Clinical trials: Rare Disease
Atherosclerotic Disease, Carotid
Post-Approval Study of the Guidant Carotid Stent Systems and Embolic Protection Systems: CAPTURE
This purpose of this study is to collect data on the FDA-approved ACCULINK(TM) Carotid Stent System and FDA 510(k)-cleared ACCUNET(TM) Embolic Protection System for the treatment of patients with atherosclerotic disease, when…
- Trial name
- NCT00180492
- Testing
- RX ACCULINK(TM) Carotid Stent System and RX ACCUNET(TM) Embolic Protection System
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- Abbott Medical Devices
Choroidal Neovascularization, Macular Edema
An Open-label Extended Clinical Protocol of Ranibizumab to Evaluate Safety and Efficacy in Rare VEGF Driven Ocular Diseases.
The pupose of this study is to evaluate the safety and the efficacy of ranibizumab in rare VEGF driven ocular diseases.
- Conditions
- Choroidal Neovascularization, Macular Edema, Glaucoma, Neovascular, Diabetic Retinopathy
- Trial name
- ECLIPSE (NCT01908816)
- Testing
- ranibizumab
- Where
- 39 sites in France
- Volunteers
- 270 people
- Sponsor
- Novartis Pharmaceuticals
Primary Hyperoxaluria Type 1, PH1
Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
This is a single-arm, open-label, single-dose dose-escalation study. It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily assess the effect…
- Trial name
- NCT07776626
- Testing
- YOLT-203
- Where
- China
- Volunteers
- 9 people
- Sponsor
- YolTech Therapeutics Co., Ltd
Non-Small Cell Lung Cancer (NSCLC), Metastatic Non-Small Cell Lung Cancer
Study of Furmonertinib in Patients With Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) With Activating, Including Uncommon, Epidermal Growth Factor Receptor (EGFR) or Human Epidermal Growth Factor Receptor 2 (HER2) Mutations
This is a Phase 1b, open-label, multi-center, dose-escalation and dose expansion study designed to evaluate the safety, pharmacokinetics (PK), and preliminary antitumor activity of furmonertinib in patients with advanced or…
- Conditions
- Non-Small Cell Lung Cancer (NSCLC), Metastatic Non-Small Cell Lung Cancer, Advanced Non-Small Cell Lung Cancer, HER2 Exon 20 Mutations
- Trial name
- NCT05364073
- Testing
- Furmonertinib
- Where
- 42 sites in United States, Australia and 9 more
- Volunteers
- 160 people
- Sponsor
- ArriVent BioPharma, Inc.
Ovarian Cancer
A Study Evaluating the Efficacy and Safety of Biomarker-Driven Therapies in Patients With Persistent or Recurrent Rare Epithelial Ovarian Tumors
This study will evaluate the efficacy and safety of multiple biomarker-selected treatments in patients with persistent or recurrent rare epithelial ovarian, fallopian tube, or primary peritoneal tumors.
- Trial name
- BOUQUET (NCT04931342)
- Testing
- Ipatasertib, Cobimetinib, Trastuzumab Emtansine
- Where
- 41 sites in United States, Australia and 11 more
- Volunteers
- 176 people
- Sponsor
- Hoffmann-La Roche
Hereditary Inclusion Body Myopathy, GNE Myopathy
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and…
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM)
- Trial name
- NCT01784679
- Where
- 5 sites in United States, Bulgaria and 3 more
- Volunteers
- 319 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Huntington Disease
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
The primary goal of this study is to evaluate the safety and pharmacodynamic effects of PTC518 compared with placebo in participants with HD.
- Trial name
- NCT05358717
- Testing
- PTC518
- Where
- 28 sites in United States, Australia and 9 more
- Volunteers
- 159 people
- Sponsor
- Novartis Pharmaceuticals