Clinical trials: Congenital Anomaly
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.
- Conditions
- Focal Segmental Glomerulosclerosis, Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis
- Trial name
- EPPIK (NCT05003986)
- Testing
- Sparsentan
- Where
- 47 sites in United States, Germany and 6 more
- Volunteers
- 67 people
- Sponsor
- Travere Therapeutics, Inc.
Pulmonary Valve Insufficiency, Complex Congenital Heart Defect
ALTERRA Post-Approval Study
This study will monitor device performance and outcomes in subjects undergoing implantation of the Edwards SAPIEN 3 Transcatheter Pulmonary Valve System with Alterra Adaptive Prestent in the post-approval setting
- Conditions
- Pulmonary Valve Insufficiency, Complex Congenital Heart Defect, Tetralogy of Fallot, Pulmonary Regurgitation
- Trial name
- NCT05378386
- Testing
- Edwards Alterra Adaptive Prestent with SAPIEN 3 Transcatheter Pulmonary Valve System
- Where
- 24 sites in United States, Israel
- Volunteers
- 150 people
- Sponsor
- Edwards Lifesciences
Epidermolysis Bullosa
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development
- Trial name
- NCT02090283
- Testing
- SD-101 dermal cream (6%)
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Scioderm, Inc.
Atopic Dermatitis
Efficacy Emollient on Xerosis in Children With Atopic Dermatitis
Atopic dermatitis is a frequent, chronic inflammatory disease influenced by local, immunological, genetic and environmental factors.
- Trial name
- NCT01467362
- Testing
- V0034CR01B, Vehicle cream, desonide 0.1% cream
- Where
- 25 sites in Estonia, France and 3 more
- Volunteers
- 251 people
- Sponsor
- Pierre Fabre Medicament
Atopic Dermatitis
Efficacy and Safety of MEDI3506 in Adult Subjects With Atopic Dermatitis
This is a research study to determine the efficacy and safety of investigational drug MEDI3506 for the treatment of adult subjects with Atopic Dermatitis.
- Trial name
- NCT04212169
- Testing
- MEDI3506
- Where
- 38 sites in United States, Australia and 4 more
- Volunteers
- 148 people
- Sponsor
- AstraZeneca
Prader-Willi Syndrome
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin).
- Trial name
- CARE-PWS (NCT03649477)
- Testing
- 3.2 mg intranasal carbetocin, 9.6 mg intranasal carbetocin
- Where
- 24 sites in United States, Australia and 1 more
- Volunteers
- 130 people
- Sponsor
- Levo Therapeutics, Inc.
Lysosomal Storage Disease, Lung Diseases
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Maroteaux-Lamy disease from blood
- Conditions
- Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia
- Trial name
- BioMaroteaux (NCT01458613)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Eczema, Dermatitis
Efficacy and Safety Study of Desloratadine (MK-4117) in Japanese Participants With Eczema/Dermatitis and Dermal Pruritus (MK-4117-202)
This is an efficacy and safety study of up to 12 weeks of desloratadine in Japanese participants with eczema/dermatitis and dermal pruritus.
- Conditions
- Eczema, Dermatitis, Dermal Pruritus
- Trial name
- NCT01916980
- Testing
- Desloratadine 5 mg
- Volunteers
- 94 people
- Sponsor
- Organon and Co
Healthy, Long QT Syndrome
Study Evaluating the Effects of Multiple Oral Doses of ERB-041 on Cardiac Repolarization in Healthy Subjects
To assess the effect of ERB-041 after multiple oral doses on cardiac repolarization as assessed by the QTc interval.
