Clinical trials: Congenital Disorder
Fabry Disease
Agalsidase Beta Long-Term Treatment Outcome for Fabry Disease Patients With IVS4 Mutation in Taiwan
This is a national, multicenter, observational, cohort study designed to assess clinical outcomes upon agalsidase beta treatment, to characterize the clinical manifestations, and to collect the natural history on male and female…
- Trial name
- NCT06052800
- Where
- 5 sites in Taiwan
- Volunteers
- 78 people
- Sponsor
- Sanofi
Eczema, Atopic Dermatitis
A Study to Evaluate CLn® BodyWash As Added Therapy in Eczema Patients
The purpose of this study is to determine if CLn BodyWash, a novel new gel cleanser formulated with surfactants and preserved with sodium hypochlorite, is effective at decreasing severity, body surface area and itching for…
- Trial name
- CLeaN (NCT01714245)
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- TopMD Skin Care, Inc.
Phenylketonurias, PKU
Evaluation of the Express Plus Range
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM.
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease
- Trial name
- express plus (NCT05051657)
- Testing
- PKU express plus
- Where
- 6 sites in United Kingdom
- Volunteers
- 28 people
- Sponsor
- Vitaflo International, Ltd
Atopic Dermatitis (AD)
A Study of Single and Multiple Ascending Doses of KBL697 in Healthy Subjects
The study is designed to investigate the safety and tolerability of KBL697 in healthy volunteers. KBL697 has been developed as a potential new treatment for atopic dermatitis (AD).
- Trial name
- NCT04056130
- Testing
- KBL697
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- KoBioLabs
Arteriovenous Fistula
Study Comparing Lutonix AV Drug Coated Balloon vs Standard Balloon for Treatment of Dysfunctional AV Fistulae
This prospective, global, multicenter, randomized, controlled study is designed to evaluate the safety and effectiveness of the Lutonix 035 AV Drug Coated Balloon PTA Catheter compared to a standard PTA Catheter in treating…
- Trial name
- NCT02440022
- Testing
- Lutonix DCB, Standard Balloon Angioplasty Catheter, Percutaneous Transluminal Angiography
- Where
- 23 sites in United States
- Volunteers
- 285 people
- Sponsor
- C. R. Bard
Eczema, Atopic Dermatitis
A Clinical Trial to Evaluate the Effects of Rejuvaskin Eczema Body Wash and Skin Recovery Cream in Adults With Eczema-Prone Skin
This is a single-arm, open-label, decentralized (fully remote) clinical trial evaluating the tolerability and self-perceived skin effects of Rejuvaskin Eczema Body Wash and Skin Recovery Cream in 35 adults aged 18-65 with…
- Conditions
- Eczema, Atopic Dermatitis, Eczema-Prone Skin
- Trial name
- NCT07700745
- Testing
- Rejuvaskin Eczema Body Wash
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Rejuvaskin
Atopic Dermatitis
Phase 2 Study of KHK4577
This study is an randomized double-blind placebo-controlled study to evaluate the efficacy and the safety of oral KHK4577 for 6 weeks in patients with atopic dermatitis. Pharmacokinetics of KHK4577 will also be assessed.
- Trial name
- NCT02004119
- Testing
- KHK4577
- Where
- Japan
- Volunteers
- 93 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Atopic Dermatitis
A Long-Term Extension Trial in Participants With Atopic Dermatitis Who Participated in Previous EDP1815 Trials
This is an Open-Label Extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of EDP1815 in participants with mild, moderate, and severe atopic dermatitis who have completed the treatment period of a…
- Trial name
- NCT05439941
- Testing
- EDP1815
- Where
- 54 sites in United States, Australia and 4 more
- Volunteers
- 287 people
- Sponsor
- Evelo Biosciences, Inc.
Afibrinogenemia, Hypofibrinogenemia
Fibrinogen Concentrate (Human) - Efficacy and Safety Study
This is a multinational, multicenter, prospective, open-label historically controlled Phase IIIb non-inferiority clinical trial on the efficacy and safety of Fibrinogen Concentrate (Human).
- Conditions
- Afibrinogenemia, Hypofibrinogenemia, Fibrinogen Deficiency
- Trial name
- NCT00916656
- Testing
- Fibrinogen Concentrate, Human (FCH), Cryoprecipitate
- Sponsor
- CSL Behring
OTOF Gene Mutation, DFNB9
A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene.
