ALN-CFB for Paroxysmal Nocturnal Hemoglobinuria

Recruiting at 1 trial location

Overseen By Clinical Trials Administrator

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Regeneron Pharmaceuticals

Must be taking: C5 inhibitors

Must not be taking: Antibiotics, antivirals, antifungals

Disqualifiers: Bone marrow transplant, meningococcal infection, others

Compensation: Varies

Number of Visits: Unknown

Duration: 6-8 weeks

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new drug, ALN-CFB, to determine its effectiveness for people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who continue to experience anemia despite other treatments. The trial aims to assess how well participants tolerate ALN-CFB and identify any side effects. Participants will receive either the drug or a placebo, which resembles the drug but contains no active ingredients. The study seeks individuals with PNH who have been on a stable dose of a C5 inhibitor and continue to have anemia. As a Phase 1/Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial requires participants to continue taking a stable dose of a C5 inhibitor medication they are already on. It does not specify if you need to stop any other medications.

Is there any evidence suggesting that ALN-CFB is likely to be safe for humans?

Research has shown that ALN-CFB is being tested for safety in people with Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells are destroyed, often requiring treatments to prevent the immune system from attacking these cells. ALN-CFB is a new treatment that aims to reduce this destruction by targeting a specific protein in the blood.

Currently, no detailed reports exist on side effects or negative reactions from ALN-CFB in humans. As the study is in its early stages, the main goal is to assess how well people tolerate the treatment, with close monitoring for any side effects.

Since this is an early-phase study, the focus is on determining if the drug is safe enough for further testing. While early studies often indicate if a treatment is generally safe, more research is needed to fully understand the potential risks and benefits for patients. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for PNH?

Most treatments for Paroxysmal Nocturnal Hemoglobinuria (PNH) work by inhibiting the complement system to prevent the destruction of red blood cells, with eculizumab and ravulizumab being common options. However, ALN-CFB works differently by targeting the complement factor B protein, which plays a crucial role in the alternative pathway of the complement system. This novel approach could potentially offer a more effective way to manage PNH by providing a new mechanism of action that may lead to better control of the disease. Researchers are excited about ALN-CFB because it represents a promising shift in how we might treat PNH, offering hope for improved outcomes for patients who may not respond optimally to current therapies.

What evidence suggests that ALN-CFB might be an effective treatment for Paroxysmal Nocturnal Hemoglobinuria?

Research shows that ALN-CFB is an experimental drug designed to target Complement Factor B (CFB), a part of the immune system. By blocking CFB, ALN-CFB might help reduce symptoms for people with Paroxysmal Nocturnal Hemoglobinuria (PNH), especially those who still experience anemia despite using a C5 inhibitor. Early results suggest that ALN-CFB can lower CFB levels in the blood, potentially preventing the destruction of red blood cells—a major issue in PNH. While more results are needed, targeting CFB remains a promising research area for this condition. Participants in this trial will receive either ALN-CFB in a single-ascending dose escalation or a placebo to evaluate its effectiveness and safety. ^{1 2 3 4 6}

Who Is on the Research Team?

Clinical Trial Management

Principal Investigator

Regeneron Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who still have anemia despite taking a C5 inhibitor. Participants must have confirmed PNH diagnosis, been on a stable dose of C5 inhibitor for at least 24 weeks, and have hemoglobin ≤10.5 g/dL plus high reticulocyte count.

Inclusion Criteria

I have been diagnosed with PNH confirmed by previous tests.

I have been on a stable dose of a C5 inhibitor for at least 24 weeks.

My hemoglobin is 10.5 g/dL or less, and I've had anemia before this check-up.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ALN-CFB or placebo to assess safety, tolerability, and pharmacokinetics

6-8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ALN-CFB

Trial Overview The study tests the safety and effectiveness of ALN-CFB, an experimental drug targeting Complement Factor B, against a placebo in people with PNH on C5 inhibitors but still anemic. It measures side effects, ALN-CFB blood levels over time, and its impact on Complement Factor B protein levels.

How Is the Trial Designed?

Group I: Single-Ascending Dose Escalation Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Regeneron Pharmaceuticals

Lead Sponsor

Trials

690

Recruited

948,000+

Founded

1988

Headquarters

Tarrytown, USA

Known For

Precision medicine

Top Products

Dupixent, EYLEA, Libtayo, Praluent

Leonard Schleifer

Regeneron Pharmaceuticals

Chief Executive Officer since 1988

MD and PhD in Medicine

George Yancopoulos

Regeneron Pharmaceuticals

Chief Medical Officer since 1997

MD from Harvard Medical School

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT07187401

A First-in-Human Safety and Efficacy Study of ALN-CFB, ... This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are ...

2. trial.medpath.com trial.medpath.com/clinical-trial/4818efed2bd726d0/nct07187401-aln-cfb-sirna-complement-factor-b-paroxysmal-nocturnal-hemoglobinuria-anemia

A First-in-Human Safety and Efficacy Study of ALN-CFB, a ... This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10112829/

Complement Inhibition in Paroxysmal Nocturnal ... Results from TRIUMPH and SHEPHERD provide evidence that eculizumab therapy for PNH is effective, safe, and well-tolerated in a standardized ...

4. tipranks.com tipranks.com/news/company-announcements/regeneron-pharmaceuticals-advances-with-aln-cfb-study-for-pnh-treatment

Regeneron Pharmaceuticals Advances with ALN-CFB ... ' This study aims to assess the safety and tolerability of ALN-CFB in adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who continue to ...

5. ctv.veeva.com ctv.veeva.com/study/a-first-in-human-safety-and-efficacy-study-of-aln-cfb-a-small-interfering-rna-sirna-targeting-com

A First-in-Human Safety and Efficacy Study of ALN-CFB, a ... This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) ...

6. go.drugbank.com go.drugbank.com/conditions/DBCOND0028737

Paroxysmal Nocturnal Hemoglobinuria (PNH) ... 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 ...