Clinical trials: Genetic Factors
- Condition Genetic Factors
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Cystic Fibrosis
Study to Validate the Instructions for Use of TOBI® Podhaler™ in Cystic Fibrosis Patients
The purpose of this actual use human factors (HF) study is to validate the approved US TOBI Podhaler Instructions for Use (IFU), by establishing that the IFU effectively communicates the information necessary to achieve safe and…
- Trial name
- NCT02178540
- Testing
- Tobi Podhaler
- Where
- 13 sites in United States
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Genetic Disorder, Turner Syndrome
Growth Response in Girls With Turner Syndrome
This trial is conducted in Europe. The aim of the trial is to evaluate growth response of two somatropin dose regimens in girls with Turner Syndrome.
- Trial name
- NCT01734486
- Testing
- somatropin
- Where
- 15 sites in France
- Volunteers
- 31 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Fc factOrs and Real-World hemophiliA Patient-ReporteD Outcomes
The primary objective of the study is to evaluate the effectiveness of prophylactic treatment with recombinant Factor VIII Fc fusion protein (rFVIIIFc) and recombinant Factor IX Fc fusion protein (rFIXFc) therapy as assessed by…
- Trial name
- FORWARD (NCT02392156)
- Testing
- rFVIIIFc, rFIXFc, non-Fc FVIII replacement products
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to <12 years) who have severe hemophilia A or…
- Trial name
- ATLAS-KIDS (NCT07285460)
- Testing
- Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC)
- Where
- 37 sites in United States, Belgium and 10 more
- Volunteers
- 85 people
- Sponsor
- Sanofi
Toxicity
Case Control Study of Pharmacogenomic Factors Associated With Hepatocellular Injury Following Exposure to Lapaquistat Acetate
The purpose of this study is to examine the genetic contribution to the mechanism of lapaquistat acetate- induced hepatic abnormalities.
- Trial name
- NCT00890448
- Testing
- Pharmacogenomic whole blood sampling
- Where
- 19 sites in United States, Argentina and 12 more
- Volunteers
- 66 people
- Sponsor
- Takeda
Lymphoma, T-Cell, Peripheral
A Retrospective Study of Clinical, Phenotypic and Genetic Factors of Peripheral T-Cell Lymphomas
The purpose of this study is to establish the distribution of peripheral T-cell lymphocyte (PTCL) subtypes by re-analysis and re-classification of samples according to the 2008 World Health Organization (WHO) classification of…
- Trial name
- NCT02788916
- Testing
- No Intervention
- Where
- 10 sites in Spain
- Volunteers
- 198 people
- Sponsor
- Takeda
Atopic Dermatitis
Comprehending Atopic Risk Elements
Skin barrier impairment and familial atopy have been implicated as crucial risk factors for the development of atopic dermatitis (AD).
- Trial name
- CARE (NCT04325451)
- Where
- 2 sites in Israel
- Volunteers
- 252 people, healthy volunteers welcome
- Sponsor
- MYOR Ltd.
Cystic Fibrosis
Human Factors Study to Validate the User Interface of TOBI Podhaler Using Placebo Capsules
The purpose of this study is to validate the user interface of TOBI Podhaler by establishing that the product can support safe and effective use for the intended users
- Trial name
- NCT03502070
- Testing
- Tobi Podhaler
- Where
- 9 sites in United States
- Volunteers
- 47 people
- Sponsor
- Mylan Inc.
SCAR
An Exploratory Study of Genetic and Clinical Factors for Serious Skin Reactions Among Users of Eslicarbazepine Acetate
The reason for this study is to understand if people with certain genes are predisposed to develop severe skin reactions after they are administered Eslicarbazepine Acetate.
- Trial name
- NCT02520557
- Testing
- Blood or Saliva
- Where
- United States
- Volunteers
- 121 people
- Sponsor
- Sumitomo Pharma America, Inc.
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
Hereditary Angioedema
Safety and Pharmacokinetics of GNR-038 in Healthy Volunteers
It is an open-label dose-escalating study in sequential cohorts to assess safety and pharmacokinetics of GNR-038.
- Trial name
- NCT04557319
- Testing
- GNR-038, 25 МЕ/kg, GNR-038, 50 МЕ/kg, GNR-038, 100 МЕ/kg
- Where
- Russia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM