Clinical trials: Musculoskeletal Deformity
FSHD, FSHD1
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- FSHD, FSHD1, FSHD2, FMD
- Trial name
- FORTITUDE-OLE (NCT06547216)
- Testing
- AOC 1020
- Where
- 17 sites in United States, Canada and 1 more
- Volunteers
- 84 people
- Sponsor
- Avidity Biosciences, Inc.
Pain
Efficacy and Safety Study of 4975 to Manage Pain of Bunionectomy
The primary purpose of this study is to compare the analgesic efficacy of a single intraoperative administration of 4975 vs. Placebo in the management of acute postoperative pain in patients undergoing bunionectomy.
- Trial name
- NCT00656578
- Testing
- 4975
- Where
- 6 sites in United States
- Volunteers
- 300 people
- Sponsor
- Anesiva, Inc.
Gout
Efficacy and Safety of Oral Febuxostat in Participants With Gout
The purpose of this study is to compare the efficacy and safety of febuxostat, once Daily (QD), to allopurinol in subjects with hyperuricemia and gout.
- Trial name
- CONFIRMS (NCT00430248)
- Testing
- Febuxostat, Allopurinol
- Where
- 258 sites in United States
- Volunteers
- 2,269 people
- Sponsor
- Takeda
Becker Muscular Dystrophy
A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight <50 kg) daily…
- Trial name
- NCT05166109
- Testing
- Vamorolone
- Where
- 2 sites in United States, Italy
- Volunteers
- 46 people
- Sponsor
- ReveraGen BioPharma, Inc.
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Muscular Dystrophies
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to explore long-term safety, tolerability and efficacy of GSK2402968 in DMD subjects who previously participated in either DMD114117 or DMD114044.
- Trial name
- NCT01480245
- Testing
- GSK2402968
- Where
- 59 sites in Argentina, Australia and 22 more
- Volunteers
- 233 people
- Sponsor
- GlaxoSmithKline
Noonan Syndrome
French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
This is a non-interventional registry of children treated with Norditropin® for short stature due to Noonan Syndrome (NS). This study aims to provide data on long-term growth evolution and safety of Norditropin® as well as Health…
- Trial name
- NCT05308927
- Testing
- Norditropin
- Where
- 3 sites in France
- Volunteers
- 221 people
- Sponsor
- Novo Nordisk A/S
DiGeorge Syndrome, Hypoparathyroidism
Parathyroid and Thymus Transplantation in DiGeorge #931
This study has three primary purposes: to assess parathyroid function after parathyroid transplantation in infants with Complete DiGeorge syndrome; to assess immune function development after transplantation; and, to assess…
- Conditions
- DiGeorge Syndrome, Hypoparathyroidism, Complete DiGeorge Syndrome
- Trial name
- NCT00566488
- Testing
- Thymus/Parathyroid Transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Gout
MPC-004 for the Treatment of an Acute Gout Flare
This study is a multicenter, randomized, double-blind, placebo-controlled, parallel group, dose-comparison to determine the efficacy and safety of a standard-dose of colchicine (4.8 mg) versus low-dose colchicine (1.8 mg) or…
- Trial name
- AGREE (NCT00506883)
- Testing
- High Dose Colchicine (4.8 mg total dose), Low Dose Colchicine (1.8mg total dose)
- Where
- 83 sites in United States
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- Takeda
Duchenne Muscular Dystrophy
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03039686
- Testing
- RO7239361
- Where
- 44 sites in United States, Argentina and 11 more
- Volunteers
- 166 people
- Sponsor
- Hoffmann-La Roche
Basal Cell Nevus Syndrome
Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Basal Cell Nevus Syndrome (Gorlin Syndrome)
This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with Gorlin…
- Trial name
- NCT03703310
- Testing
- Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
- Where
- 49 sites in United States, Belgium and 8 more
- Volunteers
- 174 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Pectus Excavatum
Pectus Excavatum Camouflage
The clinical study is testing a new treatment for pectus excavatum called "custom-made 3D printed scaffold-based soft tissue reconstruction".
