Clinical trials: Newborn Diseases
Pregnancy Complications
Study of Drug Concentration of Ondansetron and Cefazolin in Mothers and Neonates
This study proposes to look at the pharmacokinetics of two drugs (Cefazolin and ondansetron) given routinely to pregnant women who are planning to deliver via cesarean section.
- Trial name
- NCT01357369
- Testing
- Phlebotomy
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Stanford University
Anemia, Sickle Cell
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes.
- Trial name
- Arginine (NCT00513617)
- Testing
- Arginine
- Where
- 17 sites in United States
- Volunteers
- 128 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Muscular Dystrophy, Dilated Cardiomyopathy
The Cardiovascular Genetic and Therapeutic Implications of Muscular Dystrophy
This study will have significant impact on muscular dystrophy patients as it promotes early screening for heart disease. With early identification, beneficial medical therapy can be started sooner, resulting in restoring and…
- Conditions
- Muscular Dystrophy, Dilated Cardiomyopathy, Heart Failure
- Trial name
- NCT00518817
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Baylor College of Medicine
Hailey Hailey Disease
M. Hailey-Hailey: hSPCA1 Expression and Skin Structure Upon Laser Therapy
Hailey-Hailey disease is a genetic acantholytic dermatosis characterized by continuous erosion of the skin that results in a burning, painful sensation and restricts the patient in daily life.
- Trial name
- NCT03849989
- Testing
- Skin biopsy
- Where
- Netherlands
- Volunteers
- 11 people
- Sponsor
- Maastricht University Medical Center
Fabry Disease
Safety, Efficacy, & PK of PRX-102 in Patients With Fabry Disease Administered Intravenously Every 4 Weeks
This open-label switchover study will assess the safety, efficacy, and pharmacokinetics of pegunigalsidase alfa (PRX-102) 2 mg/kg administered every 4 weeks for 52 weeks in Fabry patients previously treated with ERT: agalsidase…
- Trial name
- BRIGHT (NCT03180840)
- Testing
- Pegunigalsidase alfa
- Where
- 14 sites in United States, Belgium and 5 more
- Volunteers
- 30 people
- Sponsor
- Protalix
Healthy Participants, Atopic Dermatitis
FIH Study to Evaluate the Tolerability of PF-07832837 in Healthy Adults and Patients
The purpose of the study is to evaluate the safety, tolerability, and pharmacokinetics of escalating single and repeat doses of PF-07832837 in healthy participants and in participants with moderate to severe atopic dermatitis.
- Trial name
- NCT06564389
- Testing
- PF-07832837
- Where
- 5 sites in United States
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- Pfizer
Thyroid Eye Disease
A Trial Investigating Lu AG22515 in Adult Participants With Moderate-to-Severe Thyroid Eye Disease
This trial will evaluate the effects of Lu AG22515 in adult men and women with moderate-to-severe thyroid eye disease (TED). TED is an autoimmune condition closely related to Graves' Disease.
- Trial name
- NCT06557850
- Testing
- Lu AG22515
- Where
- 6 sites in Bosnia and Herzegovina, Bulgaria and 1 more
- Volunteers
- 20 people
- Sponsor
- H. Lundbeck A/S
Autoimmune Lymphoproliferative Syndrome, Lymphoma
Fluorodeoxyglucose Imaging Studies to Detect Lymphoma
Background: Autoimmune lymphoproliferative syndrome (ALPS) is a genetic disorder of the lymph system. People with ALPS often have swollen lymph nodes, especially in the neck and armpit.
- Trial name
- NCT01672918
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Congenital Hemophilia A
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
This is an international, multicenter, open-label study to assess the efficacy, safety, and pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe hemophilia A.
- Trial name
- NCT02093897
- Testing
- rVIII-SingleChain
- Where
- 37 sites in United States, Australia and 17 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Cystic Fibrosis, Nontuberculous Mycobacterium Infection
Healthcare-associated Links in Transmission of Nontuberculous Mycobacteria in Cystic Fibrosis
Sources of NTM infection and modes of transmission among CF patients are poorly understood. Healthcare-associated transmission of NTM among CF patients has been suspected and is of growing concern for CF Centers.
- Trial name
- HALTNTM (NCT04024423)
- Testing
- Epidemiologic investigation
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Jewish Health
PIK3CA-Related Overgrowth Spectrum
A Study of the Epidemiology and Hospital Management of Patients With PROS in France
The purpose of this study was to estimate the incidence and prevalence of a group of genetic disorders known as PIK3CA-Related Overgrowth Spectrum (PROS) in France.
