Active Pregnancy, Children & Inherited Conditions NIHR-supported project Infection & Immunity

A Phase III, randomized, double-blind, parallel-group, placebo-controlled study to evaluate the pharmacokinetics, pharmacodynamics, efficacy, and safety of IV Anifrolumab in pediatric participants 5 to < 18 years of age with moderate to severe active systemic lupus erythematosus while on background standard of care therapy. (BLOSSOM)

Funder NIHR (part of DHSC)
Recipient organisation NIHR Great Ormond Street Hospital Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Jan 2025 — Oct 2029

In plain English

AI plain-English summary

Children with lupus are about to receive a drug already proven safe and effective in adults, in a clinical trial that will determine the right dose for their bodies. Systemic lupus erythematosus (SLE) is an autoimmune disease where the immune system attacks the body’s own organs, causing damage and a wide range of symptoms. Anifrolumab works by dampening this immune overactivation. While the drug has been tested in adults, its effects in children remain unconfirmed. Children metabolise drugs differently, so simply using adult doses could be ineffective or unsafe. This trial will first test the drug in a small group of older, heavier children to see how it is distributed and eliminated, then adjust the dose for younger, lighter children. After the correct dose is confirmed, more children will receive monthly infusions of either anifrolumab or a placebo for nearly a year, followed by a period where all participants receive the active drug. If successful, the research could give children with lupus a targeted treatment option that reduces organ damage and symptoms, rather than relying solely on broad immunosuppressants. That would mean fewer hospital visits, less long-term harm, and a better quality of life for a group currently without a paediatric-specific therapy.

View original technical description
The immune system usually defends the person against external agents, like infections, but when a person has a disease called Systemic Lupus Erythematosus (SLE), the immune system also attacks the person’s own body, causing damage to organs and several different symptoms. Anifrolumab is a medication that reduces the activation of the immune system, reducing in this way the SLE manifestations. Prior studies already proved that Anifrolumab is effective and safe in the treatment of adults with SLE, but the effects of this medication in children are not confirmed yet. This study is to verify how Anifrolumab will act in children, how the medication is distributed and eliminated. For that, about 100 children from 5 to less than 18 years old will join the study. The study will start with 8 children from 12 to less than 18 years old, with more than 40Kg, who will receive one infusion of the medication or placebo (an infusion with saline solution, without medication) and the distribution and elimination of the medication will be evaluated, to confirm if children should receive the same dose of adults, or if dose should be adjusted. These children will continue in the study to receive medication or placebo in monthly infusions through 48 weeks. After dose confirmation in this group, other group of 8 children from more than 5 and less than 18 years old, with less than 40Kg and more than 15Kg will receive the dose assigned to the previous group, to confirm if additional dose adequation is needed for the group with less than 40Kg. After dose of Anifrolumab is confirmed, additional patients will be recruited in both groups of age and weight, to receive medication or placebo in monthly infusions through 52 weeks, plus additional 52 weeks where all patients will receive Anifrolumab monthly.

Researchers

Elena Moraitis (Principal Investigator)

Related Research

Grants with similar aims, by meaning.

A Phase III, randomized, double-blind, parallel-group, placebo-controlled study to evaluate the pharmacokinetics, pharmacodynamics, efficacy, and safety of IV Anifrolumab in pediatric participants 5 to < 18 years of age with moderate to severe active systemic lupus erythematosus while on background standard of care therapy.
A Phase III, Randomized, Double-blind, Parallel-group, Placebo controlled Study to Evaluate Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of IV Anifrolumab in Pediatric Participants 5 to <18 Years of Age with Moderate to Severe Active Systemic Lupus Erythematosus While on Background Standard of Care Therapy
Phase III, Randomised, double-blind, parallel-group, placebo-controlled study to evaluate the pharmacokinetics, pharmacodynamics, efficacy, and safety of IV Anifrolumab in paediatric participants 5 to < 18 years of age with moderate to severe active Systemic Lupus Erythematosus (SLE).While on background standard of care therapy
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 3 Study Evaluating the Efficacy and Safety of Subcutaneous Anifrolumab in Adult Patients with Systemic Lupus Erythematosus
BLOSSOM: A Study of Anifrolumab in Pediatric Participants with Systemic Lupus Erythematosus

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