- Conditions
- OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders
- Trial name
- AUDIOGENE (NCT06370351)
- Testing
- SENS-501 administration
- Where
- 2 sites in Australia, France
- Volunteers
- 12 people
- Sponsor
- Sensorion
Spinocerebellar Degeneration
Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase III study to evaluate the efficacy and safety of KPS-0373 given once daily after breakfast in 142 patients with spinocerebellar…
- Trial name
- NCT07040137
- Testing
- KPS-0373
- Where
- Japan
- Volunteers
- 142 people
- Sponsor
- Kissei Pharmaceutical Co., Ltd.
Retinitis Pigmentosa
Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
This study is a multicenter trial performed in Japan, consisting of a comparative study period and a continuous administration period.
- Trial name
- NCT01786395
- Testing
- UF-021
- Where
- 34 sites in Japan
- Volunteers
- 202 people
- Sponsor
- R-Tech Ueno, Ltd.
Vascular Anomalies
A Study of Clinical Practice and Real-world Care of Patients Treated With mTOR, PI3K, and MEK Inhibitors for Extracranial Vascular Anomalies
This study aims to describe real-world patient characteristics, treatment patterns, and adverse events associated with targeted therapies used in patients with complex vascular anomalies.
- Trial name
- NCT07767877
- Volunteers
- 200 people
- Sponsor
- Novartis Pharmaceuticals
Congenital Myotonic Dystrophy
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
This treatment plan is limited to a single patient with Congenital Myotonic Dystrophy, who is ineligible or otherwise unable to participate in ongoing clinical trials.
- Trial name
- NCT07119775
- Testing
- Tideglusib
- Sponsor
- AMO Pharma Limited
Duchenne Muscular Dystrophy
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
The GalaxyDMD study is a global Phase 3, open-label, treatment extension study to evaluate the safety, tolerability, and durability of effect in long-term dosing of edasalonexent in pediatric patients with a genetically confirmed…
- Trial name
- GalaxyDMD (NCT03917719)
- Testing
- Edasalonexent
- Where
- 23 sites in United States, Australia and 4 more
- Volunteers
- 130 people
- Sponsor
- Catabasis Pharmaceuticals
Cystic Fibrosis
Drug-Drug Interaction Study of VX-809 and VX-770 in Healthy Subjects
The purpose of this study is to assess the pharmacokinetics and safety of co-administration of VX-770 and VX-809 in healthy adults.
- Trial name
- NCT00966602
- Testing
- VX-809, VX-770, VX-809 & VX-770
- Where
- Netherlands
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cystic Fibrosis
A Study Assessing KB407 for the Treatment of Cystic Fibrosis
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
- Trial name
- NCT05504837
- Testing
- KB407 (Nebulization)
- Where
- 6 sites in United States
- Volunteers
- 20 people
- Sponsor
- Krystal Biotech, Inc.
Prader-Willi Syndrome
A Pharmacokinetic and Safety Study of CSTI-500 in Subjects With Prader-Willi Syndrome
The purpose of this Phase 1 study is to evaluate the pharmacokinetics (PK) and safety of a single dose of CSTI-500 10 mg in subjects with Prader-Willi syndrome (PWS) between 13 and 50 years of age with a genetically confirmed…
- Trial name
- NCT05504395
- Testing
- CSTI-500
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- ConSynance Therapeutics
Patent Foramen Ovale, Stroke
PFO ACCESS Registry
Closure of Patent Foramen Ovale with the AMPLATZER® PFO OCCLUDER in patients with at least two cryptogenic strokes due to presumed paradoxical embolism through a patent foramen ovale and who have failed conventional therapy.
- Trial name
- NCT00583401
- Testing
- Device closure with the AMPLATZER PFO Occluder
- Sponsor
- Abbott Medical Devices
Severe Hemophilia A
Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A
This study was divided into four stages: screening period, main trial period, extension period and follow-up period. In the main trial, both groups received FRSW107 prophylactic therapy.
- Trial name
- NCT06136507
- Testing
- FRSW107
- Where
- 13 sites in China
- Volunteers
- 76 people
- Sponsor
- Hangzhou Gensciences Biopharmaceutical Co., Ltd.
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