Clinical trials: Defects
Leber Congenital Amaurosis (LCA), Eye Diseases
Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy…
- Conditions
- Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
- Trial name
- NCT02946879
- Testing
- AAV OPTIRPE65
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 15 people
- Sponsor
- MeiraGTx UK II Ltd
Pain, Multiple Sclerosis
A Study to Evaluate the Effects of Cannabis Based Medicine in Patients With Pain of Neurological Origin
To investigate the ability of a cannabis based medicine extract to relieve chronic refractory pain of neurological origin.
- Trial name
- NCT01606176
- Testing
- GW-1000-02
- Where
- United Kingdom
- Volunteers
- 70 people
- Sponsor
- Jazz Pharmaceuticals
Split-thickness Skin Graft Donor Sites
Clinical Study on Veloderm for the Treatment of Split-thickness Skin Graft Donor Sites
This study is a multi-center, randomized, open and paralleled positive control study for evaluating the safety and efficacy of Veloderm in treatment of split-thickness skin graft donor sites.
- Trial name
- NCT01512017
- Testing
- Crystalline cellulose simple occlusive dressing, Vaseline
- Volunteers
- 96 people
- Sponsor
- Lee's Pharmaceutical Limited
Knee Cartilage Defects
Phase 3 Pivotal Trial Comparing CARTISTEM® and Surgical Comparator for Knee Cartilage Lesions and Osteoarthritis
This study will test whether CARTISTEM is safe and works better than debridement (a surgery that cleans out damaged tissue) for people with knee cartilage damage and osteoarthritis.
- Trial name
- CARTISTEM23-01 (NCT07339111)
- Testing
- CARTISTEM® + Debridement, Debridement
- Where
- 35 sites in United States
- Volunteers
- 300 people
- Sponsor
- Medipost, Inc.
Muscular, Ventricular
Emergency/Compassionate Use - Muscular VSD Occluder
Expanded access to the AMPLATZER Muscular VSD Occluder
- Conditions
- Muscular, Ventricular, Septal, Defects
- Trial name
- NCT00590382
- Testing
- AMPLATZER Muscular VSD Occluder
- Sponsor
- Abbott Medical Devices
Long QT Syndrome (LQTS) 2
Evaluation of the Efficacy and Safety of THRV-1268 in Participants With Long QT Syndrome Type 2 (LQTS2) and Type 3 (LQTS3)
The goal of this clinical trial is to learn whether the study drug THRV-1268 can safely and effectively shorten the QT interval in people diagnosed with Long QT Syndrome Type 2 (LQTS 2) and Long QT Syndrome Type 3 (LQTS 3).
- Trial name
- Wave II (NCT07277582)
- Testing
- baseline lead-in, THRV-1268
- Where
- 9 sites in United States
- Volunteers
- 84 people
- Sponsor
- Thryv Therapeutics, Inc.
Trisomy 13, Trisomy 18
Prenatal Non-invasive Aneuploidy Test Utilizing SNPs Trial
This prospective blinded study will assess the diagnostic capability of an informatics enhanced SNP based technology (Parental Support) to identify pregnant women who are carrying a fetus with an aneuploidy from fee floating DNA…
- Conditions
- Trisomy 13, Trisomy 18, Trisomy 21, Aneuploidy
- Trial name
- PreNATUS (NCT01545674)
- Testing
- Blood Draw
- Where
- 29 sites in United States, Canada and 4 more
- Volunteers
- 937 people, healthy volunteers welcome
- Sponsor
- Natera, Inc.
Congenital Heart Disease, Tetralogy of Fallot
Study of the Native Outflow Tract Transcatheter Pulmonary Valve (TPV)
The purpose of this early feasibility study is to determine how a new transcatheter pulmonary valve will move and perform once implanted in the right ventricular outflow tract.
- Trial name
- NCT01762124
- Testing
- Native Outflow Tract TPV
- Where
- 3 sites in United States, Canada
- Volunteers
- 20 people
- Sponsor
- Medtronic Heart Valves
Cartilage Defects of the Knee
Study to Evaluate a New Cartilage Regeneration Platform for Knee Cartilage Repair
The goal of this clinical trial is to learn if a new treatment works to help regrow knee cartilage in people with knee cartilage damage.
- Trial name
- TCW (NCT07639424)
- Testing
- MAECM transplantation, Micronfracture
- Where
- 13 sites in South Korea
- Volunteers
- 104 people
- Sponsor
- ROKIT Healthcare
Congenital Heart Disease, Advanced Heart Failure
TORCH-LATAM-003: Regional Advanced-Therapy Pathways for Congenital Heart Disease
TORCH-LATAM-003 is a pragmatic stepped-wedge cluster randomized trial evaluating a regional pathway for children and adults with congenital heart disease and advanced cardiac and/or respiratory failure who may require heart…
- Conditions
- Congenital Heart Disease, Advanced Heart Failure, Respiratory Failure, Heart Transplantation
- Trial name
- TORCH-003 (NCT07837024)
- Testing
- TORCH Congenital Advanced Therapies Pathway (TORCH-CATP)
- Volunteers
- 360 people
- Sponsor
- CardioVision International Tech, S.A.S. de C.V.
