Clinical trials: Long-term Outcomes
- Condition Long-term Outcomes
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Greece
This is a National, Multicentre, Observational Registry to study adherence and long term outcomes of therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospitals in Greece and…
- Trial name
- ECOS GRE (NCT01363674)
- Testing
- Easypod™
- Where
- Greece
- Volunteers
- 180 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Hereditary Angioedemas
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice.
- Trial name
- GREAT (NCT07001280)
- Testing
- No intervention
- Where
- 22 sites in United States, Austria and 3 more
- Volunteers
- 200 people
- Sponsor
- CSL Behring
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in the UK
This is a National, Multicentre, Observational Registry to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospital in the UK and…
- Trial name
- ECOS UK (NCT01263457)
- Testing
- Easypod
- Where
- 6 sites in United Kingdom
- Volunteers
- 191 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Heart Disease
PULSTA TPV Observational Study
The purpose of the study is to determine the long-term follow-up clinical outcomes, effectiveness, and safety data of the PULSTA Transcatheter Pulmonary Valve (TPV) implantation in a real-world situation
- Trial name
- NCT07747207
- Where
- 9 sites in Germany, Italy and 4 more
- Volunteers
- 200 people
- Sponsor
- Taewoong Medical Co., Ltd.
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Argentina
This is an Argentinian, Multicentre, Observational Study to evaluate Adherence and Long Term Outcomes of Therapy in paediatric subjects using easypod™ electromechanical device for growth hormone treatment and to assess the level…
- Trial name
- ECOS ARG (NCT01582334)
- Testing
- easypod™
- Where
- Argentina
- Volunteers
- 68 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Aphakia
A Real-World Evidence (RWE) Long-Term Follow-Up Study to Assess Outcomes of Alcon Monofocal Intraocular Lenses
The purpose of this post-market clinical follow-up (PMCF) study is to describe the long-term safety and performance of AcrySof Single-Piece and AcrySof Multi-Piece monofocal intraocular lenses (IOLs).
- Trial name
- NCT05799950
- Testing
- AcrySof single-piece IOL, AcrySof multi-piece IOL
- Where
- 7 sites in United States, Puerto Rico
- Volunteers
- 138 people
- Sponsor
- Alcon Research
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
In patients with Alagille syndrome (ALGS), the key objectives are to evaluate the tolerability, long-term safety (including possible liver toxicity) and long-term efficacy of Livmarli treatment.
- Trial name
- NCT07290257
- Testing
- Livmarli Oral Product
- Where
- 15 sites in Belgium, France and 6 more
- Volunteers
- 230 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Primary Immune Deficiency
Multicenter Study of Long-Term Clinical Outcomes of Subcutaneous Immune Globulin IgPro20 in Subjects With Primary Immunodeficiency (Japan Study)
The purpose of this study is to assess the long-term efficacy, tolerability, and safety of IgPro20 in subjects with primary immunodeficiency (PID) as an extension to the preceding follow-up study ZLB07_001CR (NCT01458171).
- Trial name
- NCT01461018
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Middle East
This is a Middle East, Multicenter, Observational Study to evaluate Adherence and Long Term Outcomes of Therapy in pediatric subjects using easypod™ electromechanical device for growth hormone treatment and to assess the level of…
- Trial name
- ECOS UAE (NCT02015286)
- Testing
- easypod™
- Where
- Germany
- Volunteers
- 7 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Retinopathy of Prematurity (ROP)
Extension Study to Evaluate the Long-term Outcomes of Subjects in Study 20090
This is a follow-up study to evaluate the long term outcome of babies treated in the FIREFLEYE study.
- Trial name
- FIREFLEYE next (NCT04015180)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 55 sites in Argentina, Belgium and 22 more
- Volunteers
- 100 people
- Sponsor
- Bayer
Type 1 Diabetes
Long-Term Outcomes of Teplizumab in Routine Clinical Care
This is an observational, prospective cohort study designed to evaluate the outcomes after teplizumab treatment in participants with Stage 2 Type 1 Diabetes (T1D) for delaying the onset of Stage 3 T1D.
