Cemdisiran Clinical Trials (2026): 15 Studies for Paroxysmal Nocturnal Hemoglobinuria & Healthy

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

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

15 Cemdisiran clinical trials are catalogued on Hipa.ai from the AACT mirror of ClinicalTrials.gov, spanning 7 indications (including Paroxysmal Nocturnal Hemoglobinuria & Healthy) and 1,825 total planned enrollment. 6 Phase 3 Cemdisiran trials and 5 Phase 2 are listed. 4 Cemdisiran clinical trials are actively recruiting at 143 U.S. sites across 31 states. Most recent activity: NCT06541704 (May 2032).

Where to Participate: All Cemdisiran Trial Sites in the U.S. (143 sites across 31 states)

Every actively recruiting Cemdisirantrial 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
AZBarnet Dulaney Perkins Eye Center-OphthamalogyChandler85224NCT06541704Map
AZAssociated Retina ConsultantsPhoenix85020NCT06541704Map
AZBarnet Dulaney Perkins Eye CenterPhoenix85016NCT06541704Map
AZRetinal Research InstitutePhoenix85053NCT06541704Map
AZRetina Macula Institute of ArizonaScottsdale85255NCT06541704Map
AZRetina Associates of TuscanTucson85712NCT06541704Map
ARRetina Partners of Northwest Arkansas, PLLCSpringdale72764NCT06541704Map
CARetina Specialists of Beverly Hills- Department of OphthalmologyBeverly Hills90212NCT06541704Map
CARetina Vitreous Associates Medical GroupBeverly Hills90211NCT06541704Map
CAEye Medical Center of FresnoFresno93720NCT06541704Map
CASalehi Retina Institute dba Retina Associates of Southern CaliforniaHuntington Beach92647NCT06541704Map
CAUniversity of California IrvineIrvine92617NCT06541704Map
CAUniversity of California San Diego, Jacobs Retina CenterLa Jolla92093NCT06541704Map
CASouth Coast Retina CenterLong Beach90807NCT06541704Map
CAJules Stein Eye Institute in David Geffen School of Medicine at UclaLos Angeles90095NCT06541704Map
CAKaiser Permanente Los Angeles Medical CenterLos Angeles90027NCT06541704Map
CARetinal Consultants Medical Group Inc ModestoModesto95356NCT06541704Map
CANorthern California Retina Vitreous Associates Medical Group, Inc.Mountain View94040NCT06541704Map
CACalifornia Retina ConsultantsOxnard93036NCT06541704Map
CACalifornia Eye Specialists Medical Group Inc.Pasadena91107NCT06541704Map
CARetina Consultants San DiegoPoway92064NCT06541704Map
CACalifornia Eye Specialists Medical Group, Inc.Redlands92373NCT06541704Map
CAOptic Eye CareRowland Heights91748NCT06541704Map
CARetinal Consultants Medical Group (RCMG) - Greenback Lane Retina CenterSacramento95841NCT06541704Map
CAUC Davis Health Eye CenterSacramento95817NCT06541704Map
CAVrmg IncSacramento95825NCT06541704Map
CAWest Coast Retina Medical Group, Inc.San Francisco94109NCT06541704Map
CAOrange County Retina Medical GroupSanta Ana92705NCT06541704Map
CAMacula Retina Vitreous Research InstituteTorrance90503NCT06541704Map
COUniversity of ColoradoAurora80045NCT06541704Map
CORetina Consultants of Southern ColoradoColorado Springs80909NCT06541704Map
CORetina Specialists of Colorado - DenverDenver80222NCT06541704Map
COSouthwest Retina Research CenterDurango81303NCT06541704Map
COColorado Retina - LakewoodLakewood80228NCT06541704Map
COEye care Center of Northern Colorado doing business as Advanced Vision Research InstituteLongmont80503NCT06541704Map
FLAdvanced Retina Institute-OphthalmologyBonita Springs34134NCT06541704Map
FLAdvanced ResearchCoral Springs33067NCT06541704Map
FLRetina Group of FloridaFort Lauderdale33308NCT06541704Map
FLClearVista Clinical Research, Retina Center Tampa BayHudson34667NCT06541704Map
FLFlorida Retina Institute, Jacksonville Perimeter Park- OphthalmologyJacksonville32216NCT06541704Map
FLFlorida Retina ConsultantsLakeland33805NCT06541704Map
FLBascom Palmer Eye InstituteNaples34103NCT06541704Map
FLFlorida Retina Institute - OrlandoOrlando32806NCT06541704Map
FLRetina Care SpecialistsPalm Beach Gardens33410NCT06541704Map
FLEye Associates of PinellasPinellas Park33782NCT06541704Map
FLRetina Group of Florida, RAS-Sarasota Retina Center-OphthalmologySarasota34232NCT06541704Map
FLRetina Vitreous Associates of Florida Saint PetersburgSt. Petersburg33711NCT06541704Map
FLUniversity of South Florida OphthalmologyTampa33612NCT06541704Map
FLCenter for Retina and Macular DiseaseWinter Haven33880NCT06541704Map
GASoutheast Retina CenterAugusta30909NCT06541704Map
GAGeorgia Retina - MariettaMarietta30060NCT06541704Map
ILRetinal Vitreal ConsultantsChicago60616NCT06541704Map
ILThe University of ChicagoChicago60637NCT06541704Map
ILUniversity of Illinois at ChicagoChicago60612NCT06541704Map
ILUniversity Retina and Macula Associates PC - LemontLemont60439NCT06541704Map
ILIllinois Retina AssociatesOak Park60304NCT06541704Map
ILSpringfield Clinic Main Campus EastSpringfield62703NCT06541704Map
KSRetina Associates, LLCLenexa66215NCT06541704Map
KYUniversity of LouisvilleLouisville40202NCT06541704Map
LARetina Associate New OrleansMetairie70001NCT06541704Map
LARoss Davis RetinaMetairie70006NCT06541704Map
LAOchsner HealthNew Orleans70121NCT06541704Map
