Study of subcutaneous anifrolumab pharmacokinetics, pharmacodynamics and safety in children aged 5 to <18 years with systemic lupus erythematosus

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What is this study about?

Systemic Lupus Erythematosus is an autoimmune condition in which the body’s immune system mistakenly attacks its own organs and tissues, causing inflammation, joint pain, skin rashes, and fatigue. The trial uses the medication Anifrolumab, which is given as a subcutaneous injection (a shot placed just under the skin) to children aged 5 to under 18 years who have this disease.

The purpose of the study is to learn how the drug moves through the body, how it changes the immune response, and how safe it is for young patients. Participants will receive the medication at scheduled visits over several months, and blood samples will be taken to measure the drug’s level in the bloodstream (pharmacokinetics) and its effect on immune activity, including the activity of type I interferon (a protein that can drive inflammation) and other markers such as anti‑DNA antibodies and complement proteins that help assess disease activity. Doctors will also watch for any side effects throughout the study to evaluate safety, while the overall design remains open‑label, meaning all participants know they are receiving the active medication.

1 enrollment and consent

After agreeing to participate, you will sign a consent form that explains the study and your rights.

The study staff will confirm that you meet the age requirement (5 to less than 18 years) and have a diagnosis of systemic lupus erythematosus.

2 baseline assessments

Before receiving any medication, you will undergo a series of baseline evaluations. These include blood draws to measure antibodies, complement proteins, and other laboratory values.

The results provide a reference point for later comparisons during the study.

3 first dose of anifrolumab

You will receive a single subcutaneous injection of the study drug, Anifrolumab, using a pre‑filled syringe containing 120 mg of the medication.

The injection is given under the skin, typically in the abdomen or thigh, by a trained study staff member.

The dose is exactly 120 mg; the frequency and total number of doses are defined by the study protocol.

4 regular follow‑up visits and dosing

After the first injection, you will return to the study site for scheduled visits. At each visit you may receive additional injections of Anifrolumab as specified by the protocol.

During these visits, blood samples will be taken to monitor how the drug moves through your body (pharmacokinetics) and how it affects your immune system (pharmacodynamics).

The study will continue these visits for at least 12 weeks and may extend to 52 weeks, depending on the protocol schedule.

5 week 12 assessment

At approximately week 12, a comprehensive assessment will be performed.

Blood tests will measure the lowest drug concentration before the next dose (Ctrough,ss) and evaluate the suppression of a specific gene pattern called the type I interferon 21‑gene signature.

Additional laboratory measurements will include anti‑double‑stranded DNA antibodies and complement levels (C3, C4, CH50).

These results help determine the drug’s effect and safety after several doses.

6 extended monitoring up to week 52

You will continue to receive study medication and attend follow‑up visits through week 52.

At week 52, the same laboratory assessments performed at week 12 will be repeated to evaluate longer‑term effects.

The study team will also check for the presence of antibodies that might develop against Anifrolumab (anti‑drug antibodies).

7 study completion

After the final visit at week 52, the study will be concluded for you.

All final safety data and laboratory results will be recorded.

You will be thanked for your participation, and any remaining study medication will be discontinued.

Who Can Join the Study?

Be between 5 years old and younger than 18 years when you sign the consent form.
Have a doctor‑confirmed diagnosis of Systemic Lupus Erythematosus (SLE) that meets the 2019 EULAR/ACR criteria and has been present for at least 12 weeks before signing the consent form.
Be taking at least one of the usual lupus medicines for at least 4 weeks before signing the consent form.
Oral glucocorticoids (a steroid medicine that reduces inflammation) at a dose no higher than 1 mg per kilogram of body weight per day or 40 mg of prednisone—or an equivalent medicine—per day, whichever is lower.
Antimalarial drugs such as hydroxychloroquine, chloroquine, or quinacrine, taken for at least 4 weeks. These medicines help control lupus even though they were originally made to treat malaria.
One immunosuppressant (a drug that weakens the immune system to stop it from attacking the body) for at least 4 weeks. Allowed options and their maximum daily doses are:
– Azathioprine up to 200 mg per day,
– Mycophenolate mofetil up to 3 g per day or mycophenolic acid up to 2.16 g per day,
– Methotrexate (taken by mouth, injection under the skin, or injection into muscle) up to 25 mg per week,
– Mizoribine up to 150 mg per day,
– Tacrolimus up to 0.2 mg per kilogram of body weight per day.
Only one of these immunosuppressants may be used at a time, but glucocorticoids and/or antimalarials can be used together with it.
At the screening visit, have a disease activity score called SLEDAI‑2K of 6 or higher, which indicates moderate to severe active lupus. (SLEDAI‑2K is a questionnaire doctors use to measure how active the disease is.)
Weigh at least 15 kg (about 33 pounds) at the screening visit.

