Pozelimab Clinical Trials (2026): 18 Studies for Paroxysmal Nocturnal Hemoglobinuria & CD55-deficient Protein-losing Enteropathy
Hipa.ai Research · Source: ClinicalTrials.gov / AACT
Synced daily from ClinicalTrials.gov via AACT. Last sync: .
18 Pozelimab clinical trials are catalogued on Hipa.ai from the AACT mirror of ClinicalTrials.gov, spanning 10 indications (including Paroxysmal Nocturnal Hemoglobinuria & CD55-deficient Protein-losing Enteropathy) and 1,903 total planned enrollment. 6 Phase 3 Pozelimab trials and 2 Phase 2, 1 Phase 4 are listed. 6 Pozelimab clinical trials are actively recruiting at 148 U.S. sites across 31 states. Most recent activity: NCT06541704 (May 2032).
Where to Participate: All Pozelimab Trial Sites in the U.S. (148 sites across 31 states)
Every actively recruiting Pozelimabtrial 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 |
|---|---|---|---|---|
| AZ | Barnet Dulaney Perkins Eye Center-Ophthamalogy | Chandler85224 | NCT06541704 | Map |
| AZ | Associated Retina Consultants | Phoenix85020 | NCT06541704 | Map |
| AZ | Barnet Dulaney Perkins Eye Center | Phoenix85016 | NCT06541704 | Map |
| AZ | Retinal Research Institute | Phoenix85053 | NCT06541704 | Map |
| AZ | Retina Macula Institute of Arizona | Scottsdale85255 | NCT06541704 | Map |
| AZ | Retina Associates of Tuscan | Tucson85712 | NCT06541704 | Map |
| AR | Retina Partners of Northwest Arkansas, PLLC | Springdale72764 | NCT06541704 | Map |
| CA | Retina Specialists of Beverly Hills- Department of Ophthalmology | Beverly Hills90212 | NCT06541704 | Map |
| CA | Retina Vitreous Associates Medical Group | Beverly Hills90211 | NCT06541704 | Map |
| CA | Eye Medical Center of Fresno | Fresno93720 | NCT06541704 | Map |
| CA | Salehi Retina Institute dba Retina Associates of Southern California | Huntington Beach92647 | NCT06541704 | Map |
| CA | University of California Irvine | Irvine92617 | NCT06541704 | Map |
| CA | University of California San Diego, Jacobs Retina Center | La Jolla92093 | NCT06541704 | Map |
| CA | South Coast Retina Center | Long Beach90807 | NCT06541704 | Map |
| CA | Jules Stein Eye Institute in David Geffen School of Medicine at Ucla | Los Angeles90095 | NCT06541704 | Map |
| CA | Kaiser Permanente Los Angeles Medical Center | Los Angeles90027 | NCT06541704 | Map |
| CA | Retinal Consultants Medical Group Inc Modesto | Modesto95356 | NCT06541704 | Map |
| CA | Northern California Retina Vitreous Associates Medical Group, Inc. | Mountain View94040 | NCT06541704 | Map |
| CA | California Retina Consultants | Oxnard93036 | NCT06541704 | Map |
| CA | California Eye Specialists Medical Group Inc. | Pasadena91107 | NCT06541704 | Map |
| CA | Retina Consultants San Diego | Poway92064 | NCT06541704 | Map |
| CA | California Eye Specialists Medical Group, Inc. | Redlands92373 | NCT06541704 | Map |
| CA | Optic Eye Care | Rowland Heights91748 | NCT06541704 | Map |
| CA | Retinal Consultants Medical Group (RCMG) - Greenback Lane Retina Center | Sacramento95841 | NCT06541704 | Map |
| CA | UC Davis Health Eye Center | Sacramento95817 | NCT06541704 | Map |
| CA | Vrmg Inc | Sacramento95825 | NCT06541704 | Map |
| CA | West Coast Retina Medical Group, Inc. | San Francisco94109 | NCT06541704 | Map |
| CA | Orange County Retina Medical Group | Santa Ana92705 | NCT06541704 | Map |
