Clinical trials: Congenital Deformity
Duchenne Muscular Dystrophy
Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
The VILT-502 study is Non-interventional Study(United States)/Low-intervention Clinical Trial (Canada) of Viltolarsen administered intravenously once weekly for 10 years to boys with DMD who complete the NS-065/NCNP-01-202 study.
- Trial name
- NCT04687020
- Testing
- Viltolarsen
- Where
- 5 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- NS Pharma, Inc.
Immunologic Deficiency Syndrome
Phase 3 Study of Immune Globulin Intravenous (Human)IVIG-SN™ in Subjects With Primary Immunodeficiency
The purpose of this study is to evaluate the safety, efficacy and pharmacokinetics of Immune Globulin Intravenous (Human) IVIG-SN™ in subjects with primary immunodeficiency diseases.
- Trial name
- NCT01406470
- Testing
- Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™
- Where
- 11 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Green Cross Corporation
Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Crinecerfont in Participants With Classic Congenital Adrenal Hyperplasia (CAH) Who Are Less Than 4 Years Old
The main objective of this study is to assess the safety and tolerability of crinecerfont in pediatric participants 3 months to <4 years of age with CAH.
- Trial name
- NCT07536269
- Testing
- Crinecerfont
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Neurocrine Switzerland GmbH
Myotonic Dystrophy Type 1, DM1
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy…
- Conditions
- Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia
- Trial name
- HARBOR-OLE (NCT07008469)
- Testing
- Del-desiran (AOC 1001)
- Where
- 35 sites in United States, Canada and 8 more
- Volunteers
- 199 people
- Sponsor
- Avidity Biosciences, Inc.
Cystic Fibrosis
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele or phenotypically…
- Trial name
- NCT04126473
- Testing
- ELX-02, Ivacaftor
- Where
- 9 sites in Australia, Germany and 1 more
- Volunteers
- 17 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Behcet's Disease, Behcet Syndrome
Clinical Study of TA-650 in Patients With Behcet's Disease (BD) With Special Lesions
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of TA-650 in patients with Behcet's disease ( BD ) with special lesions after the administration of TA-650 at a dosage of 5 mg/kg in weeks 0, 2…
- Conditions
- Behcet's Disease, Behcet Syndrome, Neuro-Behcet's Disease
- Trial name
- NCT01532570
- Testing
- TA-650
- Where
- 6 sites in Japan
- Volunteers
- 18 people
- Sponsor
- Tanabe Pharma Corporation
Diabetic Retinopathy, Retinal Vein Occlusion
Computer Aided Diagnosis of Multiple Eye Fundus Diseases From Color Fundus Photograph
Blindness can be caused by many ocular diseases, such as diabetic retinopathy, retinal vein occlusion, age-related macular degeneration, pathologic myopia and glaucoma.
- Conditions
- Diabetic Retinopathy, Retinal Vein Occlusion, Retinal Artery Occlusion, Central Serous Chorioretinopathy
- Trial name
- NCT04723160
- Testing
- Software assisted imaging diagnosis
- Where
- 5 sites in China
- Volunteers
- 748 people, healthy volunteers welcome
- Sponsor
- Visionary Intelligence Ltd.
Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa
Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
This trial will create a skin graft, which the investigators call "LEAES," using the patient's own skin cells that have been genetically engineered in the lab to express a missing protein called type VII collagen.
- Trial name
- NCT01263379
- Testing
- LZRSE-Col7A1 Engineered Autologous Epidermal Sheets
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Abeona Therapeutics, Inc
Acute Gouty Arthritis
Canakinumab in the Treatment of Gouty Arthritis Flare(s) and Prevention of New Flares in Patients With Chronic Kidney Disease
The purpose of this study is to confirm the efficacy and safety results obtained in a chronic kidney disease (CKD) subgroup of patients participating in Phase III studies in a larger, independent patient population with difficult…
- Trial name
- NCT01593527
- Testing
- ACZ885, Triamcinelone acetonide
- Sponsor
- Novartis Pharmaceuticals
Friedreich's Ataxia
A Study Investigating the Safety and Tolerability of Deferiprone in Patients With Friedreich's Ataxia
The primary objective of this study is to demonstrate the safety and tolerability of deferiprone in subjects with Friedreich's ataxia (FRDA).
- Trial name
- NCT00530127
- Testing
- deferiprone
- Where
- 6 sites in Australia, Belgium and 4 more
- Volunteers
- 80 people
- Sponsor
- ApoPharma
Dilated Cardiomyopathy
A Study to Investigate Safety, Tolerability, and Pharmacokinetics of Oral AZD0233 Compared With Placebo in Healthy Adult Participants.
