How many Iptacopan clinical trials are there?

20 Iptacopan clinical trials are catalogued on Hipa.ai, sourced from the AACT mirror of ClinicalTrials.gov. 7 are actively recruiting participants. 5 are marked completed.

Are Iptacopan clinical trials recruiting in 2026?

Yes — 7 Iptacopan trials are actively recruiting in 2026, with 68 U.S. sites across 29 states. See the recruiting trials and the per-state site list further down this page.

What conditions is Iptacopan being studied for?

Iptacopan is being studied for Atypical Hemolytic Uremic Syndrome, Paroxysmal Nocturnal Hemoglobinuria, Paroxysmal Nocturnal Hemoglobinuria (PNH), Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis, C3 Glomerulopathy (C3G) and 17 more. The trials are grouped by indication on this page.

What phase is Iptacopan in?

Iptacopan clinical trials span 9 Phase 3, 6 Phase 2, 1 Phase 1. The phase × status matrix at the top of this page breaks down counts by phase and recruitment status.

Who sponsors Iptacopan clinical trials?

The lead sponsor across the most Iptacopan trials is Novartis Pharmaceuticals, with 2 additional lead sponsors listed in the data. Each trial row links to the full study detail page including sponsor information.

Iptacopan Clinical Trials (2026): 20 Studies for Atypical Hemolytic Uremic Syndrome & Paroxysmal Nocturnal Hemoglobinuria

Creator: Hipa.ai Research
Published: 2026-04-27T18:42:32.487Z

Hipa.ai Research · Source: ClinicalTrials.gov / AACT

Synced daily from ClinicalTrials.gov via AACT. Last sync: July 17, 2026.

20 Iptacopan clinical trials are catalogued on Hipa.ai from the AACT mirror of ClinicalTrials.gov, spanning 18 indications (including Atypical Hemolytic Uremic Syndrome & Paroxysmal Nocturnal Hemoglobinuria) and 1,602 total planned enrollment. 9 Phase 3 Iptacopan trials and 6 Phase 2 are listed. 9 Iptacopan clinical trials are actively recruiting at 68 U.S. sites across 29 states. Most recent activity: NCT06517758 (May 2032).

Where to Participate: All Iptacopan Trial Sites in the U.S. (68 sites across 29 states)

Every actively recruiting Iptacopantrial site, sorted by state then city. Each row links to the trial detail page (eligibility, contacts, full study record). Sites no longer enrolling at the location level are excluded. ClinicalTrials.gov / AACT does not provide street-level addresses; the map link uses the facility's geocoded coordinates where available.

State	Facility	City	Trial	Map
AL	University of Alabama at Birmingham	Birmingham35294	NCT06797518	Map
AZ	Honor Health Research Institute	Scottsdale85258	NCT06517758	Map
CA	Fullerton Neuro and Headache Ctr	Fullerton92835	NCT06517758	Map
CA	Ronald Reagan UCLA Medical Center	Los Angeles90095	NCT05755386	Map
CA	Ronald Reagan UCLA Medical Center	Los Angeles90095	NCT05268289	Map
CA	UCLA Medical Center	Los Angeles90095	NCT06797518	Map
CA	Univ Cali Irvine ALS Neuromuscular	Orange92868	NCT05755386	Map
CA	SC3 Research Pasadena	Pasadena91105	NCT06517758	Map
CA	California Pacific Medical Center	Sacramento94115	NCT06517758	Map
CA	UCSF	San Francisco94115	NCT05755386	Map
CA	Olive View UCLA Medical Center	Sylmar91342	NCT05755386	Map
CO	Childrens Hospital Colorado	Aurora80045	NCT05755386	Map
CO	Childrens Hospital Colorado	Aurora80045	NCT04817618	Map
CO	Childrens Hospital Colorado	Aurora80045	NCT06994845	Map
CO	University of Colorado Denver	Aurora80045	NCT05268289	Map
FL	Royal Research Corp	Hollywood33021	NCT05268289	Map
FL	Mayo Clinic Jacksonville	Jacksonville32224	NCT05268289	Map
FL	Nicklaus Childrens Hospital	Miami33155	NCT05755386	Map
FL	Nicklaus Childrens Hospital	Miami33155	NCT04817618	Map
FL	Central Florida Kidney Specialists	Orlando32806	NCT06797518	Map
FL	Florida Kidney Physicians	Riverview33578	NCT05268289	Map
FL	Florida Kidney Physicians	Riverview33578	NCT05268289	Map
GA	Emory University School of Medicine-Winship Cancer Institute	Atlanta30322	NCT05755386	Map
GA	Augusta University Georgia	Augusta30912	NCT06517758	Map
GA	Georgia Nephrology Research Inst	Lawrenceville30046	NCT06797518	Map
ID	CaRe Research	Chubbuck83202	NCT06797518	Map
IL	University of Chicago Medical Centr	Chicago60637	NCT06517758	Map
IL	Prairie Heart Institute	Springfield62769	NCT06517758	Map
IA	University of Iowa Health Care	Iowa City52242-1091	NCT04817618	Map
IA	University of Iowa Health Care	Iowa City52242	NCT04817618	Map
KS	Wichita Community Clcl Onco Program	Wichita67214	NCT05268289	Map
LA	Ochsner Health System	New Orleans70121	NCT05268289	Map
MD	Mid Atlantic Epilepsy and Sleep Ctr	Bethesda20817-1807	NCT06517758	Map
MA	Boston Univ School of Medicine	Boston02118	NCT05755386	Map
MA	Brigham and Womens Hosp Harvard Med School	Boston02115	NCT05268289	Map
MA	Massachusetts General Hospital	Boston02114	NCT05755386	Map
MA	Massachusetts General Hospital	Boston02114	NCT06517758	Map
MI	Henry Ford Health System	Detroit48202	NCT06517758	Map
MN	University of Minnesota	Minneapolis55455	NCT05755386	Map
MO	Washington University in St Louis	St Louis63110	NCT05755386	Map
NV	DaVita Clinical Research	Las Vegas89146	NCT06797518	Map
NM	University of New Mexico	Albuquerque87131-0001	NCT05755386	Map
NY	Stony Brook Internists PC	East Setauket11733	NCT05268289	Map
NY	Col Uni Med Center New York Presby	New York10032	NCT05755386	Map
NC	Duke University Medical Center	Durham27710	NCT06517758	Map
OH	University Of Cincinnati	Cincinnati45267	NCT05755386	Map
OH	Cleveland Clinic Foundation	Cleveland44195	NCT05268289	Map
OH	University Hospitals Cleveland Medical Center	Cleveland44106	NCT06797518	Map
OH	OSU Wexner Medical Center	Columbus43210	NCT06797518	Map
OR	OHSU Dept of Nephrology	Portland97239	NCT05755386	Map
PA	Childrens Hospital of Philadelphia	Philadelphia19104-4399	NCT06994845	Map
PA	Penn Presbyterian Medical Center	Philadelphia19104	NCT06517758	Map
PA	Temple University	Philadelphia19140	NCT05268289	Map
PA	Univ of Pennsylvania Medical Center	Philadelphia19104	NCT05755386	Map
SC	MUSC Health Lancaster Med Ctr	Lancaster29720	NCT05755386	Map
TN	Vanderbilt University Medical CenterX	Nashville37221	NCT06517758	Map
TX	UT Southwestern Medical Center	Dallas75235	NCT05755386	Map
TX	Nerve and Muscle Center of Texas	Houston77030	NCT06517758	Map
TX	Prolato Clinical Research Center	Houston77054	NCT05755386	Map
TX	Prolato Clinical Research Center	Houston77054	NCT06797518	Map
TX	Prolato Clinical Research Center	Houston77054	NCT05268289	Map
TX	Baylor Scott and White Research	Temple76502	NCT05755386	Map
UT	Prim Childrens Hosp Inv Pharm	Salt Lake City84113	NCT06994845	Map
UT	University of Utah	Salt Lake City84113	NCT05755386	Map
VA	Virginia Commonwealth University	Richmond23298	NCT05268289	Map
WI	Center for Neurological Disorders G	Greenfield53228-1321	NCT06517758	Map
WI	University of Wisconsin	Madison53792	NCT05755386	Map
WI	University of Wisconsin	Madison53792	NCT04817618	Map

Iptacopan clinical trials at a glance: phase × recruitment status

Phase	recruiting	active not recruiting	completed	terminated	not yet recruiting	available	Total
P1			1				1
P2	2	2	1	1			6
P3	5	1	3				9
N/A		1			2	1	4

Recruiting Iptacopan clinical trials (9)

Studies currently enrolling participants. Click any NCT ID to view the trial detail page (eligibility, sites, contacts).

NCT ID	Title	Phase	Indication	Enrollment	Started
NCT06517758	A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.	P3	Generalized Myasthenia Gravis	146	Jul 2024
NCT06994845	Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN	P3	Primary Immunoglobulin A Nephropathy (IgAN)	31	Nov 2025
NCT07347990	Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy	N/A	Thrombotic Microangiopathy	30	Jan 2026
NCT05935215	Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)	P3	Atypical Hemolytic Uremic Syndrome	50	Feb 2024
NCT05755386	APPARENTStudy of Efficacy and Safety of Iptacopan in Participants With IC-MPGN	P3	IC-MPGN	106	Oct 2023
NCT05268289	Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V	P2	Lupus Nephritis	240	Aug 2022
NCT06797518	Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN	P2	Glomerulonephritis, IGA	20	Feb 2025
NCT04817618	APPEAR-C3GStudy of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.	P3	C3G	98	Jul 2021
NCT06847607	AIHAA Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)	N/A	AIHA - Warm Autoimmune Hemolytic Anemia	20	Mar 2025

Iptacopan clinical trials by indication

Atypical Hemolytic Uremic Syndrome (2)

NCT	Acronym	Phase	Status	Title
NCT05935215	—	P3	recruiting	Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT04889430	APPELHUS	P3	completed	Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Paroxysmal Nocturnal Hemoglobinuria (2)

NCT	Acronym	Phase	Status	Title
NCT04747613	—	P3	active not recruiting	Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT05630001	APPULSE	P3	completed	Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.

Thrombotic Microangiopathy (1)

NCT	Acronym	Phase	Status	Title
NCT07347990	—	N/A	not yet recruiting	Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy

Primary Immunoglobulin A Nephropathy (IgAN) (1)

NCT	Acronym	Phase	Status	Title
NCT06994845	—	P3	recruiting	Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN

Hemoglobinuria, Paroxysmal (1)

NCT	Acronym	Phase	Status	Title
NCT06903234	PNH	N/A	active not recruiting	Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry

AIHA - Warm Autoimmune Hemolytic Anemia (1)

NCT	Acronym	Phase	Status	Title
NCT06847607	AIHA	N/A	not yet recruiting	A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)

Glomerulonephritis, IGA (1)

NCT	Acronym	Phase	Status	Title
NCT06797518	—	P2	recruiting	Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN

Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis (1)

NCT	Acronym	Phase	Status	Title
NCT06388941	—	P2	active not recruiting	Iptacopan in Patients With ANCA Associated Vasculitis

Generalized Myasthenia Gravis (1)

NCT	Acronym	Phase	Status	Title
NCT06517758	—	P3	recruiting	A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.

IC-MPGN (1)

NCT	Acronym	Phase	Status	Title
NCT05755386	APPARENT	P3	recruiting	Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN

Lupus Nephritis (1)

NCT	Acronym	Phase	Status	Title
NCT05268289	—	P2	recruiting	Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V

Age-Related Macular Degeneration (1)

NCT	Acronym	Phase	Status	Title
NCT05230537	—	P2	active not recruiting	A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration

Immune Thrombocytopenia (ITP) (1)

NCT	Acronym	Phase	Status	Title
NCT05086744	LNP023	P2	terminated	Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

Hepatic Impairment (1)

NCT	Acronym	Phase	Status	Title
NCT05078580	PK	P1	completed	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.

C3G (1)

NCT	Acronym	Phase	Status	Title
NCT04817618	APPEAR-C3G	P3	recruiting	Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

Paroxysmal Nocturnal Hemoglobinuria (PNH) (1)

NCT	Acronym	Phase	Status	Title
NCT04820530	APPOINT-PNH	P3	completed	Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis (1)

NCT	Acronym	Phase	Status	Title
NCT03439839	PNH	P2	completed	Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

C3 Glomerulopathy (C3G) (1)

NCT	Acronym	Phase	Status	Title
NCT05222412	—	N/A	available	Managed Access Programs for LNP023, Iptacopan

Iptacopan pivotal trial highlights (Phase 2/3/4)

Phase-2/3/4 studies with named acronyms — the registration-grade trials most cited in label discussions and Wikipedia.

Acronym	NCT	Phase	Status	Indication	Enrollment	PubMed
APPARENT	NCT05755386	P3	recruiting	IC-MPGN	106	—
APPEAR-C3G	NCT04817618	P3	recruiting	C3G	98	—
APPELHUS	NCT04889430	P3	completed	Atypical Hemolytic Uremic Syndrome	34	—
APPULSE	NCT05630001	P3	completed	Paroxysmal Nocturnal Hemoglobinuria	52	—
APPOINT-PNH	NCT04820530	P3	completed	Paroxysmal Nocturnal Hemoglobinuria (PNH)	40	—
LNP023	NCT05086744	P2	terminated	Immune Thrombocytopenia (ITP)	19	—
PNH	NCT03439839	P2	completed	Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis	16	—

Terminated and withdrawn Iptacopan trials (1)

Studies that ended before completion. Reasons are filed in each trial's ClinicalTrials.gov entry.

NCT05086744terminatedBasket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

All Iptacopan trials (20)

Sorted by most recent activity (completion → start → posted date). Each row links to the trial detail page on Hipa.ai.

NCT ID	Acronym	Title	Phase	Status	Enrollment	Sponsor	Started
NCT06517758		A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.	P3	recruiting	146	Novartis Pharmaceuticals	Jul 2024
NCT06994845		Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN	P3	recruiting	31	Novartis Pharmaceuticals	Nov 2025
NCT07347990		Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy	N/A	not yet recruiting	30	First Affiliated Hospital of Zhejiang University	Jan 2026
NCT06903234	PNH	Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registry	—	active not recruiting	200	Novartis Pharmaceuticals	Mar 2025
NCT05935215		Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)	P3	recruiting	50	Novartis Pharmaceuticals	Feb 2024
NCT05755386	APPARENT	Study of Efficacy and Safety of Iptacopan in Participants With IC-MPGN	P3	recruiting	106	Novartis Pharmaceuticals	Oct 2023
NCT05268289		Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V	P2	recruiting	240	Novartis Pharmaceuticals	Aug 2022
NCT06797518		Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgAN	P2	recruiting	20	Novartis Pharmaceuticals	Feb 2025
NCT04747613		Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria	P3	active not recruiting	208	Novartis Pharmaceuticals	Jul 2021
NCT04817618	APPEAR-C3G	Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.	P3	recruiting	98	Novartis Pharmaceuticals	Jul 2021
NCT06388941		Iptacopan in Patients With ANCA Associated Vasculitis	P2	active not recruiting	84	Novartis Pharmaceuticals	Aug 2024
NCT05230537		A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration	P2	active not recruiting	170	Novartis Pharmaceuticals	Feb 2022
NCT06847607	AIHA	A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)	N/A	not yet recruiting	20	Bing Han	Mar 2025
NCT04889430	APPELHUS	Efficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy	P3	completed	34	Novartis Pharmaceuticals	Jan 2022
NCT05630001	APPULSE	Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.	P3	completed	52	Novartis Pharmaceuticals	Apr 2023
NCT05086744	LNP023	Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders	P2	terminated	19	Novartis Pharmaceuticals	Dec 2021
NCT04820530	APPOINT-PNH	Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy	P3	completed	40	Novartis Pharmaceuticals	Jul 2021
NCT05078580	PK	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.	P1	completed	38	Novartis Pharmaceuticals	Nov 2021
NCT03439839	PNH	Study of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	P2	completed	16	Novartis Pharmaceuticals	Apr 2018
NCT05222412		Managed Access Programs for LNP023, Iptacopan	—	available	0	Novartis Pharmaceuticals	—

Iptacopan clinical trials: frequently asked questions

How many Iptacopan clinical trials are there?: 20 Iptacopan clinical trials are catalogued on Hipa.ai, sourced from the AACT mirror of ClinicalTrials.gov. 7 are actively recruiting participants. 5 are marked completed.
Are Iptacopan clinical trials recruiting in 2026?: Yes — 7 Iptacopan trials are actively recruiting in 2026, with 68 U.S. sites across 29 states. See the recruiting trials and the per-state site list further down this page.
What conditions is Iptacopan being studied for?: Iptacopan is being studied for Atypical Hemolytic Uremic Syndrome, Paroxysmal Nocturnal Hemoglobinuria, Paroxysmal Nocturnal Hemoglobinuria (PNH), Anti-Neutrophil Cytoplasm Antibodies (ANCA) Associated Vasculitis, C3 Glomerulopathy (C3G) and 17 more. The trials are grouped by indication on this page.
What phase is Iptacopan in?: Iptacopan clinical trials span 9 Phase 3, 6 Phase 2, 1 Phase 1. The phase × status matrix at the top of this page breaks down counts by phase and recruitment status.
Who sponsors Iptacopan clinical trials?: The lead sponsor across the most Iptacopan trials is Novartis Pharmaceuticals, with 2 additional lead sponsors listed in the data. Each trial row links to the full study detail page including sponsor information.

Sources and methodology

Trial inventory derived from the AACT mirror of ClinicalTrials.gov. Trials are filtered to those naming Iptacopanas an intervention. Phase, status, enrollment, sponsor, and dates are taken from the source `studies` table; primary indication is the first listed condition. Acronyms are extracted from each trial's brief title where present. PubMed links are populated for trials whose published primary outcomes have been indexed in our evidence cache.

Last refreshed July 17, 2026.

Related pages on Hipa.ai:

Iptacopan overview