A study of ruxolitinib as first treatment for children with haemophagocytic lymphohistiocytosis (HLH)

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What is this study about?

This study focuses on haemophagocytic lymphohistiocytosis (HLH), a severe condition affecting the immune system in children where the body produces too many activated immune cells. The study will evaluate the effectiveness of ruxolitinib (also known as Jakavi) when used as a first treatment option along with corticosteroids in children who have primary HLH.

The medication being tested, Jakavi, comes in tablet form in different strengths (5 mg, 10 mg, 15 mg, and 20 mg) and is taken by mouth. The purpose of this research is to determine how well patients survive until they can receive a Haematopoietic Stem Cell Transplantation, which is a procedure where damaged bone marrow is replaced with healthy cells.

The treatment period will last up to 8 weeks, during which patients will receive daily doses of the medication. The maximum daily dose that may be given is 100 mg, and the total amount of medication received during the entire study will not exceed 5,750 mg. This is a Phase 2 study that will help researchers understand how effective this treatment approach is for children with this serious condition.

1 Initial treatment phase

You will receive Jakavi (ruxolitinib) tablets in combination with corticosteroids as the first treatment for your condition

The medication will be taken by mouth (oral route)

Jakavi comes in different strengths: 5 mg, 10 mg, 15 mg, and 20 mg tablets

2 Treatment duration

The treatment will continue until you are ready for Hematopoietic Stem Cell Transplantation (HSCT), which is a procedure to replace damaged bone marrow with healthy cells

Your doctor will monitor your progress throughout the treatment period

3 Safety measures

If you are of childbearing age, you must use effective contraception during the study

Male participants must continue contraception for 90 days after the end of study

Female participants must continue contraception for 30 days after the end of study

4 Study completion

The study is expected to run until March 2028

Your participation will end when you proceed to stem cell transplantation or if other factors require discontinuation of treatment

Who Can Join the Study?

Age between 0 to 22 years
Must have confirmed HLH syndrome through either:
- Confirmed genetic diagnosis showing predisposition to primary HLH (including abnormal protein expression or family history), or
- At least 5 of these conditions:
  - Fever
  - Enlarged spleen
  - Low blood cell counts in at least two types of blood cells
  - High levels of fats in blood or low blood clotting proteins
  - Presence of certain cells consuming other blood cells
  - Decreased immune system function
  - High levels of ferritin (an iron-storing protein) in blood
  - Presence of activated immune cells
Must not have received any previous treatment specifically for HLH syndrome
If able to have children, must agree to use effective birth control during the study and:
- For males: continue for 90 days after study ends
- For females: continue for 30 days after study ends
Must provide written informed consent (parent/guardian consent for minors)
Must have social security coverage

Who Cannot Join the Study?

Patients who have already received treatment for haemophagocytic lymphohistiocytosis (HLH) other than corticosteroids
Patients younger than 2 years or older than 18 years of age
Patients with severe liver dysfunction (poor liver function that significantly affects daily activities)
Patients with active infections that are not controlled by treatment
Patients who have received organ transplants
Patients with known allergies to Ruxolitinib or similar medications
Patients with severe heart conditions that could affect their ability to participate safely
Pregnant or breastfeeding females
Patients currently participating in other clinical trials
Patients with significant kidney problems (poor kidney function that requires special medical care)
Patients unable to follow study procedures or attend scheduled visits
Patients with other serious medical conditions that could interfere with the study treatment

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Hopital Beaujon	Clichy	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	02.01.2025

Trial locations

Investigated drugs:

Ruxolitinib

Ruxolitinib is a medication that helps control the immune system’s response. It belongs to a group of drugs called JAK inhibitors. In this trial, it’s being tested as a first-line treatment for children with a rare condition affecting the immune system. The medication works by reducing inflammation and controlling overactive immune responses in the body.

Corticosteroids are anti-inflammatory medications that help reduce inflammation and suppress immune system activity. They are commonly used to treat various inflammatory conditions and autoimmune disorders. In this trial, they are used in combination with Ruxolitinib to help control the immune system’s response.

Investigated diseases:

Haemophagocytic lymphohistiocytosis

Haemophagocytic Lymphohistiocytosis (HLH) – A severe inflammatory condition where the immune system becomes overactive and damages the body’s tissues and organs. The condition occurs when certain white blood cells (histiocytes and lymphocytes) build up in organs like the liver, spleen, and bone marrow. These cells begin to destroy other blood cells, leading to organ enlargement and potential dysfunction. In children, the disease can develop rapidly, causing fever, enlarged liver and spleen, and decreased blood cell counts. HLH can be inherited (primary) or triggered by infections, autoimmune conditions, or other diseases (secondary).

Trial ID:

2024-516105-23-01

Trial Phase:

Therapeutic exploratory (Phase II)

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A study of ruxolitinib as first treatment for children with haemophagocytic lymphohistiocytosis (HLH)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?