Clinical trials: Fetal Developmental Abnormality
Turner's Syndrome
An Open-Label, Multi-centre, Phase III Study of Local Tolerability of ZOMACTON 10MG
Children with growth failure due to inadequate secretion of growth hormone or growth retardation due to Turner's syndrome are individually dosed and treated for 12 weeks
- Trial name
- NCT00250250
- Testing
- ZOMACTON
- Where
- 6 sites in Czechia, France and 1 more
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- Ferring Pharmaceuticals
CHD - Congenital Heart Disease
Home-based Cardiovascular Rehabilitation in Young Patients With Congenital Heart Disease: the "Muscle Your Heart" Program
Standard cardiovascular rehabilitation is performed in a medical care and rehabilitation centre. However, it may not be adapted to the patient's profile and there are few medical care and rehabilitation centres specialized in…
- Trial name
- Muscletoncoeur (NCT07115589)
- Testing
- Muscle Your Heart home-based program
- Where
- France
- Volunteers
- 21 people
- Sponsor
- University Hospital, Bordeaux
Usher Syndromes
Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.
- Trial name
- SLO RP (NCT04355689)
- Testing
- NPI-001
- Where
- 4 sites in Australia
- Volunteers
- 49 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Funnel Chest
Cardiorespiratory Assessment and Quality of Life After Surgical Correction of Congenital Wall Malformations
The main objective of this study is to characterize the cardiorespiratory functional gain in patients with pectus excavatum following corrective surgery.
- Trial name
- HeartSoar (NCT03770390)
- Testing
- Surgical correction of pectus excavatum
- Where
- France
- Volunteers
- 43 people
- Sponsor
- University Hospital, Montpellier
Ehlers-Danlos Syndrome
Origins and Impact of EDS in Connective Tissues and Skin
Ehlers-Danlos Syndrome (EDS) is an inherited disease of collagen, found in connective tissues, such as skin. EDS patients suffer from joint and skin problems (skin hyperextensibility, joint hypermobility) along with a large range…
- Trial name
- NCT02721797
- Testing
- Orthopaedic & Gynaecology surgery
- Where
- United Kingdom
- Volunteers
- 35 people
- Sponsor
- University College, London
Cystic Fibrosis
Clinical Outcomes of Triple Combination Therapy in Severe Cystic Fibrosis Disease.
This is an observational study of people with severe cystic fibrosis that are eligible for Vertex's triple combination therapy through its expanded access.
- Trial name
- NCT04038710
- Testing
- Triple combination therapy
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Jewish Health
Sickle Cell Disease
Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
- Trial name
- GBT_HOPE (NCT03036813)
- Testing
- voxelotor
- Where
- 62 sites in United States, Canada and 10 more
- Volunteers
- 449 people
- Sponsor
- Pfizer
Congenital Heart Disease, Pulmonary Hypertension
Early Postoperative Triple Targeted Therapy in Patients With CHD-PAH in the "Grey Zone"
The purpose of this study is to observe the efficacy of early application of triple targeted drug therapy, including trilostane, in patients with congenital heart disease who are in the "gray zone" of preoperative evaluation…
- Trial name
- NCT07844213
- Testing
- Control group (ambrisentan plus tadalafil or macitentan plus tadalafil), Treatment group (ambrisentan plus tadalafil plus treprostinil or macitentan plus tadalafil plus treprostinil)
- Where
- China
- Volunteers
- 30 people
- Sponsor
- China National Center for Cardiovascular Diseases
Fed
Bioavailability Study of Desloratadine 5 mg Tablet of Dr. Reddy's Under Fed Conditions
The purpose of this study is to compare the rate and extent of absorption of Dr. Reddy's Desloratadine 5 mg tablet to that of Clarinex® 5 mg tablet in healthy subjects under fed conditions.
