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A C5 Inhibitor-controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy (ACCESS-1)

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ravulizumab
Pozelimab
Cemdisiran
Eculizumab
Sponsored by
Regeneron Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring PNH

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes described in the protocol
  2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms described in the protocol
  3. LDH level ≥2 × ULN at the screening visit

Key Exclusion Criteria:

  1. Prior treatment with a complement inhibitor within 6 months prior to screening visit, unless patient was treated with eculizumab or ravulizumab and has documented C5 variant R885H/C in which case there is no exclusion of such patients
  2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
  3. Body weight <40 kilograms at screening visit
  4. Planned use of any complement inhibitor therapy other than study drugs during the treatment period
  5. Not meeting meningococcal vaccination requirements for ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit
  6. Any contraindication for receiving Neisseria meningitidis vaccination
  7. Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab prescribing information, where available, or national guidelines/local practice or if necessary when vaccination is less than 2 weeks from study treatment initiation)
  8. 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
  9. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Sites / Locations

  • The Oncology Institute of Hope and InnovationRecruiting
  • Toronto General HospitalRecruiting
  • George Papanikolaou HospitalRecruiting
  • Semmelweis EgyetemRecruiting
  • Fondazione Policlinico Universitario A GemelliRecruiting
  • Azienda Ospedaliera Città della Salute e della Scienza di TorinoRecruiting
  • Hematology Citta della Salute e della Scienza di TorinoRecruiting
  • Japanese Red Cross Aichi Medical Center Nagoya Daini HospitalRecruiting
  • Ogaki Municipal HospitalRecruiting
  • University of Tsukuba HospitalRecruiting
  • Panasonic Health Insurance Organization Matsushita Memorial HospitalRecruiting
  • NTT Medical Center TokyoRecruiting
  • Pusan National University HospitalRecruiting
  • Ajou University Medical CenterRecruiting
  • Gachon University Gil Medical CenterRecruiting
  • Korea University HospitalRecruiting
  • Severance HospitalRecruiting
  • Samsung Medical Center - PPDSRecruiting
  • Ewha Womans University Mokdong HospitalRecruiting
  • St. Vincent HospitalRecruiting
  • Hospital Tengku Ampuan AfzanRecruiting
  • Hospital AmpangRecruiting
  • Queen Elizabeth Hospital - Kota KinabaluRecruiting
  • Hospital Universitario Dr. Jose Eleuterio GonzálezRecruiting
  • Klinika Hematologii, Szpital Uniwersytecki Nr 2 im. Jana Biziela w BydgoszczyRecruiting
  • Uniwersyteckie Centrum KliniczneRecruiting
  • Instytut Hematologii i TransfuzjologiiRecruiting
  • Prof Dr I Chiricuta Institute of OncologyRecruiting
  • Filantropia Municipal Clinical HospitalRecruiting
  • Targu-Mures Emergency Clinical County HospitalRecruiting
  • National University HospitalRecruiting
  • Hospital Universitario BasurtoRecruiting
  • Hospital Clinic de BarcelonaRecruiting
  • Changhua Christian HospitalRecruiting
  • Hualien Tzu Chi HospitalRecruiting
  • Kaohsiung Medical University - Chung-Ho Memorial HospitalRecruiting
  • China Medical University Hospital - PPDSRecruiting
  • Tri-Service General HospitalRecruiting
  • Chang Gung HospitalRecruiting
  • National Taiwan University HospitalRecruiting
  • Rajavithi HospitalRecruiting
  • Songklanagarind Hospital Prince of Songkla UniversityRecruiting
  • King Chulalongkorn Memorial HospitalRecruiting
  • Chiang Mai UniversityRecruiting
  • Srinagarind HospitalRecruiting
  • Istanbul Universitesi Istanbul Tip Fakultesi HastanesiRecruiting
  • Ege Universitesi Tip Fakultesi HastanesiRecruiting
  • St James University HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Cohort A

Cohort B

Arm Description

Randomized 1:1

Randomized 1:1

Outcomes

Primary Outcome Measures

Percent change in lactate dehydrogenase (LDH)
Cohort A
Transfusion avoidance
Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
Maintenance of adequate control of hemolysis
Cohort B LDH ≤1.5 × ULN

