Clinical trials: Fetal Developmental Abnormality
HIV Infection
HIV and Fat Accumulation
This is a research study to see whether fat accumulation either under the skin or in the body's organs, for example, the liver, improves in men and women who take a drug called telmisartan.
- Trial name
- MATH (NCT01088295)
- Testing
- Telmisartan
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- University of California, Los Angeles
Cystic Fibrosis
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
The study evaluates the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF participants 2 to less than (<) 24 months of age.
- Trial name
- NCT05882357
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 19 sites in Australia, Canada and 5 more
- Volunteers
- 67 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Tumors With Aberrations in ALK, Anaplastic Large Cell Lymphoma
Ceritinib Rare Indications Study in ALK+ Tumors
This is Proof-of-Concept (POC) study to assess the preliminary antitumor activity and safety and tolerablity using ceritinib (LDK378) in the treatment of life threatening tumors that are characterized by ALK genetic alteration…
- Conditions
- Tumors With Aberrations in ALK, Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumor, Glioblastoma
- Trial name
- NCT02465528
- Testing
- Ceritinib (LDK378)
- Where
- 14 sites in Czechia, Denmark and 6 more
- Volunteers
- 22 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease & Thalassemia
Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
- Trial name
- NCT01590628
- Testing
- NiCord
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Gamida Cell ltd
Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 Years
This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.
- Trial name
- EARLY (NCT01082367)
- Testing
- TOBI
- Where
- 19 sites in Canada, Egypt and 8 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the…
- Trial name
- NCT02709109
- Testing
- VX-371 + HS, Hypertonic saline, Orkambi
- Where
- 40 sites in United States, France and 2 more
- Volunteers
- 147 people
- Sponsor
- Parion Sciences
Chronic Iron Overload Due to Transfusion-dependant Anemias
Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance
- Trial name
- NCT02125877
- Testing
- Deferasirox dispersible tablet, Defearisox film-coated tablet
- Where
- 51 sites in United States, Argentina and 14 more
- Volunteers
- 173 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
This study is a multiple within participant crossover study to evaluate the effect of ivacaftor on lung function in participants aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of…
- Trial name
- NCT01685801
- Testing
- Ivacaftor
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Atopic Dermatitis, Atopic Disorders
A Community-based Assessment of Skin Care, Allergies, and Eczema
Atopic dermatitis (AD) affects over 9 million children in the U.S. and often heralds the development of asthma, food allergy, skin infections and neurodevelopmental disorders.
- Conditions
- Atopic Dermatitis, Atopic Disorders, Eczema, Atopic Eczema
- Trial name
- CASCADE (NCT03409367)
- Testing
- Participant choice of over-the-counter emollients: Vaseline, Vanicream, CeraVe Healing Ointment, CeraVe cream, Cetaphil cream
- Where
- 4 sites in United States
- Volunteers
- 1,260 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Skin Cancer, Skin Condition
Non-Linear Imaging of Skin In Vivo
The overall objective of this study is to investigate the potential for the VIO System to display microscopic skin structure in people of different age, sex, race, and skin health.
- Conditions
- Skin Cancer, Skin Condition, Skin Diseases, Skin Lesion
- Trial name
- VISION (NCT05410964)
- Testing
- VIO System
- Where
- United States
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- Enspectra Health
Long QT Syndrome, Cardiac Repolarization
Effect of Beta-blocker Therapy on QTc Response in Exercise and Recovery in Normal Subjects
Background. In congenital long QT syndrome type 1 (LQT1), episodes of ventricular tachycardia are usually triggered by exercise and can be prevented in most patients by beta-blocker therapy.
- Trial name
- NCT00588965
- Testing
- Propranolol LA
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- MetroHealth Medical Center
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
Bronchopulmonary Dysplasia, Bronchopulmonary Dysplasia Associated Pulmonary Hypertension
Enteral L Citrulline Supplementation in Preterm Infants - Safety, Efficacy and Dosing
Oral L-citrulline supplementation may prevent and/or decrease the severity of chronic lung disease associated with pulmonary hypertension in preterm infants.
- Conditions
- Bronchopulmonary Dysplasia, Bronchopulmonary Dysplasia Associated Pulmonary Hypertension, Pulmonary Hypertension, Premature Birth
- Trial name
- NCT03649932
- Testing
- Enteral L-citrulline
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- The University of Texas Medical Branch, Galveston
Thalassemia
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells.
- Trial name
- THALLO (NCT00578292)
- Testing
- Busulfan, Fludarabine, Campath 1H
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
Ventricular Tachycardia, Ventricular Fibrillation
Pediatric Lead Extractability and Survival Evaluation (PLEASE)
This is a randomized, prospective clinical trial comparing 2 different types of implantable cardioverter defibrillator (ICD) leads in children and patients with congenital heart disease.
- Conditions
- Ventricular Tachycardia, Ventricular Fibrillation, Cardiomyopathy, Long QT Syndrome
- Trial name
- NCT00335036
- Testing
- ICD lead implant, Thin leads
- Where
- United States
- Volunteers
- 748 people
- Sponsor
- Charles Berul
Hemophilia A
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Prospective, multinational, non-interventional post-authorisation study to collect additional clinical data and to ensure consistency in the long-term between the outcome from pre-authorisation clinical studies (in 135 previously…
- Trial name
- NCT02962765
- Where
- 39 sites in United States, Argentina and 11 more
- Volunteers
- 80 people
- Sponsor
- Octapharma
Cleft Palate
Efficacy of Oral Versus Intravenous Acetaminophen for Primary Pediatric Cleft Palate Repair
The purpose of this investigator-initiated study is to determine whether acetaminophen is an effective pain reliever for primary cleft palate repair in children and possesses opioid sparing effects.
- Trial name
- NCT01500109
- Testing
- Oral acetaminophen, Ofirmev®, Opioid only
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Loma Linda University
Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, study of sebelipase alfa in infants with rapidly progressive lysosomal acid lipase deficiency (LAL-D). Eligible participants received once-weekly infusions of sebelipase alfa for up to 3 years.
- Trial name
- NCT02193867
- Testing
- Sebelipase Alfa
- Where
- 5 sites in United States, Finland and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hemophilia B
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
- Trial name
- NCT05164471
- Testing
- verbrinacogene setparvovec
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Spur Therapeutics
Papillon-Lefevre Disease
Cytokines in Papillon-Lefèvre Syndrome
Papillon-Lefèvre syndrome (PLS) is characterised by aggressively progressive periodontitis combined with palmo-plantar hyperkeratosis. It is caused by "loss of function" mutations in the cathepsin C gene.
- Trial name
- NCT01116934
- Where
- Germany
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Goethe University
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