RecruitingPHASE3INTERVENTIONAL

Randomized Controlled Trial in Patients on Long-term Colchicine With Colchicine-resistant Familial Mediterranean Fever (FMF) to Evaluate the Efficacy of On-demand Anakinra Treatment for Painful Attacks in Patients Who Refuse Continuous Daily Therapy

This study is for people aged 6 and over with Familial Mediterranean Fever (FMF) whose symptoms aren't fully controlled by colchicine, even at the highest dose. It tests a treatment called Anakinra, which targets specific inflammation. Instead of taking Anakinra every day, this trial explores if taking it only when a painful FMF attack starts, until symptoms improve, works well. This is for people who prefer not to take daily injections. The study will compare this 'on-demand' Anakinra approach (alongside daily colchicine) with taking pain relief medicines when an attack occurs (also alongside daily colchicine). The aim is to find out if this focused treatment can effectively manage FMF flare-ups.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Assistance Publique - Hôpitaux de Paris

Enrolment target

Start

04 Jun 2024

Estimated completion

04 Dec 2026

FMF

What is this study about?

Familial Mediterranean Fever (FMF) is a lifelong condition that causes repeated episodes of fever, tummy pain, joint pain, or skin rashes. These are often called 'attacks' or 'flare-ups'. For many years, the main treatment has been a daily medicine called colchicine, which helps prevent these attacks. However, for about 1 to 2 out of every 10 people, colchicine doesn't fully control their FMF, and they continue to have painful flare-ups. These people are considered to have 'colchicine-resistant' FMF.

Recently, newer medicines called 'biologics' have become available for FMF, including one called Anakinra. These medicines work by calming down the part of the body's defence system that causes the inflammation in FMF. Usually, if recommended by a doctor, Anakinra is given as a daily injection. However, some people might not want to inject themselves every day, or they might have other concerns about long-term daily treatment.

This study is looking into a different way to use Anakinra for these difficult-to-treat FMF attacks. Instead of daily injections, it wants to see if taking Anakinra only when a painful FMF attack begins – and stopping once the attack has settled – can still be effective. This 'on-demand' approach could offer more flexibility. The trial will compare this on-demand Anakinra, alongside your usual daily colchicine, against taking standard pain relief medicines during an attack, also with daily colchicine. The goal is to find a good treatment option for those who struggle with ongoing FMF attacks and prefer not to take continuous daily injections.

Key takeaways

Tests 'on-demand' Anakinra for FMF attacks.
For those with FMF not fully controlled by colchicine.
Compares it to standard pain relief during attacks.
Aims for better control without daily biologic injections.
Participating means continuing daily colchicine.

Who may be eligible?

To be able to join this study, you must be at least 6 years old and have a confirmed diagnosis of FMF. Your FMF must also be considered 'colchicine-resistant', meaning you still get FMF attacks at least once a month, even though you're taking the highest dose of colchicine your doctor recommends.

Importantly, this study is specifically for people who don't want to take the daily injections of Anakinra as a continuous treatment, but are open to taking it just when an attack starts. You also shouldn't have any ongoing infections, especially tuberculosis, or a history of getting infections very often. If you are pregnant or breastfeeding, or are already taking other specific anti-inflammatory biologic medicines, you won't be able to join.

Your study doctor will carefully check if these and other specific health requirements are met to make sure the study is safe and right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 6 years old or older?
Do you have a confirmed diagnosis of FMF?
Do you still get FMF attacks at least once a month even with daily colchicine?
Are you unwilling to take daily biologic injections for FMF?
Are you not currently pregnant or breastfeeding?
Do you not have active tuberculosis or frequent severe infections?

Answer every question to see your result.

What does participation involve?

If you decide to participate, you will continue to take your daily colchicine as prescribed. You will then be randomly assigned to one of two groups: either the 'on-demand Anakinra' group or the 'pain relief' group. Your daily colchicine will continue in both cases. If you are in the 'on-demand Anakinra' group, you will take an Anakinra injection (100mg) once a day as soon as you feel an FMF attack starting. You will continue this daily injection until your symptoms are better, for a maximum of 7 days. If you are in the 'pain relief' group, you will take standard pain relief medicines as needed when an attack starts. The study will involve regular visits or check-ins with the study team to monitor your FMF attacks, how you're feeling, and any side effects. The total duration of your participation in the study will be decided by the study team, but generally, these types of studies run for several months.

Potential risks and benefits

Participating in this trial might offer you the potential benefit of better control over your FMF attacks, especially if your current treatment isn't fully effective. Taking Anakinra on demand could lead to fewer or less severe attacks, improving your quality of life. However, like all medicines, Anakinra can have side effects, such as reactions at the injection site or an increased risk of infections, although these are usually mild. The alternative group will use regular pain relief, which also carries potential side effects. It's important to weigh these carefully. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Service de Médecine interne Hopital Tenon
Verified postcode
Paris, France· Recruiting

Common questions

What is Familial Mediterranean Fever (FMF)?

FMF is a genetic condition that causes repeated episodes of inflammation, leading to fever, tummy pain, joint pain, or skin rashes.

What does 'colchicine-resistant' mean?

It means your FMF attacks aren't fully controlled by colchicine, even when you're taking the highest dose your doctor recommends.

What is Anakinra?

Anakinra is a medicine that helps calm down inflammation in your body. It's given by injection.

Why is this study looking at 'on-demand' Anakinra?

This study wants to see if taking Anakinra only when an FMF attack happens, rather than every day, is still effective for people who prefer not to have daily injections.

Do I need to take daily colchicine if I join the study?

Yes, you will continue to take your daily colchicine throughout the study, whichever group you are in.

How to find out more

Léa SAVEY

lea.savey@aphp.fr 0033 1 56 01 67 91 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.