Clinical trials: Newborn Diseases
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
Advanced Solid Tumors
Phase I Trial of Oral PX-866
This study is being conducted to determine the safety and maximally tolerated dose of PX-866 when given orally on two different schedules: daily on days 1-5 and 8-12 of a 28 day cycle and daily on days 1-28 of a 28 day cycle.
- Trial name
- NCT00726583
- Testing
- PX-866
- Where
- 2 sites in United States
- Volunteers
- 90 people
- Sponsor
- Cascadian Therapeutics Inc.
Sleep Disorders
Efficacy and Safety of Circadin® in the Treatment of Sleep Disturbances in Children With Neurodevelopment Disabilities
The purpose of this study is to establish the efficacy and safety of Circadin in children with neurodevelopmental disorders and to determine the dose, this randomized, placebo-controlled study is planned to evaluate the efficacy…
- Trial name
- NCT01906866
- Testing
- Circadin 2/5/10 mg
- Where
- 28 sites in United States, Finland and 3 more
- Volunteers
- 125 people
- Sponsor
- Neurim Pharmaceuticals Ltd.
Hemophilia
First-in-Human and Proof-of-Mechanism Study of ARC19499 Administered to Hemophilia Patients
The purpose of this study is to examine the safety, tolerability and the way the body handles various single and multiple doses of ARC19499 in patients with hemophilia.
- Trial name
- NCT01191372
- Testing
- ARC19499
- Where
- United Kingdom
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire
Von Willebrand Diseases
Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)
The VIVID study is structured in a master protocol format comprised of multiple parts that evaluate intravenous (IV) and subcutaneous (SC) VGA039 in healthy volunteers and subjects with von Willebrand Disease (VWD) and other…
- Trial name
- NCT05776069
- Testing
- VGA039
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 116 people, healthy volunteers welcome
- Sponsor
- Vega Therapeutics, Inc
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease
To collect characteristics of patients with ADPKD across a broad population, over time to better understand disease progression (signs, symptoms and outcomes).
- Trial name
- OVERTURE (NCT01430494)
- Where
- 266 sites in United States, Argentina and 18 more
- Volunteers
- 3,409 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Cytomegalovirus Retinitis, HIV Infections
A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )
To determine the clinically safe and effective dose of intravitreal ISIS 2922 alone and as an additive antiviral therapy to ganciclovir in AIDS patients with cytomegalovirus (CMV) retinitis.
- Trial name
- NCT00002156
- Testing
- Fomivirsen sodium, Ganciclovir
- Where
- 18 sites in United States
- Volunteers
- 194 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Cow Milk Allergy
Symptoms Based Awareness Confirmation Study - CoMiSS Validation
The Cow's Milk-related Symptom Score (CoMiSS) tool has been developed to define a score to recognize cow's milk related symptoms in infants and young children.
- Trial name
- MOSAIC (NCT03004729)
- Testing
- CoMiSS questionnaire
- Where
- 10 sites in China
- Volunteers
- 300 people
- Sponsor
- Société des Produits Nestlé (SPN)
Acute Gouty Arthritis, Gout Flare
Safety and Efficacy of ABP-745 in Participants With an Acute Gout Flare
A randomized, double-blind, international multicenter, colchicine and placebo-controlled study to evaluate the efficacy and safety of ABP-745 in subjects with acute gout.
- Conditions
- Acute Gouty Arthritis, Gout Flare, Gout Flares, Acute Gout Flare
- Trial name
- NCT07145229
- Testing
- ABP-745 Dose A, ABP-745 Dose B, Colchicine
- Where
- 38 sites in United States, Australia and 1 more
- Volunteers
- 479 people
- Sponsor
- Atom Therapeutics Co., Ltd
Lennox Gastaut Syndrome, Dravet Syndrome
Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics.
