Clinical trials: Genetic Diseases
Coronary Artery Disease, Chest Pain
Coronary Obstruction Detection by Molecular Personalized Gene Expression (Corus CAD or ASGES)
To validate the use of Corus CAD (Age/Sex/Gene Expression score - ASGES) blood assay in subjects who are referred for the work-up of coronary artery disease.
- Conditions
- Coronary Artery Disease, Chest Pain, Cardiovascular Diseases, Coronary Heart Disease
- Trial name
- COMPASS (NCT01117506)
- Testing
- Corus CAD (ASGES)
- Where
- 7 sites in United States
- Volunteers
- 581 people
- Sponsor
- CardioDx
Dermatitis, Atopic
Tacrolimus Ointment Long Term Safety in Young Children With Atopic Dermatitis
The objective of this study is to assess the safety and efficacy of 0.03% tacrolimus ointment as long-term treatment in paediatric patients with atopic dermatitis.
- Trial name
- NCT00560326
- Testing
- Tacrolimus Ointment 0.03%
- Where
- 8 sites in Canada, Finland and 3 more
- Volunteers
- 50 people
- Sponsor
- Astellas Pharma Inc
Alpha-Mannosidosis
Lamazym Aftercare Study
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis whom previously participated in Lamazym-trial.
- Trial name
- rhLAMAN-09 (NCT01908725)
- Testing
- Lamazym
- Where
- Denmark
- Volunteers
- 8 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Duchenne Muscular Dystrophy, Cardiomyopathy
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
Male participants with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All participants will be at least 12 years of age.
- Trial name
- HOPE (NCT02485938)
- Testing
- Allogeneic Cardiosphere-Derived Cells (CAP-1002)
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Capricor Inc.
Atopic Dermatitis, Healthy Participants
A Study of BYN-001 in Healthy Adults and in Adults With Moderate to Severe Atopic Dermatitis
This first-in-human study evaluates BYN-001, a humanized IgG1 monoclonal antibody targeting interleukin-25 (IL-25), a cytokine implicated in atopic dermatitis.
- Trial name
- BYN-001-101 (NCT07746817)
- Testing
- BYN-001
- Where
- Australia
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- Bionyra Pharma
Friedreich Ataxia, Cardiomyopathy, Secondary
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia
This is a Phase 1/2, open-label, dose-ascending, multicenter study of the safety and efficacy of LX2006 for participants who have Friedreich's Ataxia with evidence of cardiomyopathy.
- Trial name
- NCT05445323
- Testing
- Low dose LX2006, Mid Dose LX2006, High Dose LX2006
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Lexeo Therapeutics
Atopic Dermatitis (AD)
A Study Evaluating BFB759 in Moderate to Severe Atopic Dermatitis
This is a double-blind, placebo-controlled study where subjects are participating for approximately 36 to 40 weeks. The study compares how well BFB759 works and how safe it is compared with a placebo.
- Trial name
- COMPASS 2-AD (NCT07105488)
- Testing
- BFB759
- Where
- 88 sites in United States, Bulgaria and 5 more
- Volunteers
- 225 people
- Sponsor
- Bluefin Biomedicine, Inc.
Cryopyrin-Associated Periodic Syndromes
Kineret CAPS Post Authorisation Study
A non-interventional, post authorization safety study to evaluate the safety of Kineret in the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) in routine clinical care with regard to serious infections, malignancies…
- Trial name
- NCT02326376
- Testing
- anakinra (Kineret)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Swedish Orphan Biovitrum
Atopic Dermatitis (AD)
A Study of NOR-101 in Healthy Adults and Adults With Moderate-to-Severe Atopic Dermatitis
This Phase 1 study will assess NOR-101, an investigational study drug, in two parts. Part 1 is a randomized, blinded study comparing single and multiple doses of NOR-101 with placebo in healthy adults.
- Trial name
- POLARIS-1 (NCT07904689)
- Testing
- NOR-101
- Where
- Australia
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- North Immunology Inc.
Atopic Dermatitis
Study to Assess the Safety and Efficacy of Lebrikizumab (LY3650150) in Adolescent Participants With Moderate-to-Severe Atopic Dermatitis
This is an open-label, single arm study of 52 weeks duration. The study will assess the safety and efficacy of lebrikizumab in adolescent participants (≥12 to <18 years weighing ≥40 kilograms) with moderate-to-severe atopic…
- Trial name
- ADore (NCT04250350)
- Testing
- Lebrikizumab
- Where
- 70 sites in United States, Australia and 2 more
- Volunteers
- 206 people
- Sponsor
- Eli Lilly and Company
Primary Immunodeficiency
An Open-label, Multicenter Study to Assess the Pharmacokinetics (PK), Safety, and Tolerability of Subcutaneous IgPro20 in Immunoglobulin (IG) Treatment-naïve Participants With Primary Immunodeficiency (PID)
This is an open-label, multicenter, phase 4 study in IG treatment-naïve participants with PID, conducted in the United States (US), to assess the PK, safety, and tolerability of IgPro20.
- Trial name
- NCT07076446
- Testing
- IgPro
- Where
- 8 sites in United States
- Volunteers
- 8 people
- Sponsor
- CSL Behring
Alpha 1-Antitrypsin Deficiency
Study of DCR-A1AT in Healthy Adult Volunteers
This is a research study to test an experimental study drug (belcesiran, also known as DCR-A1AT). This drug is being tested to see if it helps people with a rare condition known as Alpha-1 Antitrypsin Deficiency, or A1ATD.
- Trial name
- NCT04174118
- Testing
- belcesiran
- Where
- 2 sites in New Zealand, Sweden
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to determine if a subcutaneous dose of DX-88 (ecallantide; an investigational product) is safe and relieves symptoms of HAE in patients suffering from moderate to severe acute attacks of HAE.
- Trial name
- NCT00262080
- Testing
- ecallantide, Phosphate Buffer Saline (PBS),
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Shire
Healthy
A Study to Learn How Different Amounts of the Study Medicine Called PF-08049820 Are Tolerated and Act in the Body in Healthy Adults
The purpose of this study is to learn about the safety of the study medicine called PF-08049820 in healthy adults. The study will also see: how the body processes the study medicine and if food affects the amount of study…
- Trial name
- NCT06686797
- Testing
- PF-08049820
- Where
- United States
- Volunteers
- 71 people, healthy volunteers welcome
- Sponsor
- Pfizer
Uncontrolled Gout
Pegloticase and Methotrexate Co-administered in Participants With Uncontrolled Gout Who Previously Failed Pegloticase Monotherapy
This is a Phase 4, multicenter, open-label trial of pegloticase with methotrexate (MTX) in adult participants with uncontrolled gout who were previously treated with pegloticase without a concomitant immunomodulator and stopped…
- Trial name
- NCT04772313
- Testing
- Pegloticase, Methotrexate (MTX)
- Where
- 15 sites in United States
- Volunteers
- 11 people
- Sponsor
- Amgen
Cystic Fibrosis
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
- Trial name
- NCT06237335
- Testing
- RCT2100, Ivacaftor
- Where
- 23 sites in United States, France and 3 more
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- ReCode Therapeutics
Duchenne Muscular Dystrophy
Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy
A Multi-center, Randomized, Double-blind, Placebo-controlled, Phase 1/2 Trial to Evaluate the Efficacy and Safety of EN001 in Patients with Duchenne Muscular Dystrophy
- Trial name
- NCT06328725
- Testing
- EN001
- Where
- 2 sites in South Korea
- Volunteers
- 88 people
- Sponsor
- ENCell
Rare Genetic Disorders
Collection and Storage of Human Biospecimens for Research Into Rare Diseases and Medical Conditions
The objective of this protocol is to enable collection of biospecimens to facilitate current and future multidisciplinary research in rare genetic disorders and medical conditions.
- Trial name
- NCT02365376
- Where
- United States
- Sponsor
- BioMarin Pharmaceutical
Wrinkle
BTL-785F Device for Improving the Structure of Facial Muscles
This study will evaluate the clinical safety and the performance of the BTL-785F system equipped with the BTL-785-7 applicator for non-invasive facial remodeling and investigate its effect on muscles and wrinkles.
- Trial name
- NCT05524740
- Testing
- BTL-785-7
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- BTL Industries Ltd.
Skin Laxity
Post-Market Lower Eyelid Treatment With Renuvion in Greece
This is a prospective, multi-center, non-randomized, single-arm study of up to 15 subjects treated with the Renuvion APR System.
- Trial name
- NCT05605691
- Testing
- Renuvion APR System
- Where
- Greece
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Apyx Medical
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