- Trial name
- NCT00316459
- Testing
- ERB-041
- Where
- United States
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Sickle Cell Anemia
Prasugrel Versus Placebo in Adult Sickle Cell Disease
The purpose of this trial is to assess the safety of Prasugrel in adult patients with sickle cell disease (SCD) by monitoring the rate and severity of hemorrhagic events requiring medical intervention compared to placebo for 30…
- Trial name
- NCT01167023
- Testing
- Prasugrel
- Where
- 14 sites in United States
- Volunteers
- 62 people
- Sponsor
- Eli Lilly and Company
Atopic Dermatitis
Evaluation of FB-401 in Children, Adolescents and Adults (2 Years and Older) With Mild to Moderate Atopic Dermatitis
The purpose of this study is to evaluate the potential improvement in atopic dermatitis signs and symptoms following the application of FB-401 in patients 2 years or older with mild to moderate atopic dermatitis.
- Trial name
- NCT04504279
- Testing
- FB-401
- Where
- 15 sites in United States
- Volunteers
- 154 people
- Sponsor
- Forte Biosciences, Inc.
Spinal Fusion, Cervical Vertebral Fusion
Study of Titanium-Coated PEEK Cages for Degenerative Disc Disease
Prospective Follow-up study of CeSPACE XP coated PEEK cages for the treatment of degenerative cervical disc disease
- Trial name
- NCT03565224
- Where
- Spain
- Volunteers
- 50 people
- Sponsor
- Aesculap AG
Friedreich Ataxia
A Study to Assess Variation in Potential Biomarkers in Friedreich Ataxia
To test the variability of specific ribonucleic acid (RNA) and proteins as well as frataxin levels in samples of blood and buccal cells taken directly from patients with Friedreich's ataxia (FRDA) in order to confirm potential…
- Trial name
- NCT04255680
- Testing
- Buccal Swabs and Blood Draws
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Larimar Therapeutics, Inc.
Hemophilia B
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events.
- Trial name
- Honey-B (NCT05568459)
- Testing
- Non-Interventional
- Where
- 17 sites in United States, Canada and 2 more
- Volunteers
- 11 people
- Sponsor
- Regeneron Pharmaceuticals
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study…
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Trial name
- NCT02698579
- Testing
- No interventional drug product utilized in this follow-up study
- Where
- 13 sites in United States, Argentina and 7 more
- Volunteers
- 64 people
- Sponsor
- Genetix Biotherapeutics Inc.
Epidermolysis Bullosa
A Study of the Efficacy and Safety of ABH001 in the Treatment of Patients With Epidermolysis Bullosa Who Have Wounds That Are Not Healing
The purpose of this study is to evaluate the efficacy and safety of ABH001 in the treatment of patients with epidermolysis bullosa who have wounds that are not healing.
- Trial name
- NCT01749306
- Testing
- ABH001, Control wound treatment
- Where
- 14 sites in United States, Austria and 6 more
- Volunteers
- 1 people
- Sponsor
- Shire Regenerative Medicine, Inc.
Huntington's Disease
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).
- Trial name
- NCT03664804
- Testing
- No Study Drug was Administered in this Study
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Atopic Dermatitis, Atopic Dermatitis Eczema
The Efficacy of NTU 101 Lactic Acid Bacteria Powder in the Adjuvant Improvement of Atopic Dermatitis Clinical Trial
This study is a randomized, double-blind, placebo-controlled, parallel-controlled trial (14 weeks in total), divided into three periods (screening, treatment, and discontinuation follow-up)
- Trial name
- NCT05719649
- Testing
- Lactic acid bacteria NTU 101
- Volunteers
- 72 people
- Sponsor
- SunWay Biotech Co., LTD.
Neurofibromatosis Type 1
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN.
- Trial name
- INSPIRE-NF1 (NCT06541847)
- Testing
- HLX-1502
- Where
- 14 sites in United States
- Volunteers
- 25 people
- Sponsor
- Healx Limited
Lower Urinary Tract Symptoms, Bladder Outlet Obstruction
A Study of Mirabegron (YM178) in Men With Lower Urinary Tract Symptoms (LUTS) and Bladder Outlet Obstruction (BOO)
This study examined the safety, tolerability, and efficacy of mirabegron (YM178) compared to placebo.
- Trial name
- NCT00410514
- Testing
- Mirabegron
- Where
- 27 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- Astellas Pharma Inc
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