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Cemdisiran

Cemdisiran is an investigational drug being studied in clinical trials for the treatment of various rare blood disorders, including atypical hemolytic uremic syndrome (aHUS), paroxysmal nocturnal hemoglobinuria (PNH), and IgA nephropathy (IgAN). These trials aim to evaluate the safety, efficacy, and potential benefits of Cemdisiran, either alone or in combination with other drugs, in managing these challenging conditions.

Table of Contents

What is Cemdisiran?

Cemdisiran, also known as ALN-CC5, is an investigational drug being developed to treat several rare blood disorders and kidney diseases[1]. It belongs to a class of medications called small interfering RNA (siRNA) therapeutics, which work by targeting specific genes in the body[2]. Cemdisiran is not yet approved for general use and is currently being studied in clinical trials to determine its safety and effectiveness.

How Does Cemdisiran Work?

Cemdisiran works by reducing the production of a protein called complement component 5 (C5) in the liver[4]. C5 is part of the body’s complement system, which is a group of proteins that help the immune system fight infections. In some rare diseases, the complement system becomes overactive and can damage healthy cells. By lowering C5 levels, Cemdisiran aims to reduce this harmful activity and alleviate symptoms of certain diseases[5].

Conditions Treated by Cemdisiran

Cemdisiran is being investigated for the treatment of several rare conditions, including:

  • Atypical Hemolytic Uremic Syndrome (aHUS): A rare disorder that causes abnormal blood clots to form in small blood vessels throughout the body, leading to kidney damage[1].
  • Paroxysmal Nocturnal Hemoglobinuria (PNH): A rare blood disorder where red blood cells break apart easily, leading to anemia and other complications[5].
  • IgA Nephropathy (IgAN): A kidney disease caused by buildup of an antibody called immunoglobulin A (IgA) in the kidneys, which can lead to inflammation and kidney damage[3].
  • Generalized Myasthenia Gravis (gMG): An autoimmune disorder that causes muscle weakness and fatigue[2].

Clinical Trials and Research

Cemdisiran is currently being studied in several clinical trials to evaluate its safety and effectiveness. These trials are investigating various aspects of the drug, including:

  • How well it controls symptoms of different diseases[1][3]
  • Its effects on blood tests and other markers of disease activity[2]
  • How it compares to existing treatments[4]
  • Its long-term safety and effectiveness[10]

Researchers are also studying how Cemdisiran works in combination with other medications, particularly a drug called Pozelimab[5].

How is Cemdisiran Administered?

Cemdisiran is typically administered as a subcutaneous (SC) injection, which means it is injected just under the skin[1]. In most clinical trials, it is given once every four weeks (Q4W)[10]. The exact dosage and frequency may vary depending on the specific condition being treated and the individual patient’s needs.

Combination Therapy with Pozelimab

Many of the ongoing clinical trials are studying Cemdisiran in combination with another drug called Pozelimab (also known as REGN3918)[5][7]. Pozelimab is an antibody that also targets the complement system. The combination of these two drugs is being investigated to see if it provides better results than either drug alone, particularly in treating conditions like Paroxysmal Nocturnal Hemoglobinuria (PNH)[10].

Potential Side Effects and Safety

As Cemdisiran is still in the research phase, all of its potential side effects are not yet known. Clinical trials are carefully monitoring patients for any adverse events (unwanted side effects) that may occur during treatment[10]. Some of the aspects being closely watched include:

  • Serious adverse events (SAEs): These are significant medical events that may require hospitalization or lead to other serious health issues[10].
  • Changes in blood tests: Researchers are monitoring how the drug affects various blood components and markers of disease activity[5].
  • Immune system reactions: The body’s response to the drug, including the potential development of antibodies against Cemdisiran[10].

It’s important to note that the safety profile of Cemdisiran is still being established through these clinical trials. Patients considering participating in a clinical trial should discuss the potential risks and benefits with their healthcare provider[10].

