Clinical trials: Neonatal Outcome
- Condition Neonatal Outcome
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Growth Hormone Disorder, Growth Hormone Deficiency in Children
Observational Prospective Study on Patients Treated With Norditropin®
This observational study is conducted globally. The aim of the study is to investigate the effectiveness and safety of real-life treatment with Norditropin®.
- Conditions
- Growth Hormone Disorder, Growth Hormone Deficiency in Children, Adult Growth Hormone Deficiency, Genetic Disorder
- Trial name
- NCT00960128
- Testing
- somatropin
- Where
- 23 sites in Argentina, Belgium and 21 more
- Volunteers
- 21,249 people
- Sponsor
- Novo Nordisk A/S
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
Asphyxia Neonatorum
Biomarkers And Neurological Outcome in Neonates 2
Follow-up of participants of BANON1 study at age of at least 2 years. BANON1 (ClinicalTrials.gov ID: NCT03357250): Validation of biomarkers based on 24 months neuro-developmental outcome data in a human population for their…
- Trial name
- BANON2 (NCT04714775)
- Where
- 13 sites in Germany, Turkey (Türkiye)
- Volunteers
- 500 people
- Sponsor
- InfanDx AG
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
- Trial name
- NCT02131623
- Where
- 5 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
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
Fabry Disease
Fabry Outcome Survey (FOS)
The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease.
- Trial name
- FOS (NCT03289065)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Shire
Pregnancy Complications, Pregnancy Outcome
BioThrax® (Anthrax) Vaccine in Pregnancy Registry
The purpose of this study is to determine if inadvertent receipt of the BioThrax vaccine during pregnancy is independently associated with adverse maternal, pregnancy, or infant health outcomes.
- Conditions
- Pregnancy Complications, Pregnancy Outcome, Congenital Abnormalities
- Trial name
- NCT01653392
- Testing
- Observational Intervention
- Where
- United States
- Volunteers
- 98 people, healthy volunteers welcome
- Sponsor
- Emergent BioSolutions
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
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
Osteoarthritis of Hip, Congenital Hip Dysplasia
Clinical and Radiographic Outcomes of the Corin Tri-Fit Total Hip Replacement
The study will evaluate the radiographs (x-rays) of 100 patients with a TriFIT total hip at 2 weeks, 3 months, 6 months, 12 months, and yearly following surgery to see if there has been any movement or wear in the hip stem over…
- Conditions
- Osteoarthritis of Hip, Congenital Hip Dysplasia, Avascular Necrosis
- Trial name
- NCT02162186
- Where
- Canada
- Volunteers
- 37 people
- Sponsor
- Corin
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum
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
Gaucher Disease
Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received.
- Trial name
- NCT03291223
- Where
- United States
- Volunteers
- 1,257 people
- Sponsor
- Shire
Hemophilia
A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B
The purpose of this non-interventional, observational, real-world study is to find out what are the real-world experiences of people living with hemophilia A or B[OK1.1] who start the study medicine (called marstacimab) in…
- Trial name
- NCT07859527
- Testing
- Marstacimab
- Volunteers
- 100 people
- Sponsor
- Pfizer
Hemophilia A
ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
The purpose of the study is to document the natural history of hemophilia A disease and long-term outcomes in terms of effectiveness, safety and quality of life in participants receiving Antihemophilic Factor (Recombinant) …
- Trial name
- AHEAD (NCT02078427)
- Testing
- ADVATE, ADYNOVI
- Where
- 110 sites in Australia, Austria and 20 more
- Volunteers
- 951 people
- Sponsor
- Baxalta now part of Shire
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
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.
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
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
RESULT: Reliable, Emergent Solution Using Liprotamase Treatment
Liprotamase powder is a non-porcine, soluble and stable mixture of biotechnology-derived lipase, protease, and amylase digestive enzymes.
- Trial name
- NCT03051490
- Testing
- Liprotamase, porcine PERT
- Where
- 50 sites in United States, Hungary and 5 more
- Volunteers
- 140 people
- Sponsor
- Anthera Pharmaceuticals