Clinical trials: Long Term Outcomes
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
Non-small Cell Lung Cancer (NSCLC)
Long-Term Safety Outcomes and First-Line Treatment Patterns in Patients With Non-Small Cell Lung Cancer and Programmed Death-1 (Pd-L1) <1%
The purpose of this study is to assess the treatment-related adverse events and associated healthcare resource use in programmed death ligand 1 (PD-L1) negative individuals diagnosed with advanced/metastatic non-small cell lung…
- Trial name
- NCT07215962
- Testing
- Nivolumab + ipilimumab, Nivolumab + ipilimumab + platinum-based chemotherapy, Immuno-oncology-based therapy (excluding nivolumab-based regimens) with chemotherapy
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Bristol-Myers Squibb
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.
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States (LEAP-US)
The objective of this 5-year, prospective, observational cohort study is to evaluate the long-term safety and clinical outcomes of patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC)…
- Trial name
- LEAP (NCT06193928)
- Testing
- Livmarli
- Where
- 8 sites in United States
- Volunteers
- 70 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Dyslipidemia
Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk Open-label Extension
The primary clinical hypothesis is that long-term exposure of evolocumab will be safe and well tolerated in subjects with clinically evident atherosclerotic cardiovascular disease (CVD).
- Trial name
- FOURIER OLE (NCT02867813)
- Testing
- Evolocumab
- Where
- 198 sites in United States, Czechia and 5 more
- Volunteers
- 5,035 people
- Sponsor
- Amgen
Non-melanoma Skin Cancer
Post-market Multi-Center RETRO-Prospective Study to Assess Long-term Clinical Outcomes NMSC Patients Treated With eBx
The purpose of this retrospective-prospective study is to evaluate lesions after treatment for BCC or SCC NMSC in order to gain a better understanding of the durability of the treatment, and risk of late toxicities for this…
- Trial name
- NCT05257486
- Testing
- Xoft eBx
- Where
- 2 sites in United States
- Volunteers
- 183 people, healthy volunteers welcome
- Sponsor
- Xoft, 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
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
Excessive Daytime Sleepiness, Narcolepsy
Study to Assess Patient Reported Outcomes With Armodafinil Treatment for Excessive Sleepiness in Adults With Narcolepsy or Obstructive Sleep Apnea/Hypopnea Syndrome
The purpose of this study is to assess patient reported outcomes with armodafinil treatment in terms of improvement in sleepiness, satisfaction with treatment, impact on ability to engage in life activities (ie, daily or work and…
- Conditions
- Excessive Daytime Sleepiness, Narcolepsy, Obstructive Sleep Apnea/Hypopnea Syndrome (OSAHS)
- Trial name
- NCT00228566
- Testing
- Armodafinil
- Where
- 43 sites in United States
- Volunteers
- 247 people
- Sponsor
- Cephalon
Wet Age-related Macular Degeneration (wAMD)
Safety Evaluation of China Patients: Long-term Treatment Outcome of Conbercept Ophthalmic Injection in Real Word（STONE）
This is a post-marketing drug safety monitoring study in a prospective manner, and data collection will be performed in a registration-follow-up manner.
- Trial name
- NCT03054818
- Where
- 49 sites in China
- Volunteers
- 4,000 people
- Sponsor
- Chengdu Kanghong Biotech Co., Ltd.
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Sweden
This is a Swedish, Multicentre, Observational Registry to study Adherence and Long Term Outcomes of Therapy in paediatric subjects using Easypod™ electromechanical device for growth hormone treatment to assess the level of…
- Trial name
- ECOS SWE (NCT01307059)
- Testing
- easypod™
- Where
- 3 sites in Sweden
- Volunteers
- 152 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Diabetes Mellitus, Type 2
RECORD: Rosiglitazone Evaluated for Cardiac Outcomes and Regulation of Glycaemia in Diabetes
This study is a phase 3b, multicentre, randomised, open label, parallel group study. A 4-week run-in period will be followed by a median of 6 years of treatment with study medication in addition to continuation of background…
- Trial name
- RECORD (NCT00379769)
- Testing
- Rosiglitazone, Sulfonylurea, Metformin
- Where
- 446 sites in Australia, Belgium and 23 more
- Volunteers
- 4,447 people
- Sponsor
- GlaxoSmithKline
Wilson Disease
Study to Assess Long-Term Outcomes of Trientine in Wilson Disease Patients Withdrawn From Therapy With d-Penicillamine
A study to review Wilson disease patients who have previously been prescribed d- Penicillamine but were changed to trientine as treatment for their disease, and to follow them for a further 12 months.