Secondary Outcome Measures

Maintenance of adequate control of hemolysis
Cohort A LDH ≤1.5 × ULN
Breakthrough hemolysis
Cohort A and B LDH ≥2 × ULN per the protocol
Adequate control of hemolysis
Cohort A and B LDH ≤1.5 × ULN
Hemoglobin stabilization
Cohort A and B Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level per the protocol
Normalization of LDH
Cohort A and B LDH ≤1.0 × ULN per the protocol
Transfusion avoidance
Cohort A Not requiring an RBC transfusion as per protocol algorithm based on post-baseline hemoglobin values.
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.
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."
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."
Percent change in LDH
Cohort B
Rate of RBC transfused
Cohort A and B Per protocol algorithm
Number of units of RBC transfused
Cohort A and B Per protocol algorithm
Time to first LDH ≤1.5 × ULN
Cohort A and B
Time to first LDH ≤1.0 × ULN
Cohort A and B
Percentage of days with LDH ≤1.5 × ULN
Cohort A and B
Change in hemoglobin levels
Cohort A and B
Incidence and severity of treatment emergent serious adverse events (SAEs)
Cohort A and B
Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
Cohort A and B
Incidence and severity of TEAEs leading to treatment discontinuation
Cohort A and B
Change in total CH50
Cohort A and B
Percent change in total CH50
Cohort A and B
Concentration of total C5 in plasma
Cohort A and B
Concentrations of total pozelimab in serum
Cohort A and B
Concentrations of cemdisiran in plasma
Cohort A and B
Concentrations of total ravulizumab in serum
Cohort A
Concentrations of total eculizumab in serum
Cohort B
Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
Cohort A and B
Incidence of treatment emergent ADAs to cemdisiran
Cohort A and B

Full Information

First Posted
November 12, 2021
Last Updated
September 14, 2023
Sponsor
Regeneron Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT05133531
Brief Title
A C5 Inhibitor-controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy
Acronym
ACCESS-1
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 1, 2022 (Actual)
Primary Completion Date
March 1, 2027 (Anticipated)
Study Completion Date
March 1, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regeneron Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH and how the combination compares with 2 existing treatments, one called ravulizumab and the other called 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 your 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)

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
PNH

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
190 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Cohort A
Arm Type
Experimental
Arm Description
Randomized 1:1
Arm Title
Cohort B
Arm Type
Experimental
Arm Description
Randomized 1:1
Intervention Type
Drug
Intervention Name(s)
Ravulizumab
Other Intervention Name(s)
ALXN1210, Ultomiris
Intervention Description
Administered Intravenous (IV) per the protocol
Intervention Type
Drug
Intervention Name(s)
Pozelimab
Other Intervention Name(s)
REGN3918
Intervention Description
Administered IV and subcutaneous (SC) per the protocol
Intervention Type
Drug
Intervention Name(s)
Cemdisiran
Other Intervention Name(s)
ALN-CC5
Intervention Description
Administered SC per the protocol
Intervention Type
Drug
Intervention Name(s)
Eculizumab
Other Intervention Name(s)
Soliris
Intervention Description
Administered IV per the protocol
Primary Outcome Measure Information:
Title
Percent change in lactate dehydrogenase (LDH)
Description
Cohort A
Time Frame
From baseline to week 26
Title
Transfusion avoidance
Description
Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
Time Frame
From post-baseline day 1 through week 26
Title
Maintenance of adequate control of hemolysis
Description
Cohort B LDH ≤1.5 × ULN
Time Frame
From week 8 through week 26, inclusive
Secondary Outcome Measure Information:
Title
Maintenance of adequate control of hemolysis
Description
Cohort A LDH ≤1.5 × ULN
Time Frame
From week 8 through week 26, inclusive
Title
Breakthrough hemolysis
Description
Cohort A and B LDH ≥2 × ULN per the protocol
Time Frame
From post-baseline day 1 through week 26
Title
Adequate control of hemolysis
Description
Cohort A and B LDH ≤1.5 × ULN
Time Frame
From week 8 through week 26, inclusive
Title
Hemoglobin stabilization
Description
Cohort A and B Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level per the protocol
Time Frame
From day 1 (post-baseline) through week 26
Title
Normalization of LDH
Description
Cohort A and B LDH ≤1.0 × ULN per the protocol
Time Frame
Between week 8 through week 26, inclusive
Title
Transfusion avoidance
Description
Cohort A Not requiring an RBC transfusion as per protocol algorithm based on post-baseline hemoglobin values.
Time Frame
Day 1 through week 26
Title
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
Description
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.
Time Frame
From baseline to week 26
Title
Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30)
Description
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."
Time Frame
Change from baseline to week 26
Title
Change in global health status (GHS)/QoL scale score on the EORTC-QLC-C30
Description
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."
Time Frame
From baseline to week 26
Title
Percent change in LDH
Description
Cohort B
Time Frame
From baseline to week 26
Title
Rate of RBC transfused
Description
Cohort A and B Per protocol algorithm
Time Frame
Post-baseline Day 1 through week 26
Title
Number of units of RBC transfused
Description
Cohort A and B Per protocol algorithm
Time Frame
Post-baseline Day 1 through week 26
Title
Time to first LDH ≤1.5 × ULN
Description
Cohort A and B
Time Frame
Up to Week 26
Title
Time to first LDH ≤1.0 × ULN
Description
Cohort A and B
Time Frame
Up to Week 26
Title
Percentage of days with LDH ≤1.5 × ULN
Description
Cohort A and B
Time Frame
Between week 8 and week 26, inclusive
Title
Change in hemoglobin levels
Description
Cohort A and B
Time Frame
From baseline to week 26
Title
Incidence and severity of treatment emergent serious adverse events (SAEs)
Description
Cohort A and B
Time Frame
Up to 26 weeks
Title
Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
Description
Cohort A and B
Time Frame
Up to 26 weeks
Title
Incidence and severity of TEAEs leading to treatment discontinuation
Description
Cohort A and B
Time Frame
Up to 26 weeks
Title
Change in total CH50
Description
Cohort A and B
Time Frame
From baseline to week 26
Title
Percent change in total CH50
Description
Cohort A and B
Time Frame
From baseline to week 26
Title
Concentration of total C5 in plasma
Description
Cohort A and B
Time Frame
Up to 60 weeks
Title
Concentrations of total pozelimab in serum
Description
Cohort A and B
Time Frame
Up to 60 weeks
Title
Concentrations of cemdisiran in plasma
Description
Cohort A and B
Time Frame
Up to 60 weeks
Title
Concentrations of total ravulizumab in serum
Description
Cohort A
Time Frame
Up to 34 weeks
Title
Concentrations of total eculizumab in serum
Description
Cohort B
Time Frame
Up to 30 weeks
Title
Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
Description
Cohort A and B
Time Frame
Up to 52 weeks
Title
Incidence of treatment emergent ADAs to cemdisiran
Description
Cohort A and B
Time Frame
Up to 52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: 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 Key Exclusion Criteria: 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 for ravulizumab (Cohort A) or eculizumab (Cohort B) according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit Any contraindication for receiving Neisseria meningitidis vaccination 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 vaccination is less than 2 weeks from study treatment initiation) 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Clinical Trials Administrator
Phone
844-734-6643
Email
clinicaltrials@regeneron.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trial Management
Organizational Affiliation
Regeneron Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
The Oncology Institute of Hope and Innovation
City
Whittier
State/Province
California
ZIP/Postal Code
90603
Country
United States
Individual Site Status
Recruiting
Facility Name
Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 2C4
Country
Canada
Individual Site Status
Recruiting
Facility Name
George Papanikolaou Hospital
City
Thessaloniki
ZIP/Postal Code
570 10
Country
Greece
Individual Site Status
Recruiting
Facility Name
Semmelweis Egyetem
City
Budapest
ZIP/Postal Code
1089
Country
Hungary
Individual Site Status
Recruiting
Facility Name
Fondazione Policlinico Universitario A Gemelli
City
Roma
State/Province
Lazio
ZIP/Postal Code
168
Country
Italy
Individual Site Status
Recruiting
Facility Name
