Clinical trials: Neonatal Outcome
Nervous System Malformations, Arthropathy, Neurogenic
Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease
This study will evaluate the safety and effectiveness of anakinra (Kineret) for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurological, cutaneous and…
- Conditions
- Nervous System Malformations, Arthropathy, Neurogenic, Urticaria, Papilledema
- Trial name
- NCT00069329
- Testing
- anakinra
- Where
- United States
- Volunteers
- 43 people
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Retinopathy of Prematurity (ROP)
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
- Trial name
- PEDAL (NCT02386839)
- Testing
- rhIGF-1/rhIGFBP-3
- Where
- 16 sites in United States, Italy and 4 more
- Volunteers
- 76 people
- Sponsor
- Shire
Pediatric Disorder, Neonatal Disease
Neonatal Intensive Care Unit Virtual Family-Centered Rounds
The aim of this study is to evaluate the impact of virtual family-centered rounds in the neonatal intensive care unit on parental and neonatal outcomes.
- Trial name
- NCT05762835
- Testing
- Virtual family-centered rounds (FCR)
- Where
- United States
- Volunteers
- 514 people
- Sponsor
- University of California, Davis
Propionic Acidemia, Methylmalonic Acidemia
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
Background: Very few drugs exist that treat hyperammonemia, specifically PA and MMA. Diet restrictions and alternate pathway agents are the current primary treatments, but they frequently fail to prohibit brain damage.
- Trial name
- NCT01597440
- Testing
- N-carbamylglutamate, Standard of Care
- Where
- 7 sites in United States
- Volunteers
- 1 people
- Sponsor
- Mendel Tuchman
Gaucher Disease
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from…
- Trial name
- NCT03625882
- Where
- 35 sites in Japan
- Volunteers
- 63 people
- Sponsor
- Takeda
Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia
Short-Term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia
The overall objective of this drug trial is to determine whether the treatment of acute hyperammonemia with N-carbamyl-L-glutamate (NCG, Carglumic acid) in propionic acidemia (PA), methylmalonic acidemia (MMA), late-onset CPS1…
- Conditions
- Propionic Acidemia, Type I and/or Type II, Methylmalonic Acidemia, Carbamoyl-Phosphate Synthase I Deficiency Disease, Ornithine Carbamoyltransferase Deficiency
- Trial name
- STO (NCT01599286)
- Testing
- Carbaglu
- Where
- 9 sites in United States
- Volunteers
- 35 people
- Sponsor
- Mendel Tuchman
Hemophilia A
A Study of TAK-660 in Surgical Procedures for People With Hemophilia A.
This study is about a factor VIII medicine called Adynovate (TAK-660) used during surgery for people with hemophilia A who have low blood levels of factor VIII.
- Trial name
- NCT04941898
- Testing
- PEGylated Recombinant Factor VIII
- Where
- Japan
- Volunteers
- 16 people
- Sponsor
- Takeda
Acute Undifferentiated Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Bioelectrical Impedance Measurement for Predicting Treatment Outcome in Patients With Newly Diagnosed Acute Leukemia
This clinical trial studies bioelectrical impedance measurement for predicting treatment outcome in patients with newly diagnosed acute leukemia.
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
- Trial name
- NCT01869777
- Testing
- bioelectric impedance analysis
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- Wake Forest University Health Sciences
Bronchopulmonary Dysplasia
Trial of Late Surfactant for Prevention of Bronchopulmonary Dysplasia
The purpose of this study is to determine if late doses of Infasurf surfactant given when patients are receiving inhaled nitric oxide will interact to improve surfactant function and increase survival without BPD in treated…
- Trial name
- TOLSURF (NCT01022580)
- Testing
- Infasurf surfactant (ONY, Inc.)
- Where
- 21 sites in United States
- Volunteers
- 511 people
- Sponsor
- Roberta Ballard
Friedreich's Ataxia
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.
