Clinical trials: Neonatal Outcomes
Hemophilia A, Hemophilia B
Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
- Trial name
- NCT02740413
- Volunteers
- 86 people
- Sponsor
- Pfizer
Sickle Cell Disease
A Study to Learn About Sickle Cell Disease In Adult Patients
The purpose of this clinical trial is to evaluate the performance of the sickle cell disease (SCD) electronic diary in people with SCD who are on treatment that will change SCD and those not on such a treatment.
- Trial name
- NCT05407805
- Testing
- Electronic Diary
- Where
- 4 sites in United States
- Volunteers
- 98 people
- Sponsor
- Pfizer
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
Familial Amyloid Polyneuropathy, ATTR-PN
An Extension of Study Fx-005 Evaluating Long-Term Safety And Clinical Outcomes Of Fx-1006A In Patients With Transthyretin Amyloid Polyneuropathy
This study is designed to determine the long-term safety and tolerability of Fx-1006A as well as the effects of Fx-1006A on clinical outcomes in patients with ATTR-PN.
- Trial name
- NCT00791492
- Testing
- Fx-1006A
- Where
- 11 sites in Argentina, Brazil and 4 more
- Volunteers
- 86 people
- Sponsor
- Pfizer
Fragile X Syndrome
Study of SPG601 in Adult Men With Fragile X Syndrome
This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
- Trial name
- NCT06413537
- Testing
- SPG601
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Spinogenix
Sanfilippo Syndrome
Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 9 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01299727
- Testing
- rhHNS-10 mg, rhHNS-45 mg, rhHNS-90 mg
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire
Familial Adenomatous Polyposis (FAP)
Observational Familial Adenomatous Polyposis Registry Study In Patients Receiving Celecoxib Compared to Control Patients
This is a registry-based observational study assessing clinical outcomes in FAP patients receiving celecoxib compared with historical/concurrent registry patients who have not received celecoxib.
- Trial name
- NCT00151476
- Testing
- Celecoxib, Routine Medical Care
- Where
- 4 sites in United States, Canada and 2 more
- Volunteers
- 68 people
- Sponsor
- Pfizer
Pompe Disease, Glycogen Storage Disease Type II (GSD-II)
Growth and Development Study of Alglucosidase Alfa
Pompe disease (also known as glycogen storage disease Type II) is a rare autosomal recessive metabolic muscle disease caused by the deficiency of acid α glucosidase (GAA), an enzyme that degrades lysosomal glycogen.
- Conditions
- Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease
- Trial name
- NCT00486889
- Testing
- alglucosidase alfa
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase
Study TKT024EXT was a long-term, single-arm, open-label extension of Study TKT024, a one year Phase 2/Phase 3 registration study.
- Trial name
- NCT00630747
- Testing
- Idursulfase
- Where
- 52 sites in United States, Brazil and 8 more
- Volunteers
- 94 people
- Sponsor
- Shire
Complication of Renal Dialysis, End Stage Renal Disease
A Study Testing the Use of a Perivascular Sirolimus Formulation (Sirogen) in ESRD Patients Undergoing AV Fistula Surgery
The primary study objective is to evaluate the benefit of the Sirolimus eluting Collagen implant (SeCI; Sirogen), a single dose prophylactic treatment delivered intraoperatively at the time of surgical creation of an…
- Conditions
- Complication of Renal Dialysis, End Stage Renal Disease, End Stage Kidney Disease, ESRD
- Trial name
- ACCESS2 (NCT05425056)
- Testing
- Sirolimus
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 136 people
- Sponsor
- Vascular Therapies, Inc.
Fabry Disease
Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease
This study will evaluate safety and clinical outcomes of treatment with Replagal in adult participants with Fabry disease who have completed Study TKT028 (NCT00864851).
- Trial name
- NCT01124643
- Testing
- Replagal
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Shire
Hemophilia A, Hemophilia B
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.
- Trial name
- JOIN-us (NCT05856266)
- Testing
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US), Haemophilia Joint Health Score (HJHS)
- Where
- 56 sites in Bulgaria, Croatia and 9 more
- Volunteers
- 10 people
- Sponsor
- Swedish Orphan Biovitrum
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.
Complication of Renal Dialysis, End Stage Renal Disease
Trial to Evaluate the Sirolimus-Eluting Collagen Implant on AV Fistula Outcomes
The objective of this study is to evaluate efficacy and safety outcomes following use of the Sirolimus-eluting Collagen Implant (SeCI) in subjects undergoing surgical creation of an AV fistula for vascular access (index…
- Conditions
- Complication of Renal Dialysis, End Stage Renal Disease, End Stage Kidney Disease, ESRD
- Trial name
- ACCESS (NCT02513303)
- Testing
- Sirolimus, AV Fistula Surgery, Sirolimus-eluting Collagen Implant (SeCI)
- Where
- 4 sites in United States
- Volunteers
- 269 people
- Sponsor
- Vascular Therapies, Inc.
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Gaucher Disease
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care.
- Trial name
- SWITCH (NCT04718779)
- Testing
- Digital Engagement Application (GD App), No Intervention
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Takeda
Choroideremia
Efficacy and Safety of BIIB111 for the Treatment of Choroideremia
The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).
- Trial name
- STAR (NCT03496012)
- Testing
- BIIB111
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 169 people
- Sponsor
- Biogen
Hunter Syndrome
Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094
This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT…
- Trial name
- NCT02412787
- Testing
- Idursulfase-IT, Elaprase
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 56 people
- Sponsor
- Shire
Respiratory Syncytial Virus Infections
A Study of Outcomes and Events of Interest in Pregnant Women, Neonates and Infants and of RSV Surveillance
The purpose of this study is to assess pregnancy outcomes, and maternal, as well as neonatal events of interest in healthy pregnant women and their new-borns.
- Trial name
- PEPNI (NCT03614676)
- Testing
- Blood sample collection, Cord blood sample collection, Maternal Diary Card
- Where
- 40 sites in Argentina, Bangladesh and 8 more
- Volunteers
- 4,493 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Hunter Syndrome, Mucopolysaccharidosis II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
The objective of this study is to determine the safety of once weekly dosing of idursulfase 0.5 mg/kg administered by intravenous (IV) infusion for male Hunter syndrome patients ≤ 5 years old.
- Conditions
- Hunter Syndrome, Mucopolysaccharidosis II, MPS II
- Trial name
- NCT00607386
- Testing
- Idursulfase
- Where
- 3 sites in Brazil, Poland and 1 more
- Volunteers
- 28 people
- Sponsor
- Shire