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A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy ...
To evaluate the long-term safety and tolerability of AOC 1001 in DM1 patients.THIS STUDY IS ENROLLING BY INVITATION ONLY - The study will invite participants with DM1 who completed ...
Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of Dyne-101 Administered to Participants with Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).THIS STUDY ...
A Multicenter, Open-Label, Phase 1/2, Dose-Escalation and Subsequent Safety Extension Study of Subcutaneous KK8123 in Adult Patients with X-Linked Hypophosphatemia
The purpose of this study is to test the safety and effectiveness of an investigational drug called KK8123 in adults with X-linked hypophosphatemia. THIS STUDY IS ENROLLING BY INVITATION ONLY - ...
Kysa-6: a Phase 2/3, Open-Label, Randomized, Controlled, Multicenter Study of Kyv-101, an Autologous Fully Human Anti-Cd19 Chimeric Antigen Receptor T-Cell (Cd19 Car T) Therapy, Versus ...
Myasthenia gravis (MG) is a chronic autoimmune disease that affects the neuromuscular junction and is characterized by muscle weakness. B cells play a role in MG, and the disease is ...
1010002807 a Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regime with Campath-1H Fludarabine and Melphalan
The hypothesis for this study is that a preparative regimen that maximizes host immunosuppression without myeloablation will be well tolerated and sufficient for engraftment of donor hematopoietic cells. It is ...
1704113280 1704113280 1704113280 Prevention of Clot in Orthopaedic Trauma (PREVENT CLOT): a Randomized Pragmatic Trial Comparing the Complications and Safety of Blood Clot Prevention Medicines ...
The purpose of this research study is to help determine the best treatment for preventing blood clots that are formed following trauma that could potentially lead to death. At the ...
A Phase 3, Open-Label, Multicenter, Randomized Study to Evaluate the Efficacy and Safety of Romosozumab Compared with Bisphosphonates in Children and Adolescents with Osteogenesis Imperfecta ...
The primary objective of this study is to evaluate the effect of romosozumab treatment for 12-months compared with bisphosphonate(s) on the number of clinical fractures at 12-months; the number of ...
A Phase 3, Multicenter, Open-Label, Basket, LTE Study to Evaluate the Safety of Guselkumab in Pediatric Participants with Crohn's Disease, Ulcerative Colitis, or Juvenile Psoriatic ...
The purpose of this study is to evaluate long-term safety of subcutaneous guselkumab in pediatric participants with moderately to severely active ulcerative colitis, or moderately to severely active Crohn's disease, ...
An Interventional, Phase 3 Extension Study to Investigate Long-Term Safety and Tolerability of Tolebrutinib in Participants with Relapsing Multiple Sclerosis, Primary Progressive Multiple Sclerosis, or ...
This is a Phase 3 extension, global, multicenter study to assess the long-term safety and tolerability of tolebrutinib in adult participants (aged ≥18 years) with RMS, PPMS, or NRSPMS who ...
Master Protocol of Two Independent, Randomized, Double-Blind, Phase 3 Studies Comparing Efficacy and Safety of Frexalimab (SAR441344) to Teriflunomide in Adult Participants with Relapsing Forms ...
The purpose of each study is to independently measure the annualized relapse rate (ARR) with administration of frexalimab compared to a daily oral dose of teriflunomide in male and female ...