Clinical trials: Adult Congenital Heart Disease
Solid Tumors
Dose Escalation of RMC-4630 Monotherapy in Relapsed/Refractory Solid Tumors
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) profiles of escalating doses of RMC-4630 monotherapy in adult participants with relapsed/refractory solid tumors…
- Trial name
- NCT03634982
- Testing
- RMC-4630
- Where
- 12 sites in United States
- Volunteers
- 133 people
- Sponsor
- Revolution Medicines, Inc.
Exocrine Pancreatic Insuficiency
Determine the Prevalence of Exocrine Pancreatic Insufficiency (EPI) in Pediatric and Adult Participants With Alagille Syndrome After Liver Transplantation
The goal of this clinical trial is to determine the prevalence of exocrine pancreatic insufficiency (EPI) in a population of pediatric and adult participants with Alagille Syndrome (ALGS) post liver transplant.
- Trial name
- NCT07335523
- Testing
- Fecal Fat
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Digestive Care, Inc.
Arrhythmogenic Right Ventricular Cardiomyopathy
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.
- Trial name
- RIDGE-1 (NCT06228924)
- Testing
- TN-401
- Where
- 7 sites in United States
- Volunteers
- 15 people
- Sponsor
- Tenaya Therapeutics
Pulmonary Arterial Hypertension
Adult Congenital Heart Disease Registry (QuERI)
Multi-center, observational, U.S.-based longitudinal program. Data will be collected prospectively for 3 years. Individual physician feedback will be provided on data collected with the purpose of improving the management of…
- Trial name
- NCT01659411
- Testing
- Observational
- Volunteers
- 217 people
- Sponsor
- Actelion
Non-small Cell Lung Cancer, Colorectal Cancer
A Study of JAB-3312 in Adult Patients With Advanced Solid Tumors in China
This is a Phase 1, open-label dose-escalation study to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) and assess the DLT of JAB-3312.
- Conditions
- Non-small Cell Lung Cancer, Colorectal Cancer, Pancreatic Ductal Carcinoma, Esophageal Squamous Cell Carcinoma
- Trial name
- NCT04121286
- Testing
- JAB-3312
- Where
- 5 sites in China
- Volunteers
- 17 people
- Sponsor
- Allist Pharmaceuticals, Inc.
Dilated Cardiomyopathy
A Study to Investigate Safety, Tolerability, and Pharmacokinetics of Oral AZD0233 Compared With Placebo in Healthy Adult Participants.
The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of AZD0233 following single and multiple ascending dose (SAD and MAD) administration in healthy participants.
- Trial name
- NCT06381466
- Testing
- AZD0233
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Primary Ciliary Dyskinesia
A Study to Assess the Safety, Tolerability, Ciliary Rescue, and Pharmacodynamics of RCT1100 in Adults With PCD
This is the second in-human study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
- Trial name
- NCT06600425
- Testing
- RCT1100
- Where
- 2 sites in United Kingdom
- Volunteers
- 7 people
- Sponsor
- ReCode Therapeutics
Fabry Disease
4D-310 in Adults With Fabry Disease and Cardiac Involvement
This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration.
- Trial name
- NCT05629559
- Testing
- 4D-310
- Where
- 4 sites in Australia, Taiwan
- Volunteers
- 18 people
- Sponsor
- 4D Molecular Therapeutics
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.
Primary Ciliary Dyskinesia
A Longitudinal, Observational Study of Primary Ciliary Dyskinesia in Adults
The goal of this observational study is to characterize clinical measures and biomarkers of airway disease in adults with primary ciliary dyskinesia (PCD) and in a group of healthy volunteers (HV) to establish normative values.
- Trial name
- NCT05685186
- Testing
- Spirometry, Multiple Breath Washout (MBW), Mucociliary Clearance (MCC)
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- ReCode Therapeutics
Long QT Syndrome Type 3
Effect of Eleclazine on Shortening of the QT Interval, Safety, and Tolerability in Adults With Long QT Syndrome Type 3
The primary objective of this study is to evaluate the effect of oral eleclazine on mean daytime QTcF interval after 24 weeks of treatment with elecalzine in participants with long QT syndrome Type 3.
- Trial name
- NCT02300558
- Testing
- Eleclazine
- Where
- 12 sites in United States, Canada and 6 more
- Volunteers
- 41 people
- Sponsor
- Gilead Sciences
Congenital Heart Disease
TORCH-LATAM: A Regional Pathway to Improve Transplant Evaluation for Congenital Heart Disease
Congenital heart disease can progress to advanced cardiac or cardiopulmonary failure requiring heart, heart-lung, or, in selected congenital conditions, bilateral lung transplantation with cardiac repair.