Azienda Ospedaliera Città della Salute e della Scienza di Torino
City
Torino
State/Province
Piemonte
ZIP/Postal Code
10126
Country
Italy
Individual Site Status
Recruiting
Facility Name
Hematology Citta della Salute e della Scienza di Torino
City
Turin
ZIP/Postal Code
10126
Country
Italy
Individual Site Status
Recruiting
Facility Name
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
City
Nagoya
State/Province
Aichi
ZIP/Postal Code
466-8650
Country
Japan
Individual Site Status
Recruiting
Facility Name
Ogaki Municipal Hospital
City
Ogaki city
State/Province
Gifu
ZIP/Postal Code
503-8502
Country
Japan
Individual Site Status
Recruiting
Facility Name
University of Tsukuba Hospital
City
Tsukuba-shi
State/Province
Ibaraki
ZIP/Postal Code
305-8576
Country
Japan
Individual Site Status
Recruiting
Facility Name
Panasonic Health Insurance Organization Matsushita Memorial Hospital
City
Moriguchi-city
State/Province
Osaka
ZIP/Postal Code
570-8540
Country
Japan
Individual Site Status
Recruiting
Facility Name
NTT Medical Center Tokyo
City
Shinagawa-ku
State/Province
Tokyo
ZIP/Postal Code
141-8625
Country
Japan
Individual Site Status
Recruiting
Facility Name
Pusan National University Hospital
City
Busan
ZIP/Postal Code
49241
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Ajou University Medical Center
City
Gyeonggi-do
ZIP/Postal Code
16499
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Gachon University Gil Medical Center
City
Incheon
ZIP/Postal Code
21565
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Korea University Hospital
City
Seoul
ZIP/Postal Code
02841
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Severance Hospital
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Samsung Medical Center - PPDS
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Ewha Womans University Mokdong Hospital
City
Seoul
ZIP/Postal Code
7895
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
St. Vincent Hospital
City
Suwon
ZIP/Postal Code
16247
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Hospital Tengku Ampuan Afzan
City
Kuantan
State/Province
Pahang
ZIP/Postal Code
25200
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Hospital Ampang
City
Ampang
State/Province
Selangor
ZIP/Postal Code
68000
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Queen Elizabeth Hospital - Kota Kinabalu
City
Kota Kinabalu
ZIP/Postal Code
88586
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Dr. Jose Eleuterio González
City
Monterrey
State/Province
Nuevo León
ZIP/Postal Code
64460
Country
Mexico
Individual Site Status
Recruiting
Facility Name
Klinika Hematologii, Szpital Uniwersytecki Nr 2 im. Jana Biziela w Bydgoszczy
City
Bydgoszcz
ZIP/Postal Code
85-168
Country
Poland
Individual Site Status
Recruiting
Facility Name
Uniwersyteckie Centrum Kliniczne
City
Gdansk
ZIP/Postal Code
80-214
Country
Poland
Individual Site Status
Recruiting
Facility Name
Instytut Hematologii i Transfuzjologii
City
Warszawa
ZIP/Postal Code
02-776
Country
Poland
Individual Site Status
Recruiting
Facility Name
Prof Dr I Chiricuta Institute of Oncology
City
Cluj-Napoca
State/Province
Cluj
ZIP/Postal Code
400015
Country
Romania
Individual Site Status
Recruiting
Facility Name
Filantropia Municipal Clinical Hospital
City
Craiova
State/Province
Dolj
ZIP/Postal Code
200143
Country
Romania
Individual Site Status
Recruiting
Facility Name
Targu-Mures Emergency Clinical County Hospital
City
Targu Mures
State/Province
Mures
ZIP/Postal Code
540136
Country
Romania
Individual Site Status
Recruiting
Facility Name
National University Hospital
City
Singapore
ZIP/Postal Code
119074
Country
Singapore
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Basurto
City
Bilbao
State/Province
Vizcaya
ZIP/Postal Code
48013
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
ZIP/Postal Code
8036
Country
Spain
Individual Site Status
Recruiting
Facility Name
Changhua Christian Hospital
City
Changhua City
ZIP/Postal Code
50006
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Hualien Tzu Chi Hospital
City
Hualien City
ZIP/Postal Code
97002
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Kaohsiung Medical University - Chung-Ho Memorial Hospital
City
Kaohsiung
ZIP/Postal Code
807
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
China Medical University Hospital - PPDS
City
Taichung
ZIP/Postal Code
40447
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Tri-Service General Hospital
City
Taipei
ZIP/Postal Code
11490
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Chang Gung Hospital
City
Taoyuan City
ZIP/Postal Code
33305
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
National Taiwan University Hospital
City
Tapei City
ZIP/Postal Code
100229
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Rajavithi Hospital
City
Ratchathewi
State/Province
Krung Thep Maha Nakhon-Bangkok
ZIP/Postal Code
10400
Country
Thailand
Individual Site Status
Recruiting
Facility Name
Songklanagarind Hospital Prince of Songkla University
City
Hat Yai
State/Province
Songkhla
ZIP/Postal Code
90110
Country
Thailand
Individual Site Status
Recruiting
Facility Name
King Chulalongkorn Memorial Hospital
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Individual Site Status
Recruiting
Facility Name
Chiang Mai University
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
Individual Site Status
Recruiting
Facility Name
Srinagarind Hospital
City
Khon Kaen
ZIP/Postal Code
40000
Country
Thailand
Individual Site Status
Recruiting
Facility Name
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
City
Istanbul
ZIP/Postal Code
34418
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Ege Universitesi Tip Fakultesi Hastanesi
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Individual Site Status
Recruiting
Facility Name
St James University Hospital
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
IPD Sharing Time Frame
When Regeneron has received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication, has made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry), has the legal authority to share the data, and has ensured the ability to protect participant privacy.
IPD Sharing Access Criteria
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
IPD Sharing URL
https://vivli.org/

Learn more about this trial

A C5 Inhibitor-controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

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