Clinical trials: Congenital Diseases
Atopic Dermatitis
Evaluation of NM26-2198 in Healthy Subjects and in Patients With Moderate-to-severe Atopic Dermatitis (AD)
This is a randomized, double-blind, placebo-controlled, single- and multiple ascending dose study of subcutaneous (SC) administration of NM26-2198 in healthy volunteers and adult patients with moderate to-severe AD to evaluate…
- Trial name
- NCT05859724
- Testing
- NM26-2198
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Yellow Jersey Therapeutics AG
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Dyslipidemias, Familial Hypercholesterolemia
Study of ARO-ANG3 in Healthy Volunteers and in Dyslipidemic Patients
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetcs and pharmacodynamics of single- and multiple doses of ARO-ANG3 in healthy adult volunteers and in dyslipidemic patients including familial…
- Conditions
- Dyslipidemias, Familial Hypercholesterolemia, Hypertriglyceridemia
- Trial name
- NCT03747224
- Testing
- ARO-ANG3, sterile normal saline (0.9% NaCl)
- Where
- 6 sites in Australia, New Zealand
- Volunteers
- 93 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Patent Foramen Ovale
Precutaneous High Risk Patent Foramen Ovale to Treat Migraine Headaches
The foramen ovale, a kind of physiologic channel in the interatrial septum in the heart at embryonic stage, is closed normally at 5-7 months after birth.
- Trial name
- NCT02777359
- Testing
- the transcatheter closure of PFO
- Where
- China
- Volunteers
- 100 people
- Sponsor
- First Affiliated Hospital Xi'an Jiaotong University
TSC, Tuberous Sclerosis Complex
Long-term, Prospective Study Evaluating Clinical and Molecular Biomarkers of Epileptogenesis in a Genetic Model of Epilepsy - Tuberous Sclerosis Complex
The primary objective of clinical part of EPISTOP project is to identify the clinical and molecular biomarkers of epileptogenesis in a prospective clinical study of patients with TSC.
- Conditions
- TSC, Tuberous Sclerosis Complex, Epilepsy
- Trial name
- EPISTOP (NCT02098759)
- Testing
- epilepsy early diagnosis protocol
- Where
- 9 sites in Austria, Belgium and 6 more
- Volunteers
- 100 people
- Sponsor
- Sergiusz Jozwiak
Wiskott-Aldrich Syndrome
Gene Therapy for WAS
This is a phase I/II study to evaluate the safety and efficacy of Hematopoietic Stem Cell genetherapy for the Wiskott-Aldrich Syndrome.
- Trial name
- NCT01347346
- Testing
- Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Genethon
Acoustic Schwannoma, Adult Anaplastic Meningioma
Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas
RATIONALE: Monoclonal antibodies, such as bevacizumab, can block tumor growth in different ways. Some block the ability of tumor cells to grow and spread.
- Conditions
- Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma
- Trial name
- NCT01125046
- Testing
- bevacizumab
- Where
- 5 sites in United States
- Volunteers
- 50 people
- Sponsor
- Northwestern University
Polycystic Liver Disease, Hepatomegaly
Open-Label Extension of LOCKCYST Trial
Positive data originating from two polycystic liver patients treated with somatostatin analogues, showed a volume reduction of 38.3% and 14.9%.
- Conditions
- Polycystic Liver Disease, Hepatomegaly, Liver Diseases, Polycystic Kidney
- Trial name
- LOCKCYST-ext (NCT00771888)
- Testing
- lanreotide
- Where
- 2 sites in Belgium, Netherlands
- Volunteers
- 54 people
- Sponsor
- Radboud University Medical Center
Jaundice Neonatal
The Risk of Hyperbilirubinemia in Term Newborns After Placental Transfusion
Delayed cord clamping (DCC) has been a subject of extensive research for the last couple of years. Based on published data, numerous neonatal benefits have been suggested such as increased hemoglobin and ferritin levels both at…
- Trial name
- NCT02625688
- Testing
- Delayed cord clamping, Cord milking, Early cord clamping
- Where
- Poland
- Volunteers
- 400 people
- Sponsor
- Princess Anna Mazowiecka Hospital, Warsaw, Poland
Sickle Cell Disease
Medication Adherence in Youth With Sickle Cell Disease (SCD)
Youth diagnosed with sickle cell disease (SCD) may have difficulty taking medication as prescribed (adherence). Hydroxyurea (HU) is one medication that youth may take to help manage SCD.