| CA | Macula Retina Vitreous Research Institute | Torrance90503 | NCT06541704 | Map |
| CO | University of Colorado | Aurora80045 | NCT06541704 | Map |
| CO | Retina Consultants of Southern Colorado | Colorado Springs80909 | NCT06541704 | Map |
| CO | Retina Specialists of Colorado - Denver | Denver80222 | NCT06541704 | Map |
| CO | Southwest Retina Research Center | Durango81303 | NCT06541704 | Map |
| CO | Colorado Retina - Lakewood | Lakewood80228 | NCT06541704 | Map |
| CO | Colorado Retina Associates | Lakewood80228 | NCT07230834 | Map |
| CO | Eye care Center of Northern Colorado doing business as Advanced Vision Research Institute | Longmont80503 | NCT06541704 | Map |
| FL | Advanced Retina Institute-Ophthalmology | Bonita Springs34134 | NCT06541704 | Map |
| FL | Advanced Research | Coral Springs33067 | NCT06541704 | Map |
| FL | Retina Group of Florida | Fort Lauderdale33308 | NCT06541704 | Map |
| FL | ClearVista Clinical Research, Retina Center Tampa Bay | Hudson34667 | NCT06541704 | Map |
| FL | Florida Retina Institute, Jacksonville Perimeter Park- Ophthalmology | Jacksonville32216 | NCT06541704 | Map |
| FL | Florida Retina Consultants | Lakeland33805 | NCT06541704 | Map |
| FL | Bascom Palmer Eye Institute | Naples34103 | NCT06541704 | Map |
| FL | Florida Retina Institute - Orlando | Orlando32806 | NCT06541704 | Map |
| FL | Retina Care Specialists | Palm Beach Gardens33410 | NCT06541704 | Map |
| FL | Eye Associates of Pinellas | Pinellas Park33782 | NCT06541704 | Map |
| FL | Retina Group of Florida, RAS-Sarasota Retina Center-Ophthalmology | Sarasota34232 | NCT06541704 | Map |
| FL | Retina Vitreous Associates of Florida Saint Petersburg | St. Petersburg33711 | NCT06541704 | Map |
| FL | University of South Florida Ophthalmology | Tampa33612 | NCT06541704 | Map |
| FL | Center for Retina and Macular Disease | Winter Haven33880 | NCT06541704 | Map |
| GA | Southeast Retina Center | Augusta30909 | NCT06541704 | Map |
| GA | Georgia Retina - Marietta | Marietta30060 | NCT06541704 | Map |
| IL | Retinal Vitreal Consultants | Chicago60616 | NCT06541704 | Map |
| IL | The University of Chicago | Chicago60637 | NCT06541704 | Map |
| IL | University of Illinois at Chicago | Chicago60612 | NCT06541704 | Map |
| IL | University Retina and Macula Associates PC - Lemont | Lemont60439 | NCT06541704 | Map |
| IL | Illinois Retina Associates | Oak Park60304 | NCT06541704 | Map |
| IL | Springfield Clinic Main Campus East | Springfield62703 | NCT06541704 | Map |
| KS | Retina Associates, LLC | Lenexa66215 | NCT06541704 | Map |
| KY | University of Louisville | Louisville40202 | NCT06541704 | Map |
| LA | Retina Associate New Orleans | Metairie70001 | NCT06541704 | Map |
| LA | Ross Davis Retina | Metairie70006 | NCT06541704 | Map |
| LA | Ochsner Health | New Orleans70121 | NCT06541704 | Map |
| LA | Eye Assoc. of NE LA dba Haik Humble Eye Ctr | West Monroe71291 | NCT06541704 | Map |
| LA | Eye Associates of Northeast Louisiana dba Haik Humble Eye Center | West Monroe71291 | NCT06541704 | Map |
| MD | The Retina Care Center | Baltimore21209 | NCT06541704 | Map |
| MD | The Retina Group of Washington | Chevy Chase20815 | NCT06541704 | Map |
| MD | Cumberland Valley Retina Consultants-Ophthalmology | Hagerstown21740 | NCT06541704 | Map |
| MD | Mid Atlantic Retina Specialist | Hagerstown21740 | NCT06541704 | Map |
