Clinical trials: Genetic Conditions
Primary Hyperoxaluria Type 1, PH1
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
- Trial name
- NCT07587021
- Testing
- YOLT-203
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- YolTech Therapeutics Co., Ltd
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF
US Benchmarking Clinical Study
The goal of this observational study is to measure shear wave velocity (SWV) in patients with non-reduced left ventricular ejection fraction (LVEF) heart failure and left ventricular hypertrophy, with or without transthyretin…
- Conditions
- HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy
- Trial name
- EMS-CT-1 (NCT07215715)
- Where
- 3 sites in United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- eMyosound SAS
Alpha-1 Antitrypsin Deficiency
Respreeza® Self-administration and Learning Program (AmAREtTI Study)
According to the Respreeza® Summary of Product Characteristics, the initial infusions must be administered under the supervision of a health professional experienced in the treatment of alpha-1 antitrypsin deficiency, although…
- Trial name
- AmAREtTI (NCT04262284)
- Where
- 11 sites in France
- Volunteers
- 15 people
- Sponsor
- CSL Behring
Dilated Cardiomyopathy (DCM)
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.
- Trial name
- NCT07137338
- Testing
- RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)
- Where
- 3 sites in United States
- Volunteers
- 8 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Achromatopsia
Clinical and Genetic Characterization of Individuals With Achromatopsia
The purpose of this study is to identify individuals with achromatopsia caused by mutations in the CNGB3 gene and characterize their clinical condition using several tests of visual function every 6 months for up to 1.5 years.
- Trial name
- NCT01846052
- Where
- 5 sites in United States
- Volunteers
- 56 people
- Sponsor
- Beacon Therapeutics
Psoriasis, Atopic Dermatitis
An Exploratory Study to Evaluate a Digital Intervention to Disrupt Scratching in Atopic Dermatitis and Psoriasis
CT-100 is a platform that provides interactive, software based therapeutic components that may be used as part of a treatment in future software-based prescription digital therapeutics.
- Trial name
- NCT06275659
- Testing
- CT-100-004-A, CT-100-004-B
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Click Therapeutics, Inc.
Arrhythmogenic Cardiomyopathy, PKP2-ACM
Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant
The primary objective of this Phase 1/2 long-term follow-up (LTFU) study is to assess the long-term safety and tolerability of LX2020 for the treatment of arrhythmogenic cardiomyopathy (ACM) due to a plakophilin-2 gene (PKP2)…
- Conditions
- Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
- Trial name
- GRIT-PKP2 (NCT07050160)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Lexeo Therapeutics
Primary Lateral Sclerosis, Adult, 1
"Individual Patient Expanded Access IND to Treat Primary Lateral Sclerosis"
This Individual Patient Expanded Access IND has been created as requested by an 83-year-old man who suffers Primary Lateral Sclerosis and for which the drugs currently approved are not providing an improvement over the…
- Trial name
- NCT04825613
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Saccular and Fusiform Aneurysms, Pseudoaneurysm
Targeting Real World Usage In Stroke Treatment
TRUST Registry is an observational, prospective, long-term, post-market surveillance registry of subjects treated with WallabyPhenox flow modulation devices, stent systems, bifurcation aneurysm implants including their HPC…
- Conditions
- Saccular and Fusiform Aneurysms, Pseudoaneurysm, Vascular Dissection, Perforation
- Trial name
- TRUST (NCT07474935)
- Testing
- Aneurysm Treatment with a Neurovascular Flow Diverter, Dissection Treatment with a Neurovascular Flow Diverter, Perforation Treatment with a Neurovascular Flow Diverter
- Where
- 13 sites in France, Germany and 3 more
- Volunteers
- 5,000 people
- Sponsor
- Phenox GmbH
Alpha1-Antitrypsin Deficiency
Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)
This is a multi-center, open-label study to evaluate the long-term safety of weekly intravenous (IV) infusions of 60 mg/kg alpha1-PI (human), modified process (Alpha-1 MP) in adult participants with Alpha1 Antitrypsin Deficiency…
- Trial name
- NCT02870348
- Testing
- Alpha-1 MP
- Where
- 3 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
ITCH, Atopic Dermatitis
Obtaining Descriptive Classifications of Pruritus and Assessing Change in Pruritus Over Time in Atopic Dermatitis Patients Using Topical Roflumilast Cream.
