Paroxysmal Nocturnal Hemoglobinuria study: ALN-CFB
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 to see if it helps people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who still have low red blood cell counts while taking other treatments. is designed to lower levels of Complement Factor B (CFB) in the blood.
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 testing whether a new drug called ALN-CFB is safe. It also looks at how well it works for low red blood cells.
- Who it's for
- You may be able to join if you have PNH and still have low red blood cells while on current treatment.
- What's involved
- You would get a shot of the new drug or a pretend treatment. You would have blood tests to check for side effects and drug levels.
- Time commitment
- The study involves visits over time to check your health and blood levels.
- 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 is enrolling
- 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, but 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?
This study is enrolling now at 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 enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.