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Not yet recruitingRare disease

Study of VSA012 Sodium in Paroxysmal Nocturnal Hemoglobinuria Patients Who Are Complement Inhibitor‑Naïve or Have Persistent Anemia on C5 Inhibitor Therapy

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study involves people with Paroxysmal Nocturnal Hemoglobinuria, a rare condition in which the immune system destroys red blood cells, leading to anemia, fatigue, and an increased risk of blood clots. The investigational product is called VSA012 Injection, a medication given as a small shot under the skin (subcutaneous injection). The drug is being tested in individuals who have never used a complement‑inhibiting therapy, who stopped such therapy recently, or who continue to have anemia despite stable treatment with a C5 complement inhibitor.

The purpose of the study is to assess the safety and early effectiveness of the test medication in this patient group. Participants will receive a series of injections over several months and will attend regular clinic visits where blood samples are taken and simple checks such as heart rhythm (12‑lead ECG) and vital signs are recorded. Blood tests will include measurements of LDH, a marker that rises when red blood cells break down, and other indicators of how the drug moves through the body (pharmacokinetics) and how it works (pharmacodynamics). The study will also monitor for serious heart or vessel problems, known as MAVEs, and will track changes in anemia, need for blood transfusions, and quality‑of‑life scores. All procedures are designed to be straightforward and to minimize inconvenience.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Baseline Assessments

    After you join the study, clinical staff will record your medical history and perform a physical examination.

    You will have blood tests, a 12‑lead electrocardiogram (ECG), and measurements of vital signs such as blood pressure and body weight.

    These baseline results will be used to compare later safety and effectiveness findings.

  2. Step 2

    First VSA012 Injection

    You will receive the investigational medicine, VSA012 injection, as a subcutaneous (under the skin) injection.

    The exact amount of the drug is defined by the study protocol; the specific dose is not disclosed in the public description.

    The injection is administered by a qualified healthcare professional in a clinical setting.

  3. Step 3

    Safety Monitoring Visits

    Following each injection, you will attend regular visits where staff will check for any side effects or adverse events.

    Safety checks include repeat blood tests, ECG, vital‑sign measurements, physical examination, and body‑weight recording.

    Any serious or unexpected problems will be documented and evaluated promptly.

  4. Step 4

    Efficacy Evaluation Visits

    At scheduled visits, the study will measure how well the treatment works for your condition.

    Key measurements include the change in lactate dehydrogenase (LDH) levels, hemoglobin (Hb) concentration, and whether you need blood transfusions.

    Additional assessments may track biomarkers related to hemolysis and clotting risk, as well as quality‑of‑life scores such as the FACIT‑F questionnaire.

  5. Step 5

    Additional Doses (If Required)

    Depending on the study protocol, you may receive more than one dose of VSA012 injection over the trial period.

    Each additional dose will be given subcutaneously and followed by the same safety and efficacy assessments described above.

  6. Step 6

    End‑of‑Study Assessments

    When the study period ends, you will undergo a final set of evaluations similar to the baseline and monitoring visits.

    These final assessments will summarize safety data, overall drug exposure, and the overall effect on your disease signs and symptoms.

Who can join the trial?

6 criteria

  • Must have a confirmed diagnosis of Paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that can cause anemia and blood clots.
  • Must be male or female.
  • Must be within the age ranges allowed by the study, which include both children and adults.
  • Must be able to follow the study visits and procedures, or have a parent or guardian do so if under the legal age.
  • Must be considered a vulnerable participant (someone who may need extra protection) and still meet the study’s safety requirements.
  • Must be a patient with the condition, not a healthy volunteer.

Who cannot join the trial?

2 criteria

  • If you meet any of the exclusion criteria that are listed in the study protocol for Groups 1‑4 or Group 6, you cannot take part in the trial. (Exclusion criteria are specific conditions or factors that would make a person unsuitable for the study.)
  • If you meet any of the exclusion criteria that are listed in the study protocol for Groups 5 or Group 7, you cannot take part in the trial.
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Investigated drugs

VSA012 Injection is an experimental medication being tested for people with paroxysmal nocturnal hemoglobinuria (PNH). It is given as a small amount under the skin (subcutaneous injection). The drug is designed to work on the body’s complement system, a part of the immune system that can mistakenly destroy red blood cells in PNH. In this study, researchers are looking at how safe the injection is, how well people can tolerate it, how the drug moves through the body, how it affects the disease process, and whether it can improve symptoms such as anemia. Participants include those who have never used other complement‑inhibiting medicines, those who stopped such medicines recently, and those who still have low blood counts even while on stable complement‑inhibitor therapy.

What is already known about the treatment

VSA012 Injection - VSA012 Injection is given as a subcutaneous injection, meaning it is placed under the skin with a small needle. It is an experimental medicine currently being studied in early‑phase clinical trials for safety and effectiveness in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The drug works by blocking a protein called C5 in the immune system, which helps stop the destruction of red blood cells. It belongs to the class of complement inhibitors, a type of biologic therapy.

Investigated diseases

Paroxysmal nocturnal hemoglobinuria (PNH) - Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder in which the body's immune system attacks its own red blood cells, causing them to break down prematurely. This breakdown releases hemoglobin into the bloodstream, especially during the night or early morning. Over time, the loss of red blood cells can lead to persistent low blood counts and fatigue. Some people experience episodes of increased cell destruction that may come and go. The condition can also affect the blood's ability to clot, leading to occasional clot formation. As the disease continues, symptoms may become more frequent or noticeable.
Trial detailsLast updated 17 Oct 2026
Age18+ yearsPhasePhase ITrial ID2026-526187-21-00Protocol codeVSA012-1002Estimated enrolment12 patients

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