- Trial name
- NCT02143076
- Testing
- Demographic Questionnaire, Medical Adherence Measure Questionnaire, Acceptability Questionnaire
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Hypophosphatasia
Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT02496689
- Testing
- asfotase alfa
- Where
- 5 sites in United States, France
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pregnancy; Spina Bifida, Fetal, Environmental Exposure
Understanding Effects of Folic Acid on the Methylosome and Transcriptome of Women With Spina Bifida Affected Pregnancies
Folic acid is currently considered to be the standard of care for primary and secondary prevention of spina bifida, but the mechanisms underlying folic acid's benefits are unknown.
- Trial name
- NCT05500690
- Testing
- Folic Acid Tablet
- Where
- Bangladesh
- Volunteers
- 69 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Obstructive Sleep Apnea, Fetal Growth Restriction
Sleep Apnea and Fetal Growth Restriction
This study aims to evaluate the association between obstructive sleep apnea (OSA) and fetal growth restriction (FGR) and to assess the role of auto-titrated positive airway pressure (aPAP) as antenatal therapy in these patients.
- Conditions
- Obstructive Sleep Apnea, Fetal Growth Restriction, Pregnancy Related
- Trial name
- SAFER (NCT04084990)
- Testing
- S9 VPAP Adapt
- Where
- 3 sites in United States, Israel
- Volunteers
- 3 people
- Sponsor
- Washington University School of Medicine
Congenital Disorders
Comparison of NIRS and Serum Lactate to Predict Poor Post-operative Outcomes
Mortality after neonatal cardiac repair remains significant, and neonates who have undergone operative repair of congenital heart disease may experience sudden and unexpected demise.
- Trial name
- NCT00485524
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's Healthcare of Atlanta
Prader-Willi Syndrome
Study of Tesomet With Open-label Extension in Subjects With Prader-Willi Syndrome
This study will evaluate the safety and efficacy of Tesomet (tesofensine + metoprolol) in subjects with PWS.
- Trial name
- PWS (NCT05198362)
- Testing
- Tesomet
- Where
- United States
- Sponsor
- Saniona
Urea Cycle Disorder
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years.
- Trial name
- THRIVE (NCT01948427)
- Where
- United States
- Volunteers
- 203 people
- Sponsor
- Amgen
Limb Girdle Muscular Dystrophies
Motor Parameters in Patients With Limb Girdle Muscular Dystrophy
The primary objective of the study is to perform 2-year follow up with motor parameters evolution using instrumental assessments in patients with limb girdle muscular dystrophie, and to identify which motor parameters are…
- Trial name
- EIDY (NCT04772027)
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Congenital Heart Disease
Pulmonary Capillary Recruitment in Fontan Patients
This study evaluates the effects of pulmonary vasodilator therapy on pulmonary capillary blood flow by measuring the functional capillary surface area (FCSA) at baseline and post nitric oxide inhalation, through the injection of…
- Trial name
- NCT04292483
- Testing
- Nitric Oxide
- Where
- Canada
- Volunteers
- 15 people
- Sponsor
- Jewish General Hospital
Atopic Dermatitis
Effects of Lactobacillus Reuteri Plus Vitamin D3 in Children With Atopic Dermatitis
The purpose of this study is to evaluate in a randomized, double-blinded, controlled trial, whether a new food supplement containing Lactobacillus reuteri DSM 17938 and vitamin D3 (Reuterin® D3) may improve the SCORAD in…
- Trial name
- NCT02945683
- Testing
- Reuterin D3
- Where
- Italy
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Noos S.r.l.
Atopic Dermatitis
Efficacy of Fecal Microbial Transplantation Treatment in Adults With Atopic Dermatitis
Prospective double-blinded placebo-controlled study, among adult patients suffering from moderate-to-severe atopic dermatitis (AD), insufficiently responsive to topical and systemic treatment.
- Trial name
- NCT04283968
- Testing
- Fecal Microbial Transplantation
- Where
- 2 sites in Israel
- Volunteers
- 50 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
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