| MA | Massachusetts Eye And Ear Infirmary | Boston02114 | NCT06541704 | Map |
| MA | Opthalmic Consultants of Boston | Boston02114 | NCT06541704 | Map |
| MA | Ophthalmic Consultants of Boston | East Weymouth02189 | NCT07230834 | Map |
| MA | New England Retina Consultants, PC | Springfield01107 | NCT06541704 | Map |
| MA | Vitreo-Retinal Associates PC | Worcester01605 | NCT06541704 | Map |
| MI | Retina Associates of Michigan | Grand Blanc48439 | NCT06541704 | Map |
| MS | Mississippi Retina Associates | Madison39110 | NCT06541704 | Map |
| MS | Deep Blue Retina Clinical Research PLLC | Southaven38671 | NCT06541704 | Map |
| MO | Retina Research Institute, LLC | St Louis63144 | NCT06541704 | Map |
| NJ | Mid Atlantic Retina - Cherry Hill | Cherry Hill08034 | NCT06541704 | Map |
| NJ | New Jersey Retina | Teaneck07666 | NCT06541704 | Map |
| NJ | New Jersey Retina | Toms River08755 | NCT06541704 | Map |
| NM | Vision Research Center at Eye Associates of New Mexico | Albuquerque87109 | NCT06541704 | Map |
| NY | Sight MD | Brentwood11717 | NCT06541704 | Map |
| NY | Retina Vitreous Surgeons of Central New York, PC | Liverpool13088 | NCT06541704 | Map |
| NY | New York Ear & Eye- East 85th | New York10028 | NCT06541704 | Map |
| NY | Northwell Health- Manhattan, Ear, Eye and Throat Hospital- Ophthalmology | New York10065 | NCT06541704 | Map |
| NY | Retina Associates of New York, Upper East Side | New York10075 | NCT06541704 | Map |
| NY | Seeta Eye center | Poughkeepsie12603 | NCT06541704 | Map |
| NC | Western Carolina Retinal Associates | Asheville28803 | NCT06541704 | Map |
| NC | NC Retina Associates | Cary27511 | NCT06541704 | Map |
| NC | Graystone Eye | Hickory28602 | NCT06541704 | Map |
| NC | North Carolina Retina Associates | Wake Forest27587 | NCT06541704 | Map |
| NC | Piedmont Retina Specialist | Winston-Salem27103 | NCT06541704 | Map |
| OH | Cincinnati Eye Institute | Cincinnati45242 | NCT06541704 | Map |
| OH | University Hospitals Cleveland Medical Center | Cleveland44106 | NCT06541704 | Map |
| OH | The Ohio State University | Columbus43212 | NCT06541704 | Map |
| OH | Midwest Retina, Inc | Dublin43016 | NCT06541704 | Map |
| OK | Retina Vitreous Center - Edmond | Edmond73013 | NCT06541704 | Map |
| OK | Tulsa Retina Consultants | Tulsa74114 | NCT06541704 | Map |
| OR | Verum Research LLC | Eugene97401 | NCT06541704 | Map |
| OR | EyeHealth Northwest | Portland97225 | NCT06541704 | Map |
| OR | OHSU-Casey Eye Institute, Marquam Hill-Ophthalmology | Portland97239 | NCT06541704 | Map |
| OR | Retina Northwest, The Highlands Building | Portland97221 | NCT06541704 | Map |
| OR | Cascade Medical Research Institute LLC | Springfield97477 | NCT06541704 | Map |
| PA | Mid Atlantic Retina | Bethlehem18017 | NCT06541704 | Map |
| PA | Erie Retinal Surgery & Erie Retina Research | Erie16507 | NCT06541704 | Map |
| PA | Mid Atlantic Retina, Wills Eye Hospital | Philadelphia19107 | NCT06541704 | Map |
| PA | Vision Research Solutions - the Eye Institute | Philadelphia19141 | NCT06541704 | Map |
| PA | Sewickley Eye Group | Sewickley15143 | NCT06541704 | Map |
| SC | Retina Consultants of Carolina | Greenville29605 | NCT06541704 | Map |
| SC | Charleston Neuroscience Institute LLC | Ladson29456 | NCT06541704 | Map |