Aspect Details
Drug Name Cemdisiran (also known as ALN-CC5)
Mechanism of Action Reduces C5 mRNA in the liver, lowering circulating C5 protein levels and inhibiting terminal complement pathway activity
Administration Subcutaneous injection
Conditions Studied Atypical Hemolytic Uremic Syndrome (aHUS), Paroxysmal Nocturnal Hemoglobinuria (PNH), IgA Nephropathy (IgAN)
Key Outcomes Measured Safety, tolerability, pharmacokinetics, effects on disease markers (e.g., LDH, hemoglobin, proteinuria), quality of life improvements
Combination Therapy Studied alone and in combination with Pozelimab in some trials
Trial Phases Phase 2 studies for various conditions
Patient Populations Adults with rare blood disorders, some trials specifically for Japanese participants
Notable Assessments Complement activity (CH50), breakthrough hemolysis, transfusion requirements, fatigue scores

FAQ

  • What is Cemdisiran? Cemdisiran is an investigational drug designed to reduce the level of C5 mRNA in the liver, which in turn lowers the levels of circulating C5 protein. This action inhibits the terminal complement pathway activity, potentially preventing damage to blood cells and organs in certain rare blood disorders.
  • What conditions is Cemdisiran being studied for? Cemdisiran is being studied in clinical trials for several rare blood disorders, including atypical hemolytic uremic syndrome (aHUS), paroxysmal nocturnal hemoglobinuria (PNH), and IgA nephropathy (IgAN).
  • How is Cemdisiran administered? In the clinical trials, Cemdisiran is typically administered as a subcutaneous (under the skin) injection. The dosing schedule varies depending on the specific trial and condition being studied.
  • Is Cemdisiran being studied alone or in combination with other drugs? Cemdisiran is being studied both as a monotherapy (used alone) and in combination with other drugs, such as Pozelimab, depending on the specific trial and condition being investigated.
  • What are the main goals of the Cemdisiran clinical trials? The main goals of the Cemdisiran clinical trials are to evaluate its safety, tolerability, efficacy, and pharmacokinetics (how the drug moves through the body). Researchers are also assessing its effects on various disease markers and symptoms, as well as its potential to improve patients' quality of life.

Ongoing Clinical Trials on Cemdisiran

  • Study of Pozelimab and Cemdisiran for Adults with Geographic Atrophy from Age-Related Macular Degeneration

    Recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Hungary Italy Poland +1

  • Long-term safety and effectiveness study of pozelimab and cemdisiran combination treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH)

    Recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Greece Hungary Italy Poland Romania Spain

  • Study of Pozelimab and Cemdisiran in Adults with Paroxysmal Nocturnal Hemoglobinuria Not Responding Well to Current C5 Inhibitor Treatment

    Not yet recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Poland Spain

  • Study on the Safety and Effectiveness of Pozelimab and Cemdisiran for Adults with Paroxysmal Nocturnal Hemoglobinuria Not Recently Treated with Complement Inhibitors

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Greece Hungary Italy Poland Romania Spain

Glossary

  • Atypical Hemolytic Uremic Syndrome (aHUS): A rare, life-threatening disease characterized by the formation of blood clots in small blood vessels throughout the body, leading to kidney damage and failure.
  • Paroxysmal Nocturnal Hemoglobinuria (PNH): A rare blood disorder where red blood cells break apart prematurely, leading to various complications such as anemia, blood clots, and organ damage.
  • IgA Nephropathy (IgAN): A kidney disease caused by the buildup of immunoglobulin A (IgA) in the kidneys, which can lead to inflammation and kidney damage over time.
  • Complement System: A part of the immune system that helps or 'complements' the ability of antibodies and immune cells to clear microbes and damaged cells from the body.
  • C5: A protein that plays a crucial role in the complement system of the immune response.
  • Thrombotic Microangiopathy (TMA): A condition characterized by the formation of blood clots in small blood vessels throughout the body, which can lead to organ damage.
  • Lactate Dehydrogenase (LDH): An enzyme found in nearly all living cells that is often used as a marker of tissue damage or disease.
  • Hemoglobin: A protein in red blood cells that carries oxygen throughout the body.
  • Proteinuria: The presence of excess proteins in the urine, which can be a sign of kidney disease.
  • Pharmacokinetics: The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Pharmacodynamics: The study of how a drug affects the body, including its mechanism of action and relationship between drug concentration and effect.
  • Anti-Drug Antibodies (ADA): Antibodies produced by the immune system in response to a therapeutic drug, which can potentially reduce the drug's effectiveness or cause side effects.

References

  1. https://clinicaltrials.gov/study/NCT03303313
  2. https://clinicaltrials.gov/study/NCT05070858
  3. https://clinicaltrials.gov/study/NCT03841448
  4. https://clinicaltrials.gov/study/NCT03999840
  5. https://clinicaltrials.gov/study/NCT04888507
  6. https://clinicaltrials.gov/study/NCT06028594
  7. https://clinicaltrials.gov/study/NCT04811716
  8. https://clinicaltrials.gov/study/NCT04601844
  9. https://clinicaltrials.gov/study/NCT04940364
  10. https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effectiveness-of-pozelimab-and-cemdisiran-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh/