A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors

Launched by HOFFMANN-LA ROCHE · Jun 12, 2020

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called crovalimab to see how safe and effective it is compared to an existing treatment called eculizumab for people with a condition known as Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a rare blood disorder that can cause symptoms like fatigue, dark-colored urine, and blood clots. The researchers aim to enroll about 190 participants who have been receiving eculizumab or another similar medication for at least three months and have certain blood test results that meet the study’s criteria.

To be eligible for this trial, participants must weigh at least 40 kg and have a confirmed diagnosis of PNH. Women who can become pregnant need to agree to use contraception during the study and for a period after treatment. Throughout the study, participants will attend regular visits where they will receive either crovalimab or continue their current treatment. This trial is currently recruiting participants, and it’s important to note that those with certain medical conditions or who are pregnant or breastfeeding cannot join. Overall, this study aims to gather valuable information that could help improve treatment options for individuals with PNH.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Body weight ≥ 40 kg at screening (pediatric participants with body weight \< 40 kg)
• • Treated with eculizumab or ravulizumab for PNH for at least 3 months prior to Day 1
• • Lactate Dehydrogenase Levels ≤ 2x the upper limit of normal (ULN) at screening
• • Willingness and ability to comply with all study visits and procedures
• • Documented diagnosis of PNH, confirmed by high sensitivity flow cytometry
• • Vaccination against Neisseria meningitidis serotypes A, C, W, and Y \< 3 years prior to initiation of study treatment; or, if not previously done, vaccination administered no later than one week after the first drug administration
• • Women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception during the treatment period and for 10.5 months after the final dose of crovalimab or for 3 months after the final dose of eculizumab (or longer if required by the local product label)
• Exclusion Criteria:
• • History of allogeneic bone marrow transplantation
• • History of myelodysplastic syndrome with Revised International Prognostic Scoring System (IPSS-R) prognostic risk categories of intermediate, high and very high
• • Pregnant or breastfeeding, or intending to become pregnant during the study, within 10.5 months after the final dose of crovalimab, or 3 months after the final dose of eculizumab (or longer if required by the local product label)
• • Participation in another interventional treatment study with an investigational agent or use of any experimental therapy within 28 days of screening or within 5 half-lives of that investigational product, whichever was greater: participants enrolled in an eculizumab or ravulizumab interventional study are eligible provided they fulfill eligibility (e.g., are willing and able to comply with the study assessments) and stop their participation in current trial before randomisation/enrolment
• • Positive for Active Hepatitis B and C infection (HBV/HCV)
• • Concurrent disease, treatment, procedure, or surgery or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose any additional risk for the participant, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study
• • History of or ongoing cryoglobulinemia at screening