Clinical trials: Genetic Disorders
Cystic Fibrosis
Pharmacokinetics of Tedizolid Phosphate in Cystic Fibrosis
The proposed study is designed to characterize the pharmacokinetics of intravenous and oral tedizolid in patients with Cystic Fibrosis.
- Trial name
- NCT02444234
- Testing
- Tedizolid PO, Tedizolid IV
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Southern California
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B.
- Trial name
- NCT04286412
- Testing
- Nonacog alfa
- Where
- 5 sites in India
- Volunteers
- 25 people
- Sponsor
- Pfizer
Dermatitis, Atopic
A Study for AR100DP1 in Mild to Moderate Atopic Dermatitis (AD)
This is a Phase I/IIa Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of AR100DP1 in Health Subjects, and Subjects with Mild to Moderate Atopic Dermatitis.
- Trial name
- NCT04220411
- Testing
- AR100DP1
- Where
- Taiwan
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Arjil Pharmaceuticals LLC
Osteoporosis
Efficacy Study of Zoledronic Acid and Teriparatide Combination Therapy in Women With Osteoporosis
The purpose of this study is to assess the effects of zoledronic acid administered at the same time with teriparatide compared to zoledronic acid alone and teriparatide alone on bone mineral density (BMD) gain in the lumbar spine…
- Trial name
- NCT00439244
- Testing
- Zoledronic acid, Teriparatide
- Where
- 33 sites in United States, Belgium and 2 more
- Volunteers
- 412 people
- Sponsor
- Novartis
Mixed Hyperlipidemia
Lipid Efficacy and Safety in Participants With Mixed Hyperlipidemia (MK-0524B-024)
This is a 12-week clinical trial in participants with mixed hyperlipidemia to study the effects of MK-0524B on lipids.The primary hypothesis is that MK-0524B (dosed as MK-0524A coadministered with simvastatin) will be superior to…
- Trial name
- NCT00289900
- Testing
- MK-0524A, Atorvastatin, Simvastatin
- Volunteers
- 2,340 people
- Sponsor
- Merck Sharp & Dohme LLC
Cystic Fibrosis, Methicillin-resistant Staphylococcus Aureus
Early Methicillin-resistant Staphylococcus Aureus (MRSA) Therapy in Cystic Fibrosis (CF)
Purpose: There has been a recent, rapid increase in prevalence of Methicillin-resistant Staphylococcus aureus (MRSA) among patients with Cystic Fibrosis (22% across US CF centers in 2009).
- Trial name
- STAR-Too (NCT01349192)
- Testing
- Rifampin, Trimethoprim/Sulfamethoxazole, Minocycline
- Where
- 13 sites in United States
- Volunteers
- 47 people
- Sponsor
- University of North Carolina, Chapel Hill
Phenylketonuria (PKU)
Effects of Kuvan on Melatonin Secretion
This study examines the effect of tetrahydrobiopterin (Kuvan) and Large Neutral Amino Acid (LNAA) therapy on melatonin and dopamine levels in individuals with Phenylketonuria (PKU).
- Trial name
- NCT01617070
- Testing
- Kuvan, Large Neutral Amino Acid Therapy
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Southern California
Unverricht-Lundborg Disease
Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease (ULD) in Adolescents and Adults
The study will compare the efficacy and safety of Brivaracetam with placebo in patients with Unverricht- Lundborg Disease (ULD).
- Trial name
- NCT00368251
- Testing
- BRV 2.5 mg, BRV 25 mg, BRV 50 mg
- Where
- 18 sites in United States, Canada and 6 more
- Volunteers
- 56 people
- Sponsor
- UCB Pharma
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03811028
- Testing
- SOBI003
- Where
- 3 sites in United States, Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Sickle Cell Disease
Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization
This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU).
- Trial name
- NCT04080167
- Testing
- InCharge Health mobile application, HU Toolbox mobile application
- Where
- 7 sites in United States
- Volunteers
- 382 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
MRSA, Cystic Fibrosis
AeroVanc in the Treatment of Methicillin-resistant Staphylococcus Aureus Infection in Patients With Cystic Fibrosis
This is a multi-center, randomized phase III study to evaluate the clinical effectiveness of AeroVanc in persistent methicillin-resistant Staphylococcus aureus (MRSA) infection in patients with cystic fibrosis (CF).
