A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor
← catalyst calendarNCT07187401 · readout in 1,198 d
Sponsored by Regeneron Pharmaceuticals (industry) · REGN — their whole pipeline →.
Phase
Phase 1/2
Status
Recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
24estimated
Sites
8
Countries
Canada, South Korea, United Kingdom
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2026-02-11 | actual | When the study began enrolling |
| Primary completion | 2029-11-29 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2031-07-15 | estimated | The whole study's end, after follow-up |
| First posted | 2025-09-23 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-09 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Persistent Anemia
Interventions
- Drug: ALN-CFB
- Drug: Placebo
Primary outcomes
what the primary-completion date above is the date OFOccurrence of Treatment-Emergent Adverse Events (TEAEs)
measured Through 365 Days
Severity of TEAEs
measured Through 365 Days
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