Clinical trials: Neonatal Outcome
Persistent Fetal Circulation Syndrome
Sildenofil in Persistent Pulmonary Hypertension in Newborns
This study hopes to evaluate the effectiveness of early combined use of Sildenafil and nitric oxide (iNO) in newborns with Persistent pulmonary hypertension (PPHN) and or hypoxemic respiratory failure and assess whether this…
- Trial name
- Sildeno (NCT01558466)
- Testing
- Sildenafil, diluent
- Where
- Qatar
- Volunteers
- 100 people
- Sponsor
- Hamad Medical Corporation
Down Syndrome, Edwards Syndrome
Clinical Performance of the MaterniT21 PLUS LDT in Multiple Gestation Pregnancies
This study will evaluate the clinical performance of massively parallel sequencing (MPS) using the MaterniT21 PLUS LDT in the detection of fetal aneuploidy in circulating cfDNA extracted from a maternal blood sample obtained from…
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Turner Syndrome
- Trial name
- NCT02226315
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Sequenom, Inc.
Fabry Disease
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natural history on male and female…
- Trial name
- NCT06052800
- Where
- 5 sites in Taiwan
- Volunteers
- 78 people
- Sponsor
- Sanofi
Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I
Limb-Girdle Video Assessment
The purpose of this study is to develop a new remote-based video assessment outcome measure for Limb-Girdle Muscular Dystrophy (LGMD) trials. The overall objectives for this study are: 1.
- Trial name
- NCT05528757
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- The Emmes Company, LLC
Gout
Allopurinol Outcome Study
This is a study of allopurinol in gout patients with hyperuricemia that will evaluate the safety and serum urate (sUA) lowering capability of allopurinol as a urate lowering therapy (ULT) for up to six months.
- Trial name
- LASSO (NCT01391325)
- Testing
- Allopurinol
- Where
- 173 sites in United States, Australia and 5 more
- Volunteers
- 1,735 people
- Sponsor
- Ardea Biosciences, 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
Malaria
Study to Assess Safety and Clinical Responses With Coartem® Baby Dispersible Tablets in Paediatric Patients Weighing 2 to <5 kg With Uncomplicated Malaria
This study is being conducted to further evaluate the safety and clinical outcomes of Coartem® Baby (artemether/lumefantrine 5 mg/60 mg) in neonates and infants weighing ≥2 kg to <5 kg with uncomplicated malaria due to Plasmodium…
- Trial name
- NCT07713602
- Testing
- Coartem® Baby
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Fragile-X Syndrome
EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes
This prospective observational study (registry) on Fragile X syndrome (FXS) is designed to evaluate characteristics, management and patient and caregiver-related outcomes the quality of life of Families and patients with FXS and…
- Trial name
- EXPLAIN (NCT01711606)
- Where
- Germany
- Volunteers
- 75 people
- Sponsor
- Novartis Pharmaceuticals
Preterm Delivery
A Randomized Trial Comparing the Impact of One Versus Two Courses of Antenatal Steroids (ACS) on Neonatal Outcome
The hypothesis is that administration of two courses of antenatal corticosteroids, compared to one course, will show a 40% reduction in the incidence of composite neonatal morbidity in patients delivering prior to 34 weeks'…
- Trial name
- ACS (NCT00201643)
- Testing
- Betamethasone or Dexamethasone (2nd course of ACS)
- Where
- 22 sites in United States
- Volunteers
- 437 people
- Sponsor
- Obstetrix Medical Group
Protein C Deficiency
Retrospective Study to Capture Dosing and Treatment Outcome Data in Participants With Severe Congenital Protein C Deficiency Who Were Treated With Protein C Concentrate Under an Emergency Use IND
This is a data collection study with the purpose of capturing dosing and treatment outcome data in participants with severe congenital protein C deficiency who were treated with protein C concentrate under an Emergency Use…
- Trial name
- NCT00161720
- Testing
- Protein C Concentrate (Human) Vapor Heated
- Where
- 10 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baxalta now part of 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
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
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
Haemophilia B
Post-marketing Surveillance (Use Result Surveillance) With Refixia®
The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B.
- Trial name
- NCT03875547
- Testing
- Refixia®
- Where
- 23 sites in Denmark, Japan
- Volunteers
- 28 people
- Sponsor
- Novo Nordisk A/S
Huntington Disease
PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD
This study will evaluate the efficacy and safety of pridopidine 45mg twice daily (BID) in patients with early stage manifest Huntington Disease (HD).
- Trial name
- NCT04556656
- Testing
- Pridopidine
- Where
- 58 sites in United States, Austria and 9 more
- Volunteers
- 499 people
- Sponsor
- Prilenia
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
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
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