The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A. The study duration for each participant is approximately 12 months.
This is a phase 2b, randomized, double-blind, 3-arm study for the treatment of Crohn's disease. The primary objective of this study is to assess the efficacy of different doses of SAR442970 compared with placebo in participants with moderate to severe Crohn's disease. The total study duration is up to 168 …
This is a parallel, Phase 2/Phase 3, 3-arm study to investigate the efficacy, safety, and tolerability of subcutaneous (SC) treatment with lunsekimig compared with placebo in adult participants (aged 40 to 80 years, inclusive) with inadequately controlled Chronic obstructive pulmonary disease (COPD) characterized by an eosinophilic phenotype. Participation to the …
This is a parallel, Phase 2, two-arm study to assess the efficacy and safety of 14-days intravenous (IV) infusion of teplizumab treatment. Teplizumab has been approved by FDA to delay the onset of Stage 3 Type 1 Diabetes (T1D) in adults and pediatric patients aged 8 years and older with …
The purpose of this study is to assess the safety and immunogenicity of vYF in adults aged 18 years and over in Japan. Study details include: The study duration will be up to approximately 1 month. One single dose of vYF will be administrated subcutaneously at the 1st visit. The …
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The …
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients …
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary …
