Clinical trials: Musculoskeletal Abnormalities
Hyperuricemia, Gout
Phase IIa Study of ALLN-346 (Engineered Urate Oxidase) in Subjects With Hyperuricemia, Gout and Chronic Kidney Disease
The purpose of this Phase IIa study is to evaluate the safety, tolerability and pharmacodynamics of ALLN-346 in subjects with hyperuricemia and gout, and with mild to moderate chronic kidney disease.
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Trial name
- NCT04987294
- Testing
- ALLN-346
- Where
- 17 sites in United States
- Volunteers
- 19 people
- Sponsor
- Allena Pharmaceuticals
Duchenne Muscular Dystrophy (DMD)
Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)
This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- ELEVATE-LTE (NCT07682129)
- Testing
- ENTR-601-44, ENTR-601-45
- Where
- 17 sites in Belgium, Italy and 3 more
- Volunteers
- 80 people
- Sponsor
- Entrada Therapeutics, Inc.
Duchenne Muscular Dystrophy
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and…
- Trial name
- ESSENCE (NCT02500381)
- Testing
- SRP-4045, SRP-4053
- Where
- 76 sites in United States, Argentina and 22 more
- Volunteers
- 228 people
- Sponsor
- Sarepta Therapeutics, Inc.
Tumor or Lymphoma
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of JNJ-42756493 in Adult Participants With Advanced or Refractory Solid Tumors or Lymphoma
The purpose of this study is to evaluate the safety, pharmacokinetics (study of what the body does to a drug), and pharmacodynamics (study of what a drug does to the body) of JNJ-42756493, a pan-fibroblast growth factor receptor…
- Trial name
- NCT01703481
- Testing
- JNJ-42756493: Part 1, JNJ-42756493: Part 2, JNJ-42756493: Part 3
- Where
- 37 sites in United States, France and 1 more
- Volunteers
- 188 people
- Sponsor
- Janssen Research & Development, LLC
Gout, Hyperuricemia
Effect of Tigulixostat on the Pharmacokinetics of Theophylline
A phase 1, open-label, drug-drug interaction study to evaluate the effect of multiple doses of tigulixostat on the pharmacokinetics of single-dose theophylline in healthy adult volunteers.
- Trial name
- NCT06189404
- Testing
- Tigulixostat, Theophylline
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- LG Chem
Muscular Dystrophy, Duchenne, Ambulatory Care
Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
The aim of this Phase III study was to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
- Trial name
- DELOS (NCT01027884)
- Testing
- Idebenone
- Where
- 23 sites in United States, Austria and 8 more
- Volunteers
- 65 people
- Sponsor
- Santhera Pharmaceuticals
Gout, Chronic Kidney Disease (CKD)
A Phase 4 Safety and Efficacy Study to Evaluate Lesinurad 200 mg in Participants With Gout and Renal Impairment
This study evaluates the safety and efficacy of lesinurad administered with an XOI versus a placebo plus an XOI in gout participants who have moderate renal impairment and who are not at target level of serum urate (sUA).
- Trial name
- NCT03226899
- Testing
- Lesinurad, XOI, colchicine
- Where
- 116 sites in United States, Czechia and 2 more
- Volunteers
- 242 people
- Sponsor
- Ironwood Pharmaceuticals, Inc.
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH
The purpose of this study is to assess the safety, pharmacokinetics and efficacy of KRN23 in pediatric Chinese patients with XLH
- Trial name
- NCT04842032
- Testing
- KRN23
- Where
- 5 sites in China
- Volunteers
- 28 people
- Sponsor
- Kyowa Kirin Co., Ltd.
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Foot Deformities, Acquired, Disorder of Joint of Foot
A Clinical Study of Outcomes in Foot and Ankle Bone Grafting Using map3® Cellular Allogeneic Bone Graft
This is a single arm pilot study in patients requiring surgical fusion in the foot or ankle. Patients will receive map3® Cellular Allogeneic Bone Graft containing donor matched stem cells.
