Clinical trials: Neonatal Outcome
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
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
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.
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05690 (Care Program) (P05710)
The objective of this follow-up study is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of multifollicular growth in women undergoing controlled ovarian stimulation (COS) prior to in vitro…
- Trial name
- Care (NCT00702624)
- Testing
- Corifollitropin alfa, recFSH (follitropin beta), gonadatropin releasing hormone (GnRH) antagonist ganirelix
- Volunteers
- 113 people
- Sponsor
- Organon and Co
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
Hypophosphatasia
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support…
- Trial name
- NCT04195763
- Testing
- asfotase alfa
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05693 (P05713)
The objective of follow-up study P05713 is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of monofollicular growth in women who underwent ovulation induction (OI) in base study P05693…
- Trial name
- Care (NCT00702338)
- Testing
- corifollitropin alfa, recombinant Follicle Stimulating Hormone (recFSH), human Chorion Gonadotropin (hCG)
- Volunteers
- 1 people
- Sponsor
- Organon and Co
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447993
- Testing
- Low Dose NT-501, High Dose NT-501
- Where
- 11 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurotech Pharmaceuticals
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
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
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.
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
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial 38833 (P05783)
The objective of this trial is to evaluate whether corifollitropin alfa (MK-8962, Org 36286) treatment for the induction of multifollicular growth in women undergoing Controlled Ovarian Stimulation (COS) prior to in vitro…
- Trial name
- Care (NCT00702520)
- Testing
- Corifollitropin alpha (MK-8962, Org 36286) 100 ug, Corifollitropin alpha (MK-8962, Org 36286) 150 ug, Triptorelin
- Volunteers
- 15 people
- Sponsor
- Organon and Co
Atopic Eczema
Study to Investigate Skin Conditions and Patient Assessment of LAS 41002 in the Treatment of Atopic Eczema
The aim of the study is an intra-individual comparison of skin conditions in addition to a patient based assessment of product appearance in the topical treatment of atopic eczema.
- Trial name
- NCT01119313
- Testing
- LAS 41002, Active
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Almirall, S.A.
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