Clinical trials: Congenital Diseases
Port-Wine Stain
Pathogenic Mechanisms of Port Wine Stain and Repository of Port Wine Stain Biopsy Samples
The purpose of this study is to investigate the pathogenic mechanisms of Port Wine Stain, collect biopsy samples and blood samples to characterize exosomes and metabolites from Port Wine Stain.
- Trial name
- NCT02051101
- Testing
- Biopsy sample from Port Wine Stain Birthmark
- Where
- United States
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- University of California, Irvine
Grown-up Congenital Heart Disease (GUCH)
Effect of Short-term Oxygen During CPET in GUCH
In a randomized, sham-controlled crossover trial the investigators will test whether supplemental oxygen given during cardiopulmonary exercise testing will improve exercise performance and physiological parameters in patients…
- Trial name
- NCT04076501
- Testing
- Oxygen application
- Where
- Switzerland
- Volunteers
- 7 people
- Sponsor
- University of Zurich
Preeclampsia, Preterm Birth
Biobank on Prematurity, Preeclampsia and Other Pregnancy Complications
The Biobank includes data and biological specimens of women from three original studies: 1) First-trimester Prediction of Preeclampsia (PREDICTION Study, NCT02189148), 2) Pre-Eclampsia And growth Retardation, an evaluative…
- Conditions
- Preeclampsia, Preterm Birth, Pregnancy Complications, Fetal Anomalies
- Trial name
- NCT02744365
- Testing
- Observational
- Where
- Canada
- Volunteers
- 7,845 people, healthy volunteers welcome
- Sponsor
- CHU de Quebec-Universite Laval
Granulomatous Disease, Chronic, Job's Syndrome
Posaconazole to Treat Invasive Fungal Infections
This study will evaluate the safety and effectiveness of posaconazole for treating invasive fungal infections. New therapies for these infections are needed for patients who do not respond, to or cannot tolerate, standard…
- Conditions
- Granulomatous Disease, Chronic, Job's Syndrome, Infection
- Trial name
- NCT00033982
- Testing
- Posaconazole
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Pseudoxanthoma Elasticum
Phenotypic Expressions in a French Pseudoxanthoma-Elasticum Cohort
The cohort is intended to study the phenotypic expressions of the pseudoxanthoma elasticum (PXE) disease in various tissues (eye, skin, arteries, etc).
- Trial name
- NCT01446380
- Where
- France
- Volunteers
- 350 people
- Sponsor
- University Hospital, Angers
Brugada Syndrome
Right Ventricle Morphology and Hemodynamics in BrS
The study purpose is to evaluate the morphological, functional and electrophysiological characteristics of the right ventricle before and after ajmaline in patients diagnosed with Brugada syndrome as well as to correlate CMR…
- Trial name
- RV-BrS (NCT03524079)
- Testing
- Ajmaline 17-(Chloroacetate) Monohydrochloride
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- IRCCS Policlinico S. Donato
Osteogenesis Imperfecta (OI)
A Dose REgimen-Finding Study of AGA2115 in Chinese Patients With Osteogenesis ImpeRfecta (EIR)
This study is to evaluate the safety and efficacy of AGA2115 at three different dose regimens in Chinese adults and adolescents with Type I, III, or IV Osteogenesis imperfecta (OI).
- Trial name
- EIR (NCT07557446)
- Testing
- AGA2115
- Where
- 5 sites in China
- Volunteers
- 48 people
- Sponsor
- Angitia Biopharmaceuticals Guangzhou Limited
Eisenmenger Syndrome
Transcatheter Valve Implantation in Patients With Dysfunctional Left and Right Sided Heart Valves
A growing body of data, suggests that transcatheter valves are effective when implanted in other locations (mitral, aortic, tricuspid) and in high pressure environments.
- Trial name
- NCT02119442
- Testing
- Transcather Valve Implantation
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- University of California, Los Angeles
Turner Syndrome
Social Cognition and Turner Syndrome
Monocentric multidisciplinary study (psychologists, endocrinologists, psychiatrists, and molecular biologists) to characterize social cognition in adolescents with Turner syndrome (TS).
- Trial name
- COGNITUR (NCT01687842)
- Testing
- Evaluation of 45,X Turner syndrome patients
- Where
- France
- Volunteers
- 25 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cystic Fibrosis
CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)
This is a Phase 2 study with primary objective of looking whether YPT-01 phage therapy reduces sputum bacterial load in cystic fibrosis subjects with Pseudomonas aeruginosa.
