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
- -
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.
- Conditions
- Interventions
- First Posted Date
- 2024-07-24
- Last Posted Date
- 2024-11-20
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 146
- Registration Number
- NCT06517758
- Locations
- 🇺🇸
Fullerton Neuro and Headache Ctr, Fullerton, California, United States
🇺🇸SC3 Research Pasadena, Pasadena, California, United States
🇺🇸Superior Associates in Research LLC, Hialeah, Florida, United States
Iptacopan in Patients With ANCA Associated Vasculitis
- Conditions
- Interventions
- First Posted Date
- 2024-04-29
- Last Posted Date
- 2024-10-08
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 78
- Registration Number
- NCT06388941
- Locations
- 🇹🇷
Novartis Investigative Site, Pendik Istanbul, Turkey
Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
- First Posted Date
- 2023-07-07
- Last Posted Date
- 2024-11-14
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 50
- Registration Number
- NCT05935215
- Locations
- 🇬🇧
Novartis Investigative Site, London, United Kingdom
Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN
- First Posted Date
- 2023-03-06
- Last Posted Date
- 2024-10-01
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 68
- Registration Number
- NCT05755386
- Locations
- 🇺🇸
Ronald Reagan UCLA Medical Center UCLA Connie Frank Clinic, Los Angeles, California, United States
🇺🇸Univ Cali Irvine ALS Neuromuscular Tustin, Orange, California, United States
🇺🇸UCSF Main Centre, San Francisco, California, United States
Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.
- First Posted Date
- 2022-11-29
- Last Posted Date
- 2024-11-25
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 52
- Registration Number
- NCT05630001
- Locations
- 🇺🇸
City Of Hope National Med Center, Duarte, California, United States
🇺🇸Lakes Research, Miami Lakes, Florida, United States
🇺🇸Huntsman Cancer Institute Univ of Utah, Salt Lake City, Utah, United States
Managed Access Programs for LNP023, Iptacopan
- First Posted Date
- 2022-02-03
- Last Posted Date
- 2024-10-28
- Lead Sponsor
- Novartis Pharmaceuticals
- Registration Number
- NCT05222412
Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders
- Conditions
- Interventions
- First Posted Date
- 2021-10-21
- Last Posted Date
- 2024-06-07
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 19
- Registration Number
- NCT05086744
- Locations
- 🇬🇧
Novartis Investigative Site, London, United Kingdom
🇺🇸Massachusetts General Hospital, Boston, Massachusetts, United States
Pharmacokinetic (PK) and Safety Study of Iptacopan (LNP023) in Participants With Mild, Moderate, and Severe Hepatic Impairment Compared to Matched Control Healthy Participants With Normal Hepatic Function.
- First Posted Date
- 2021-10-14
- Last Posted Date
- 2023-07-24
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 38
- Registration Number
- NCT05078580
- Locations
- 🇺🇸
Novartis Investigative Site, San Antonio, Texas, United States
Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy
- First Posted Date
- 2021-05-17
- Last Posted Date
- 2024-12-12
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 50
- Registration Number
- NCT04889430
- Locations
- 🇺🇸
Uni Of Alabama At Birmingham, Birmingham, Alabama, United States
🇺🇸Univ of California at Los Angeles, Los Angeles, California, United States
🇺🇸Univ Cali Irvine ALS Neuromuscular, Orange, California, United States
Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.
- First Posted Date
- 2021-03-26
- Last Posted Date
- 2024-08-09
- Lead Sponsor
- Novartis Pharmaceuticals
- Target Recruit Count
- 98
- Registration Number
- NCT04817618
- Locations
- 🇺🇸
Albany Medical Center Department of Medicine, Albany, New York, United States
🇺🇸University of Iowa Health Care, Iowa City, Iowa, United States
🇺🇸Childrens Hospital Colorado, Aurora, Colorado, United States
Drug Development Updates
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