Clinical trials: Musculoskeletal Abnormalities
Fused Vertebrae, Ankylosing Spondylitis
Clinical Investigation of MONTAGE in Adults With Spinal Deformity Undergoing Pedicle Subtraction Osteotomy
This study evaluates the difference in postoperative bleeding between two study groups, FDA cleared MONTAGE Settable Resorbable Hemostatic Bone Putty and standard of care (no bone hemostat) during pedicle subtraction osteotomy…
- Conditions
- Fused Vertebrae, Ankylosing Spondylitis, Sagittal Deformities, Thoracolumbar Kyphosis
- Trial name
- NCT04125147
- Testing
- Experimental: Montage Bone Hemostat
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Abyrx, Inc.
Achondroplasia
Bioequivalence Study to Compare Two Injection Devices for BMN 111 in Healthy Participants
The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.
- Trial name
- NCT05813314
- Testing
- BMN 111 administration via Injector Pen, BMN 111 administration via vial and syringe
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
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
Duchenne Muscular Dystrophy
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the…
- Trial name
- NCT01753804
- Testing
- Observational study
- Where
- 16 sites in United States, Argentina and 8 more
- Volunteers
- 269 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy (DMD)
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Hypophosphatasia (HPP)
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
- Trial name
- NCT01203826
- Testing
- Asfotase Alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Gout, Hyperuricemia
Tigulixostat, Phase 3 Study, Placebo Controlled in Gout Patients
The aim of this 6-month randomized multi-regional double-blind parallel group placebo-controlled phase 3 study is to assess the efficacy and safety of three different doses of Tigulixostat in gout patients with hyperuricemia.
- Conditions
- Gout, Hyperuricemia, Gout Flare, Tophi
- Trial name
- EURELIA1 (NCT05586958)
- Testing
- Tigulixostat
- Where
- 52 sites in United States, Georgia and 2 more
- Volunteers
- 354 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
FSHD - Facioscapulohumeral Muscular Dystrophy
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
The goal of this clinical trial is to learn about how an umbilical cord lining-derived stem cell product (ULSC) performs when treating Facioscapulohumeral Muscular Dystrophy (FSHD) 1 or 2.
- Trial name
- NCT07086521
- Testing
- ULSC
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Restem, LLC.
Bunion, Post-operative Pain
CPL-01 in the Management of Postoperative Pain After Bunionectomy
Subjects receive study drug during bunionectomy and are followed for pain and opioid use, with the hypothesis that those who receive CPL-01 will have less pain and less opioid use than either control arm.
- Trial name
- NCT05831449
- Testing
- Local anesthetic injection of CPL-01, Naropin, 0.5% Injectable Solution, Morphine rescue medication
- Where
- United States
- Volunteers
- 616 people
- Sponsor
- Cali Pharmaceuticals LLC