Who Cannot Join the Study?

Having active, severe kidney disease caused by lupus (classified as ISN/RPS or WHO Class III or IV ± Class V) with a lot of protein in the urine (proteinuria) when standard treatment is not enough.
Having active, severe, or unstable brain or nervous system lupus problems, such as aseptic meningitis (brain lining inflammation without infection), cerebral vasculitis (blood vessel inflammation in the brain), myelopathy (spinal cord damage), demyelinating syndromes (damage to nerve coverings), confusion, loss of consciousness, psychosis, stroke, cranial nerve problems, ongoing seizures (status epilepticus), loss of coordination (cerebellar ataxia), or damage to multiple nerves (mononeuritis multiplex).
Having any other serious illness, disorder, or finding that the doctor believes could increase risk, make participation difficult, or affect the study’s results.
Having a current or recent shingles (herpes zoster) infection that has not fully healed within 12 weeks before signing the consent form, or getting shingles between signing the consent and the first study day.
Having taken any commercially available biologic medication (a drug made from living cells) within the required wash‑out period, which is five times the drug’s half‑life, before signing the consent.
Having received B‑cell depleting chemotherapy drugs such as rituximab or obinutuzumab within 26 weeks before signing the consent.
Having taken any other experimental drug (investigational agent, either a small molecule or a biologic) within five half‑lives or the expected immune‑affecting period before signing the consent.
Taking any prohibited medication listed in the study’s appendix without completing the required wash‑out period before signing the consent.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Katholieke Universiteit te Leuven	Leuven	Belgium
Unidade Local De Saúde De Santa Maria, E.P.E.	Lisbon	Portugal

Other Sites

Site Name	City	Country
Centre Hospitalier Regional De La Citadelle	Liege	Belgium
Universite Catholique de Louvain (UCL) – Cliniques Universitaires Saint-Luc	Brussels	Belgium
Uaucxzutfsol Zrfsyolgvf Gnob	Gent	Belgium
Cyarhm Hnznunxagi Upjkzlfpvuamf Dn Stmyg Aiulznh Ehrhpm	Porto	Portugal

Trial status

Country	Status	Recruitment Start
Belgium	Not yet recruiting	18.11.2026
Portugal	Not yet recruiting	18.11.2026

Trial locations

Investigated Drugs:

Anifrolumab

Saphnelo is the study medication that contains the active ingredient anifrolumab. It is given as a subcutaneous injection, which means it is injected just under the skin. In this trial, children aged 5 to under 18 years who have systemic lupus erythematosus (SLE) receive Saphnelo so researchers can learn how the drug moves through the body, how it affects the disease, and whether it is safe for young patients.

Systemic Lupus Erythematosus – An autoimmune disorder in which the immune system mistakenly attacks healthy tissues throughout the body. It can cause inflammation of the skin, joints, kidneys, heart, lungs, and nervous system. Symptoms often appear in episodes that flare up and then improve, and new organs may become involved over time. The disease may start with mild signs such as joint pain or rash and can progress to affect internal organs, leading to more complex clinical features.

Trial ID:

2025-524578-41-00

Protocol code:

D3465C00008

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of subcutaneous anifrolumab pharmacokinetics, pharmacodynamics and safety in children aged 5 to <18 years with systemic lupus erythematosus

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?