- Trial name
- EpiPROS (NCT07222423)
- Where
- United States
- Volunteers
- 3,605 people
- Sponsor
- Novartis Pharmaceuticals
Autosomal Dominant Polycystic Kidney Disease
Somatostatin In Patients With Autosomal Dominant Polycystic Kidney Disease And Moderate To Severe Renal Insufficiency
The general aim of the trial is to assess the efficacy of one year treatment with long-acting somatostatin analogue (Octreotide LAR) compared with placebo in slowing kidney and liver growth rate in patients with ADPKD and…
- Trial name
- ALADIN2 (NCT01377246)
- Testing
- Octreotide-LAR, Saline solution.
- Where
- 6 sites in Italy
- Volunteers
- 100 people
- Sponsor
- Mario Negri Institute for Pharmacological Research
Lipoprotein Lipase Deficiency, Familial Hyperlipoproteinemia Type 1
Glybera Registry, Lipoprotein Lipase Deficient (LPLD) Patients
Lipoprotein lipase deficiency (LPLD) is a rare autosomal recessive disorder, characterized by loss-of function mutations in the LPL gene, leading to the inability to produce functionally active lipoprotein lipase (LPL).
- Conditions
- Lipoprotein Lipase Deficiency, Familial Hyperlipoproteinemia Type 1, Familial Hyperchylomicronemia
- Trial name
- GENIALL (NCT03293810)
- Testing
- Observational study
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- UniQure Biopharma B.V.
Epilepsy, Epilepsy, Generalized
Clobazam in Patients With Lennox-Gastaut Syndrome
The purpose of this study is to evaluate the safety and efficacy of clobazam as adjunctive therapy in the treatment of seizures which lead to drop attacks (drop seizures) in patients 2 to 60 years of age with Lennox-Gastaut…
- Conditions
- Epilepsy, Epilepsy, Generalized, Seizures
- Trial name
- NCT00518713
- Testing
- Clobazam Low Dose, Clobazam Medium Dose, Clobazam High Dose
- Where
- 53 sites in United States, Australia and 3 more
- Volunteers
- 238 people
- Sponsor
- Lundbeck LLC
Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome
Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies
The purpose of this study is to identify genes associated with impaired development and function of the cranial nerves and brainstem, which may result in misalignment of the eyes (strabismus) and related conditions.
- Conditions
- Congenital Fibrosis of Extraocular Muscles, Duane Retraction Syndrome, Duane Radial Ray Syndrome, Mobius Syndrome
- Trial name
- NCT03059420
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Cystic Fibrosis
Resting Energy Expenditure in Patients With Cystic Fibrosis: A Pilot Study
Cystic fibrosis is an inherited disorder that causes severe damage to the lungs, digestive system, and other organs in the body.
- Trial name
- NCT03699982
- Testing
- Daily calorie target based on resting energy expenditure
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- CAMC Health System
Fanconi Anemia
Corrective Exercise and Rehabilitation in Fanconi Anemia: A Case Study
Introduction: Fanconi anemia (FA) is a rare autosomal recessive DNA repair disorder characterized by congenital malformations, progressive bone marrow failure, and reduced quality of life.
- Trial name
- NCT06958380
- Testing
- Corrective exercise and rehabilitation
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Pardis Specialized Wellness Institute
Lynch Syndrome
Protocol for Non-invasive Gynaecological Surveillance in Lynch Syndrome
Lynch syndrome is the most common inherited condition that raises a person's risk of getting certain cancers. It happens because of changes in genes that normally fix damaged DNA.
- Trial name
- PREDI-ENDO (NCT07877779)
- Where
- United Kingdom
- Volunteers
- 1,450 people
- Sponsor
- University of Edinburgh
Epilepsy, Focal Cortical Dysplasia
First-in-human Experience Using Novel Ultraflexible Low-impedance Electrode Arrays: an IDEAL Stage 1 Study
The goal of this first-in-human study is to evaluate a novel ultraflexible microelectrode in children undergoing neurosurgery to remove tissue that causes epilepsy (seizures) in focal cortical dysplasia (FCD) or long-term…
- Trial name
- NCT06116279
- Testing
- Insertion of electrode during planned neurosurgery for epilepsy
- Where
- United Kingdom
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Incidence of Cholangiocarcinoma on Choledochal Cysts, Postoperative Complications in Choledochal Cysts
Analysis of the Incidence of Cholangiocarcinoma on Choledochal Cyst in a Western Center
Choledochal cysts are congenital cystic dilatations of the intra and/or extrahepatic bile duct. They have been considered a rare entity of the child population.
- Conditions
- Incidence of Cholangiocarcinoma on Choledochal Cysts, Postoperative Complications in Choledochal Cysts, Choledochal Cyst Monitoring
- Trial name
- QuiCo (NCT05404503)
- Where
- Spain
- Volunteers
- 300 people
- Sponsor
- María Teresa Moreno Asencio
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