- Conditions
- Foot Deformities, Acquired, Disorder of Joint of Foot, Fracture of Foot, Deformity of Bone
- Trial name
- NCT02161016
- Testing
- map3
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- RTI Surgical
Gout, Hyperuricemia
Tigulixostat, Phase 3 Study, Allopurinol Controlled in Gout Patients
The aim of this 12-month randomized multi-regional double-blind parallel group allopurinol and placebo-controlled phase 3 study is to assess the efficacy and safety of three different doses of Tigulixostat in gout patients with…
- Conditions
- Gout, Hyperuricemia, Gout Flare, Tophi
- Trial name
- EURELIA2 (NCT05586971)
- Testing
- Tigulixostat, Allopurinol
- Where
- 279 sites in United States, Argentina and 19 more
- Volunteers
- 2,202 people
- Sponsor
- LG Chem
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Joint Disease
PMCF Study for PROFEMUR® Am Femoral Components and PROCOTYL® O HA Coated Acetabular Components
MicroPort Orthopedics (MPO) is conducting this post market clinical follow-up (PMCF) study to evaluate the safety and efficacy of its total hip arthroplasty (THA) and resurfacing components marketed in the European Union (EU).
- Trial name
- NCT02507258
- Testing
- PROFEMUR® Am Femoral Stem w/ PROCOTYL® O Shell
- Where
- France
- Sponsor
- MicroPort Orthopedics Inc.
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Degenerative Disc Disease, Kyphosis
Post-Market Clinical Follow-up Study of A-SPINE's Products
This is a post-market clinical follow-up study that use questionnaires such as Visual Analog Scale (VAS), the Neck Disability Index (NDI), or the Oswestry Disability Index (ODI) to compare the clinical improvement effects before…
- Conditions
- Degenerative Disc Disease, Kyphosis, Scoliosis, Trauma
- Trial name
- NCT04848376
- Testing
- (1) SmartLoc (2) SmartLoc Evo (3) Winloc(4) Vigor PEEK Cervical Disc Spacer (5) Combo C (6) Polymer Lumbar Disc Spacer (7) X'Plo (8) Rainboo (9) Combo L
- Where
- Taiwan
- Volunteers
- 200 people
- Sponsor
- A-Spine Asia Co., Ltd.
PTCH1 or SMO Activated Solid and Hematologic Tumors
LDE225 for Patients With PTCH1 or SMO Mutated Tumors
The purpose of this signal seeking study is to determine whether treatment with LDE225 demonstrates sufficient efficacy in hedgehog pathway-mutated solid tumors and/or hematologic malignancies to warrant further study
- Trial name
- SIGNATURE (NCT02002689)
- Testing
- LDE225
- Where
- 10 sites in United States
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Duchenne Muscular Dystrophy
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
- Trial name
- NCT04956289
- Testing
- Viltolarsen
- Where
- 8 sites in United States, China and 4 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Arthritis (Hip), Hip Dysplasia, Congenital
Clinical and Radiological Outcomes of the ROMAX Hip Resurfacing System
The goal of this observational study is to collect clinical and radiological data to monitor the safety and performance of the ROMAX hip resurfacing system.
- Trial name
- NCT06775704
- Testing
- Hip resurfacing system
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Medacta International SA
Hepatocellular Carcinoma
A Study of LY2157299 in Participants With Advanced Hepatocellular Carcinoma
The main purpose of this study is to evaluate the safety and effectiveness of the study drug known as LY2157299 in participants with hepatocellular carcinoma.
- Trial name
- NCT02178358
- Testing
- LY2157299, Sorafenib
- Where
- 16 sites in China, Hong Kong and 3 more
- Volunteers
- 132 people
- Sponsor
- Eli Lilly and Company
Adolescent Idiopathic Scoliosis
Comparing Pedicle Screw Systems for the Treatment of Adolescent Paediatric Spine Deformity
Evaluation of the efficacy of the K2M MESA Rail™ Deformity System at improving coronal and sagittal correction of the adolescent idiopathic spine deformity in comparison to the side loading DePuy Synthes USS II pedicle screw…
- Trial name
- NCT02590380
- Testing
- MESA Rail Deformity System, DePuy Synthes USS II System
- Where
- United Kingdom
- Sponsor
- K2M, Inc.