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Long‑Term Safety and Tolerability of Oral Iptacopan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria or IgA Nephropathy

InvestigationalNo placebo
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What is this trial about?

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

The study focuses on two rare conditions: Paroxysmal Nocturnal Hemoglobinuria, a blood disorder in which red blood cells are destroyed, and IgA nephropathy, a kidney disease that causes protein to leak into the urine. The treatment being examined is an oral capsule called iptacopan (code name LNP023) taken once daily.

The aim is to evaluate the long‑term safety and tolerability of the medication when given alone after completion of earlier pediatric studies. The design is open‑label and single‑arm, meaning all participants receive the drug and both the study team and participants know the treatment being used.

Participants who have finished the previous trials will continue to take the medication for an extended period, attending regular visits where health checks, blood tests, and kidney assessments are performed. Blood tests will monitor hemoglobin, the protein that carries oxygen, and a laboratory marker called LDH that can indicate red cell breakdown. Kidney function will be evaluated using a measurement called eGFR and a urine test called UPCR that measures protein loss. These visits occur at scheduled intervals over several years to watch for side effects and overall well‑being.

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

    Enrollment and consent

    After completing the previous pediatric study, you will be asked to join the extension program. you will review information about the study and sign a consent form that explains the purpose, procedures, and your rights.

    The study team will confirm that you meet the eligibility criteria for the extension.

  2. Step 2

    Baseline assessments

    Before taking any study medication, you will undergo a series of evaluations that include a physical exam, vital‑sign measurements (such as blood pressure and heart rate), and laboratory tests to check blood counts, kidney function, and other safety parameters.

    These results establish a reference point (baseline) for later comparisons.

  3. Step 3

    Start of medication

    You will begin taking iptacopan, a medication provided as 400 mg oral hard gelatin capsules. the dose is taken by mouth as directed by the study and will be continued for the duration of the extension program.

    The medication is intended to be taken regularly, and you will be instructed on the exact timing (for example, once daily).

  4. Step 4

    Regular safety visits

    Throughout the study period, you will attend scheduled visits where the study team will perform safety assessments. these include checking vital signs, repeating laboratory tests, and asking about any side effects or new health concerns.

    The frequency of these visits will be defined by the study protocol and will continue until the end of the study.

  5. Step 5

    Ongoing medication adherence and reporting

    You are expected to take the study medication exactly as prescribed and to keep a record of each dose.

    If you experience any adverse events (unwanted symptoms) or changes in your health, you should report them to the study team at the next visit or as instructed.

  6. Step 6

    Final visit and study completion

    At the end of the extension program (the end‑of‑treatment or end‑of‑study visit), you will undergo a final set of evaluations similar to the baseline assessments.

    The study team will review all safety data collected during the trial and will discuss any remaining questions you may have.

Who can join the trial?

5 criteria

  • Informed consent signed by a parent or legal guardian (and possibly the child’s own agreement, called assent) is required before joining the study.
  • The patient must have finished the treatment part of a previous Novartis pediatric study using the medicine iptacopan for any disease that involves the immune system’s complement pathway.
  • The patient must be willing and able to attend all scheduled doctor visits, follow the treatment plan, and complete any other study procedures.
  • The patient must have up‑to‑date vaccinations against Neisseria meningitidis (a bacteria that can cause meningitis), Streptococcus pneumoniae (a bacteria that can cause pneumonia), and Haemophilus influenzae (a bacteria that can cause serious infections); any needed booster shots must be given.
  • The doctor must judge that the patient may benefit from continued use of iptacopan and that the patient has been clinically stable (meaning their disease has not gotten worse) while on the medication.

Who cannot join the trial?

3 criteria

  • Having any other health problem (comorbidity), especially an active infection that spreads throughout the body (systemic bacterial, viral or fungal infection) or having cancer (malignancy) that the doctor thinks could increase your risk or affect the study results.
  • Having a history of repeated serious infections caused by “encapsulated organisms” (bacteria with a protective coating), such as the germs that cause meningitis (Neisseria meningitidis), pneumonia (Streptococcus pneumoniae) or certain throat infections (Haemophilus influenzae).
  • Being a woman who could become pregnant (women of child‑bearing potential) and not using a reliable birth‑control method during the study and for one week after stopping the study drug. Reliable methods include total abstinence (if it matches your usual lifestyle), surgeries that remove the ovaries or uterus, tubal ligation, an intrauterine device (IUD), hormonal pills, implants, injections, or condoms used with spermicide.
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Investigated drugs

iptacopan is an oral medication that comes in hard gelatin capsules. In this study, participants take iptacopan by mouth as the only treatment (monotherapy) after finishing earlier pediatric trials. The purpose of giving iptacopan is to monitor how safe it is and how well patients tolerate it when used for a long period of time. This medication is being studied because it may help manage the condition it is designed for, and it has been given a special status for rare diseases, known as an orphan drug.

What is already known about the treatment

IPTACOPAN - It is taken by mouth as a hard gelatin capsule, usually 400 mg once daily. The drug is an approved orphan medication that is being studied for long‑term safety in rare diseases such as paroxysmal nocturnal hemoglobinuria and IgA nephropathy. It works by blocking a protein called factor B in the complement system, which reduces the damage caused by an over‑active immune response. IPTACOPAN belongs to the class of complement factor B inhibitors, a type of targeted therapy that modulates the immune system.

Investigated diseases

  • Paroxysmal nocturnal hemoglobinuria

    A rare disorder in which red blood cells are unusually sensitive to destruction, causing episodes of hemolysis that can occur at night or during stress. The breakdown of red cells releases hemoglobin into the bloodstream, leading to anemia and occasional dark urine. Over time, the condition may cause the body to produce new red cells more rapidly, which can increase the workload on the bone marrow. Patients often notice fatigue and shortness of breath as the disease progresses.

  • IgA nephropathy

    A kidney disease characterized by the buildup of immunoglobulin A (IgA) in the glomeruli, the filtering units of the kidneys. This deposition triggers inflammation that can cause blood and protein to appear in the urine. As the inflammation continues, the filtering ability of the kidneys may gradually decline, leading to reduced kidney function. The disease often progresses slowly, with occasional episodes of increased urinary abnormalities.

Trial detailsLast updated 8 Oct 2026
Age0-17PhasePhase IIITrial ID2026-525677-39-00Protocol codeCLNP023I12001BEstimated enrolment46 patientsSponsorNovartis Pharma AG

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