- Trial name
- NCT05451108
- Testing
- PCL Pectus Scaffold implantation and autologous fat grafting
- Where
- Australia
- Volunteers
- 7 people
- Sponsor
- BellaSeno Pty Ltd
Knee Osteoarthritis, Bone; Deformity, Congenital
COREV 2.0 PMCF-Study on Manual and Navigated Revision Knee Patients
The Columbus® Revision endoprosthetic system is bearing the CE-mark. This means that within the scope of this observational study the patient will not be subjected to any additional stressful, study-related medical examinations…
- Conditions
- Knee Osteoarthritis, Bone; Deformity, Congenital, Prosthesis Failure, Prosthesis-Related Infections
- Trial name
- COREV (NCT05113303)
- Testing
- Navigated Revision Total Knee Arthroplasty, Manual Revision Total Knee Arthroplasty
- Where
- Spain
- Volunteers
- 100 people
- Sponsor
- Aesculap AG
Bunionectomy, Hallux Valgus
Phase 3, Sciatic Nerve Block With EXPAREL for Subjects Undergoing Bunionectomy
The study is conducted sequentially in two parts. Part A: The purpose is to obtain information on pharmacokinetic (PK) profile, pharmacodynamics (PD), efficacy, safety, and to assess the performance of 266 mg EXPAREL vs 133 mg…
- Trial name
- NCT05157841
- Testing
- Bupivacaine liposome injectable suspension 266 mg, Bupivacaine HCl, Bupivacaine liposome injectable suspension 133 mg
- Where
- 7 sites in United States
- Volunteers
- 185 people
- Sponsor
- Pacira Pharmaceuticals, Inc
Muscular Dystrophy, Duchenne
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
- Trial name
- NCT05429372
- Testing
- PF-06939926
- Where
- 13 sites in United States, Australia
- Volunteers
- 10 people
- Sponsor
- Pfizer
Blepharospasm, Hemifacial Spasm
Post Marketing Surveillance Study of Dysport
The purpose of this study is to provide further information regarding the risks and benefits of Dysport in marketed indications.
- Conditions
- Blepharospasm, Hemifacial Spasm, Cervical Dystonia, Spasmodic Torticollis
- Trial name
- NCT00210431
- Where
- 36 sites in France, Germany and 1 more
- Volunteers
- 783 people
- Sponsor
- Ipsen
Spinal Muscular Atrophy, Spine Deformity
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
- Conditions
- Spinal Muscular Atrophy, Spine Deformity, Scoliosis
- Trial name
- PIERRE (NCT05866419)
- Testing
- ThecaFlex DRx System
- Where
- 21 sites in United States, Germany and 3 more
- Volunteers
- 90 people
- Sponsor
- Alcyone Therapeutics, Inc
Duchenne Muscular Dystrophy
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.
- Trial name
- NCT04060199
- Testing
- Viltolarsen
- Where
- 40 sites in United States, Australia and 17 more
- Volunteers
- 77 people
- Sponsor
- NS Pharma, Inc.
Advanced Stage Solid Tumors
First in Human Dose Escalation Study of Vactosertib (TEW-7197) in Subjects With Advanced Stage Solid Tumors
The phase I dose escalation study will investigate the safety, tolerability, and pharmacokinetics of the TGF-β pathway inhibitor TEW 7197 in subjects with advanced, refractory solid tumors.
- Trial name
- NCT02160106
- Testing
- TEW-7197
- Where
- 3 sites in United States
- Volunteers
- 35 people
- Sponsor
- MedPacto, Inc.
Gout
Review of Safety Using Rilonacept in Preventing Gout Exacerbations (RE-SURGE)
The purpose of this clinical research study is to determine the safety and effectiveness of an experimental drug called rilonacept in subjects with gout who are on urate-lowering therapy.
- Trial name
- NCT00856206
- Testing
- Rilonacept
- Where
- 124 sites in United States, Germany and 4 more
- Volunteers
- 1,315 people
- Sponsor
- Regeneron Pharmaceuticals