- Trial name
- NCT03181932
- Testing
- Vancomycin inhalation powder
- Where
- 71 sites in United States, Canada
- Volunteers
- 188 people
- Sponsor
- Savara Inc.
Neurofibromatosis Type 1, Brain Tumor
Feasibility and Clinically Application of Magnetic Resonance Fingerprinting
This study will look at the feasibility of using magnetic resonance fingerprinting (MRF) in children, adolescents and young adults (AYA) with and without brain tumors.
- Conditions
- Neurofibromatosis Type 1, Brain Tumor, Glioma
- Trial name
- NCT02387840
- Testing
- Magnetic Resonance Imaging, Magnetic Resonance Fingerprinting
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Case Comprehensive Cancer Center
Ophthalmopathy, Thyroid-Associated
Rituximab (RTX) Therapy in Patients With Active TAO
Thyroid Associated Ophthalmopathy is condition affecting the eyes of about 10% of patients with Graves disease. Its combination of protrusion affecting the looks of the patient and pain is often severely affecting the quality of…
- Trial name
- NCT02378298
- Testing
- Rituximab, Iv Methylprednisolone, peroral methylprednisolone and Methotrexate
- Where
- 5 sites in Sweden
- Volunteers
- 38 people
- Sponsor
- Göteborg University
Atopic Dermatitis
Study to Evaluate Long-Term Safety of ASN002 in Subjects With Moderate to Severe Atopic Dermatitis
This is a randomized open label study to evaluate the long term safety of ASN002 in subjects with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT03654755
- Testing
- ASN002
- Where
- 38 sites in United States, Canada
- Volunteers
- 162 people
- Sponsor
- Asana BioSciences
Cystic Fibrosis
Ease of Use and Microbial Contamination of Tobramycin Inhalation Powder (TIP) Versus Nebulised Tobramycin Inhalation Solution (TIS) and Nebulised Colistimethate (COLI)
The purpose of this interventional Phase IV study was to explore the ease of use of TIP and prevalence of microbial contamination of the T-326 Inhaler compared with TIS and colistimethate administered via nebuliser for the…
- Trial name
- NCT01844778
- Testing
- Tobramycin Inhalation Powder, Tobramycin inhalation solution, Colistimethate
- Where
- 21 sites in Germany, Ireland and 3 more
- Volunteers
- 60 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
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 homozygous for the F508del mutation (F/F).
- Trial name
- NCT03460990
- Testing
- VX-659/TEZ/IVA, TEZ/IVA, IVA
- Where
- 46 sites in United States, Australia and 4 more
- Volunteers
- 116 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Atopic Dermatitis (AD)
Study to Determine the Safety and Effectiveness of Dupilumab for Treatment of Atopic Dermatitis (AD)
The primary objective of the study was to assess the efficacy of Dupilumab, compared to placebo, in adult patients with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT01979016
- Testing
- Dupilumab, Background treatment
- Where
- 4 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Regeneron Pharmaceuticals
Prostate Cancer, Prostate Cancer, Familial
Clinical Validity and Utility of Genomic-targeted Chemoprevention of PCa: Aim 4a
This study was designed to compare the efficacy, perception, decision making, and cost-effectiveness of genomic and non-genomic approaches for risk assessment of prostate cancer and for chemoprevention of prostate cancer.
- Conditions
- Prostate Cancer, Prostate Cancer, Familial, Hereditary Prostate Cancer
- Trial name
- NCT02381015
- Testing
- Genetic Risk Score: Number Format, Genetic Risk Score: Number + Pictograph, Family History: Number Format
- Volunteers
- 700 people, healthy volunteers welcome
- Sponsor
- Endeavor Health
Cardiomyopathy, Hypertrophic
Study of Efficacy of Oral Sacubitril/Valsartan in Adult Patients With Non-obstructive Hypertrophic Cardiomyopathy
The purpose of this study was to determine if LCZ696 can improve functional capacity (via improved peak VO2) in non-obstructive hypertrophic cardiomyopathy (HCM) patient population over the course of 50 weeks of treatment.
- Trial name
- NCT04164732
- Testing
- LCZ696
- Where
- 18 sites in United States, Germany and 4 more
- Volunteers
- 46 people
- Sponsor
- Novartis Pharmaceuticals