LAEye Assoc. of NE LA dba Haik Humble Eye CtrWest Monroe71291NCT06541704Map
LAEye Associates of Northeast Louisiana dba Haik Humble Eye CenterWest Monroe71291NCT06541704Map
MDThe Retina Care CenterBaltimore21209NCT06541704Map
MDThe Retina Group of WashingtonChevy Chase20815NCT06541704Map
MDCumberland Valley Retina Consultants-OphthalmologyHagerstown21740NCT06541704Map
MDMid Atlantic Retina SpecialistHagerstown21740NCT06541704Map
MAMassachusetts Eye And Ear InfirmaryBoston02114NCT06541704Map
MAOpthalmic Consultants of BostonBoston02114NCT06541704Map
MANew England Retina Consultants, PCSpringfield01107NCT06541704Map
MAVitreo-Retinal Associates PCWorcester01605NCT06541704Map
MIRetina Associates of MichiganGrand Blanc48439NCT06541704Map
MSMississippi Retina AssociatesMadison39110NCT06541704Map
MSDeep Blue Retina Clinical Research PLLCSouthaven38671NCT06541704Map
MORetina Research Institute, LLCSt Louis63144NCT06541704Map
NJMid Atlantic Retina - Cherry HillCherry Hill08034NCT06541704Map
NJNew Jersey RetinaTeaneck07666NCT06541704Map
NJNew Jersey RetinaToms River08755NCT06541704Map
NMVision Research Center at Eye Associates of New MexicoAlbuquerque87109NCT06541704Map
NYSight MDBrentwood11717NCT06541704Map
NYRetina Vitreous Surgeons of Central New York, PCLiverpool13088NCT06541704Map
NYNew York Ear & Eye- East 85thNew York10028NCT06541704Map
NYNorthwell Health- Manhattan, Ear, Eye and Throat Hospital- OphthalmologyNew York10065NCT06541704Map
NYRetina Associates of New York, Upper East SideNew York10075NCT06541704Map
NYSeeta Eye centerPoughkeepsie12603NCT06541704Map
NCWestern Carolina Retinal AssociatesAsheville28803NCT06541704Map
NCNC Retina AssociatesCary27511NCT06541704Map
NCGraystone EyeHickory28602NCT06541704Map
NCNorth Carolina Retina AssociatesWake Forest27587NCT06541704Map
NCPiedmont Retina SpecialistWinston-Salem27103NCT06541704Map
OHCincinnati Eye InstituteCincinnati45242NCT06541704Map
OHUniversity Hospitals Cleveland Medical CenterCleveland44106NCT06541704Map
OHThe Ohio State UniversityColumbus43212NCT06541704Map
OHMidwest Retina, IncDublin43016NCT06541704Map
OKRetina Vitreous Center - EdmondEdmond73013NCT06541704Map
OKTulsa Retina ConsultantsTulsa74114NCT06541704Map
ORVerum Research LLCEugene97401NCT06541704Map
OREyeHealth NorthwestPortland97225NCT06541704Map
OROHSU-Casey Eye Institute, Marquam Hill-OphthalmologyPortland97239NCT06541704Map
ORRetina Northwest, The Highlands BuildingPortland97221NCT06541704Map
ORCascade Medical Research Institute LLCSpringfield97477NCT06541704Map
PAMid Atlantic RetinaBethlehem18017NCT06541704Map
PAErie Retinal Surgery & Erie Retina ResearchErie16507NCT06541704Map
PAMid Atlantic Retina, Wills Eye HospitalPhiladelphia19107NCT06541704Map
PAVision Research Solutions - the Eye InstitutePhiladelphia19141NCT06541704Map
PASewickley Eye GroupSewickley15143NCT06541704Map
SCRetina Consultants of CarolinaGreenville29605NCT06541704Map
SCCharleston Neuroscience Institute LLCLadson29456NCT06541704Map
SCCharleston Neuroscience Institute, LLCMt. Pleasant29464NCT06541704Map
SCPalmetto Retina CenterWest Columbia29169NCT06541704Map
SDBlack Hills Regional Eye InstituteRapid City57701NCT06541704Map
TNTennessee Retina, PCNashville37203NCT06541704Map
TNVanderbilt Eye InstituteNashville37232NCT06541704Map
TXRetina Research Institute of TexasAbilene79606NCT06541704Map
TXAustin Neuromuscular CenterAustin78759NCT06479863Map
TXAustin Neuromuscular CenterAustin78759NCT06479863Map
TXAustin Research Center for RetinaAustin78705NCT06541704Map
TXAustin Retina AssociatesAustin78705NCT06541704Map
TXRetina Consultants TexasBellaire77401NCT06541704Map
TXStar Retina PLLCBurleson76028NCT06541704Map
TXRetina Foundation of the SouthwestDallas75231NCT06541704Map
TXTexas Retina AssociatesDallas75231NCT06541704Map
TXUniversity of Texas Southwestern Medical CenterDallas75390NCT06541704Map
TXRetina Consultants of TexasKaty77494NCT06541704Map
TXValley Retina Institute, P.A.McAllen78503NCT06541704Map
TXAustin Retina AssociatesRound Rock78681NCT06541704Map
TXMedical Center Ophthalmology AssociatesSan Antonio78240NCT06541704Map
TXRetina Associates of South TexasSan Antonio78240NCT06541704Map
TXRetina Consultants of TexasSan Antonio78240NCT06541704Map
TXRetina Consultants of Texas Brown Retina InstituteSan Antonio78251NCT06541704Map
TXRetina Center of TexasSouthlake76092NCT06541704Map
TXRetina Consultants TexasThe Woodlands77384NCT06541704Map
TXTyler Retina Research Institute- OphthalmologyTyler75703NCT06541704Map
UTRetina Associates Of Utah Retina SpecialistSalt Lake City84107NCT06541704Map
UTRocky Mountain Retina ConsultantsSalt Lake City84107NCT06541704Map
UTSalt Lake Retina- OphthalmologyWest Jordan84088NCT06541704Map
VTUniversity of VermontBurlington05401NCT06541704Map
VASoutheastern Retina AssociatesBristol24201NCT06541704Map
VARetina Group of WashingtonFairfax22031NCT06541704Map
VAPiedmont Eye CenterLynchburg24502NCT06541704Map
VAVirginia Eye InstituteRichmond23226NCT06541704Map
WASpokane Eye Clinical ResearchSpokane99204NCT06541704Map