| SC | Charleston Neuroscience Institute, LLC | Mt. Pleasant29464 | NCT06541704 | Map |
| SC | Palmetto Retina Center | West Columbia29169 | NCT06541704 | Map |
| SD | Black Hills Regional Eye Institute | Rapid City57701 | NCT06541704 | Map |
| TN | Tennessee Retina, PC | Nashville37203 | NCT06541704 | Map |
| TN | Vanderbilt Eye Institute | Nashville37232 | NCT06541704 | Map |
| TX | Retina Research Institute of Texas | Abilene79606 | NCT06541704 | Map |
| TX | Austin Neuromuscular Center | Austin78759 | NCT06479863 | Map |
| TX | Austin Neuromuscular Center | Austin78759 | NCT06479863 | Map |
| TX | Austin Research Center for Retina | Austin78705 | NCT06541704 | Map |
| TX | Austin Retina Associates | Austin78705 | NCT06541704 | Map |
| TX | Austin Retina Associates | Austin78705 | NCT07230834 | Map |
| TX | Retina Consultants Texas | Bellaire77401 | NCT06541704 | Map |
| TX | Star Retina PLLC | Burleson76028 | NCT06541704 | Map |
| TX | Retina Foundation of the Southwest | Dallas75231 | NCT06541704 | Map |
| TX | Retina Foundation of the Southwest | Dallas75231 | NCT07230834 | Map |
| TX | Texas Retina Associates | Dallas75231 | NCT06541704 | Map |
| TX | University of Texas Southwestern Medical Center | Dallas75390 | NCT06541704 | Map |
| TX | Retina Consultants of Texas | Katy77494 | NCT06541704 | Map |
| TX | Valley Retina Institute, P.A. | McAllen78503 | NCT06541704 | Map |
| TX | Austin Retina Associates | Round Rock78681 | NCT06541704 | Map |
| TX | Medical Center Ophthalmology Associates | San Antonio78240 | NCT06541704 | Map |
| TX | Retina Associates of South Texas | San Antonio78240 | NCT06541704 | Map |
| TX | Retina Consultants of Texas | San Antonio78240 | NCT06541704 | Map |
| TX | Retina Consultants of Texas Brown Retina Institute | San Antonio78251 | NCT06541704 | Map |
| TX | Retina Center of Texas | Southlake76092 | NCT06541704 | Map |
| TX | Retina Consultants Texas | The Woodlands77384 | NCT06541704 | Map |
| TX | Retina Consultants Texas | The Woodlands77384 | NCT07230834 | Map |
| TX | Tyler Retina Research Institute- Ophthalmology | Tyler75703 | NCT06541704 | Map |
| UT | Retina Associates Of Utah Retina Specialist | Salt Lake City84107 | NCT06541704 | Map |
| UT | Rocky Mountain Retina Consultants | Salt Lake City84107 | NCT06541704 | Map |
| UT | Salt Lake Retina- Ophthalmology | West Jordan84088 | NCT06541704 | Map |
| VT | University of Vermont | Burlington05401 | NCT06541704 | Map |
| VA | Southeastern Retina Associates | Bristol24201 | NCT06541704 | Map |
| VA | Retina Group of Washington | Fairfax22031 | NCT06541704 | Map |
| VA | Piedmont Eye Center | Lynchburg24502 | NCT06541704 | Map |
| VA | Virginia Eye Institute | Richmond23226 | NCT06541704 | Map |
| WA | Spokane Eye Clinical Research | Spokane99204 | NCT06541704 | Map |
Pozelimab clinical trials at a glance: phase × recruitment status
| Phase | recruiting | active not recruiting | completed | terminated | no longer available | approved for marketing | Total |
|---|---|---|---|---|---|---|---|
| Early P1 | 1 | 1 | |||||
| P1 | 1 | 3 | 4 | ||||
| P2 | 2 | 2 | |||||
| P2/P3 | 1 | 1 | |||||
| P3 | 3 | 2 | 1 | 6 | |||
| P4 | 1 | 1 | |||||
| N/A | 1 | 2 | 3 |
Recruiting Pozelimab clinical trials (6)
Studies currently enrolling participants. Click any NCT ID to view the trial detail page (eligibility, sites, contacts).
| NCT ID | Title | Phase | Indication | Enrollment | Started |
|---|---|---|---|---|---|
| NCT06541704 | SIENNAA Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy | P3 | Age-related Macular Degeneration (AMD) | 975 | Oct 2024 |
| NCT07154745 | PNHA Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently | P3 | Paroxysmal Nocturnal Hemoglobinuria | 35 | May 2026 |
| NCT07142343 | A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease | P4 | CHAPLE Disease | 5 | Mar 2026 |
| NCT05744921 | ACCESS-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 Works | P3 | Paroxysmal Nocturnal Hemoglobinuria | 202 | Mar 2023 |
| NCT07230834 | VIENNAIntravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants | P1 | Geographic Atrophy (GA) | 54 | Jan 2026 |
| NCT06479863 | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis | Early P1 | Sporadic Inclusion Body Myositis (sIBM) | 10 | Aug 2024 |
Pozelimab clinical trials by indication
Paroxysmal Nocturnal Hemoglobinuria (7)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT07154745 | PNH | P3 | recruiting | A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently |
| NCT05744921 | ACCESS-EXT | P3 | recruiting | A 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 |
| NCT05133531 | ACCESS-1 | P3 | active not recruiting | A 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 |
| NCT04811716 | — | P2 | completed | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy |
| NCT06028594 | — | N/A | no longer available | Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria |
| NCT05131204 | ACCESS 2 | P3 | terminated | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria |
| NCT04888507 | — | P2 | completed | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy |
Healthy (2)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT04940364 | — | P1 | completed | A 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 |
| NCT04601844 | — | P1 | completed | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers |
CD55-deficient Protein-losing Enteropathy (2)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT04209634 | — | P2/P3 | completed | Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease) |
| NCT05232110 | — | N/A | approved for marketing | Compassionate Use of Pozelimab |
CHAPLE Disease (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT07142343 | — | P4 | recruiting | A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease |
Geographic Atrophy (GA) (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT07230834 | VIENNA | P1 | recruiting | Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants |
Age-related Macular Degeneration (AMD) (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT06541704 | SIENNA | P3 | recruiting | A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy |
Sporadic Inclusion Body Myositis (sIBM) (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT06479863 | — | Early P1 | recruiting | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis |
Generalized Myasthenia Gravis (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT05070858 | NIMBLE | P3 | active not recruiting | A 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 |
Healthy Volunteer (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT04491838 | — | P1 | completed | Study of a Single Subcutaneous Dose of Pozelimab Produced From Two Different Manufacturing Processes in Healthy Adult Participants |
CD55-Deficient Protein-Losing Enteropathy (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT06003881 | CHAPLE Disease | N/A | approved for marketing | Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy |
Pozelimab 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 |
|---|---|---|---|---|---|---|
| SIENNA | NCT06541704 | P3 | recruiting | Age-related Macular Degeneration (AMD) | 975 | — |
| PNH | NCT07154745 | P3 | recruiting | Paroxysmal Nocturnal Hemoglobinuria | 35 | — |
| ACCESS-EXT | NCT05744921 | P3 | recruiting | Paroxysmal Nocturnal Hemoglobinuria | 202 | — |
| NIMBLE | NCT05070858 | P3 | active not recruiting | Generalized Myasthenia Gravis | 288 | PMID 42030965 |
| ACCESS-1 | NCT05133531 | P3 | active not recruiting | Paroxysmal Nocturnal Hemoglobinuria | 202 | — |
| ACCESS 2 | NCT05131204 | P3 | terminated | Paroxysmal Nocturnal Hemoglobinuria | 3 | — |
Terminated and withdrawn Pozelimab trials (1)
Studies that ended before completion. Reasons are filed in each trial's ClinicalTrials.gov entry.
