An adaptive Phase I/II study of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH)
Phase 1
Completed
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Registration Number
- JPRN-jRCT2080223462
- Lead Sponsor
- CHUGAI PHARMACEUTICAL CO., LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 44
Inclusion Criteria
Male or female patients between the age of 18 and 75 years
-A documented GPI-deficient PNH clone size of >= 10%
-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 (Part 2, 4)
-Patients receive regular infusions of eculizumab (Part 3, 4)
Exclusion Criteria
-Known or suspected hereditary complement deficiency.
-History of meningococcal meningitis.
-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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method safety<br>pharmacodynamics<br>Safety(adverse events), Pharmacodynamics(Complement activity, C5)
- Secondary Outcome Measures
Name Time Method efficacy<br>pharmacokinetics<br>Efficacy(LDH, free Hemoglobin), Pharmacokinetics