Cemdisiran clinical trials at a glance: phase × recruitment status

Phaserecruitingactive not recruitingcompletedterminatedwithdrawnno longer availableTotal
Early P111
P122
P22125
P33216
N/A11

Recruiting Cemdisiran clinical trials (4)

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

NCT IDTitlePhaseIndicationEnrollmentStarted
NCT06541704SIENNAA Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic AtrophyP3Age-related Macular Degeneration (AMD)975Oct 2024
NCT07154745PNHA Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyP3Paroxysmal Nocturnal Hemoglobinuria35May 2026
NCT05744921ACCESS-EXTA Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it WorksP3Paroxysmal Nocturnal Hemoglobinuria202Mar 2023
NCT06479863Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body MyositisEarly P1Sporadic Inclusion Body Myositis (sIBM)10Aug 2024

Cemdisiran clinical trials by indication

Paroxysmal Nocturnal Hemoglobinuria (7)

NCTAcronymPhaseStatusTitle
NCT07154745PNHP3recruitingA Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
NCT05744921ACCESS-EXTP3recruitingA Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
NCT05133531ACCESS-1P3active not recruitingA Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
NCT04811716P2completedPozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
NCT06028594N/Ano longer availableCompassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT05131204ACCESS 2P3terminatedEfficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT04888507P2completedPozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy

Healthy (2)

NCTAcronymPhaseStatusTitle
NCT04940364P1completedA Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy Volunteers
NCT04601844P1completedSafety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers

Atypical Hemolytic Uremic Syndrome (2)

NCTAcronymPhaseStatusTitle
NCT03999840DANCEP2withdrawnEculizumab to Cemdisiran Switch in aHUS
NCT03303313ALN-CC5P2withdrawnA Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome

Age-related Macular Degeneration (AMD) (1)

