Clinical trials: Newborn Diseases
Vitamin D Deficiency, Sickle Cell Disease
Vitamin D Status in Children With Sickle Cell Disease Living in Lyon, France
Vitamin D deficiency may be under-diagnosed in sickle cell disease French children. Therefore, the investigator need an epidemiologic study about the prevalence of vitamin D deficiency in this specific population.
- Trial name
- EVADDREP (NCT04694976)
- Testing
- 25-OH Vitamin D measure
- Where
- France
- Volunteers
- 130 people
- Sponsor
- Hospices Civils de Lyon
Hereditary Angioedema, HAE
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Trial name
- APeX-S (NCT03472040)
- Testing
- BCX7353
- Where
- 87 sites in United States, Australia and 18 more
- Volunteers
- 387 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Hemorrhagic Telangiectasia
A Study to Evaluate ALN-6400 in Healthy Volunteers and Patients With Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in healthy volunteers evaluate the efficacy, safety, tolerability and PD of…
- Trial name
- InsigHHT (NCT06659640)
- Testing
- ALN-6400
- Where
- 16 sites in United States, Australia and 4 more
- Volunteers
- 89 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B
The Study's Primary Objective is to evaluate the pharmacokinetics, safety (acute effects associated with infusions, and inhibitor development) and efficacy (breakthrough bleeding and control of hemorrhaging during prophylaxis) of…
- Trial name
- NCT01271868
- Testing
- IB1001
- Where
- 10 sites in United States, India and 1 more
- Volunteers
- 9 people
- Sponsor
- Medexus Pharma, Inc.
Fetal Macrosomia
Estimation of Fetal Weight by MR Imaging to PREdict Neonatal MACROsomia (PREMACRO Study)
Macrosomia and growth restriction are important causes of perinatal morbidity, at or near to term. However, clear identification of 'at risk' foetuses is difficult and clinical estimates of fetal weight are poor.
- Trial name
- NCT02713568
- Testing
- Ultrasound examination, Magnetic resonance examination
- Where
- Belgium
- Volunteers
- 2,413 people, healthy volunteers welcome
- Sponsor
- Brugmann University Hospital
Molar Hypomineralization
Intraosseus Injection Using Extra-short Needle Combined With Infiltration in Pediatric Patients With Molar Incisor Hypomineralization
Background: Effective pain control is crucial in managing pediatric dental patients. Molar-incisor hypomineralization (MIH), marked by enamel defects and dentin hypersensitivity, often hinders effective local anesthesia.
- Trial name
- NCT07239050
- Testing
- intraosseus anesthesia, Conventional nerve block
- Where
- Egypt
- Volunteers
- 26 people
- Sponsor
- Alexandria University
Crohn's Disease
A Study to Test the Effect of CDP870 in the Treatment of Crohn's Disease Over 26 Weeks, Comparing CDP870 to a Dummy Drug (Placebo)
A 26 week study to examine the efficacy, safety and pharmacokinetics of CDP870 in Crohn's disease
- Trial name
- NCT00152490
- Testing
- Certolizumab Pegol (CDP870)
- Volunteers
- 604 people
- Sponsor
- UCB Pharma
Healthy
Study of PF 04965842 Effect on MATE1/2K Activity in Healthy Participants
This is a Phase 1, randomized, 2 way crossover, open label study of the effect of PF-04965842 on metformin (a probe for MATE1/2K activity) PK in healthy adult participants.
- Trial name
- NCT03796182
- Testing
- Metformin, PF-04965842
- Where
- Belgium
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Blood Disease, Cerebrovascular Accident
Stroke Prevention in Sickle Cell Anemia (STOP 2)
To determine how long blood transfusions are needed for primary stroke prevention. Also, to determine the duration of risk associated with abnormal transcranial Doppler ultrasound (TCD) and to determine the specificity of the…
- Conditions
- Blood Disease, Cerebrovascular Accident, Anemia, Sickle Cell
- Trial name
- NCT00006182
- Testing
- Blood Transfusion
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
CMT, CMT (Charcot Marie Tooth Disease)
TREMOR IN CHARCOT-MARIE-TOOTH
Tremor is a symptom that has already been described in many case reports and case series concerning patients with Charcot-Marie-Tooth (CMT) disease.