- Trial name
- NCT02426905
- Testing
- trientine dihydrochloride
- Where
- 4 sites in Germany, Greece and 2 more
- Volunteers
- 90 people
- Sponsor
- Univar BV
Parkinson's Disease (PD)
Real-World Study of ABBV-951 Subcutaneous Infusion to Assess Change in Disease Activity in Adult Participants With Parkinson's Disease
Parkinson's disease (PD) is a neurological condition, which affects the brain. PD gets worse over time, but how quickly it progresses varies a lot from person to person.
- Trial name
- ROSSINI (NCT06107426)
- Where
- 68 sites in United States, Australia and 8 more
- Volunteers
- 427 people
- Sponsor
- AbbVie
Pulmonary Arterial Hypertension
A Prospective Non-interventional Observational Study to Observe Long-term Treatment and Outcomes in Pulmonary Arterial Hypertension (PAH) Patients
The goal of this observational study is to learn about the long-term development and outcomes of different treatment patterns of patients who initially participated in the TripleTRE study.
- Trial name
- TripleTRE-NIS (NCT06445673)
- Where
- 18 sites in Austria, Czechia and 7 more
- Volunteers
- 110 people
- Sponsor
- AOP Orphan Pharmaceuticals AG
Kidney Transplant Rejection
TruGraf® Long-term Clinical Outcomes Study
This is a prospective, multi-center, observational study. Subjects will have OmniGraf™ (TruGraf® and TRAC™) testing at study enrollment and thereafter every 3 months.
- Trial name
- NCT04491552
- Testing
- Patients monitored with TruGraf and TRAC testing
- Where
- 36 sites in United States
- Volunteers
- 2,000 people
- Sponsor
- Transplant Genomics, Inc.
Health-related Quality of Life, Pain
Outcomes During Digitally Delivered Osteoarthritis Treatment
Objectives: To examine changes in health-related quality of life (HRQL), pain and function among patients with knee or hip osteoarthritis (OA) participating in digitally delivered exercise and education treatment for up to 12 or…
- Conditions
- Health-related Quality of Life, Pain, Function, Osteoarthritis, Knee
- Trial name
- NCT05026580
- Testing
- Joint Academy exercise and education smart phone application
- Where
- Sweden
- Sponsor
- Joint Academy
Growth Disorders
To Assess the Level of Adherence of Subjects Receiving SAIZEN® Via Easypod™ in Spain
This is a Spanish, Multicentre, Observational Study to study adherence and long term outcomes of therapy in paediatric subjects using easypod™ electromechanical device for growth hormone treatment to assess the level of adherence…
- Trial name
- ECOS ESP (NCT01376921)
- Testing
- easypod™
- Where
- Spain
- Volunteers
- 300 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Glaucoma, Cataract
Observational Study to Evaluate Long Term Outcomes for Ocular Hypertension and Glaucoma Patients Treated With the SpyGlass Bimatoprost Implant System / IOL Combination
The goal of this clinical trial is to further observe and learn if the SpyGlass Pharma Bimatoprost Implant System / IOL Combination works to treat cataracts and either ocular hypertension or glaucoma.
- Conditions
- Glaucoma, Cataract, Ocular Hypertension
- Trial name
- NCT07154810
- Where
- Honduras
- Volunteers
- 23 people
- Sponsor
- SpyGlass Pharma, Inc.
Prostate Cancer (Adenocarcinoma)
Registry to Evaluate Long-Term Outcomes in Patients Who Undergo 68-Ga-PSMA-11 PET/CT Imaging Evaluations
The purpose of this observational research registry is to understand the real-world use of Illuccix PET/CT in prostate cancer, and how results from this testing impact patient's treatment and prostate cancer journey over time.
- Trial name
- NCT06655064
- Testing
- None- Observational
- Where
- 9 sites in United States
- Volunteers
- 120 people
- Sponsor
- Telix Pharmaceuticals (Innovations) Pty Limited