Clinical trials: Newborn Diseases
Atopic Dermatitis
Real-world Effectiveness Study of Long-term Treatment With Dupilumab in Participants ≥6 Years With Atopic Dermatitis
This is a Prospective, non-interventional (NIS) observational study in patients (≥6 years) with atopic dermatitis (AD) receiving dupilumab for the prospective evaluation of signs and symptoms, quality of life and disease control.
- Trial name
- PROTYPE2 (NCT06039241)
- Where
- 55 sites in Germany
- Volunteers
- 572 people
- Sponsor
- Sanofi
Cystic Fibrosis
Study Assessing PTI-428 Safety, Tolerability, Pharmacokinetics and Effect in Subjects With Cystic Fibrosis
The study population is comprised of adult subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation and are currently receiving background treatment with tezacaftor/ivacaftor for a minimum of 1 month prior…
- Trial name
- NCT03591094
- Testing
- PTI-428
- Where
- 26 sites in United States
- Volunteers
- 40 people
- Sponsor
- Proteostasis Therapeutics, Inc.
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.
Familial Adenomatous Polyposis
Effect of EPA-FFA on Polypectomy in Familial Adenomatous Polyposis
2 Year randomised, double-blind, placebo-controlled, parallel group study to determine the safety and efficacy of EPA-FFA gastro resistant capsules in FAP.
- Trial name
- NCT03806426
- Testing
- Eicosapentaenoic acid free fatty acid (EPA-FFA)
- Where
- Italy
- Volunteers
- 204 people
- Sponsor
- S.L.A. Pharma AG
Gaucher Disease, Type 1
A Gene Therapy Study in Patients With Gaucher Disease Type 1
This study is a first-in-human, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1.
- Trial name
- GALILEO-1 (NCT05324943)
- Testing
- FLT201
- Where
- 10 sites in United States, Brazil and 4 more
- Volunteers
- 10 people
- Sponsor
- Spur Therapeutics
Atopic Dermatitis
Phase 2 Study of OPA-15406 Ointment in Pediatric Patients With Atopic Dermatitis
To evaluate the safety, efficacy (dose response) and pharmacokinetics of 0.3% and 1% OPA-15406 when applied twice daily for 4 weeks in pediatric patients with atopic dermatitis.
- Trial name
- NCT03018691
- Testing
- OPA-15406
- Where
- 4 sites in Japan
- Volunteers
- 73 people
- Sponsor
- Otsuka Pharmaceutical Co., Ltd.
Mucopolysaccharidosis III-B
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.
- Trial name
- NCT06488924
- Testing
- JR-446
- Where
- 4 sites in Japan
- Volunteers
- 10 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Duchene Muscular Dystrophy
A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine…
- Trial name
- PROVIDUS (NCT07127978)
- Where
- 9 sites in United States
- Volunteers
- 300 people
- Sponsor
- ITF Therapeutics LLC
Dermatitis, Atopic
An Observational Study Investigating the Treatment Satisfaction of Atopic Dermatitis Patients Who Have Switched to Ointment (W/O Emulsion) Formulation of Very Strong Class Topical Corticosteroids
This study intends to investigate patient satisfaction of treatment with Ointment (W/O emulsion) formulations of Potency Class II topical corticosteroids (TCs) for atopic dermatitis (AD) patients in Japan.
- Trial name
- Ointment TS-AD (NCT02627989)
- Testing
- Diflucortolone valerate (Nerisona), Diflucortolone valerate (Texmeten)
- Where
- Japan
- Volunteers
- 55 people
- Sponsor
- LEO Pharma
Dermatitis, Atopic
Treatment and Control of Atopic Dermatitis With 0.1% Tacrolimus Ointment
Long-term tacrolimus ointment based regimen comprising of up to 6 weeks of initial twice daily treatment and subsequent twice weekly prophylactic application can effectively treat active lesions of atopic dermatitis and prevent…
- Trial name
- NCT00480610
- Testing
- tacrolimus ointment
- Where
- 22 sites in Austria, Belgium and 11 more
- Volunteers
- 226 people
- Sponsor
- Astellas Pharma Inc
Cystic Fibrosis
Study of Ataluren (PTC124) in Cystic Fibrosis
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study…
- Trial name
- NCT02107859
- Testing
- Ataluren
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 61 people
- Sponsor
- PTC Therapeutics
Polycystic Kidney, Autosomal Recessive
A Safety, Pharmacokinetic, Single Ascending Dose Study of Tesevatinib in Pediatric Subjects With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
This study evaluates safety and tolerability of a single ascending dose of a tesevatinib liquid formulation administered to pediatric subjects with ARPKD.
- Trial name
- NCT03096080
- Testing
- Tesevatinib
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Kadmon Corporation, LLC
Dermatitis, Atopic Dermatitis
Efficacy and Safety Study of Apremilast in Subjects With Moderate to Severe Atopic Dermatitis
A study to evaluate the efficacy and safety of apremilast (CC-10004) in subjects with moderate to severe atopic dermatitis
- Trial name
- NCT02087943
- Testing
- Apremilast
- Where
- 31 sites in United States, Canada and 1 more
- Volunteers
- 191 people
- Sponsor
- Amgen
Intracranial Arteriovenous Malformations
Non-adhesive Liquid Embolic System in the Embolization of Cerebral Arteriovenous Malformations
This clinical trial is to verify the safety and effectiveness of the Non-adhesive Liquid Embolic System(NALES) produced by Suzhou Hengrui Hongyuan Medical Technology Co., Ltd.
- Trial name
- NCT05058482
- Testing
- embolism
- Where
- 13 sites in China
- Volunteers
- 116 people
- Sponsor
- Suzhou Hengruihongyuan Medical Technology Co. LTD
Hemophilia B
Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
A Phase 1/2, open-label, dose-finding safety study of single ascending doses of DTX101 in adult males with moderate/severe to severe hemophilia B.
- Trial name
- NCT02618915
- Testing
- DTX101
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Rheumatoid Arthritis, Osteoarthritis
A Multi-centre Study to Assess the Long-term Performance of the Summit™ Hip in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Summit™ hip in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00208390
- Testing
- Summit Tapered Hip System
- Where
- 5 sites in China, Hong Kong and 3 more
- Volunteers
- 275 people
- Sponsor
- DePuy International
Arrhythmogenic Right Ventricular Cardiomyopathy
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.
- Trial name
- RIDGE-1 (NCT06228924)
- Testing
- TN-401
- Where
- 7 sites in United States
- Volunteers
- 15 people
- Sponsor
- Tenaya Therapeutics
Myotonic Dystrophy 1
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy.
- Trial name
- NCT02858908
- Testing
- Tideglusib
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- AMO Pharma Limited
Mucopolysaccharidosis I, Hurler's Syndrome
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Trial name
- NCT00144768
- Testing
- laronidase
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Atopic Dermatitis
Efficacy and Safety of Orally Administered DS107 in Adult Patients With Moderate to Severe Atopic Dermatitis
The objective of this study is to compare the efficacy and safety of orally administered DS107 (2g) versus placebo in the treatment of moderate to severe Atopic Dermatitis (AD).
- Trial name
- NCT03817190
- Testing
- DS107
- Where
- 50 sites in United States, Austria and 3 more
- Volunteers
- 219 people
- Sponsor
- DS Biopharma
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