 more
- Volunteers
- 297 people
- Sponsor
- Icagen
Prader-Willi Syndrome, Hyperphagia
Oxytocin vs. Placebo for the Treatment Hyperphagia in Children and Adolescents With Prader-Willi Syndrome
The investigators propose a randomized double-blind 8 week treatment trial of intranasal oxytocin (IN-OXT) vs. placebo in 24 subjects aged 5 to 18 years with PWS in order to assess IN-OXT's affect on (1) Eating behaviors (2)…
- Trial name
- OXT-PWS (NCT02629991)
- Testing
- Intranasal Oxytocin (IN-OXT)
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Montefiore Medical Center
Dermatitis, Atopic, Eczema
A Study of Lebrikizumab in Combination With Topical Corticosteroids in Patients With Atopic Dermatitis (AD) That Are Not Adequately Controlled With or Are Non-eligible for Cyclosporine
The main purpose of this study is to evaluate the efficacy of lebrikizumab compared with placebo in participants not adequately controlled with cyclosporine or for whom cyclosporine is not medically advisable up to Week 16.
- Trial name
- ADvantage (NCT05149313)
- Testing
- Lebrikizumab
- Where
- 48 sites in Austria, Belgium and 6 more
- Volunteers
- 331 people
- Sponsor
- Almirall, S.A.
Fabry Disease
Evaluation of Phenotypic Variability in Fabry Disease
Cerebrovascular events, such as stroke, are a devastating complication of Fabry disease that results in part from storage of complex lipids in both large and small vessels.
- Trial name
- NCT03145779
- Where
- United States
- Sponsor
- Boston Children's Hospital
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
Abnormalities, Blood Coagulation Disorder
Study of Abnormal Blood Clotting in Children With Stroke
Effective treatment and prevention strategies for childhood stroke and porencephaly can only be developed once the causes are understood.
- Conditions
- Abnormalities, Blood Coagulation Disorder, Brain Disease, Cerebrovascular Accident
- Trial name
- NCT00001927
- Where
- United States
- Volunteers
- 130 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Bronchopulmonary Dysplasia
Mesenchymal Stem Cell Therapy for Bronchopulmonary Dysplasia in Preterm Babies
Bronchopulmonary Dysplasia (BPD) is the most frequent disease related to a premature birth, 15-50% of very low birth newborns (<1500 gr.) will develop BPD.
- Trial name
- NCT02443961
- Testing
- Mesenchymal Stem Cell (MSC) therapy
- Where
- 4 sites in Spain
- Volunteers
- 10 people
- Sponsor
- Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal
Heart Septal Defects, Ventricular, Atrioventricular Septal Defect
Aberrations in Carnitine Homeostasis in Congenital Heart Disease With Increased Pulmonary Blood Flow
Infants with congenital heart disease and increased pulmonary blood flow have altered carnitine homeostasis that is associated with clinical outcomes; and L-carnitine treatment will attenuate these alterations and improve…
- Trial name
- L-carn (NCT01825369)
- Testing
- IV L-carnitine
- Where
- United States
- Sponsor
- University of California, San Francisco
Normal Middle Ears., Otitis Media Ears.
Observation of Gas Exchange in the Middle Ear
Observation of fluid behavior over a perforated eardrum. A comparison of the behavior of the gas in normal vs. different kinds of otitis media ears.
- Trial name
- NCT00794105
- Where
- Israel
- Sponsor
- Shaare Zedek Medical Center
Colorectal Cancer, Precancerous Condition
Celecoxib in Preventing Colorectal Cancer in Young Patients With a Genetic Predisposition for Familial Adenomatous Polyposis
RATIONALE: Chemoprevention is the use of certain drugs to keep cancer from forming. The use of celecoxib may keep polyps and colorectal cancer from forming in patients with familial adenomatous polyposis.
- Trial name
- NCT00685568
- Testing
- celecoxib
- Where
- 4 sites in United States
- Volunteers
- 22 people
- Sponsor
- M.D. Anderson Cancer Center
Heart Defects, Congenital, Adolescent
Rehabilitation of Adolescents and Young Adults With Congenital Heart Diseases
The investigators aim to measure the impact of a rehabilitation program in congenital cardiology in terms of health related quality of life.
