Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
New research demonstrates machine learning can optimize drug dosing, identifies biomarkers of treatment response, and validates prognostic criteria for long-term kidney survival in pediatric lupus nephritis patients.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07347990 |
Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy |
NOT_YET_RECRUITING | NA |
| NCT07279610 |
N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy |
ACTIVE_NOT_RECRUITING | PHASE2/PHASE3 |
| NCT07059026 |
Thrombotic Microangiopathy (TMA) Associated With Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) In Adult Patients |
RECRUITING | |
| NCT05991245 |
French National Cohort MATRIX "Renal and Systemic Thrombotic Microangiopathy" |
UNKNOWN | |
| NCT04845022 |
Incidence of Snakebite Associated Thrombotic Microangiopathy & Role of Peripheral Blood Smear as a Predictor of Clinical Outcome |
UNKNOWN | |
| NCT04743804 |
Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger |
TERMINATED | PHASE3 |
| NCT04557735 |
Study of Ravulizumab in Pediatric Participants With HSCT-TMA |
COMPLETED | PHASE3 |
| NCT04543591 |
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant |
COMPLETED | PHASE3 |
| NCT02604420 |
Identification and Treatment of Thrombotic Microangiopathies in Allogeneic Stem Cell Transplants |
COMPLETED | |
| NCT02355782 |
OMS721 Compassionate Use in Patients With Thrombotic Microangiopathy |
AVAILABLE |