- NCT05131204terminatedEfficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
All Pozelimab trials (18)
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 |
|---|---|---|---|---|---|---|---|
| NCT06541704 | SIENNA | A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy | P3 | recruiting | 975 | Regeneron Pharmaceuticals | Oct 2024 |
| NCT07154745 | PNH | A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently | P3 | recruiting | 35 | Regeneron Pharmaceuticals | May 2026 |
| NCT07142343 | A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease | P4 | recruiting | 5 | Regeneron Pharmaceuticals | Mar 2026 | |
| NCT05744921 | ACCESS-EXT | A 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 | P3 | recruiting | 202 | Regeneron Pharmaceuticals | Mar 2023 |
| NCT05070858 | NIMBLE | A 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 | P3 | active not recruiting | 288 | Regeneron Pharmaceuticals | Dec 2021 |
| NCT07230834 | VIENNA | Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants | P1 | recruiting | 54 | Regeneron Pharmaceuticals | Jan 2026 |
| NCT06479863 | Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis | Early P1 | recruiting | 10 | Austin Neuromuscular Center | Aug 2024 | |
| NCT05133531 | ACCESS-1 | A 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 | P3 | active not recruiting | 202 | Regeneron Pharmaceuticals | Aug 2022 |
| NCT04209634 | Open-Label Efficacy and Safety Study of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease) | P2/P3 | completed | 10 | Regeneron Pharmaceuticals | Jan 2020 | |
| NCT04811716 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | P2 | completed | 24 | Regeneron Pharmaceuticals | Jul 2021 | |
| NCT06028594 | Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria | — | no longer available | 0 | Regeneron Pharmaceuticals | — | |
| NCT06003881 | CHAPLE Disease | Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy | — | approved for marketing | 0 | Regeneron Pharmaceuticals | — |
| NCT05131204 | ACCESS 2 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | P3 | terminated | 3 | Regeneron Pharmaceuticals | Oct 2022 |
| NCT04888507 | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | P2 | completed | 6 | Regeneron Pharmaceuticals | Jul 2021 | |
| NCT04940364 | A 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 | P1 | completed | 30 | Regeneron Pharmaceuticals | Aug 2021 | |
| NCT05232110 | Compassionate Use of Pozelimab | — | approved for marketing | 0 | Regeneron Pharmaceuticals | — | |
| NCT04601844 | Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers | P1 | completed | 19 | Regeneron Pharmaceuticals | Nov 2020 | |
| NCT04491838 | Study of a Single Subcutaneous Dose of Pozelimab Produced From Two Different Manufacturing Processes in Healthy Adult Participants | P1 | completed | 40 | Regeneron Pharmaceuticals | Aug 2020 |
Pozelimab clinical trials: frequently asked questions
- How many Pozelimab clinical trials are there?
- 18 Pozelimab clinical trials are catalogued on Hipa.ai, sourced from the AACT mirror of ClinicalTrials.gov. 6 are actively recruiting participants. 6 are marked completed.
- Are Pozelimab clinical trials recruiting in 2026?
- Yes — 6 Pozelimab trials are actively recruiting in 2026, with 148 U.S. sites across 31 states. See the recruiting trials and the per-state site list further down this page.
- What conditions is Pozelimab being studied for?
- Pozelimab is being studied for Paroxysmal Nocturnal Hemoglobinuria, CD55-deficient Protein-losing Enteropathy, Geographic Atrophy (GA), Healthy, Generalized Myasthenia Gravis and 7 more. The trials are grouped by indication on this page.
- What phase is Pozelimab in?
- Pozelimab clinical trials span 1 Phase 4, 6 Phase 3, 2 Phase 2, 4 Phase 1. The phase × status matrix at the top of this page breaks down counts by phase and recruitment status.
- Who sponsors Pozelimab clinical trials?
- The lead sponsor across the most Pozelimab trials is Regeneron Pharmaceuticals, with 1 additional lead sponsor 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 Pozelimabas 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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