Clinical trials: Congenital Deformity
Pompe Disease (Late-onset)
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric…
- Trial name
- NCT03911505
- Testing
- Cipaglucosidase Alfa, Miglustat
- Where
- 17 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- Amicus Therapeutics
Acute Hepatic Porphyria
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
This global patient registry is being conducted to characterize the natural history and real-world clinical management of patients with AHP, and to further characterize the real-world safety and effectiveness of givosiran and…
- Trial name
- ELEVATE (NCT04883905)
- Where
- 30 sites in United States, Belgium and 8 more
- Volunteers
- 150 people
- Sponsor
- Alnylam Pharmaceuticals
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Hereditary Angioedema
A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).
- Trial name
- NCT04654351
- Testing
- TAK-667
- Where
- 3 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Takeda
Dermatitis, Atopic
Crisaborole Ointment 2% Skin Biomarker Biopsy Study in Atopic Dermatitis
This study is being conducted to characterize the mechanism of action of crisaborole ointment 2%, by evaluation of efficacy and changes in key skin biomarkers in atopic dermatitis (AD) lesions treated with crisaborole ointment 2%…
- Trial name
- NCT03233529
- Testing
- Crisaborole ointment 2% BID
- Where
- Canada
- Volunteers
- 40 people
- Sponsor
- Pfizer
Eyelid Diseases, Eyelid Movement Disorders
Feasibility of Dynamic Muscle Stimulation + Radiofrequency for Improving Blink Quality in Subjects with Dry Eye Disease
To demonstrate that DMSt + RF improves eye blink quality in subjects with dry eye disease
- Conditions
- Eyelid Diseases, Eyelid Movement Disorders, Dry Eye, Dry Eye Syndromes
- Trial name
- NCT05945069
- Testing
- TriLift device
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Lumenis Be Ltd.
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
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