- Trial name
- TORCH-LATAM (NCT07799272)
- Testing
- TORCH Integrated Transplant Access Pathway (TORCH-ITAP)
- Volunteers
- 600 people
- Sponsor
- CardioVision International Tech, S.A.S. de C.V.
Non-small Cell Lung Cancer, Head and Neck Cancer
A First-in-Human Study of JAB-3068 (SHP2 Inhibitor) in Adult Patients With Advanced Solid Tumors in China
This is a Phase 1/2a, open-label, multi-center study of JAB-3068 in Patients with advanced solid tumors.This study has two phases: dose escalation phase and dose expansion phase.
- Conditions
- Non-small Cell Lung Cancer, Head and Neck Cancer, Esophageal Cancer, Other Metastatic Solid Tumors
- Trial name
- NCT03565003
- Testing
- JAB-3068
- Where
- 4 sites in China
- Volunteers
- 126 people
- Sponsor
- Jacobio Pharmaceuticals Co., Ltd.
Dental Caries
Clinical Performance of Incremental and Bulk Fill Composites in Class II Restorations
This study will examine the clinical performance of Class II restorations over a three year period with 3 composite resins - a conventional composite resin, a flowable composite resin and a bulk placed and cured composite resin.
- Trial name
- NCT02889835
- Testing
- Supreme Universal Restorative, Supreme Flowable Restorative, Bulk Fill Flowable Restorative
- Where
- United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Solventum US LLC
Friedreich Ataxia, Cardiomyopathies
A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ATI-105 in Healthy Adults
The goal of this clinical trial is to learn how ATI-105 administered to healthy participants at works in the body at different doses and with or without food.
- Conditions
- Friedreich Ataxia, Cardiomyopathies, Central Nervous System Diseases, Neuromuscular Diseases
- Trial name
- NCT07869654
- Testing
- ATI-105
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Adjuvia Therapeutics, Inc.
Primary Ciliary Dyskinesia (PCD)
Study of Inhaled RCT1100 in Adults With PCD Caused by Pathogenic Mutations in the DNAI1 Gene to Measure Mucociliary Clearance
This is a multi-dose study with RCT1100 and is designed to provide safety, tolerability and preliminary efficacy data for future clinical studies.
- Trial name
- NCT06633757
- Testing
- RCT1100
- Where
- 3 sites in United States, Denmark and 1 more
- Volunteers
- 14 people
- Sponsor
- ReCode Therapeutics
Dilated Cardiomyopathy (DCM), BAG3 Mutation Associated Dilated Cardiomyopathy
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
This is a Phase 1/2, open-label, dose-exploration and dose-expansion, clinical trial evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of a single intravenous infusion of AFTX-201 in adults with…
- Trial name
- UPBEAT (NCT07426419)
- Testing
- AFTX-201
- Where
- 5 sites in United States, Canada
- Volunteers
- 22 people
- Sponsor
- Affinia Therapeutics
Solid Tumor, Unspecified, Adult
RLY-1971 in Subjects With Advanced or Metastatic Solid Tumors
This study is a multi-center, open-label, dose escalation and expansion study of RLY-1971 in subjects with advanced or metastatic solid tumors.
- Trial name
- NCT04252339
- Testing
- RLY-1971
- Where
- 6 sites in United States
- Volunteers
- 56 people
- Sponsor
- Hoffmann-La Roche
Dilated Cardiomyopathy
A Single Ascending Dose Study Assessing the Safety, Tolerability, PK and PD of MYK-491
Up to 72 healthy volunteers will be given a single dose of MYK-491 or placebo and be monitored for safety and tolerability over a 7 day period.
- Trial name
- NCT03062956
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- MyoKardia, Inc.
Dilated Cardiomyopathy
A Study of AZD4063 in PLN R14del Dilated Cardiomyopathy
The purpose of the study is to assess the safety, tolerability and the pharmacokinetics (PK) of AZD4063 after single dose administration in participants with phospholamban (PLN) R14del dilated cardiomyopathy.
- Trial name
- PULSE (NCT07241104)
- Testing
- AZD4063
- Where
- 4 sites in Netherlands
- Volunteers
- 23 people
- Sponsor
- AstraZeneca