The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of AZD0233 following single and multiple ascending dose (SAD and MAD) administration in healthy participants.
- Trial name
- NCT06381466
- Testing
- AZD0233
- Where
- United States
- Volunteers
- 84 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Intracranial Aneurysm, Brain Arterial Disease
Safety, Performance and Usability of BALT Medical Devices: The EVIDENCE Post Marketing Clinical Follow-up Platform
BALT has designed an electronic platform to continue collecting clinical data as part of the post-marketing clinical follow-up of its devices.
- Conditions
- Intracranial Aneurysm, Brain Arterial Disease, Acute Ischemic Stroke, Peripheral Vascular Diseases
- Trial name
- EVIDENCE (NCT04927156)
- Testing
- BALT medical devices
- Where
- 12 sites in France, Germany and 1 more
- Volunteers
- 10,000 people
- Sponsor
- Balt Extrusion
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Evaluating the Safety and effectivenesS in Adult KorEaN Patients Treated With Tolvaptan for Management of Autosomal domInAnt poLycystic Kidney Disease
To evaluate the safety and therapeutic effectiveness of tolvaptan when administered to slow the progression of cyst development and renal function insufficiency in adult Korean patients diagnosed with rapidly progressive ADPKD…
- Trial name
- ESSENTIAL (NCT03949894)
- Testing
- Tolvaptan
- Where
- 18 sites in South Korea
- Volunteers
- 118 people
- Sponsor
- Korea Otsuka Pharmaceutical Co., Ltd.
Intracardiac and Septal Defects, Valve and Anulus Repair
Prospective and Non-randomized Registry of CardioCel 3D
Post-market, prospective, multi-centre, open-label, registry designed to collect prospective safety and performance data on the use of CardioCel in patients with cardiovascular disorders and in accordance with local standard of…
- Conditions
- Intracardiac and Septal Defects, Valve and Anulus Repair, Great Vessel Reconstruction, Peripheral Vessel Reconstruction
- Trial name
- NCT04175327
- Testing
- CardioCel implantation
- Where
- 6 sites in Germany, Italy and 2 more
- Volunteers
- 200 people
- Sponsor
- LeMaitre Vascular
Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia
Study to Evaluate the Safety and Efficacy of SPR001 in Subjects With Classic Congenital Adrenal Hyperplasia
This is a Phase 2 study of SPR001 for the treatment of classic CAH that will provide 12 weeks of open-label treatment to eligible subjects.
- Conditions
- Congenital Adrenal Hyperplasia, CAH - Congenital Adrenal Hyperplasia, CAH - 21-Hydroxylase Deficiency
- Trial name
- NCT03687242
- Testing
- SPR001
- Where
- 8 sites in United States
- Volunteers
- 11 people
- Sponsor
- Spruce Biosciences
Thyroid-Associated Ophthalmopathy
Lanreotide (Somatuline Autogel) in Thyroid-associated Ophthalmopathy Treatment
The main purpose of this study is to evaluate the efficacy and safety of 3 doses of Somatuline Autogel 60mg to control the muscle infiltration and edema, eyelid retraction and extraocular muscular contraction in patients with…
- Trial name
- NCT00288522
- Testing
- Lanreotide (Autogel formulation). Duration of treatment - 3 injections, 1 every 28 days
- Where
- Spain
- Volunteers
- 5 people
- Sponsor
- Ipsen
Mucopolysaccharidosis IVA (Morquio A Syndrome)
BMN 110 Phase 3B in Australian Patients
There is currently no treatment for MPS IVA other than supportive care for the clinical manifestations of the disease. Enzyme replacement therapy (ERT) with BMN 110 to replace the deficient GALNS is a potential new treatment…
- Trial name
- MOR-AUS (NCT01966029)
- Testing
- BMN 110
- Where
- 5 sites in Australia
- Volunteers
- 13 people
- Sponsor
- BioMarin Pharmaceutical
Lennox-Gastaut Syndrome
A Placebo-Controlled, Double-Blind Comparative Study of E2080 in Lennox-Gastaut Syndrome Patients (Study E2080-J081-304)
To confirm that the combination therapy of rufinamide has superior efficacy compared to placebo in patients with Lennox-Gastaut syndrome.
- Trial name
- NCT01146951
- Testing
- Rufinamide (E2080)
- Where
- 23 sites in Japan
- Volunteers
- 66 people
- Sponsor
- Eisai Limited
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Congenital Adrenal Hyperplasia
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal…
- Trial name
- CONnECT (NCT05063994)
- Testing
- Chronocort, Cortef
- Where
- 21 sites in United States, France and 1 more
- Volunteers
- 55 people
- Sponsor
- Neurocrine UK Limited
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