Paroxysmal Nocturnal HemoglobinuriaPhase 3ACCESS-1NCT05133531
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
A Phase 3 study testing Ravulizumab, Pozelimab and Cemdisiran in people with Paroxysmal Nocturnal Hemoglobinuria, run by Regeneron Pharmaceuticals at 68 sites in 24 countries.
Trial summary
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Ravulizumab, Pozelimab and Cemdisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 202 participants
- Where
- 68 sitesin 24 countries
- Study dates
- 1 Aug 2022to 26 Oct 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
- Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
- LDH level ≥2 × ULN at the screening visit
- Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol
Who cannot join
- Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- Body weight <40 kilograms at screening visit
- Planned use of any complement inhibitor therapy other than study drugs during the treatment period
- Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol.
- Any contraindication for receiving Neisseria meningitidis vaccinations (serotypes ACWY and B).
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- Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, where available, or national guidelines/local practice, or if necessary when administration of the first dose of the quadrivalent meningococcal vaccine [serotype ACWY] or the second dose of the serotype B meningococcal vaccine [when available] is less than 2 weeks prior to study treatment initiation) as described in the protocol
- Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
- Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
- Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Trial locations and contacts
68 sites in 24 countries.
68 sites in 24 countries
South Korea9 sites
Pusan National University Hospital
Busan, 49241
Gachon University Gil Medical Center
Incheon, Namdong-Gu, 21565
Korea University Hospital
Seoul, 02841
Severance Hospital
Seoul, 03722
Samsung Medical Center
Seoul, 06351
Seoul St. Mary's Hospital - The Catholic University of Korea
Seoul, 065791
Ewha Womans University Mokdong Hospital
Seoul, 07985
St. Vincent Hospital
Suwon, Gyeonggi-do, 16247
Ajou University Medical Center
Suwon, Gyeonggi-do, 16499
Taiwan9 sites
Changhua Christian Hospital
Changhua, 500-06
Hualien Tzu Chi Hospital
Hualien City, 97002
Kaohsiung Medical University Hospital
Kaohsiung City, 80756
China Medical University Hospital
Taichung, Central Taiwan, 40447
Taichung Veterans General Hospital (VGHTC)
Taichung, 4070
National Cheng Kung University Hospital
Tainan, 704
National Taiwan University Hospital
Taipei, 100229
Tri-Service General Hospital
Taipei, 114
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan, Hunan Province, 33305
India8 sites
Postgraduate Institute of Medical Education & Research (PGIMER)
Chandigarh, Punjab, 708248
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, 302017
Malabar Cancer Center, Kerala
Kannur, Kerala, 670103
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi, Kerala, 682041
King George Hospital
Lucknow, Uttar Pradesh, 226003
Sanjay Gandhi Postgraduate Institute of Medical Sciences
Lucknow, Uttar Pradesh, 226014
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra, 400022
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, National Capital Territory of Delhi, 110085
Japan5 sites
Matsushita Memorial Hospital
Moriguchi, Osaka, 570-8540
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken, 466-8650
Ogaki Municipal Hospital
Ōgaki, Gifu, 503-8502
NTT Medical Center Tokyo
Shinagawa-ku, Tokyo, 141-8625
University of Tsukuba Hospital
Tsukuba, Ibaraki, 305-8576
Spain4 sites
Hospital Clinic de Barcelona
Barcelona, 08036
Hospital Universitario Basurto
Bilbao, Vizcaya, 48013
Hospital General JM Morales Meseguer
Murcia, 30008
Hospital Clinico Universitario de Salamanca
Salamanca, 37007
Thailand4 sites
King Chulalongkorn Memorial Hospital
Bangkok, 10330
Chaing Mai University
Chiang Mai, 50200
Clinical Research Center, Faculty of Medicine, Prince of Songkla University
Hat Yai, Changwat Songkhla, 90110
Faculty of Medicine Khon Kaen University
Khon Kaen, 40002
Brazil3 sites
Centro de Estudos e Pesquisas em Hematologia e Oncologia
Santo André, São Paulo, 09060-650
A Beneficencia Portuguesa de Sao Paulo, BP Mirante
São Paulo, 01321-001
Casa de Saude Santa Marcelina
São Paulo, 08270-070
Italy3 sites
Aou Careggi
Florence, Firenze, 50139
Fondazione Policlinico Universitario A. Gemelli - IRCCS
Rome, Roma, 00168
Hematology Citta della Salute e della Scienza di Torino
Turin, 10126
Malaysia3 sites
Hospital Ampang
Ampang, Selangor, 68000
Hospital Queen Elizabeth
Kota Kinabalu, Sabah, 88586
Hospital Tg Ampuan Afzan
Kuantan, Pahang, 25200
Poland3 sites
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz, 85-168
University Clinical Center Medical University of Gdansk
Gdansk, Pomeranian Voivodeship, 80-214
Institute of Hematology and Transfusion Medicine
Warsaw, 02-776
Romania3 sites
Ion Chiricuta Oncology Institute
Cluj-Napoca, Cluj, 400015
Municipal Hospital Filantropia
Craiova, Dolj, 200143
Targu Mures Clinical County Emergency Hospital
Târgu Mureş, Mureș County, 540136
Turkey (Türkiye)2 sites
Istanbul University
Istanbul, 34418
Ege University
Izmir, 35100
United States1 site
The Oncology Institute of Hope & Innovation
Whittier, California, 90602
China1 site
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100032
Mexico1 site
Servicio de Hematologia del Hospital Universitario de la Uanl
Monterrey, Nuevo León, 64460
About this study
Official title: A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizumab.
