Clinical trials: Defects
Hypoplastic Left Heart Syndrome, Single Ventricle
Cardiac Stem/Progenitor Cell Infusion in Univentricular Physiology (APOLLON Trial)
The purpose of this study is to evaluate the efficacy and safety of intracoronary injection of JRM-001 after reconstructive surgery in pediatric patients with functional single ventricle
- Trial name
- APOLLON (NCT02781922)
- Testing
- Autologous cardiac stem cells (JRM-001)
- Where
- 4 sites in Japan
- Volunteers
- 40 people
- Sponsor
- Metcela Inc.
Vanishing Twin, Trisomy 21
Verification of Risk Assignment for Whole Chromosome Using SNP-based NIPT in Vanishing Twin Pregnancies
The purpose of this study is to collect blood samples from women carrying a vanishing twin pregnancy to further develop Natera's non-invasive prenatal screening test to provide information about possible chromosomal conditions…
- Conditions
- Vanishing Twin, Trisomy 21, Trisomy 13, Trisomy 18
- Trial name
- VANISH (NCT05004337)
- Where
- 21 sites in United States
- Volunteers
- 136 people
- Sponsor
- Natera, Inc.
Corneal Epithelium Defect
A Phase II Clinical Trial to Evaluate the Efficacy and Safety of ZKY001 Eye Drops
A multicenter, randomized, double-blind, placebo-controlled clinical trial was designed. A total of 120 subjects were randomly assigned to two experimental groups (lowe-dose ZKY001 eye drops group,Medium-dose ZKY001 eye drops…
- Trial name
- TPRK (NCT06209203)
- Testing
- lowe-dose ZKY001 eye drops, Medium-dose ZKY001 eye drops
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Zhaoke (Guangzhou) Ophthalmology Pharmaceutical Ltd.
Bone Void in the Maxillofacial Area
Filling Bone Defects/Voids With Autologous BonoFill For Maxillofacial Bone Regeneration
Phase I/II open label first in human single center clinical study, is to evaluate the safety and the efficacy of BonoFill as bone filler containing the patient own (autologous) adipose tissue derived cells (HATDCs) in…
- Trial name
- NCT02153268
- Testing
- Single Arm - Liposuction procedure and BonoFill transplantation will be done for all subjects on study
- Where
- Israel
- Volunteers
- 11 people
- Sponsor
- BonusBio Group Ltd
Microdeletion Syndromes, Trisomy 21
Development of Non-invasive Prenatal Test for Microdeletion and Other Genetic Syndromes Based on Cell Free DNA
The purpose of this study is to collect blood from families with a child who has been diagnosed with a chromosomal disorder including microdeletions in order to further develop a non-invasive prenatal screening test based on…
- Conditions
- Microdeletion Syndromes, Trisomy 21, Trisomy 18, Trisomy 13
- Trial name
- Microdel Triad (NCT02109770)
- Where
- 2 sites in United States
- Volunteers
- 216 people
- Sponsor
- Natera, Inc.
Articular Cartilage Defect, Articular Cartilage Degeneration
Phase 2 Clinical Trial of CartiLife® in the United States
To evaluate the safety and efficacy of implanting pellet-type extracellular matrix-associated autologous chondrocytes (CartiLife®) obtained by cultivating costal chondrocytes of the subject with articular cartilage defects of the…
- Trial name
- NCT04744402
- Testing
- Autologous Chondrocyte Implantation (CartiLife®)
- Where
- 5 sites in United States
- Volunteers
- 25 people
- Sponsor
- Biosolution Co., Ltd.
Epilepsy
Birth Defects Associated With Exposure to Lamotrigine in Pregnancy (EUROCAT)
In 2007 a case control study was completed within the EUROCAT European network of congenital anomaly registers to test the hypothesis that first trimester lamotrigine monotherapy exposure is associated with an increased risk of…
- Trial name
- NCT01055327
- Testing
- Lamotrigine monotherapy, No anti-epileptic drug exposure, Non lamotrigine anti-epileptic drug monotherapy
- Volunteers
- 1 people
- Sponsor
- GlaxoSmithKline
Myocardial Ischemia, Heart Defects, Congenital
Spectroscopic Assessment of Myocardial Oxygenation Changes in Pediatric Heart Surgery
The goal of this observational study is to investigate the ability of the SpectroCor Tissue Oxygenation Monitor and its accessory SpectroCor Sensor to observe and monitor changes in cardiac oxygen availability in pediatric…
- Conditions
- Myocardial Ischemia, Heart Defects, Congenital, Reperfusion Injury, Myocardial
- Trial name
- NCT07785141
- Where
- Finland
- Volunteers
- 30 people
- Sponsor
- Spectrocor
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Chondral Defect, Osteochondritis Dissecans (OCD)
A Study of MACI in Patients Aged 10 to 17 Years With Symptomatic Chondral or Osteochondral Defects of the Knee
The objective of this study is to compare the efficacy and safety of MACI® vs arthroscopic microfracture in the treatment of participants aged 10 to 17 years with symptomatic articular chondral or osteochondral defects of the…
- Conditions
- Chondral Defect, Osteochondritis Dissecans (OCD), Articular Cartilage Defect, Articular Cartilage Disorder of Knee
- Trial name
- PEAK (NCT03588975)
- Testing
- MACI, microfracture
- Where
- 15 sites in United States
- Volunteers
- 45 people
- Sponsor
- Vericel Corporation
Turner's Syndrome
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years.
