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Tests treatment safety and results for Paroxysmal Nocturnal Hemoglobinuria (PNH)
Official title 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
ClinicalTrials.gov ID: NCT07187401
What this study is testing
What is ALN-CFB?
ALN-CFB is an investigational medicine, being studied as a potential treatment for paroxysmal nocturnal hemoglobinuria (pnh).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and continue to have anemia (low red blood cell count).
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing
- Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to...
- Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol
- Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1 Key
You likely can't join if
- Has history of bone marrow transplantation or receipt of an organ transplant
- Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms
- Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2...
- Has laboratory evidence of bone marrow failure, as described in the protocol
- Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol NOTE: Other Protocol Defined...
The study team makes the final eligibility decision.
Where it's taking place
- Toronto, Ontario, Canada
- Suwon, Gyeonggi-do, South Korea
- Seoul, South Korea
- Leeds, West Yorkshire, United Kingdom
- London, United Kingdom
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, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Toronto, Ontario, Canada; Suwon, Gyeonggi-do, South Korea; Seoul, South Korea; Leeds, West Yorkshire, United Kingdom; London, United Kingdom. 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 ClinicalTrials.gov; the study team decides eligibility.