The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug".
Read more
The study is looking at several research questions, including:
- How effective is the pozelimab + cemdisiran combination compared to ravulizumab?
- How effective is pozelimab + cemdisiran combination compared to eculizumab?
- What side effects may happen from taking the study drugs?
- How much study drugs are in the blood at different times?
- Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Ravulizumab, Pozelimab, Cemdisiran, Eculizumab
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RavulizumabDrug
Also called ALXN1210, Ultomiris
Administered Intravenous (IV) per the protocol
PozelimabDrug
Also called REGN3918
Administered IV and subcutaneous (SC) per the protocol
CemdisiranDrug
Also called ALN-CC5
Administered SC per the protocol
EculizumabDrug
Also called Soliris
Administered IV per the protocol
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Experimental | Randomized 1:1 Receives: Drug: Ravulizumab; Drug: Pozelimab; Drug: Cemdisiran |
| Cohort B | Experimental | Randomized 1:1 Receives: Drug: Pozelimab; Drug: Cemdisiran; Drug: Eculizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change in lactate dehydrogenase (LDH)
Cohort A
Measured: From baseline to week 26
Transfusion avoidance
Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
Measured: From post-baseline day 1 through week 26
Adequate control of hemolysis
Cohort B LDH ≤1.5 × ULN at each visit
Measured: From week 8 through week 26, inclusive
Secondary outcomes (28)
Maintenance of adequate control of hemolysis
Cohort A and B LDH ≤1.5 × ULN
Measured: From week 8 through week 26, inclusive
Breakthrough hemolysis
Cohort A and B LDH ≥2 × ULN per the protocol
Measured: From post-baseline day 1 through week 26
Adequate control of hemolysis
Cohort A LDH ≤1.5 × ULN
Measured: From week 8 through week 26, inclusive
Hemoglobin stabilization
Cohort A and B Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level per the protocol
Measured: From day 1 (post-baseline) through week 26
Normalization of LDH
Cohort A and B LDH ≤1.0 × ULN per the protocol
Measured: Between week 8 through week 26, inclusive
Transfusion avoidance
Cohort A Not requiring an RBC transfusion as per protocol algorithm based on post-baseline hemoglobin values.
Measured: Day 1 through week 26
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
Cohort A and B FACIT-Fatigue Scale is a 13-item, self-reported PRO measure assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.
Measured: From baseline to week 26
Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30)
Cohort A and B EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
Measured: From baseline to week 26
Change in global health status (GHS)/QoL scale score on the EORTC-QLC-C30
Cohort A and B EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
Measured: From baseline to week 26
Percent change in LDH
Cohort B
Measured: From baseline to week 26
Rate of RBC transfused
Cohort A and B Per protocol algorithm
Measured: Post-baseline Day 1 through week 26
Number of units of RBC transfused
Cohort A and B Per protocol algorithm
Measured: Post-baseline Day 1 through week 26
Time to first LDH ≤1.5 × ULN
Cohort A and B
Measured: Up to Week 26
Time to first LDH ≤1.0 × ULN
Cohort A and B
Measured: Up to Week 26
Percentage of days with LDH ≤1.5 × ULN
Cohort A and B
Measured: Between week 8 and week 26, inclusive
Change in hemoglobin levels
Cohort A and B
Measured: From baseline to week 26
Incidence and severity of treatment emergent serious adverse events (SAEs)
Cohort A and B
Measured: Up to 26 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
Cohort A and B
Measured: Up to 26 weeks
Incidence and severity of TEAEs leading to treatment discontinuation
Cohort A and B
Measured: Up to 26 weeks
Change in total CH50
Cohort A and B
Measured: From baseline to week 26
Percent change in total CH50
Cohort A and B
Measured: From baseline to week 26
Concentration of total C5 in plasma
Cohort A and B
Measured: Up to 60 weeks
Concentrations of total pozelimab in serum
Cohort A and B
Measured: Up to 60 weeks
Concentrations of cemdisiran in plasma
Cohort A and B
Measured: Up to 60 weeks
Concentrations of total ravulizumab in serum
Cohort A
Measured: Up to 34 weeks
Concentrations of total eculizumab in serum
Cohort B
Measured: Up to 30 weeks
Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
Cohort A and B
Measured: Up to 60 weeks
Incidence of treatment emergent ADAs to cemdisiran
Cohort A and B
Measured: Up to 60 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Nov 2021
- Study start1 Aug 2022
- Primary completion12 Oct 2026 (estimated)
- Study completion26 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 202 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05133531
- Sponsor's protocol ID
- R3918-PNH-2021
- Other IDs
- 2020-004486-40, 2023-509657-31-00, 2023
- Acronym
- ACCESS-1
- Status last verified
- Apr 2026
- First submitted
- 12 Nov 2021
- Last updated
- 30 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy
- ClinicalTrials.gov ID
- NCT05133531
- Acronym / sponsor ID
- ACCESS-1 / R3918-PNH-2021
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 202 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 12 Oct 2026 (estimated)
- Study completion
- 26 Oct 2026 (estimated)
- First posted
- 24 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizumab.
The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug".
The study is looking at several research questions, including:
- How effective is the pozelimab + cemdisiran combination compared to ravulizumab?
- How effective is pozelimab + cemdisiran combination compared to eculizumab?
- What side effects may happen from taking the study drugs?
- How much study drugs are in the blood at different times?
- Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Experimental | Drug: Ravulizumab, Drug: Pozelimab, Drug: CemdisiranRandomized 1:1 |
| Cohort B | Experimental | Drug: Pozelimab, Drug: Cemdisiran, Drug: EculizumabRandomized 1:1 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
- Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
- LDH level ≥2 × ULN at the screening visit
- Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol
Key exclusion criteria (10 listed)
- Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- Body weight <40 kilograms at screening visit
- Planned use of any complement inhibitor therapy other than study drugs during the treatment period
- Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RavulizumabAlso: ALXN1210, UltomirisAdministered Intravenous (IV) per the protocol | Drug | Cohort A |
| PozelimabAlso: REGN3918Administered IV and subcutaneous (SC) per the protocol | Drug | Cohort A, Cohort B |
| CemdisiranAlso: ALN-CC5Administered SC per the protocol | Drug | Cohort A, Cohort B |
| EculizumabAlso: SolirisAdministered IV per the protocol | Drug | Cohort B |
Primary outcome
Percent change in lactate dehydrogenase (LDH)
Time frame: From baseline to week 26
Cohort A
Transfusion avoidance
Time frame: From post-baseline day 1 through week 26
Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
Adequate control of hemolysis
Time frame: From week 8 through week 26, inclusive
Cohort B LDH ≤1.5 × ULN at each visit
28 secondary outcomes
Maintenance of adequate control of hemolysis
Time frame: From week 8 through week 26, inclusive
Breakthrough hemolysis
Time frame: From post-baseline day 1 through week 26
Adequate control of hemolysis
Time frame: From week 8 through week 26, inclusive
Hemoglobin stabilization
Time frame: From day 1 (post-baseline) through week 26
Normalization of LDH
Time frame: Between week 8 through week 26, inclusive
Transfusion avoidance
Time frame: Day 1 through week 26
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
Time frame: From baseline to week 26
Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30)
Time frame: From baseline to week 26
Change in global health status (GHS)/QoL scale score on the EORTC-QLC-C30
Time frame: From baseline to week 26
Percent change in LDH
Time frame: From baseline to week 26
Rate of RBC transfused
Time frame: Post-baseline Day 1 through week 26
Number of units of RBC transfused
Time frame: Post-baseline Day 1 through week 26
Time to first LDH ≤1.5 × ULN
Time frame: Up to Week 26
Time to first LDH ≤1.0 × ULN
Time frame: Up to Week 26
Percentage of days with LDH ≤1.5 × ULN
Time frame: Between week 8 and week 26, inclusive
Change in hemoglobin levels
Time frame: From baseline to week 26
Incidence and severity of treatment emergent serious adverse events (SAEs)
Time frame: Up to 26 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
Time frame: Up to 26 weeks
Incidence and severity of TEAEs leading to treatment discontinuation
Time frame: Up to 26 weeks
Change in total CH50
Time frame: From baseline to week 26
Percent change in total CH50
Time frame: From baseline to week 26
Concentration of total C5 in plasma
Time frame: Up to 60 weeks
Concentrations of total pozelimab in serum
Time frame: Up to 60 weeks
Concentrations of cemdisiran in plasma
Time frame: Up to 60 weeks
Concentrations of total ravulizumab in serum
Time frame: Up to 34 weeks
Concentrations of total eculizumab in serum
Time frame: Up to 30 weeks
Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
Time frame: Up to 60 weeks
Incidence of treatment emergent ADAs to cemdisiran
Time frame: Up to 60 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2026.
- Individual participant dataSharing planned: Yes. All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
68 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ravulizumab, Pozelimab and Cemdisiran in people with Paroxysmal Nocturnal Hemoglobinuria, run by Regeneron Pharmaceuticals at 68 sites in 24 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 68 sites in South Korea, Taiwan, India, Japan, Spain and 19 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change in lactate dehydrogenase (LDH) (time frame: From baseline to week 26). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2026.