Ravulizumab
DrugAdministered Intravenous (IV) per the protocol
Other names: ALXN1210, Ultomiris
NCT Number: NCT05133531
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)
This study is active but is not currently recruiting participants.
Notify Me18 year and older
All sexes
Interventional
Phase 3
Centro de Estudos e Pesquisas em Hematologia e Oncologia, Santo André, São Paulo, Brazil
Healthy volunteers accepted: No
Only the study team can determine whether someone qualifies for participation.
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Administered Intravenous (IV) per the protocol
Other names: ALXN1210, Ultomiris
Administered IV and subcutaneous (SC) per the protocol
Other names: REGN3918
Administered SC per the protocol
Other names: ALN-CC5
Administered IV per the protocol
Other names: Soliris
Time frame: From baseline to week 26
Cohort A
Time frame: From post-baseline day 1 through week 26
Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
Time frame: From week 8 through week 26, inclusive
Cohort B LDH ≤1.5 × ULN at each visit
Time frame: From week 8 through week 26, inclusive
Cohort A and B LDH ≤1.5 × ULN
Time frame: From post-baseline day 1 through week 26
Cohort A and B LDH ≥2 × ULN per the protocol
Time frame: From week 8 through week 26, inclusive
Cohort A LDH ≤1.5 × ULN
Time frame: From day 1 (post-baseline) through week 26
Cohort A and B Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level per the protocol
Time frame: Between week 8 through week 26, inclusive
Cohort A and B LDH ≤1.0 × ULN per the protocol
Time frame: Day 1 through week 26
Cohort A Not requiring an RBC transfusion as per protocol algorithm based on post-baseline hemoglobin values.
Time frame: From baseline to week 26
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
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
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
Cohort B
Time frame: Post-baseline Day 1 through week 26
Cohort A and B Per protocol algorithm
Time frame: Post-baseline Day 1 through week 26
Cohort A and B Per protocol algorithm
Time frame: Up to Week 26
Cohort A and B
Time frame: Up to Week 26
Cohort A and B
Time frame: Between week 8 and week 26, inclusive
Cohort A and B
Time frame: From baseline to week 26
Cohort A and B
Time frame: Up to 26 weeks
Cohort A and B
Time frame: Up to 26 weeks
Cohort A and B
Time frame: Up to 26 weeks
Cohort A and B
Time frame: From baseline to week 26
Cohort A and B
Time frame: From baseline to week 26
Cohort A and B
Time frame: Up to 60 weeks
Cohort A and B
Time frame: Up to 60 weeks
Cohort A and B
Time frame: Up to 60 weeks
Cohort A and B
Time frame: Up to 34 weeks
Cohort A
Time frame: Up to 30 weeks
Cohort B
Time frame: Up to 60 weeks
Cohort A and B
Time frame: Up to 60 weeks
Cohort A and B
Regeneron Pharmaceuticals
Industry
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
Acronym: ACCESS-1
OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.
View the official ClinicalTrials.gov record (opens in a new tab)This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.
Published trials that share one or more normalized conditions with this study.
NCT04432584
Anemia, Anemia, Hemolytic
Charlotte, North Carolina, United States
View Trial DetailsNCT06298955
Anemia, Anemia, Hemolytic
Aachen, Germany
View Trial DetailsNCT04747613
Anemia, Anemia, Hemolytic
Duarte, California, United States
View Trial DetailsNCT05389449
Anemia, Anemia, Hemolytic
Los Angeles, California, United States
View Trial Details