- Trial name
- PROTI (NCT01303406)
- Testing
- Idebenone
- Where
- 6 sites in Austria, Germany and 2 more
- Volunteers
- 29 people
- Sponsor
- Santhera Pharmaceuticals
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire
Hemophilia B
Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B
Primary Objective: To evaluate the safety (acute effects associated with infusions, and inhibitor development), pharmacokinetics (PK), and efficacy with respect to breakthrough bleeding during prophylaxis and with respect to…
- Trial name
- NCT00768287
- Testing
- IB1001, nonacog alfa
- Where
- 23 sites in United States, France and 5 more
- Volunteers
- 77 people
- Sponsor
- Medexus Pharma, Inc.
Anesthesia; Adverse Effect, Congenital Heart Disease
The Effect of Anesthesia on Neurodevelopmental Outcome (NDO)
The purpose of this study is to assess whether the type of anesthesia, narcotic-based versus inhalational anesthesia administered during cardiopulmonary bypass (CPB) surgery contributes to the wide variation in neurologic…
- Conditions
- Anesthesia; Adverse Effect, Congenital Heart Disease, Neurodevelopmental Disorders
- Trial name
- NDO (NCT03882788)
- Testing
- Isoflurane, Fentanyl (high dose), Fentanyl (low dose)
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- Stanford University
Klinefelter Syndrome
Androgen Effect on Klinefelter Syndrome Motor Outcome
The purpose of this study is to evaluate the effects of low-dose androgen on the motor and cognitive development of boys with Klinefelter syndrome.
- Trial name
- NCT00348946
- Testing
- androgen oxandrolone
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Thomas Jefferson University
Hemophilia B
RIXUBIS Drug Use-Result Survey (Japan)
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT02937831
- Testing
- RIXUBIS
- Where
- 5 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Baxalta now part of Shire
Neonatal Seizures
Prophylactic Phenobarbital After Neonatal Seizures
The treatment of infants with medications after their seizures have stopped is very variable. No one knows if continuing treatment with phenobarbital for up to several months is helpful or harmful.
- Trial name
- PROPHENO (NCT01089504)
- Testing
- phenobarbital
- Where
- 18 sites in United States
- Volunteers
- 13 people
- Sponsor
- University of Rochester
Hemophilia A, Prophylaxis of Bleeding
Prophylaxis Regimen for Hemophilia A Patients
Researchers are looking for a better way to treat people who have hemophilia A. Hemophilia A is a genetic bleeding disorder that is caused by the lack of a protein in the blood called "clotting factor 8" (FVIII).
- Trial name
- PREDICT (NCT05036278)
- Testing
- Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
- Where
- 12 sites in United States
- Volunteers
- 21 people
- Sponsor
- Bayer
Congenital Heart Disease
Stress Response and Neurodevelopmental Outcome After Cardiac Surgery Utilizing CPB in Children: A Prospective, Double Blinded and Randomized Study
The overall goal of this project is to determine the role of anesthetic management in children undergoing cardiac surgery utilizing CPB in the setting of fast tracking and early extubation.
- Trial name
- NCT02492269
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Aymen N Naguib
Bronchopulmonary Dysplasia
Analysis of BPD in Premature Infants With Typical Imaging Changes
Bronchopulmonary dysplasia (BPD) is a common chronic respiratory disease in preterm infants. The increase in the survival rate of premature babies following the improvement of perinatal treatment and care has caused an increase…
- Trial name
- NCT04163822
- Testing
- no intervention
- Where
- China
- Volunteers
- 256 people
- Sponsor
- Wang Jianhui
Muscular Dystrophy, Arthrogryposis
WREX Outcome Study
This study is to evaluate how the Wilmington Robotic Exoskeleton (WREX) is working for children who are using the WREX, or have used it in the past.
- Conditions
- Muscular Dystrophy, Arthrogryposis, Spinal Muscular Atrophy
- Trial name
- NCT02218593
- Testing
- WREX orthosis
- Volunteers
- 25 people
- Sponsor
- Nemours Children's Clinic