- Trial name
- NCT01314352
- Testing
- Desloratadine
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Hepatitis C, Fibrosis
The Prevalence and Severity of HCV Infection in Thalassemia Major and Thalassemia Intermedia in Siriraj Hospital
The primary objective is to assess the prevalence and genotypes of HCV infection in thalassemia major and thalassemia intermediate patients who have received blood transfusion in the Department of Medicine, Faculty of Medicine…
- Conditions
- Hepatitis C, Fibrosis, Thalassemia
- Trial name
- NCT02904252
- Testing
- Transient elastography
- Where
- Thailand
- Volunteers
- 158 people
- Sponsor
- Mahidol University
Sickle Cell Disease
Hybrid Effectiveness-Implementation Trial of Guided Relaxation and Acupuncture for Chronic Sickle Cell Disease Pain
The investigators will conduct a hybrid type 1 effectiveness implementation trial to assess the effectiveness of acupuncture and guided relaxation on 360 people with Sickle Cell Disease (SCD), while observing and gathering…
- Trial name
- GRACE (NCT04906447)
- Testing
- Guided Relaxation, Acupuncture
- Where
- 5 sites in United States
- Volunteers
- 379 people, healthy volunteers welcome
- Sponsor
- University of Illinois Chicago
Acute Respiratory Distress Syndrome ARDS
Driving Pressure-guided Tidal Volume Ventilation in the Acute Respiratory Distress Syndrome
Acute respiratory distress syndrome (ARDS) is associated with high mortality, some of which can be attributed to ventilator-induced lung injury (VILI) when artificial ventilation is not customized to the severity of lung injury.
- Trial name
- DRIVENT (NCT06322758)
- Testing
- Tidal volume customization in the acute respiratory distress syndrome
- Volunteers
- 750 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Atopic Dermatitis
A Study to Assess the Efficacy and Safety of CMK389 in Patients With Moderate to Severe Atopic Dermatitis.
The main purpose of this phase 2 study was to assess the efficacy and safety of CMK389 in patients with atopic dermatitis.
- Trial name
- NCT04836858
- Testing
- CMK389
- Where
- 18 sites in Czechia, France and 4 more
- Volunteers
- 71 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Heart Disease (CHD)
BOND: Direct Breastfeeding to Enhance Maternal and Infant Health in Congenital Heart Disease
The primary objective of this study is to enhance the rates of direct breast feeding (DBF) among infants with congenital heart disease (CHD), and to gain insights into the implications of DBF on key metrics of child and parent…
- Trial name
- NCT07582848
- Testing
- Breastfeeding clinical practice guideline (CPG) for congenital heart disease (CHD)
- Where
- 17 sites in United States
- Volunteers
- 266 people
- Sponsor
- Carelon Research
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Retinal Hemangioblastoma, Von Hippel-Lindau Disease
Retrospective Case Series of Trans-scleral Cryotherapy for Retinal Hemangioblastoma
Background: Retinal hemangioblastoma (RH) is a tumor. It grows from the retina in the eye. It can threaten a person s vision. Trans-scleral cryotherapy is used to destroy the tumors and minimize the long-term risks of vision…
- Trial name
- NCT04458935
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Eye Institute (NEI)
Malformation Brain
The Role of Ultrasonography in Pregnancy in the Study of Fetal Central Nervous System Malformations
The aim of this observational study is to evaluate the correlation between new ultrasound patterns and fetal central nervous system (CNS) malformations.
- Trial name
- CNS_2021 (NCT06788808)
- Testing
- Comparison between groups
- Where
- Italy
- Volunteers
- 80 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Eisenmenger Syndrome, Congenital Heart Disease
Sleep-disordered Breathing in Eisenmenger Syndrome
Sleep-disordered breathing (SDB) is a wellknown comorbidity in cardiovascular disease. Knowledge about SDB in adult congenital heart disease is limited.
- Conditions
- Eisenmenger Syndrome, Congenital Heart Disease, Sleep-disordered Breathing
- Trial name
- NCT02614417
- Testing
- Polysomnography
- Volunteers
- 20 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
- Trial name
- REALIZE (NCT01524289)
- Testing
- Anacetrapib
- Volunteers
- 306 people
- Sponsor
- Merck Sharp & Dohme LLC
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen