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

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

Synced daily from ClinicalTrials.gov via AACT. Last sync: .

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.

StateFacilityCityTrialMap
ALUniversity of Alabama at BirminghamBirmingham35294NCT06797518Map
AZHonor Health Research InstituteScottsdale85258NCT06517758Map
CAFullerton Neuro and Headache CtrFullerton92835NCT06517758Map
CARonald Reagan UCLA Medical CenterLos Angeles90095NCT05268289Map
CARonald Reagan UCLA Medical CenterLos Angeles90095NCT05755386Map
CAUCLA Medical CenterLos Angeles90095NCT06797518Map
CAUniv Cali Irvine ALS NeuromuscularOrange92868NCT05755386Map
CASC3 Research PasadenaPasadena91105NCT06517758Map
CACalifornia Pacific Medical CenterSacramento94115NCT06517758Map
CAUCSFSan Francisco94115NCT05755386Map
CAOlive View UCLA Medical CenterSylmar91342NCT05755386Map
COChildrens Hospital ColoradoAurora80045NCT04817618Map
COChildrens Hospital ColoradoAurora80045NCT05755386Map
COChildrens Hospital ColoradoAurora80045NCT06994845Map
COUniversity of Colorado DenverAurora80045NCT05268289Map
FLRoyal Research CorpHollywood33021NCT05268289Map
FLMayo Clinic JacksonvilleJacksonville32224NCT05268289Map
FLNicklaus Childrens HospitalMiami33155NCT04817618Map
FLNicklaus Childrens HospitalMiami33155NCT05755386Map
FLCentral Florida Kidney SpecialistsOrlando32806NCT06797518Map
FLFlorida Kidney PhysiciansRiverview33578NCT05268289Map
FLFlorida Kidney PhysiciansRiverview33578NCT05268289Map
GAEmory University School of Medicine-Winship Cancer InstituteAtlanta30322NCT05755386Map
GAAugusta University GeorgiaAugusta30912NCT06517758Map
GAGeorgia Nephrology Research InstLawrenceville30046NCT06797518Map
IDCaRe ResearchChubbuck83202NCT06797518Map
ILUniversity of Chicago Medical CentrChicago60637NCT06517758Map
ILPrairie Heart InstituteSpringfield62769NCT06517758Map
IAUniversity of Iowa Health CareIowa City52242-1091NCT04817618Map
IAUniversity of Iowa Health CareIowa City52242NCT04817618Map
KSWichita Community Clcl Onco ProgramWichita67214NCT05268289Map
LAOchsner Health SystemNew Orleans70121NCT05268289Map
MDMid Atlantic Epilepsy and Sleep CtrBethesda20817-1807NCT06517758Map
MABoston Univ School of MedicineBoston02118NCT05755386Map
MABrigham and Womens Hosp Harvard Med SchoolBoston02115NCT05268289Map
MAMassachusetts General HospitalBoston02114NCT05755386Map
MAMassachusetts General HospitalBoston02114NCT06517758Map
MIHenry Ford Health SystemDetroit48202NCT06517758Map
MNUniversity of MinnesotaMinneapolis55455NCT05755386Map
MOWashington University in St LouisSt Louis63110NCT05755386Map
NVDaVita Clinical ResearchLas Vegas89146NCT06797518Map
NMUniversity of New MexicoAlbuquerque87131-0001NCT05755386Map
NYStony Brook Internists PCEast Setauket11733NCT05268289Map
NYCol Uni Med Center New York PresbyNew York10032NCT05755386Map
NCDuke University Medical CenterDurham27710NCT06517758Map
OHUniversity Of CincinnatiCincinnati45267NCT05755386Map
OHCleveland Clinic FoundationCleveland44195NCT05268289Map
OHUniversity Hospitals Cleveland Medical CenterCleveland44106NCT06797518Map
OHOSU Wexner Medical CenterColumbus43210NCT06797518Map
OROHSU Dept of NephrologyPortland97239NCT05755386Map
PAChildrens Hospital of PhiladelphiaPhiladelphia19104-4399NCT06994845Map
PAPenn Presbyterian Medical CenterPhiladelphia19104NCT06517758Map
PATemple UniversityPhiladelphia19140NCT05268289Map
PAUniv of Pennsylvania Medical CenterPhiladelphia19104NCT05755386Map
SCMUSC Health Lancaster Med CtrLancaster29720NCT05755386Map
TNVanderbilt University Medical CenterXNashville37221NCT06517758Map
TXUT Southwestern Medical CenterDallas75235NCT05755386Map
TXNerve and Muscle Center of TexasHouston77030NCT06517758Map
TXProlato Clinical Research CenterHouston77054NCT05268289Map
TXProlato Clinical Research CenterHouston77054NCT05755386Map
TXProlato Clinical Research CenterHouston77054NCT06797518Map
TXBaylor Scott and White ResearchTemple76502NCT05755386Map
UTPrim Childrens Hosp Inv PharmSalt Lake City84113NCT06994845Map
UTUniversity of UtahSalt Lake City84113NCT05755386Map
VAVirginia Commonwealth UniversityRichmond23298NCT05268289Map
WICenter for Neurological Disorders GGreenfield53228-1321NCT06517758Map
WIUniversity of WisconsinMadison53792NCT04817618Map
WIUniversity of WisconsinMadison53792NCT05755386Map

Iptacopan clinical trials at a glance: phase × recruitment status

Phaserecruitingactive not recruitingcompletedterminatednot yet recruitingavailableTotal
P111
P222116
P35139
N/A1214

Recruiting Iptacopan clinical trials (9)

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

NCT IDTitlePhaseIndicationEnrollmentStarted
NCT06517758A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.P3Generalized Myasthenia Gravis146Jul 2024
NCT06994845Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgANP3Primary Immunoglobulin A Nephropathy (IgAN)31Nov 2025
NCT07347990Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic MicroangiopathyN/AThrombotic Microangiopathy30Jan 2026
NCT05935215Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)P3Atypical Hemolytic Uremic Syndrome50Feb 2024
NCT05755386APPARENTStudy of Efficacy and Safety of Iptacopan in Participants With IC-MPGNP3IC-MPGN106Oct 2023
NCT05268289Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- VP2Lupus Nephritis240Aug 2022
NCT06797518Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgANP2Glomerulonephritis, IGA20Feb 2025
NCT04817618APPEAR-C3GStudy of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.P3C3G98Jul 2021
NCT06847607AIHAA Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)N/AAIHA - Warm Autoimmune Hemolytic Anemia20Mar 2025

Iptacopan clinical trials by indication

Atypical Hemolytic Uremic Syndrome (2)

NCTAcronymPhaseStatusTitle
NCT05935215P3recruitingEfficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
NCT04889430APPELHUSP3completedEfficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor Therapy

Paroxysmal Nocturnal Hemoglobinuria (2)

NCTAcronymPhaseStatusTitle
NCT04747613P3active not recruitingLong-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT05630001APPULSEP3completedSingle 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)

NCTAcronymPhaseStatusTitle
NCT07347990N/Anot yet recruitingSafety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy

Primary Immunoglobulin A Nephropathy (IgAN) (1)

NCTAcronymPhaseStatusTitle
NCT06994845P3recruitingStudy to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN

Hemoglobinuria, Paroxysmal (1)

NCTAcronymPhaseStatusTitle
NCT06903234PNHN/Aactive not recruitingPost-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)

NCTAcronymPhaseStatusTitle
NCT06847607AIHAN/Anot yet recruitingA Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)

Glomerulonephritis, IGA (1)

NCTAcronymPhaseStatusTitle
NCT06797518P2recruitingStudy 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)

NCTAcronymPhaseStatusTitle
NCT06388941P2active not recruitingIptacopan in Patients With ANCA Associated Vasculitis

Generalized Myasthenia Gravis (1)

NCTAcronymPhaseStatusTitle
NCT06517758P3recruitingA 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)

NCTAcronymPhaseStatusTitle
NCT05755386APPARENTP3recruitingStudy of Efficacy and Safety of Iptacopan in Participants With IC-MPGN

Lupus Nephritis (1)

NCTAcronymPhaseStatusTitle
NCT05268289P2recruitingStudy of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- V

Age-Related Macular Degeneration (1)

NCTAcronymPhaseStatusTitle
NCT05230537P2active not recruitingA Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration

Immune Thrombocytopenia (ITP) (1)

NCTAcronymPhaseStatusTitle
NCT05086744LNP023P2terminatedBasket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological Disorders

Hepatic Impairment (1)

NCTAcronymPhaseStatusTitle
NCT05078580PKP1completedPharmacokinetic (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)

NCTAcronymPhaseStatusTitle
NCT04817618APPEAR-C3GP3recruitingStudy of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.

Paroxysmal Nocturnal Hemoglobinuria (PNH) (1)

NCTAcronymPhaseStatusTitle
NCT04820530APPOINT-PNHP3completedStudy 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)

NCTAcronymPhaseStatusTitle
NCT03439839PNHP2completedStudy of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

C3 Glomerulopathy (C3G) (1)

NCTAcronymPhaseStatusTitle
NCT05222412N/AavailableManaged 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.

AcronymNCTPhaseStatusIndicationEnrollmentPubMed
APPARENTNCT05755386P3recruitingIC-MPGN106
APPEAR-C3GNCT04817618P3recruitingC3G98
APPELHUSNCT04889430P3completedAtypical Hemolytic Uremic Syndrome34
APPULSENCT05630001P3completedParoxysmal Nocturnal Hemoglobinuria52
APPOINT-PNHNCT04820530P3completedParoxysmal Nocturnal Hemoglobinuria (PNH)40
LNP023NCT05086744P2terminatedImmune Thrombocytopenia (ITP)19
PNHNCT03439839P2completedParoxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis16

Terminated and withdrawn Iptacopan trials (1)

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

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 IDAcronymTitlePhaseStatusEnrollmentSponsorStarted
NCT06517758A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.P3recruiting146Novartis PharmaceuticalsJul 2024
NCT06994845Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgANP3recruiting31Novartis PharmaceuticalsNov 2025
NCT07347990Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic MicroangiopathyN/Anot yet recruiting30First Affiliated Hospital of Zhejiang UniversityJan 2026
NCT06903234PNHPost-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH Registryactive not recruiting200Novartis PharmaceuticalsMar 2025
NCT05935215Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)P3recruiting50Novartis PharmaceuticalsFeb 2024
NCT05755386APPARENTStudy of Efficacy and Safety of Iptacopan in Participants With IC-MPGNP3recruiting106Novartis PharmaceuticalsOct 2023
NCT05268289Study of Efficacy and Safety of LNP023 in Participants With Active Lupus Nephritis Class III-IV, +/- VP2recruiting240Novartis PharmaceuticalsAug 2022
NCT06797518Study to Evaluate the Impact of Iptacopan on Top of SOC on Biopsy Changes in Kidneys of Adult Patients With IgANP2recruiting20Novartis PharmaceuticalsFeb 2025
NCT04747613Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal HemoglobinuriaP3active not recruiting208Novartis PharmaceuticalsJul 2021
NCT04817618APPEAR-C3GStudy of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy.P3recruiting98Novartis PharmaceuticalsJul 2021
NCT06388941Iptacopan in Patients With ANCA Associated VasculitisP2active not recruiting84Novartis PharmaceuticalsAug 2024
NCT05230537A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular DegenerationP2active not recruiting170Novartis PharmaceuticalsFeb 2022
NCT06847607AIHAA Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)N/Anot yet recruiting20Bing HanMar 2025
NCT04889430APPELHUSEfficacy and Safety of Iptacopan (LNP023) in Adult Patients With Atypical Hemolytic Uremic Syndrome Naive to Complement Inhibitor TherapyP3completed34Novartis PharmaceuticalsJan 2022
NCT05630001APPULSESingle Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan.P3completed52Novartis PharmaceuticalsApr 2023
NCT05086744LNP023Basket Study to Assess Efficacy, Safety and PK of Iptacopan (LNP023) in Autoimmune Benign Hematological DisordersP2terminated19Novartis PharmaceuticalsDec 2021
NCT04820530APPOINT-PNHStudy of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor TherapyP3completed40Novartis PharmaceuticalsJul 2021
NCT05078580PKPharmacokinetic (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.P1completed38Novartis PharmaceuticalsNov 2021
NCT03439839PNHStudy of Safety, Efficacy, Tolerability, Pharmacokinetics and Pharmacodynamics of LNP023 in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)P2completed16Novartis PharmaceuticalsApr 2018
NCT05222412Managed Access Programs for LNP023, Iptacopanavailable0Novartis 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.

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