At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- ALN-CFB (drug), Placebo (drug)
- How long the study runs
- Study runs about 65 months (dates as stated)
- About the drug or intervention
- ALN-CFB — drug: Administered as defined in the protocol · Placebo — drug: Administered as defined in the protocol
- Patient visit burden
- Not specified by the sponsor
In plain English
This is a first-in-human study of a new medicine called ALN-CFB in adults with paroxysmal nocturnal haemoglobinuria (PNH), a rare blood condition. People joining must still have ongoing anaemia (low haemoglobin, the protein in red blood cells that carries oxygen) despite at least 24 weeks of treatment with a C5 inhibitor, a type of medicine already used for PNH. ALN-CFB is a small interfering RNA (siRNA), a type of medicine designed to switch off a specific protein called complement factor B.
Who can take part
- You have been diagnosed with PNH, confirmed by an earlier test called flow cytometry
- You have taken a steady dose of a C5 inhibitor medicine (eculizumab, an approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks before screening
- Your haemoglobin is 10.5 g/dL or below at the first screening visit, with signs of anaemia before this visit
- Your reticulocyte count (young red blood cells) is at least 100 x 10^9/L at the first screening visit
Who may not be able to
- You have had a bone marrow transplant or an organ transplant
- You have had meningococcal infection, or repeated infections with similar bacteria
- You have an active infection, or a recent infection needing antibiotic, antiviral, or antifungal treatment, within 2 weeks of screening or during screening
- Your blood tests show bone marrow failure
- You have recent or unstable medical problems not related to PNH or its complications
What taking part involves
- • Receiving ALN-CFB, a new medicine being given to people for the first time
- • Not stated — ask the trial team about how the medicine is given, the dose, or whether there is a comparison (placebo) group
Time commitment: Not stated — ask the trial team about how many visits are needed, how long the study lasts, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 18 Years and over
- Who
- All
- Number of participants
- 24
- Started
- 2026-02-11
- Last checked
- 2026-07
Plain English Summary
What is this study?
- • Testing a new treatment for paroxysmal nocturnal hemoglobinuria (pnh)
- • Phase1/Phase2 - 24 participants
- • This study is researching an experimental drug called ALN-CFB
Who can take part?
- • Ages 18 Years and over
- • Diagnosed with paroxysmal nocturnal hemoglobinuria (pnh)
Where?
- • Leeds - St. James's University Hospital
- • London - Kings College Hospital NHS Foundation Trust
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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). The aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug. The study is looking at several other research questions, including: * What side effects may happen from taking ALN-CFB * How much ALN-CFB is in the blood at different times * How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB
More detail
The protocol will be amended to describe Part B of the study after Part A data have been analyzed.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 18 Years and over
- Who can join: All genders
What the study is looking for
- ✓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...
- ✓Has red blood cell level ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the...
- ✓Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1
Who cannot take part
- ✗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,...
- ✗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
See the full criteria
Where Is This Study? (2 UK sites)
St. James's University Hospital
Leeds LS97TF, United Kingdom
Kings College Hospital NHS Foundation Trust
London SE5 9RS, United Kingdom
How to Get in Touch
Clinical Trials Administrator
Sponsor contactCONTACT