- Conditions
- Heart Defects, Congenital, Adolescent, Young Adult
- Trial name
- QUALIREHAB (NCT03690518)
- Testing
- Cardiac rehabilitation
- Where
- 17 sites in France
- Volunteers
- 142 people
- Sponsor
- University Hospital, Montpellier
Angelman Syndrome
Use of Eye Tracking to Study Social Perception Abnormalities in Children With Angelman Syndrome
Angelman syndrome (AS) is a rare neurogenetic disorder that affects approximately 1 in 15,000 children - approximately 500,000 people worldwide.
- Trial name
- EYEANGEL (NCT06737718)
- Testing
- Eye tracking, Data collection from patients' medical files, questionnaires
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hemangiomas, Vascular Malformations
Diagnosis of Hemangiomas and Vascular Malformations
To develop a non-invasive, simple and affordable, measurement technique to differentiate hemangiomas from subtypes of VMS. The purpose of this study is to determine the best office based diagnositc instrument for differentiating…
- Conditions
- Hemangiomas, Vascular Malformations, Venular Malformations, Venous Malformations
- Trial name
- NCT00577213
- Volunteers
- 56 people
- Sponsor
- University of Arkansas
Bicuspid Aortic Valve Disease
Bicuspid Echocardiac Study Team（BEST）
The Chinese Bicuspid Aortic Valve (BAV) Ultrasound imaging cohort study is a Chinese BAV ultrasound imaging cohort study. At present, a retrospective study cohort involving more than 30 hospitals has been established.
- Trial name
- BEST (NCT05656820)
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Suzhou Municipal Hospital
Atopic Dermatitis (AD)
A Phase 3 Study of MH004 Ointment in Patients With Mild to Moderate Atopic Dermatitis
This is a multicenter, randomized, double-blind, vehicle-controlled phase III study of MH004 Ointment with a 44-week open-label long-term safety extension period.
- Trial name
- NCT07185282
- Testing
- MH004 Ointment, Vehicle Ointment
- Where
- China
- Volunteers
- 375 people
- Sponsor
- Minghui Pharmaceutical (Hangzhou) Ltd
Dermatitis, Atopic, Child
Efficacy of Fermented Rice Flour for the Treatment of Atopic Dermatitis: Randomized, Double-blind Controlled Trial
This trial aims at evaluating the efficacy of a fermented rice flour for the treatment of atopic dermatitis (AD). The fermented rice flour, obtained from Lactobacillus paracasei CBA L74 (Heinz Italia SpA, Latina, Italy), does not…
- Conditions
- Dermatitis, Atopic, Child, Infant
- Trial name
- NCT03042624
- Testing
- Fermented rice, Maltodextrins
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- University of Milan
Fragile X Syndrome, Autism Spectrum Disorder
Alpha Auditory Entrainment for Cognitive Enhancement and Sensory Hypersensitivity in Youth With Developmental Disorders
Fragile X Syndrome (FXS) is a complex neurodevelopmental disorder caused by a mutation on the X chromosome. Scientists have investigated FXS extensively in both humans and animals.
- Conditions
- Fragile X Syndrome, Autism Spectrum Disorder, Autistic Disorder, Asperger Syndrome
- Trial name
- ENTRAIN (NCT06227780)
- Testing
- Alpha Auditory Entrainment
- Where
- United States
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hemophilia A, Bleeding Disorder
Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women and Post-menarchal Girls With Bleeding Disorders
Characterizing the impact and treatment of reproductive tract bleeding on women and post-menarchal girls with bleeding disorders.
- Trial name
- NCT03834727
- Where
- United States
- Volunteers
- 256 people
- Sponsor
- Oregon Health and Science University
Hemangiomas, Venous Malformations
Airway Vascular Lesions
Controversy exists in the treatment of airway hemangiomas ranging from tracheotomy, various lasers, to open removal, none of which have proven to be a gold standard.
- Conditions
- Hemangiomas, Venous Malformations, Lymphatic Malformations
- Trial name
- NCT00866827
- Testing
- Protocols for Treatment
- Volunteers
- 75 people
- Sponsor
- University of Arkansas
Severe Combined Immunodeficiency, X-Linked
Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency
Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity.
- Trial name
- NCT03601286
- Testing
- Lentiviral vector transduced CD34+ cells
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
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