- Conditions
- CMT, CMT (Charcot Marie Tooth Disease), Charcot Marie Tooth Disease (CMT), Charcot Marie Tooth Disease
- Trial name
- CMT-TREM (NCT07570459)
- Where
- Germany
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University Medical Center Goettingen
Leber's Hereditary Optic Neuropathy (LHON)
Post Authorisation Safety Study With Raxone in LHON Patients
This study is a multicentre, prospective, non-interventional post-authorisation safety study (PASS) of the clinical outcomes for patients with LHON treated with Raxone®. No medication will be provided as part of this study.
- Trial name
- PAROS (NCT02771379)
- Testing
- Idebenone
- Where
- 26 sites in Austria, France and 4 more
- Volunteers
- 229 people
- Sponsor
- Santhera Pharmaceuticals
Tourette Syndrome
Characterising Sleep Disorders in Children With Tourette Syndrome
Healthy sleep is essential for a young person's growth, development, and wellbeing. It is estimated that up to 80% of young people with Tourette Syndrome experience sleep difficulties.
- Trial name
- NCT07450079
- Volunteers
- 34 people
- Sponsor
- Steffi Baker
Cystic Fibrosis
Study About Complications of Totally Implanted Venous Access Devices (TIVADs) in People With Cystic Fibrosis (CF)
Pulmonary infections are the major cause of mortality and morbidity in cystic fibrosis (CF); patients frequently have to take antibiotics which often cannot be given orally or by aerosol but have to be administered intravenously.
- Trial name
- NCT00670579
- Where
- Italy
- Volunteers
- 80 people
- Sponsor
- University of Florence
Huntington Disease
Study of WVE-003 in Patients With Huntington's Disease
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of WVE-003 in adult patients with early-manifest HD who…
- Trial name
- NCT05032196
- Testing
- SAD: 30mg WVE-003, SAD: 60mg WVE-003, SAD: 90mg WVE-003
- Where
- 23 sites in Australia, Canada and 8 more
- Volunteers
- 47 people
- Sponsor
- Wave Life Sciences USA, Inc.
Heart Disease, Health
Molecular Forms of Plasma and Urinary B-type Natriuretic Peptide and Its N-terminal Prohormone in Health and Disease in Pediatric Cardiology
B-type natriuretic Peptide (BNP) is a cardiac hormone secreted from the cardiac myocytes in response to volume load. Plasma levels of BNP, as measured by immunoassay methods, are elevated in patients with heart diseases.
- Trial name
- NCT01603888
- Testing
- no intervention
- Where
- Israel
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization
Asphyxia Neonatorum
Resident Training Enhanced by New Innovations: Teleintubation
The overall goal of this study was to create a simulation environment with repeated practice for residents and intense, immediate feedback.
- Trial name
- NCT02572427
- Testing
- Education for intubation skills, No added education for intubation skills
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- University of Arkansas
Spinal Cord Injuries, Cerebral Palsy
Mobile Health Self-Management and Support System for Chronic and Complex Health Conditions
This study will assess the benefits of using mobile health system designed for individuals with chronic and complex health conditions (such as those with Spinal Cord Injury,Cerebral Palsy, Spina Bifida, and Traumatic Brain…
- Conditions
- Spinal Cord Injuries, Cerebral Palsy, Spinal Dysraphism, Brain Injuries
- Trial name
- NCT02592291
- Testing
- MHealth
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- University of Pittsburgh
Hypospadias
Urethrocutaneous Fistula Rate After Double Dartos and Single Dartos Tubularized Incised Plate Urethroplasty
The subjects were divided into two groups. Each group comprised 30 children. Children were randomly allocated in two groups via computer generated tables.
- Trial name
- NCT04699318
- Testing
- Group B, Group A
- Where
- Pakistan
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- King Edward Medical University
Infant, Premature, Diseases
Adjustable Fortification of Human Milk Fed to Chinese Preterm Infants
This clinical trial will compare how the individualized and fortified human milk feeding will help a premature infant grow.
- Trial name
- NCT01731613
- Testing
- human milk fortifier
- Where
- China
- Volunteers
- 51 people
- Sponsor
- Peking Union Medical College Hospital
Von Willebrand Disease
Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.
- Trial name
- NCT00816660
- Testing
- Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII), Marketed plasma-derived VWF/FVIII concentrate
- Where
- 25 sites in United States, Austria and 4 more
- Volunteers
- 32 people
- Sponsor
- Baxalta now part of Shire
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