Clinical trials: Newborn Diseases
Alpha 1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
- Trial name
- NCT04167345
- Testing
- VX-814
- Where
- 28 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Hemophilia A; Hemophilia B
A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
- Trial name
- NCT03481946
- Testing
- BAY1093884
- Where
- Israel
- Volunteers
- 6 people
- Sponsor
- Bayer
Septal Defect, Atrial
Safety and Efficacy of the GORE® Septal Occluder to Treat Ostium Secundum Atrial Septal Defects
The primary objective of the GORE® Septal Occluder Study is to evaluate the safety and efficacy of the occluder device in the treatment of transcatheter closure of ostium secundum atrial septal defects (ASDs).
- Trial name
- NCT01711983
- Testing
- GORE® CARDIOFORM Septal Occluder
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- W.L.Gore & Associates
Persistent Pulmonary Hypertension of the Newborn
Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn
This study assessed the safety and treatment effect of intravenous (IV) Remodulin as an add-on therapy in neonates with persistent pulmonary hypertension of the newborn (PPHN).
- Trial name
- NCT02261883
- Testing
- IV Remodulin
- Where
- 14 sites in United States
- Volunteers
- 42 people
- Sponsor
- United Therapeutics
Achondroplasia
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of…
- Trial name
- PROPEL3 (NCT06164951)
- Testing
- Infigratinib 0.25 mg/kg/day
- Where
- 27 sites in United States, Argentina and 8 more
- Volunteers
- 114 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Atopic Dermatitis
Safety and Efficacy of FURESTEM-AD Inj. in Patients With Moderate to Severe Chronic Atopic Dermatitis(AD)
This is multi-center, randomized, double-blind, parallel, placebo-controlled phase III clinical trial to evaluate the efficacy and safety of FURESTEM-AD Inj. for moderate to severe chronic atopic dermatitis
- Trial name
- NCT03269773
- Testing
- FURESTEM-AD Inj.
- Where
- 11 sites in South Korea
- Volunteers
- 197 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Primary Immunodeficiency
Pharmacokinetics and Safety of IVIG Nanogam 100 mg/ml
Intravenous immunoglobulin (IVIG) is used for treatment of a heterogeneous group of immune related disorders both as immune-replacement and immune-modulating therapy.
- Trial name
- Nanogam (NCT01985373)
- Testing
- Intravenous immunoglobulin infusion
- Where
- 4 sites in Netherlands
- Volunteers
- 23 people
- Sponsor
- Prothya Biosolutions
Tourette's Disorder
Safety and Tolerability Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to obtain long term efficacy, safety and tolerability data of once weekly aripiprazole in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01416441
- Testing
- Aripiprazole
- Where
- 80 sites in United States, Bulgaria and 8 more
- Volunteers
- 170 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Hemophilia B
Efficacy and Safety of AlphaNine Versus BeneFIX in Patients With Severe Hereditary Haemophilia B
The goal of this non-randomized, multi-center study in subjects with severe hereditary haemophilia B was to determine and compare the pharmacokinetic and safety profiles of BeneFIX in subjects having had 2 prior pharmacokinetic…
- Trial name
- NCT03091751
- Testing
- BeneFIX
- Where
- 3 sites in Bulgaria
- Volunteers
- 13 people
- Sponsor
- Grifols Biologicals, LLC
Muscular Dystrophies, Limb Girdle Muscular Dystrophy
Biomarker Development in LGMD2i
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD2i to support therapeutic development.
- Trial name
- MLB-01-001 (NCT04202627)
- Where
- 11 sites in United States, Denmark
- Volunteers
- 101 people
- Sponsor
- ML Bio Solutions, Inc.
Alzheimer's Disease, Huntington's Disease
A Phase I Study To Estimate The Effect Of Ketoconazole And Omeprazole On The Pharmacokinetics Of Dimebon In Healthy Subjects Who Are Normal Or Poor CYP2D6 Metabolizers
This study will evaluate the potential for a drug-drug interaction of Dimebon with ketoconazole and omeprazole, potent inhibitors of the drug metabolizing enzymes CYP3A4 and CYP2C19, respectively.
- Trial name
- NCT00931073
- Testing
- Dimebon alone, Dimebon + Ketoconazole, Dimebon + Omeprazole
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Pfizer
Gaucher Disease
Taste Evaluation of Different Liquid Formulations With Eliglustat
Primary Objective: The purpose of this study is to assess the palatability of eliglustat prototype liquid formulations in healthy subjects.
- Trial name
- NCT02422654
- Testing
- eliglustat
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Sickle Cell Disease
Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
- Trial name
- PROSPECT (NCT04930445)
- Testing
- Oxbryta® (voxelotor) 500mg Tablets
- Where
- 43 sites in United States
- Volunteers
- 265 people
- Sponsor
- Pfizer
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function.
- Trial name
- NCT00847379
- Testing
- Ataluren
- Where
- 37 sites in United States, Australia and 9 more
- Volunteers
- 173 people
- Sponsor
- PTC Therapeutics
Gout, Coronary Artery Disease
Uric Acid Study in Healthy Male Volunteers
This study is being conducted to examine the effect of AZD6140 on the levels of certain chemicals in subjects' blood and urine.
- Trial name
- NCT00738842
- Testing
- AZD6140
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Hereditary Cancer Syndromes, Clinical Decision Support
Closing the GAPS: Guideline Adherence, Prevention and Surveillance in Hereditary Cancer
The goal of this clinical trial is to see if a software platform can improve cancer screening in young adults with genetic risk for cancer. The trial will also help improve the software platform (Nest).
- Trial name
- NCT06654466
- Testing
- Nest, an electronic medical record (EMR)-integrated software platform to deliver longitudinal, genetics-based care at scale.
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Nest Genomics
Skin Laxity
Safety and Effectiveness Evaluation of the Device in Achieving Submental Lift
A prospective evaluation of the ThermiRF device in treating skin laxity under the chin and neck. Seventy male and female subjects between the age of 35 and 65 (inclusive) will be enrolled in this trial.
- Trial name
- NCT02832674
- Testing
- Percutaneous Radiofrequency single treatment
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- ThermiGen, LLC
Dermatitis, Atopic, Eczema
A Study of Lebrikizumab in Combination With Topical Corticosteroids in Patients With Atopic Dermatitis (AD) That Are Not Adequately Controlled With or Are Non-eligible for Cyclosporine
The main purpose of this study is to evaluate the efficacy of lebrikizumab compared with placebo in participants not adequately controlled with cyclosporine or for whom cyclosporine is not medically advisable up to Week 16.
- Trial name
- ADvantage (NCT05149313)
- Testing
- Lebrikizumab
- Where
- 48 sites in Austria, Belgium and 6 more
- Volunteers
- 331 people
- Sponsor
- Almirall, S.A.
Hemophilia A
Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery
Recombinant factor VIII for the prevention of bleeding in women/girls with haemophilia A undergoing major surgery
- Trial name
- NCT05936580
- Testing
- Nuwiq
- Where
- 16 sites in United States, Finland and 8 more
- Volunteers
- 28 people
- Sponsor
- Octapharma
Von Willebrand Disease
Pharmacokinetic, Safety and Tolerability Study of Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex in Type 3 Von Willebrand Disease
The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.
- Trial name
- NCT00816660
- Testing
- Recombinant von Willebrand factor : recombinant FVIII (rVWF:rFVIII), Marketed plasma-derived VWF/FVIII concentrate
- Where
- 25 sites in United States, Austria and 4 more
- Volunteers
- 32 people
- Sponsor
- Baxalta now part of Shire
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