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A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients with Paroxysmal Nocturnal Hemoglobinuria
EU CTIS ID: 2023-506287-14-00
What this study is testing
1. Part 1: Evaluate the safety and tolerability of single doses of crovalimab in healthy volunteers (HVs) Part 2, 3, and 4: Evaluate the safety and tolerability of RO7112689 for a total duration of 5 months in treatment naïve patients with PNH and PNH patients switching treatment to crovalimab Evaluate the pharmacodynamic (PD) effect of multiple doses of crovalimab on complement activity in patients with PNH Open Label Extension (OLE): Assess the long term safety of crovalimab
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Part 1 (HVs only) • Healthy male volunteers between the age of 21 and 55 years • Neisseria meningitidis vaccination against serogroups B and A, C, W, and Y • Subjects with a negative hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), hepatitis C antibody, and HIV test result are eligible for the study • Subjects who have been vaccinated against hepatitis B • Willing to comply with a non-smoking policy during the in-clinic portion of the study
- 2. Parts 2, 3, and 4 (PNH patients only) • Male or female patients between the age of 18 and 75 years • Neisseria meningitidis vaccination in accordance with most current local guidelines or Standard of Care (SOC) for patients at increased risk for meningococcal disease (Part 2 and Part 4 Arm A) • Patient has been vaccinated with Neisseria meningtidis vaccine(s) in accordance with most current local guidelines or SOC for patients at increased risk for meningococcal disease or is being revaccinated if applicable (Part 3 and Part 4 Arm B) • Stable dose for >= 28 days prior to screening of other therapies (immunosuppressant therapy, corticosteroids, iron supplements) • Negative pregnancy test for women of childbearing potential
- 3. Part 2 and Part 4 Arm A only (currently untreated PNH patients who are candidates for treatment with complement inhibitors only) • Hepatitis B patients can be enrolled if their LFT values are less than 2 × upper limit of normal (ULN) and there is no liver function impairment. • PNH patients who have not been treated with any complement inhibitor or if previously treated stopped treatment due to lack of efficacy based on a single missense C5 heterozygous mutation
- 4. Part 3 and Part 4 Arm B only (PNH patients currently treated with eculizumab only) • Subjects with a negative hepatitis B surface antigen (HBsAg), hepatitis B core antibody (HBcAb), hepatitis C antibody, and HIV test result are eligible for the study. o Subjects sero positive for HCV but adequately treated without detectable HCV RNA are eligible o Subjects who have been vaccinated against hepatitis B are eligible o Subjects sero positive for HBV but adequately treated without detectable HBV DNA are eligible • Patients are adequately controlled based on investigator opinion) • Patients receive regular infusions of eculizumab
- 5. OLE only – PNH patients • PNH patients who have completed Parts 2, 3, and 4 respectively • PNH patients who derived, in the Investigator’s opinion, benefit from treatment with crovalimab • For women who are not post-menopausal and have not undergone surgical sterilization agreement to remain abstinent or use a contraception method that results in a failure rate of <1% per year, during the treatment period and for 5.5 half-lives or at least 10.5 months after the last dose of crovalimab • Vaccination currency for Neisseria meningitidis serotypes A, C, W, Y and B should be maintained throughout the OLE, according to local guidelines or SOC as applicable in patients with complement deficiency. In the absence of clear local guidelines for Neisseria meningitidis, the Advisory Committee on Immunization Practices (ACIP) 2020 Guidelines are recommended
You likely can't join if
- 1. Parts 1, 2, 3, and 4: • Known or suspected hereditary complement deficiency • History of meningococcal meningitis • Any major episode of infection requiring hospitalization or treatment with intravenous (IV) antibiotics within 28 days prior to screening or oral antibiotics within 2 weeks prior to screening and up to first study drug administration • History of or currently active primary or secondary immunodeficiency, including known history of HIV infection • Evidence of malignant disease including myelodysplastic syndrome, or malignancies diagnosed within the previous 5 years • Pregnant or breastfeeding, or intending to become pregnant during the study, including the OLE period, within 46 weeks (approximately 10.5 months) after the final dose of crovalimab
- 2. Part 1 - HVs only: • Any clinically relevant history or the presence of moderate to severe respiratory, renal, hepatic, gastrointestinal, haematological, lymphatic, neurological, cardiovascular, psychiatric, musculoskeletal, or connective tissue disease. • Any major illness within one month before the screening examination or any febrile illness within 2 weeks prior to screening and up to first study drug administration. • Prior splenectomy • History or presence of clinically significant electrocardiogram (ECG) abnormalities or cardiovascular disease • Congenital or acquired complement deficiency • Carriers of Neisseria meningitidis based on cultures from naso-pharyngeal swabs
- 3. Parts 2, 3, and 4 - PNH patients only: • Evidence of moderate to severe concurrent renal, liver, cardiac, pulmonary or gastrointestinal disease not related to PNH as determined by the Investigator. • History of bone marrow transplantation. • Treatment with azathioprine or erythrocyte-stimulating agents within 14 days prior to first study drug administration • Splenectomy < 1 year before start of crovalimab
- 4. Parts 3 and 4 Arm B (PNH patients only): • Any evidence of seropositive auto-immune connective tissue diseases • Any evidence of active inflammatory conditions
The study team makes the final eligibility decision.
Where it's taking place
- Japan
- United States
- Switzerland
- Korea, Republic of
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Japan; United States; Switzerland; Korea, Republic of. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.