- Conditions
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- Trial name
- EpiTALY (NCT05485831)
- Testing
- Epidiolex 100 mg/mL Oral Solution
- Where
- 18 sites in Italy
- Volunteers
- 111 people
- Sponsor
- Jazz Pharmaceuticals
Venous Stenosis, Venous Occlusion
The Merit WRAPSODY AV Access Efficacy Study (WAVE)
The purpose of the study is to demonstrate the safety and efficacy of the Merit WRAPSODY Endovascular Stent Graft for treatment of stenosis or occlusion within the dialysis access outflow circuit
- Trial name
- WAVE (NCT04540302)
- Testing
- Merit WRAPSODY Endovascular Stent Graft, PTA
- Where
- 43 sites in United States, Brazil and 2 more
- Volunteers
- 357 people
- Sponsor
- Merit Medical Systems, Inc.
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
Mucopolysaccharidosis Type IIIB
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B).
- Trial name
- NCT03227042
- Where
- 9 sites in United States, Argentina and 7 more
- Volunteers
- 44 people
- Sponsor
- Allievex Corporation
Phenylketonurias
Evaluation of PKU Sphere Liquid
PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.
- Trial name
- NCT05096988
- Testing
- PKU sphere liquid
- Where
- 3 sites in United Kingdom
- Volunteers
- 19 people
- Sponsor
- Vitaflo International, Ltd
Arteriovenous Fistula
Paclitaxel-Coated Balloon for the Treatment of AVF
To evaluate the safety and efficacy of the Zylox Drug Coated Balloon (DCB) for treatment of subjects presenting with de novo or non-stented restenotic obstructive lesion of native arteriovenous dialysis fistulae (AVF) in the…
- Trial name
- AVF (NCT05088083)
- Testing
- Paclitaxel-Coated Percutaneous Transluminal Angioplasty (PTA) Balloon
- Volunteers
- 143 people
- Sponsor
- Zhejiang Zylox Medical Device Co., Ltd.
X-Linked Hypophosphatemia
Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
The primary objective of this study is to evaluate the effect of KRN23 (burosumab) therapy in improving rickets in children with XLH compared with active control (oral phosphate/active vitamin D).
- Trial name
- NCT02915705
- Testing
- burosumab, Oral Phosphate Supplement, active vitamin D
- Where
- 16 sites in United States, Australia and 5 more
- Volunteers
- 61 people
- Sponsor
- Kyowa Kirin, Inc.
VHL Gene Mutation, VHL
MK-3795 (PT2385) for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma (MK-3795-003)
The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL participants treated with MK-3795.
- Conditions
- VHL Gene Mutation, VHL, VHL Syndrome, VHL Gene Inactivation
- Trial name
- NCT03108066
- Testing
- MK-3795
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis
An Extension Trial of Inclisiran in Participants With Cardiovascular Disease and High Cholesterol
This clinical study was designed to assess the efficacy, safety, and tolerability of long-term dosing of inclisiran and evolocumab given as subcutaneous injections in participants with high cardiovascular risk and elevated…
- Conditions
- Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis, Type2 Diabetes, Familial Hypercholesterolemia
- Trial name
- ORION-3 (NCT03060577)
- Testing
- Inclisiran, Evolocumab
- Where
- 52 sites in United States, Canada and 3 more
- Volunteers
- 382 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy 1
Long-Term Follow-up Study for Patients From AVXS-101-CL-101
This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi.
- Trial name
- START (NCT03421977)
- Testing
- Onasemnogene Abeparvovec-xioi
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Gene Therapies
Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Biomarker for Transthyretin-Related Familial Amyloidotic Polyneuropathy (BioTRAP)
International, multicenter, observational, longitudinal study to identify biomarker/s for the development of a new MS-based biomarker for the early and sensitive diagnosis of Transthyretin-Related Familial Amyloidotic…
- Conditions
- Transthyretin Amyloidosis, Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin Amyloid Cardiopathy
- Trial name
- BioTRAP (NCT02713880)
- Where
- 2 sites in Germany, India
- Sponsor
- CENTOGENE GmbH Rostock
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