- Trial name
- NCT01066052
- Testing
- r-hGH
- Where
- France
- Volunteers
- 115 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
Long QT Syndrome
Effect of Oral GS-6615 on Dofetilide-Induced QT Prolongation, Safety, and Tolerability in Healthy Adults
This study will evaluate the effect of oral GS-6615 on QTc interval in healthy adults with dofetilide-induced QTc prolongation.
- Trial name
- NCT02365532
- Testing
- GS-6615, Dofetilide
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Ductus Arteriosus, Patent
NIT-OCCLUD PDA Phase II Sentinel Trial
The purpose of this study is to evaluate the safety and efficacy of the Nit-Occlud -PDA Occlusion System for transcatheter closure of PDA with minimum angiographic diameter less than 4 mm, and to compare one year safety and…
- Trial name
- NCT00828334
- Testing
- Transcatheter PDA Coil
- Where
- 15 sites in United States
- Volunteers
- 357 people
- Sponsor
- PFM Medical, Inc
Atrial Septal Defect
Lifetech CeraFlex™ ASD Closure System Post-Market Clinical Follow-Up
The objective of this post-market registry is to assess the clinical use of the Lifetech CeraFlex™ Closure System in a real-world and on-label fashion.
- Trial name
- NCT05887700
- Testing
- CeraFlex™ Atrial Septal Defect (ASD) Closure System
- Where
- 10 sites in Brazil, Greece and 1 more
- Volunteers
- 145 people
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.
Coagulation Defects
Safety Study of Spray-Dried Solvent/Detergent-Treated Plasma for Infusion in Healthy Volunteers
This study is a Phase 1, single-center, open-label, ascending dose study which will assess the safety and tolerability of 5 doses of spray-dried solvent/detergent treated plasma (Resusix®) in healthy volunteers.
- Trial name
- NCT01589666
- Testing
- Spray-dried S/D-treated Plasma
- Where
- United States
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- Entegrion, Inc.
Heart Diseases, Pulmonary Valve Regurgitation
PULSTA Transcatheter Pulmonary Valve Pre-Approval Study
The purpose of the study is to evaluate the safety and effectiveness of the PULSTA Transcatheter Pulmonary Valve (TPV) System for the treatment of congenital or acquired pulmonary valve stenosis and/or regurgitation who require…
- Conditions
- Heart Diseases, Pulmonary Valve Regurgitation, Pulmonary Valve Stenosis, Tetralogy of Fallot
- Trial name
- NCT03983512
- Testing
- PULSTA TPV System
- Where
- 11 sites in Germany, Italy and 4 more
- Volunteers
- 58 people
- Sponsor
- Taewoong Medical Co., Ltd.
Volume Defects in the Mid-face
To Evaluate the Effectiveness and Safety of YVOIRE Y-Solution 720 Injected Into the Mid-Face
To Evaluate the Effectiveness and Safety of YVOIRE Y-Solution 720 Injected Into the Mid-Face
- Trial name
- NCT04437745
- Testing
- YVOIRE Y-Solution 720
- Where
- China
- Volunteers
- 238 people, healthy volunteers welcome
- Sponsor
- LG Chem
X-Linked Retinitis Pigmentosa
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
- Trial name
- NCT04312672
- Testing
- AAV5-hRKp.RPGR
- Where
- 5 sites in United States, United Kingdom
- Volunteers
- 42 people
- Sponsor
- MeiraGTx Ocular UK Ltd
Pregnancy, Aneuploidy
Noninvasive Screening for Fetal Aneuploidy: A New Maternal Plasma Marker
The purpose of this study is to collect samples for the purpose of developing a prenatal aneuploid test using circulating cell free fetal (ccff) nucleic acid from blood samples from pregnant women who have a high-risk pregnancy…
- Conditions
- Pregnancy, Aneuploidy, Down Syndrome, Edwards Syndrome
- Trial name
- NCT00971334
- Where
- 35 sites in United States, France
- Volunteers
- 2,502 people
- Sponsor
- Sequenom, Inc.
Down Syndrome (Trisomy 21), Edwards Syndrome (Trisomy 18)
Non-Invasive Screening for Fetal Aneuploidy: A New Maternal Plasma Marker
Validate that circulating cell free fetal nucleic acid can be used to identify a direct marker for fetal aneuploidy, particularly fetal Down Syndrome (DS), that is better than surrogate markers.
- Conditions
- Down Syndrome (Trisomy 21), Edwards Syndrome (Trisomy 18), Patau Syndrome (Trisomy 13), Turner Syndrome
- Trial name
- NCT00770458
- Where
- 7 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Sequenom, Inc.