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A Phase 1/2, Multicenter, Open-Label, Dose-Escalation, Safety, Tolerability, and Clinical Activity Study of a Single Dose of JAG201 Gene Therapy Delivered Via Intracerebroventricular Administration in Participants With SHANK3 Haploinsufficiency
This is a Phase 1/2, first in human, open-label, dose-escalation study to evaluate the safety, tolerability, and clinical activity of a single dose of JAG201 a…
A Phase 2 Study to Evaluate the Safety and Efficacy of Atacicept in Multiple Autoimmune Glomerular Diseases (PIONEER)
The purpose of this study is to evaluate the safety and tolerability of atacicept in adult and adolescent participants and to measure the effect in reducing pr…
Nephrotic Syndrome Study Network Under the Rare Diseases Clinical Research Network
Minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), and Membranous nephropathy (MN), generate an enormous individual and societal financia…
A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome
This Phase 3, randomized, double-blind, parallel-group (2-arm), placebo-controlled, multicenter study will evaluate the efficacy and safety of NNZ-2591 compare…
Implementing Precision Medicine for Glomerular Diseases in the Nephrotic Syndrome Study Network (NEPTUNE)
NEPTUNE Match is an additional opportunity offered to NEPTUNE study participants to prospectively recruit and communicate patient-specific clinical trial match…
Recurrence Post-transplant Observational Study in Focal Segmental Glomerulosclerosis (FSGS) and Minimal Change Disease (MCD)
The morbidity of recurrence of focal segmental glomerulosclerosis (FSGS) and minimal change disease (MCD) after transplant is well-recognized and include conte…