Clinical trials: Congenital Malformations
Monochorionic Diamniotic Placenta, Monochorial Monoamniotic Placenta
MoDi Twin Placental Imaging
Twin-twin transfusion syndrome (TTTS) can arise in monochorionic, diamniotic (MoDi), those identical twins arising from division of a single fertilized ovum at 4-8 days gestational age (GA) or monochorionic, monoamniotic (MoMo or…
- Conditions
- Monochorionic Diamniotic Placenta, Monochorial Monoamniotic Placenta, Monochorionic Twins, Ultrasound Doppler
- Trial name
- NCT07667647
- Testing
- Verasonics Vantage 256 Ultrasound
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Carilion Clinic
Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease
Fipamezole in Neurogenic Orthostatic Hypotension
The purpose of this study is to determine whether Fipamezole is effective in the treatment of orthostatic hypotension and related symptoms in multiple system atrophy and Parkinson's disease.
- Conditions
- Symptomatic Neurogenic Orthostatic Hypotension (NOH), Parkinson's Disease, Multiple System Atrophy
- Trial name
- Foehn (NCT00758849)
- Testing
- Fipamezole
- Where
- 4 sites in France, Portugal
- Volunteers
- 24 people
- Sponsor
- Juvantia Pharma Ltd
Duchenne Muscular Dystrophy, Performance
Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance
The investigator investigated the effect of lower limb flexibility on functional performance of children with Duchenne Muscular Dystrophy.
- Conditions
- Duchenne Muscular Dystrophy, Performance, Flexibility
- Trial name
- NCT03534349
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Hacettepe University
Hernias, Diaphragmatic, Congenital
Resistance Exercise in Children With Post-operative Congenital Diaphragmatic Hernia
Congenital diaphragmatic hernia (CDH) is a life-threatening condition with long-term complications including respiratory tract infections, respiratory muscle weakness, and abnormal lung functions.
- Trial name
- hernia (NCT04900649)
- Testing
- chest resistance exercise, chest expansion exercises
- Where
- Saudi Arabia
- Volunteers
- 32 people
- Sponsor
- Qassim University
Cystic Fibrosis
A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF…
- Trial name
- NCT03447249
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 101 sites in United States, Australia and 9 more
- Volunteers
- 385 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Atopic Dermatitis
Combination Therapy for Atopic Dermatitis
Atopic dermatitis is a chronic relapsing disease with acute flares. The standard therapy is to treat acute flares using topical medications.
- Trial name
- NCT00119158
- Testing
- Combination of pimecrolimus and fluticasone, pimecrolimus
- Where
- 3 sites in United States
- Volunteers
- 90 people
- Sponsor
- Children's Hospital of Philadelphia
Infant Colic, Postpartum Depression
Measuring and Reducing Excessive Infant Crying
Excessive infant crying (EIC) is likely to increase the risk of child abuse. The investigators propose a randomized trial using an intervention based on recommendations of Karp.
- Trial name
- UTHealth THB (NCT01217658)
- Testing
- The Happiest Baby on The Block, AAP Infant Colic counseling
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The University of Texas Health Science Center, Houston
Hemoglobinopathies
Prevalence and Hematological Characteristics of Hemoglobinopathies
This study aimed to determine the prevalence and hematological characteristics of hemoglobinopathies in Mardan and surrounding districts of Khyber Pakhtunkhwa (KPK), Pakistan.
- Trial name
- NCT07180836
- Testing
- No Intervention (Observational Study)
- Where
- Pakistan
- Volunteers
- 839 people
- Sponsor
- Bacha Khan Medical College
Gout, Hyperuricemia
Post-Authorisation Safety Study of Lesinurad
Non-interventional population-based prospective cohort study in multiple databases comparing patients with gout who initiate lesinurad in combination with an existing xanthine oxidase inhibitor (XOI) (lesinurad+XOI cohort) to a…
- Trial name
- SATURATES (NCT04072471)
- Testing
- Zurampic®, Control group: xanthine oxidase inhibitor monotherapy
- Sponsor
- AstraZeneca
Vitamin D Deficiency, Cystic Fibrosis
PK/PD of Vitamin D3 in Adults With CF
Despite the extensive literature on adverse clinical outcomes associated with vitamin D deficiency, there are currently no proven treatment strategy that effectively achieves and maintains optimal serum vitamin D status in cystic…
- Trial name
- NCT03734744
- Testing
- Vitamin D3
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Southern California
Hemophilia
A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B
This study will assess the pharmacokinetics and pharmacodynamics of rhFVIIa at three dose levels. The results will help identify the most optimal doses to take forward to the Phase 2/3 studies where bleedings in hemophilia…
- Trial name
- rhFVIIa (NCT01708564)
- Testing
- rhFVIIa
- Where
- 3 sites in United States, Netherlands
- Volunteers
- 15 people
- Sponsor
- rEVO Biologics
Tourette Syndrome, Habit Reversal Training
Extinction Learning in Adults With Tourette Syndrome
Aversive sensory phenomena such as premonitory urges play a central role in the behavioral treatment model of tics. Extinction learning and extinction recall are learning processes implicated within this model, but are still…
- Conditions
- Tourette Syndrome, Habit Reversal Training, Tic Disorders, Tics
- Trial name
- NCT04498364
- Testing
- Habit Reversal Training
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Cystic Fibrosis
BioEnergetics and Metabolomics in Cystic Fibrosis
With better medical care, patients with cystic fibrosis (CF), a life-threatening disease, are enjoying longer lives. As the CF life expectancy increases, conditions such as CF-related diabetes (CFRD) become more prevalent.
- Trial name
- BEAM-CF (NCT02225899)
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Emory University
Fontan Procedure
Clinical Outcomes in Fenestrated Extra-Cardiac Fontan in Low Preoperative Risk Profiles
Evaluation of fenestration benefit in Extra-cardiac total cavo-pulmonary connection (Fontan procedure) in single ventricle anomaly, comparing clinical outcomes of patients with low preoperative risk profiles in prospective study
- Trial name
- NCT03503032
- Testing
- Extracardiac Fontan, Fontan fenestration
- Where
- Saudi Arabia
- Volunteers
- 62 people
- Sponsor
- King Faisal Specialist Hospital & Research Center
Cardiac Amyloidosis
Echocardiographic Characterization of Cardiac Amyloidosis
This is a mono-center observational ambispective study in which patients with cardiac amyloidosis evaluated at the Fondazione Policlinico Universitario Agostino Gemelli IRCCS (Rome, Italy) will be enrolled.
- Trial name
- Echo-AMY (NCT05760287)
- Where
- Italy
- Volunteers
- 255 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Gastroschisis
Sutureless vs Sutured Gastroschisis Closure
This study aims to prospectively assess outcomes of sutureless versus sutured gastroschisis closure with a randomized control trial. The parameters of this trial were determined using our retrospective study as pilot data.
- Trial name
- NCT01094587
- Testing
- Gastroschisis closure with suture, Gastroschisis closure without suture
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Stanford University
Asthma, Allergic Rhinitis
Immunologic Basis of Anti-IgE Therapy (Study II: On Patients With Asthma)
The purpose of this study is to look at measures that will help scientists understand the way Omalizumab, an FDA-approved anti-allergy medication, works.
- Conditions
- Asthma, Allergic Rhinitis, Atopic Dermatitis
- Trial name
- Xolair (NCT00367016)
- Testing
- Omalizumab
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of California, Davis
Type II Gaucher Disease
An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
This exploratory trial is to prove the tolerability and safety of VGN-R08b to treat infants with type II Gaucher disease.
- Trial name
- NCT06272149
- Testing
- VGN-R08b
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Primary Ciliary Dyskinesia, Kartagener Syndrome
Genetics of Primary Ciliary Dyskinesia
This study is designed to study DNA sequencings for mutations in a research genetic test panel of genes (which contains all 32 known and/or published genes associated with PCD).
- Trial name
- NCT02389049
- Where
- 8 sites in United States, Canada
- Volunteers
- 320 people
- Sponsor
- University of North Carolina, Chapel Hill
Congenital Factor VII Deficiency
A Clinical Study to Assess Sutacimig in Participants With Congenital Factor VII Deficiency
Open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of a single dose of sutacimig monotherapy in participants with congenital FVII deficiency (FVIID).
- Trial name
- NCT07347249
- Testing
- Sutacimig
- Where
- United Kingdom
- Volunteers
- 18 people
- Sponsor
- Hemab ApS