Articular Cartilage Lesion of the Femoral Condyle
Efficacy and Safety Study of co.Don Chondrosphere to Treat Cartilage Defects
This is a prospective, phase III, multicenter, open label, randomised clinical trial of co.don chondrosphere®, a three-dimensional autologous chondrocyte transplantation product (ACT3D-CS)compared to the procedure of…
- Trial name
- NCT01222559
- Testing
- co.don chondrosphere®, Microfracture
- Where
- 12 sites in Germany, Poland
- Volunteers
- 102 people
- Sponsor
- co.don AG
Aortic Stenosis, Bicuspid Aortic Valve (BAV)
TAVR vs. SAVR Study of VitaFlow Liberty® for Severe BAV Stenosis
To evaluate the safety and effectiveness of the Transcatheter aortic valve and retrievable delivery system (VitaFlow Liberty®) for the treatment of severe bicuspid aortic valve (BAV) stenosis.
- Trial name
- PROMIS-BAV (NCT06597188)
- Testing
- VitaFlow Liberty, Commercially available surgical bioprosthetic valve
- Where
- China
- Volunteers
- 452 people
- Sponsor
- Shanghai MicroPort CardioFlow Medtech Co., Ltd.
Neutropenia
A Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are…
- Trial name
- NCT06056297
- Testing
- Mavorixafor
- Where
- 114 sites in United States, Argentina and 23 more
- Volunteers
- 176 people
- Sponsor
- X4 Pharmaceuticals
Benign Prostatic Hyperplasia
Optilume™ BPH Catheter System in Men With Symptomatic BPH
PINNACLE is a prospective, multi-center, randomized control clinical trial to establish the safety and efficacy of the Optilume™ BPH Catheter System in the treatment of benign prostatic hyperplasia (BPH)
- Trial name
- PINNACLE (NCT04131907)
- Testing
- Optilume BPH Catheter System
- Where
- 21 sites in United States, Canada
- Volunteers
- 162 people
- Sponsor
- Urotronic Inc.
Long QT Syndrome
Study of LQT-1213 on QTc-induced Prolongation in Healthy Adult Subjects (Part1) and on Congenital Long QT in Patients Diagnosed With Type 2 or 3 Long QT Syndrome (Part 2).
Part 1: This is a Phase 1b, randomized, double-blind, crossover, dose escalation, placebo-controlled study to evaluate the effect of oral LQT-1213 on dofetilide-induced QTc prolongation in healthy adult subjects.
- Trial name
- NCT05906732
- Testing
- LQT-1213, Dofetilide 250 μg Cap
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Thryv Therapeutics, Inc.
Migraine, Patent Foramen Ovale
GORE® CARDIOFORM Septal Occluder Migraine Clinical Study
Multi-center, prospective, randomized, placebo- and sham-controlled study to evaluate the GORE® CARDIOFORM Septal Occluder for migraine headache relief
- Conditions
- Migraine, Patent Foramen Ovale, PFO - Patent Foramen Ovale
- Trial name
- RELIEF (NCT04100135)
- Testing
- Actual device PFO closure, Thienopyridine (clopidogrel or prasugrel)
- Where
- 15 sites in United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- W.L.Gore & Associates
Age Related Macular Degeneration
Correlation Between Visual Field Defects on FORESEE HOME and on Optical Coherence Tomography in Choroidal Neovascularization Subjects
The FORESEE HOME is used in the recent years to detect Age-related Macular Degeneration (AMD) lesions. The device is capable of differentiation as to stages of AMD and early detection of changes including choroidal…
- Trial name
- NCT00884637
- Where
- Israel
- Volunteers
- 15 people
- Sponsor
- Notal Vision Ltd.
Patent Ductus Arteriosus
Safety and Efficacy Study of Ibuprofen l-Lysine Solution in Premature Infants for Treatment of PDA
The purpose of this study is to determine the safety and effectiveness of ibuprofen l-lysine iv in premature infants in the early treatment of Patent Ductus Arteriosus.
- Trial name
- NCT00440804
- Testing
- ibuprofen l-lysine iv solution (NeoProfen (R) )
- Sponsor
- Farmacon
Coarctation of the Aorta, Pulmonary Artery Stenosis
Growth Trial: Study of the Renata Minima Stent
The objective of the clinical investigation is to access clinical safety and effectiveness of the Minima Stent in neonates, infants, and young children requiring intervention for common congenital vascular stenosis (i.e.…
- Trial name
- NCT05086016
- Testing
- Catheterized Stenting
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Renata Medical
Atrial Septal Defect
Cera™ ASD Occluder Post-Market Clinical Follow-Up Study
The objective of this post-market study is to: collect real-word clinical data and confirm the long-time safety and performance of the Lifetech Cera™ ASD Occluder identify previously unknown side-effects
- Trial name
- NCT06849635
- Where
- 6 sites in Poland
- Volunteers
- 139 people
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.