Clinical trials: Genetic Diseases
Hereditary Angioedema
EAP for Deucrictibant Immediate-release
This is an expanded access program (EAP) designed to allow access to deucrictibant immediate-release (IR) capsule for eligible participants with hereditary angioedema (HAE) in the United States (US) and US territories who lack…
- Trial name
- NCT07759141
- Testing
- Deucrictibant IR
- Sponsor
- Pharvaris Netherlands B.V.
Friedreich Ataxia
A Single Ascending Dose Study of DT-216P2 in Normal Healthy Participants
This purpose of the study is to evaluate the safety, tolerability and pharmacokinetics of single ascending doses of DT-216P2 administered either subcutaneously (SC) and intravenously (IV) in normal healthy participants.
- Trial name
- NCT06772870
- Testing
- DT-216P2, Saline
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Design Therapeutics, Inc.
Infections, Streptococcal
Immunization of Children Between 8 Weeks and 2 Years of Age With GSK Pneumococcal Vaccine GSK1024850A
The aim of the study is to evaluate the immunogenicity, safety and reactogenicity of GSK Biologicals' pneumococcal conjugate vaccine GSK1024850A.
- Trial name
- NCT01175083
- Testing
- GSK1024850A (Synflorix), Tritanrix-HepB/Hib, Polio Sabin
- Where
- Burkina Faso
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Dilated Cardiomyopathy
A Study to Investigate Safety, Tolerability, and Pharmacokinetics of Oral AZD0233 Compared With Placebo in Healthy Adult Participants.
The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of AZD0233 following single and multiple ascending dose (SAD and MAD) administration in healthy participants.
- Trial name
- NCT06381466
- Testing
- AZD0233
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Alpha-1 Antitrypsin Deficiency, AATD
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1 will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will consist of multiple ascending dose (MAD)…
- Trial name
- rhAAT-Fc (NCT03815396)
- Testing
- INBRX-101/rhAAT-Fc
- Where
- 10 sites in United States, New Zealand and 1 more
- Volunteers
- 31 people
- Sponsor
- Inhibrx Biosciences, Inc
Plasmodium Falciparum Malaria, Malaria
Effect of Plasmodium Falciparum Exposure and Sickle Cell Trait on Infection Rates and Kinetics After IV Administration of PfSPZ Challenge
The study is designed to establish infectivity of Plasmodium falciparum sporozoites (PfSPZ) via intravenous (IV) administration in three groups with different malaria immunity-status: 1.
- Trial name
- NCT02237586
- Testing
- PfSPZ Challenge
- Where
- Gabon
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Sanaria Inc.
Atopic Dermatitis Eczema, Atopic Dermatitis
Trends in the Epidemiology and Treatment of Atopic Dermatitis in the United Kingdom (UK)
Atopic dermatitis (AD), commonly called eczema, is one of the most frequently occurring skin conditions. It is estimated to affect around one fifth of children in developed countries and is also becoming increasingly common in…
- Conditions
- Atopic Dermatitis Eczema, Atopic Dermatitis, Eczema
- Trial name
- NCT03823794
- Testing
- No intervention
- Where
- United Kingdom
- Volunteers
- 148,166 people, healthy volunteers welcome
- Sponsor
- Momentum Data
Corneal Sensation Reduced, Corneal Dystrophy
Defining the Operating Parameters for a Rebound-esthesiometer
The purpose of this study is to define the operating parameters for a new method to measure corneal sensitivity.
- Conditions
- Corneal Sensation Reduced, Corneal Dystrophy, Corneal Degeneration, Corneal Transplant Failure
- Trial name
- NCT04384094
- Testing
- Esthesiometer measurement
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Icare Finland Oy
Dermatitis, Atopic
Protopic Ointment in Adult Atopic Eczema of the Face
Patients with facial atopic eczema and a poor response to topical corticosteroid applied either Protopic or Fluticasone ointment twice a day for 3 weeks.
- Trial name
- NCT00690105
- Testing
- tacrolimus 0.1%, fluticasone 0.005 %
- Where
- France
- Volunteers
- 577 people
- Sponsor
- Astellas Pharma Inc
Alpha 1-Antitrypsin Deficiency
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1…
- Trial name
- ChAMP (NCT00295061)
- Testing
- Alpha-1 MP, alpha-1 proteinase inhibitor (human)
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
Gout, Hyperuricemia
Open Label PK, PD and DDI of Dotinurad and Allopurinol in Gout Patients With Hyperuricemia
A open label multi-center 3-period multidose, PK/PD and drug-drug interaction (DDI) study to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of 7 days of treatment with two doses of dotinurad monotherapy, and to…
- Trial name
- NCT06056570
- Testing
- dotinurad, dotinurad + allopurinol, allopurinol
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- Urica Therapeutics Inc.
Fabry Disease
Treatment Protocol of Replagal for Patients With Fabry Disease
The study will evaluate the safety and efficacy of Replagal® (agalsidase alfa) at a dose of 0.2 mg/kg infused intravenously (IV) over 40 minutes, every other week.
- Trial name
- NCT01031173
- Testing
- agalsidase alfa
- Where
- 25 sites in United States
- Sponsor
- Shire
Hyperuricemia, Gout
Scintigraphy Study in Healthy Volunteers to Characterize the Performance of ALLN-346 Tablets
The purpose of the study is to understand the gastrointestinal transit time and disintegration behavior of the formulations in vivo in normal healthy volunteers when ALLN-346 formulations are given with light meal or in fasted or…
- Trial name
- NCT05168683
- Testing
- Each ALLN-346 treatment will be radiolabelled to contain 4 MBq 99mTc at time of dosing with light meal, Each ALLN-346 treatment will be radiolabelled to contain 4 MBq 99mTc at time of dosing in fasted/fed state
- Where
- United Kingdom
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Allena Pharmaceuticals
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Renal Impairment
Study of GBT021601 in Participants With Renal Impairment
Renal Impairment study of GBT021601.
- Trial name
- NCT05878704
- Testing
- GBT021601
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Pfizer
Primary Immune Deficiency Disorder
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
- Trial name
- NCT03988426
- Testing
- Octanorm
- Where
- 5 sites in Russia
- Volunteers
- 25 people
- Sponsor
- Octapharma
Huntington Disease
Frequency of Selected Single Nucleotide Polymorphisms in Huntington Disease Gene Expansion Carriers
For participation in this epidemiological study, a single-day visit at the study site is required. Participants will be recruited from Huntington Disease clinics, and they will be asked to answer questions regarding their…
- Trial name
- NCT06667414
- Where
- 45 sites in United States, Argentina and 10 more
- Volunteers
- 574 people
- Sponsor
- Hoffmann-La Roche
Tourette's Disorder
Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD)
The goal of the current trial is to determine efficacy and safety of once-weekly aripiprazole in reducing Total Tic Severity (TTS) score in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01418352
- Testing
- Aripiprazole
- Where
- 35 sites in United States, Bulgaria and 3 more
- Volunteers
- 83 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Mixed Hyperlipidemia
Carotid B-mode Ultrasound Study to Compare Anti-Atherosclerotic Effect of Torcetrapib/Atorvastatin to Atorvastatin.
The Torcetrapib project was terminated on December 2, 2006 due to safety findings. To look at ultrasound images taken in the carotid arteries and to look at various lipids in the blood of people with mixed hyperlipidemia
- Trial name
- RADIANCE 2 (NCT00134238)
- Testing
- torcetrapib/atorvastatin, atorvastatin
- Where
- 51 sites in United States, Canada and 4 more
- Volunteers
- 755 people
- Sponsor
- Pfizer
Hemophilia A
A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A
This study will assess the safety and tolerability of SPK-8011QQ in adult males with moderately severe to severe hemophilia A.
- Trial name
- NCT07226206
- Testing
- SPK-8011QQ
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Hoffmann-La Roche
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