This study is being done to obtain descriptive classifications of pruritus using a patient directed survey system and assess change in pruritus over time in patients with Atopic Dermatitis over 4 weeks with use of topical…
- Trial name
- NCT07560618
- Testing
- Roflumilast Cream 0.15%
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Integrative Skin Science and Research
Indolent Systemic Mastocytosis
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment
The purpose of this study is to evaluate the efficacy and safety of oral masitinib versus placebo in the treatment of patients suffering from smouldering or indolent systemic mastocytosis with severe symptoms of mast cell…
- Trial name
- NCT04333108
- Testing
- Masitinib, Best Supportive Care
- Where
- 17 sites in France, Germany and 6 more
- Volunteers
- 140 people
- Sponsor
- AB Science
Dermatitis, Atopic
Investigation of Topical SB705498 on Healthy Volunteers
A two part, randomized, double-blind, placebo controlled study to investigate the effects of topical doses of SB705498 in healthy volunteers.
- Trial name
- NCT01673529
- Testing
- SB705498
- Where
- United Kingdom
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Gout, Hyperuricemia
The Effect of Naproxen on the Pharmacokinetic Profile of LC350189 in Healthy Adults
This is a Phase 1, open-label, fixed-sequence, 3-period, 2-way drug interaction study designed to assess the pharmacokinetics, pharmacodynamics, safety, and tolerability of LC350189 and Naproxen when administered alone and in…
- Trial name
- NCT04139824
- Testing
- Xanthine Oxidase Inhibitor, nonsteroidal anti-inflammatory drug
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- LG Chem
Sickle Cell Disease
A Study of Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of…
- Trial name
- HIBISCUS (NCT04624659)
- Testing
- Etavopivat Tablets Low dose, Etavopivat Tablets High dose, Etavopivat Tablets
- Where
- 142 sites in United States, Canada and 15 more
- Volunteers
- 450 people
- Sponsor
- Forma Therapeutics, Inc.
Dermatitis, Atopic, Pruritus
Study to Evaluate ASN008 Topical Gel (TG)
This is an ascending dose escalation study to test the safety, tolerability and preliminary efficacy of ASN008 TG in first-in-human subjects
- Conditions
- Dermatitis, Atopic, Pruritus, Dermatitis Eczema
- Trial name
- NCT03798561
- Testing
- ASN008 TG
- Where
- 5 sites in United States, Canada
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Asana BioSciences
Healthy
AStudy to Evaluate the Bioavailability of PF 04965842 in Healthy Subjects
Phase 1, open label, single dose, 3 way crossover study to evaluate the bioavailability of a solid dose formulation of PF 04965842 relative to a suspension formulation under fasting conditions and the effect of food on the…
- Trial name
- NCT02163161
- Testing
- PF-04965842, PF- 04965842
- Where
- Belgium
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Atopic Dermatitis (Eczema)
The Effects of a Botanical Moisturizer and an Unscented Soap on Eczema and Itch
The purpose of this study is to examine the effects of a botanical moisturizer and an unscented soap on eczema severity, itch, mood, and various skin measures (such as skin hydration) in those with mild to moderate eczema.
- Trial name
- NCT06804070
- Testing
- Botanical Moisturizer + Unscented Soap
- Where
- Poland
- Volunteers
- 34 people
- Sponsor
- Codex Labs Corporation
Wrinkle, Acne Scars - Mixed Atrophic and Hypertrophic
AV-23-001 AVAVA MIRIA Pilot Study
This non-significant risk study is intended to demonstrate the use of the MIRIA Skin Treatment System can show improvement in dermatologic conditions currently indicated for treatment with a laser (such as, but not limited to…
- Conditions
- Wrinkle, Acne Scars - Mixed Atrophic and Hypertrophic, Pigmented Lesions, Skin Laxity
- Trial name
- NCT06557434
- Testing
- MIRIA
- Where
- 2 sites in United States
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- AVAVA, Inc.