SNY — ICGG Gaucher Disease Registry & Pregnancy Sub-registry - Clinical Trial Completion
Jan 31, 2034, 5:00:00 AM UTC
Summary
The International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry and Pregnancy Sub-registry, sponsored by Genzyme (a Sanofi company), is expected to complete on January 31, 2034. This long-term, observational study tracks clinical outcomes and natural history in patients with Gaucher disease, including a sub-registry focused on pregnancy outcomes. The registry aims to enhance understanding of disease progression, guide therapeutic interventions, and evaluate the long-term effectiveness of treatments such as imiglucerase and eliglustat. No experimental interventions are involved, and patient care is determined by treating physicians.
Company
SANOFI (SNY)NASDAQ — Healthcare: Manufacturing, Drug Manufacturers - General: Pharmaceutical Preparations
www.sanofi.comSimilar Events
Natural History Study of Infantile and Juvenile GM1 Gangliosidosis - Expected Trial Completion
A natural history study of infantile and juvenile GM1 gangliosidosis, sponsored by the University of Pennsylvania and associated with Passage Bio (PASG), is expected to complete on December 31, 2026. The observational trial aims to collect longitudinal clinical, imaging, and laboratory data from up to 40 patients to better understand disease progression and identify potential efficacy endpoints and biomarkers for future interventional studies. GM1 gangliosidosis is a rare, fatal lysosomal storage disorder with limited treatment options. The study's findings may inform the design of future clinical trials for therapeutic candidates.
trial completionMaternal and Postnatal Outcomes Study (MOS) Expected Trial Completion
The Maternal and Postnatal Outcomes Study (MOS), sponsored by Chiesi Farmaceutici, is projected to complete on December 1, 2034. This global observational registry will assess the safety of Elfabrio (pegunigalsidase alfa) in women with Fabry disease during pregnancy and breastfeeding, as well as outcomes for their infants. The study aims to collect data on maternal and infant health, pregnancy complications, delivery outcomes, and congenital malformations. Elfabrio is an enzyme replacement therapy for Fabry disease, a rare genetic disorder. The registry will enroll patients over a 10-year period, with data collected both prospectively and retrospectively.
trial completionRGX-111 Long-Term Follow-Up Clinical Trial Completion
REGENXBIO Inc. is conducting a long-term follow-up observational study for RGX-111, a gene therapy candidate targeting Mucopolysaccharidosis Type I (MPS I). The trial, registered as NCT06103487, is expected to complete on September 1, 2027. The study aims to assess the long-term safety of RGX-111 after a single intracisternal or intracerebroventricular administration in participants from previous RGX-111 trials. No new treatment is administered under this protocol. RGX-111 is an investigational gene therapy product in the rare disease category.
trial completionClinical Trial Completion for SGN-CEACAM5C
The clinical trial for SGN-CEACAM5C, an antibody-drug conjugate targeting advanced solid tumors, is expected to complete on February 14, 2030. The trial, sponsored by Seagen Inc., a subsidiary of Pfizer, aims to evaluate the safety and efficacy of the experimental drug PF-08046050 in patients with hard-to-treat solid tumors. The study includes multiple parts to determine optimal dosing and effectiveness, with a total enrollment of 494 participants.
trial completion