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Looking for participantsPhase1/Phase2

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

Sponsor: Regeneron Pharmaceuticals

NCT ID: NCT07187401

View on ClinicalTrials.gov ↗

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.

Paroxysmal Nocturnal Hemoglobinuria (PNH)Persistent Anemia

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

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
Key Inclusion Criteria: 1. Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing 2. Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as described in the protocol 3. Has hemoglobin ≤10.5 g/dL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol 4. Has peripheral blood reticulocyte count of ≥100 x 10\^9/L at screening visit 1 Key Exclusion Criteria: 1. Has history of bone marrow transplantation or receipt of an organ transplant 2. Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms 3. Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period 4. Has laboratory evidence of bone marrow failure, as described in the protocol 5. Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol NOTE: Other Protocol Defined Inclusion / Exclusion Criteria Apply

Where Is This Study? (2 UK sites)

St. James's University Hospital

Leeds LS97TF, United Kingdom

Recruiting
Hospital R&D contact (matched)

R&I Team

leedsth-tr.researchfacilitation@nhs.net0113 2060469

Kings College Hospital NHS Foundation Trust

London SE5 9RS, United Kingdom

Recruiting
Hospital R&D contact (matched)

Jasmine Palmer

kch-tr.research@nhs.net0203 299 1980

How to Get in Touch

Clinical Trials Administrator

Sponsor contact

CONTACT

844-734-6643 clinicaltrials@regeneron.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-07