Patientforskning
Logga in
Alla studier
RekryteringFAS1, FAS2INTERVENTIONELL

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

This research study is investigating a new medication called ALN-CFB. It's for adults with a rare blood disorder called Paroxysmal Nocturnal Hemoglobinuria (PNH). Specifically, the study is for people who are already taking another medicine called a C5 inhibitor, but still have low red blood cell counts (anaemia). The main goals are to find out how safe ALN-CFB is, whether it causes side effects, and how well people tolerate it compared to a placebo (a dummy drug that looks the same but contains no active medicine). Researchers will also check how the drug works in the body and if it affects certain blood protein levels.

Överblick

Status
Rekrytering
Fas
FAS1, FAS2
Sponsor
Regeneron Pharmaceuticals
Inskrivningsmål
24
Starta
11 Feb 2026
Beräknad slutförande
15 Jul 2031
Paroxysmal Nocturnal Hemoglobinuria (PNH)Persistent Anemia

Vad handlar denna studie om?

This research study is looking into a new experimental medicine called ALN-CFB. It's designed for people who have a rare blood condition called Paroxysmal Nocturnal Hemoglobinuria, often shortened to PNH. PNH can cause red blood cells to break down, leading to various symptoms, including anaemia, which means having low numbers of red blood cells.

Many people with PNH are already treated with a type of medicine called a C5 inhibitor. However, for some, even with this treatment, their anaemia can continue. This study is specifically for these individuals – those with PNH who are on a C5 inhibitor but still suffer from ongoing anaemia.

The main purpose of this study is to carefully check how safe ALN-CFB is and how well people tolerate it. To do this, some participants will receive ALN-CFB, while others will receive a placebo. A placebo looks exactly like the study medicine but doesn't contain any active drug. Researchers will also be monitoring for any side effects, how much of the drug stays in the blood, and if it changes levels of a protein called Complement Factor B.

Viktiga punkter

  • This study explores a new drug called ALN-CFB for PNH patients with unresolved anaemia.
  • It's for adults already on a C5 inhibitor but still experiencing low red blood cell counts.
  • The main goals are to check the drug's safety, side effects, and how well it's tolerated.
  • Some participants will receive the active drug, others a placebo (dummy drug).
  • Close medical monitoring will be part of the study participation.

Vem kan vara berättigad?

To join this study, you must be an adult aged 18 or over. You need to have been diagnosed with PNH, confirmed by specific blood tests. It's also important that you've been consistently taking your current PNH medication (a C5 inhibitor like eculizumab, ravulizumab, or crovalimab) for at least 24 weeks before the study starts.

Your blood tests will need to show that you still have anaemia, meaning your haemoglobin level is 10.5 g/dL or lower, and you have a certain level of developing red blood cells (reticulocytes).

You wouldn't be able to join this study if you've had a bone marrow or organ transplant, or if you've had certain serious infections, such as meningococcal infection. Active infections or recent serious infections needing treatment would also prevent you from joining. Additionally, if you have other severe medical conditions not related to your PNH, or if tests show your bone marrow isn't working properly, you might not be suitable.

Kan denna studie passa dig?

Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.

  1. Är du 18 år eller äldre?
  2. Do you have a diagnosis of PNH confirmed by medical tests?
  3. Have you been taking your current PNH C5 inhibitor medication regularly for at least 6 months?
  4. Do you still have low red blood cell counts (anaemia) despite your current PNH treatment?
  5. Have you *not* had a bone marrow or organ transplant?
  6. Do you *not* have any active, serious infections or recent infections needing ongoing treatment?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

If you join this study, you'll either receive the experimental drug, ALN-CFB, or a placebo. Neither you nor your doctor will know which one you're receiving. You'll have regular visits to the study clinic for check-ups, which will include blood tests, physical exams, and answering questions about how you’re feeling. These visits are important for doctors to monitor your health, check for side effects, and see how the study drug is working. The total time you'd be involved in the study isn't specified here, but clinical studies usually involve a series of visits over several months.

Potentiella risker och fördelar

Taking part in any clinical study has potential benefits and risks. You might benefit from the new medication if it works, and you'll be closely monitored by medical professionals. However, there's a chance you could experience side effects, just like with any medication, and there's no guarantee the treatment will improve your condition. Researchers don't yet know all the effects of ALN-CFB. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Platser (8)

  • Toronto General Hospital
    Verifierat postnummer
    Toronto, Kanada· Rekrytering
  • St. Vincent Hospital - The Catholic University of Korea
    Verifierat postnummer
    Suwon, South Korea· Rekrytering
  • The Catholic University of Korea, Seoul St. Mary's Hospital
    Verifierat postnummer
    Seoul, Sydkorea· Rekrytering
  • Seoul National University Hospital
    Verifierat postnummer
    Seoul, Sydkorea· Rekrytering
  • Severance Hospital
    Verifierat postnummer
    Seoul, Sydkorea· Rekrytering
  • Samsung Medicinskt Center
    Verifierat postnummer
    Seoul, Sydkorea· Rekrytering
  • St. James's University Hospital
    Verifierat postnummer
    Leeds, Storbritannien· Rekrytering
  • Kings College Hospital NHS Foundation Trust
    Verifierat postnummer
    London, Storbritannien· Rekrytering

Vanliga frågor

What is PNH?

PNH is a rare blood disorder where red blood cells break down too early, leading to symptoms like tiredness (from anaemia) and blood clots.

What is a C5 inhibitor?

A C5 inhibitor is a type of medicine currently used to treat PNH by stopping some of the red blood cell breakdown.

What is a 'placebo' in this study?

A placebo is a 'dummy' treatment that looks like the real study drug but contains no active medicine. It helps researchers fairly compare the new drug's effects.

Will I know if I'm getting the study drug or the placebo?

No, this study is 'blinded,' meaning neither you nor your doctors will know if you're receiving ALN-CFB or the placebo until after the study is complete, or in an emergency.

What does 'first-in-human' mean?

It means this is one of the first times this new drug is being given to people, after extensive testing in laboratories, to carefully check its safety and how it works.

Hur du får reda på mer

Clinical Trials Administrator

clinicaltrials@regeneron.com 844-734-6643 Officiell rättegångsprotokoll

Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.

Intresserad av att delta?

Registrera ditt intresse

Dela dina uppgifter och forskarteamet för "A First-in-Human Safety and Efficacy Study of ALN-CFB, a Sma…" kommer att kontakta dig om du kan vara berättigad. Prata alltid med din läkare innan du går med på att delta.

Diskussion

Gemenskapsdiskussion

Drivs av vårt forum på community.patient.info. Var vänlig och respektfull — detta är inte medicinsk rådgivning.