- Trial name
- NCT04684641
- Testing
- Standard Dose YPT-01
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
Atopic Dermatitis
Long-term Safety and Efficacy Study of Lebrikizumab (LY3650150) in Participants With Moderate-to-Severe Atopic Dermatitis (ADjoin)
This is designed to assess the long-term safety and efficacy of lebrikizumab for moderate-to-severe atopic dermatitis. It will last up to 33 months.
- Trial name
- ADjoin (NCT04392154)
- Testing
- Lebrikizumab
- Where
- 321 sites in United States, Australia and 14 more
- Volunteers
- 1,153 people
- Sponsor
- Eli Lilly and Company
Hemophilia B
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05630651
- Testing
- ZS801
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Alpha1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
- Trial name
- NCT04474197
- Testing
- VX-864
- Where
- 30 sites in United States, Canada and 4 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
AV Fistula
Tissue Markers Predictive of Early Stenosis in the Arterovenous Fistula for Hemodialysis
The study involves the recruitment of two populations. POPULATION 1 (surgical sample of excess venous segment): at least 75 consecutive patients, enrolled according to the inclusion criteria and subjected to first AVF surgery as…
- Trial name
- FAV-2019 (NCT06815159)
- Testing
- Identification any tissue markers predictive or favoring early stenosis in AVF for haemodialysis.
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Atopic Dermatitis
A Study to Evaluate the Safety and Efficacy of AK120 in Subjects With Moderate to Severe Atopic Dermatitis
This is a multicenter, randomized, open label phase II clinical study to evaluate the safety and efficacy of AK120 in the treatment of subjects with moderate to severe atopic dermatitis.
- Trial name
- NCT06092762
- Testing
- AK120
- Where
- 26 sites in China
- Volunteers
- 180 people
- Sponsor
- Akeso
Primary Ciliary Dyskinesia, Healthy
Examining Bronchial Hyperresponsiveness in Primary Ciliary Dyskinesia
The purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness".
- Trial name
- BHR (NCT07288827)
- Testing
- Lung Function Testing, Phlebotomy, Allergy skin prick testing
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Indiana University
Colic, Nephropathy
Neonatal Outcome of Children With Antenatal Colic Hyperechogenicity
Fetal colic hyper-echogenicity is a presenting symptoms for cystinuria lysinuria. A few cases of fetuses with dibasic protein intolerance (more complex prognosis) presented with colic hyper-echogenicity antenatal.
- Trial name
- COLONECHOGENE (NCT04983069)
- Where
- France
- Volunteers
- 400 people
- Sponsor
- University Hospital, Montpellier
Retinitis Pigmentosa (RP)
Investigation of Therapeutic Efficacy and Safety of UMSCs for the Management of Retinitis Pigmentosa (RP)
Retinitis pigmentosa (RP) is the most common hereditary retinal disorder (accounts for 20% of children attending blind schools in Pakistan) which causes degeneration of rod and cone photoreceptors.
- Trial name
- NCT04763369
- Testing
- Injection of stem cells in sub-tenon space of eye for the management of retinitis pigmentosa, Injection of stem cells in suprachoroidal space of eye for the management of Retinitis Pigmentosa
- Where
- Pakistan
- Volunteers
- 50 people
- Sponsor
- Jinnah Burn and Reconstructive Surgery Centre, Lahore
Lactose Intolerance
Effects Comparison of A1 and A2 Milk on Gastrointestinal Physiology, Symptoms and Cognitive Behavior
This is a single-site, double-blinded, randomized, controlled, 2X2 cross-over study aiming to compare effects of milk containing only A2 type beta casein versus milk containing both A1 and A2 beta casein proteins on the…
- Trial name
- NCT02406469
- Testing
- Oral consumption milk Tetra Pak
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- a2 Milk Company Ltd.
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
To determine the clinical benefit rate of selumetinib in combination with sirolimus in patients with unresectable or metastatic neurofibromatosis type 1 (NF1) associated or sporadic MPNST.
- Trial name
- NCT03433183
- Testing
- Selumetinib, Sirolimus
- Where
- 5 sites in United States
- Volunteers
- 21 people
- Sponsor
- Sarcoma Alliance for Research through Collaboration
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