Iptacopan
- Generic Name
- Iptacopan
- Brand Names
- Fabhalta
- Drug Type
- Small Molecule
- Chemical Formula
- C25H30N2O4
- CAS Number
- 1644670-37-0
- Unique Ingredient Identifier
- 8E05T07Z6W
- Background
Iptacopan is under investigation in clinical trial NCT04578834 (Study of Efficacy and Safety of LNP023 in Primary Iga Nephropathy Patients).
- Indication
用于治疗既往未接受过补体抑制剂（如依库珠单抗等）治疗的阵发性睡眠性血红蛋白尿症(PNH，一种罕见病)成人患者。
PNH是一种由补体介导的罕见血液疾病。PNH患者的造血干细胞PIG-A基因发生突变，导致其产生易于被补体系统过早破坏的红细胞，从而引发血管内溶血（红细胞在血管内被破坏）和血管外溶血（红细胞在脾脏和肝脏中被破坏），临床主要表现为贫血、阵发性血红蛋白尿、骨髓造血功能衰竭和血栓形成等。
- Associated Conditions
- -
- Associated Therapies
- -
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
- First Posted Date
- 2021-02-10
- Last Posted Date
- 2024-09-30
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 250
- Registration Number
- NCT04747613
- Locations
- 🇺🇸
The University of Utah, Salt Lake City, Utah, United States
🇺🇸City Of Hope, Duarte, California, United States
🇺🇸USC Norris Cancer Center, Los Angeles, California, United States
Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Phase 2
Completed
- Conditions
- Interventions
- First Posted Date
- 2018-02-20
- Last Posted Date
- 2024-06-12
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 16
- Registration Number
- NCT03439839
- Locations
- 🇮🇹
Novartis Investigative Site, Napoli, Italy
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