- Trial name
- AL1GN (NCT07360080)
- Testing
- Teplizumab
- Where
- 10 sites in United States, Israel and 1 more
- Volunteers
- 1,000 people
- Sponsor
- Sanofi
Eosinophilic Esophagitis
A Non-interventional Observational Study to Assess Long-term Efficacy and Safety of Dupilumab for the Treatment of Patients (>= 12 Years) With Eosinophilic Esophagitis Under Real-world-conditions in Germany
This is a longitudinal, observational non-interventional study (NIS) designed to characterize the patient population who receives dupilumab for eosinophilic esophagitis (EoE) treatment in a real-world setting in Germany with…
- Trial name
- ProMEAL (NCT06695897)
- Testing
- Dupilumab
- Where
- 15 sites in Germany
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Sanofi
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
Cryopyrin-associated Periodic Syndromes (CAPS), Familial Cold Autoinflam Syn (FCAS)
Clinical Outcomes and Safety: A Registry Study of Ilaris (Canakinumab) Patients
The purpose of this observational study is to collect additional information regarding long-term safety and effectiveness of Ilaris in the treatment of CAPS patients in clinical practice.
- Conditions
- Cryopyrin-associated Periodic Syndromes (CAPS), Familial Cold Autoinflam Syn (FCAS), Muckle-wells Syn (MWS), Neonatal Onset Multisystem Inflam Disease (NOMID)
- Trial name
- B-Confident (NCT01213641)
- Where
- 17 sites in United States, Austria and 3 more
- Volunteers
- 288 people
- Sponsor
- Novartis Pharmaceuticals
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
Duchenne Muscular Dystrophy
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported…
- Trial name
- DMD-001 SUMMIT (NCT06564974)
- Testing
- Vamorolone
- Where
- 33 sites in United States, Puerto Rico
- Volunteers
- 250 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Chronic Hepatitis B
Study of Entecavir in Patients With Chronic Hepatitis B Virus (HBV) Infection
The purpose of this study is to prospectively assess the long-term outcomes (benefits and risks) associated with entecavir (ETV) therapy as compared to other antivirals approved for the treatment of chronic HBV infection.
- Trial name
- NCT00388674
- Testing
- entecavir, Other anti-HBV medication
- Where
- 290 sites in United States, Argentina and 22 more
- Volunteers
- 12,522 people
- Sponsor
- Bristol-Myers Squibb
Obstetric Labour, Premature
Follow up Study to Assess Long Term Safety and Outcomes in Infants and Children Born to Mothers Participating in Retosiban Treatment Studies
The primary objective of this study is to assess the safety and outcomes of infants and children who were exposed to retosiban or comparator in utero in the Phase III spontaneous preterm labor (SPTL) treatment studies, to provide…
- Trial name
- ARIOS (NCT02292784)
- Testing
- Retosiban, Atosiban
- Where
- 26 sites in United States, Belgium and 9 more
- Volunteers
- 98 people
- Sponsor
- GlaxoSmithKline
Growth Disorders
A Canadian, Multi-centre, Observational Registry to Study Adherence and Long Term Outcomes of Therapy in Pediatric Subjects Using SAIZEN® Via Easypod™ Auto-injector for Growth Hormone Treatment
This is a Canadian, Multicentre, Observational Registry to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment from hospital and to assess…
- Trial name
- ECOS CAN (NCT01267526)
- Testing
- Easypod™
- Where
- 9 sites in Canada
- Volunteers
- 205 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Progressive Familial Intrahepatic Cholestasis
A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in South Korea
This study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) as they use odevixibat in their daily lives.
- Trial name
- NCT07185919
- Where
- 5 sites in South Korea
- Volunteers
- 10 people
- Sponsor
- Ipsen