NCTAcronymPhaseStatusTitle
NCT06541704SIENNAP3recruitingA Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy

Sporadic Inclusion Body Myositis (sIBM) (1)

NCTAcronymPhaseStatusTitle
NCT06479863Early P1recruitingEfficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis

Generalized Myasthenia Gravis (1)

NCTAcronymPhaseStatusTitle
NCT05070858NIMBLEP3active not recruitingA Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

IgA Nephropathy (IgAN) (1)

NCTAcronymPhaseStatusTitle
NCT03841448P2terminatedA Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN)

Cemdisiran 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
SIENNANCT06541704P3recruitingAge-related Macular Degeneration (AMD)975
PNHNCT07154745P3recruitingParoxysmal Nocturnal Hemoglobinuria35
ACCESS-EXTNCT05744921P3recruitingParoxysmal Nocturnal Hemoglobinuria202
NIMBLENCT05070858P3active not recruitingGeneralized Myasthenia Gravis288PMID 42030965
ACCESS-1NCT05133531P3active not recruitingParoxysmal Nocturnal Hemoglobinuria202
ACCESS 2NCT05131204P3terminatedParoxysmal Nocturnal Hemoglobinuria3
DANCENCT03999840P2withdrawnAtypical Hemolytic Uremic Syndrome0
ALN-CC5NCT03303313P2withdrawnAtypical Hemolytic Uremic Syndrome0

Terminated and withdrawn Cemdisiran trials (4)

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

All Cemdisiran trials (15)

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

NCT IDAcronymTitlePhaseStatusEnrollmentSponsorStarted
NCT06541704SIENNAA Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic AtrophyP3recruiting975Regeneron PharmaceuticalsOct 2024
NCT07154745PNHA Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyP3recruiting35Regeneron PharmaceuticalsMay 2026
NCT05744921ACCESS-EXTA Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it WorksP3recruiting202Regeneron PharmaceuticalsMar 2023
NCT05070858NIMBLEA Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia GravisP3active not recruiting288Regeneron PharmaceuticalsDec 2021
NCT06479863Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body MyositisEarly P1recruiting10Austin Neuromuscular CenterAug 2024
NCT05133531ACCESS-1A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor TreatmentP3active not recruiting202Regeneron PharmaceuticalsAug 2022
NCT03999840DANCEEculizumab to Cemdisiran Switch in aHUSP2withdrawn0Mario Negri Institute for Pharmacological ResearchJan 2021
NCT04811716Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab MonotherapyP2completed24Regeneron PharmaceuticalsJul 2021
NCT06028594Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuriano longer available0Regeneron Pharmaceuticals
NCT05131204ACCESS 2Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal HemoglobinuriaP3terminated3Regeneron PharmaceuticalsOct 2022
NCT03841448A Study of Cemdisiran in Adults With Immunoglobulin A Nephropathy (IgAN)P2terminated31Alnylam PharmaceuticalsSep 2019
NCT04888507Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab TherapyP2completed6Regeneron PharmaceuticalsJul 2021
NCT04940364A Study to Examine the Safety, Tolerability and Biological Effects of Single Doses of Subcutaneously and Intravenously Administered Pozelimab as Monotherapy and in Combination With Single Doses of Subcutaneously Administered Cemdisiran in Adult Japanese Healthy VolunteersP1completed30Regeneron PharmaceuticalsAug 2021
NCT04601844Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult VolunteersP1completed19Regeneron PharmaceuticalsNov 2020
NCT03303313ALN-CC5A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic SyndromeP2withdrawn0Alnylam PharmaceuticalsSep 2017

Cemdisiran clinical trials: frequently asked questions

How many Cemdisiran clinical trials are there?
15 Cemdisiran clinical trials are catalogued on Hipa.ai, sourced from the AACT mirror of ClinicalTrials.gov. 4 are actively recruiting participants. 4 are marked completed.
Are Cemdisiran clinical trials recruiting in 2026?
Yes — 4 Cemdisiran trials are actively recruiting in 2026, with 143 U.S. sites across 31 states. See the recruiting trials and the per-state site list further down this page.
What conditions is Cemdisiran being studied for?
Cemdisiran is being studied for Paroxysmal Nocturnal Hemoglobinuria, Healthy, Atypical Hemolytic Uremic Syndrome, Geographic Atrophy (GA), Glomerulonephritis, IgA and 6 more. The trials are grouped by indication on this page.
What phase is Cemdisiran in?
Cemdisiran clinical trials span 6 Phase 3, 5 Phase 2, 2 Phase 1. The phase × status matrix at the top of this page breaks down counts by phase and recruitment status.
Who sponsors Cemdisiran clinical trials?
The lead sponsor across the most Cemdisiran trials is Regeneron Pharmaceuticals, with 3 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 Cemdisiranas 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 .

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