Study on the Safety and Effectiveness of Ibrutinib for Children and Young Adults with Chronic Graft Versus Host Disease

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What is this study about?

This clinical trial is focused on studying a condition known as Chronic Graft Versus Host Disease (cGVHD), which can occur after a stem cell transplant. The study is investigating the use of a medication called Ibrutinib, which is being tested in different forms such as a suspension for oral use, film-coated tablets, and hard capsules. The purpose of the study is to find the right dose and to assess the safety and effects of Ibrutinib in children and young adults with cGVHD.

The study is divided into two parts. In the first part, researchers aim to determine the appropriate dose of Ibrutinib for children aged 1 to under 12 years. In the second part, the focus is on understanding how the body processes the medication and its safety in participants aged 1 to under 22 years. Participants in the study may have moderate or severe cGVHD that has not responded to previous treatments. Some participants may also be newly diagnosed with cGVHD and require systemic immunosuppression, which is a treatment to reduce the activity of the immune system.

Throughout the study, participants will receive either Ibrutinib or a placebo. The study will monitor the response to the treatment over a period of time, including the duration of response and overall survival rate. Additionally, the study will observe the growth and development of pediatric participants. The trial is expected to continue until November 2025, with the aim of providing valuable information on the use of Ibrutinib in treating cGVHD in younger patients.

1 joining the study

Upon joining the study, the patient will be informed about the trial’s purpose and procedures. Consent will be obtained from the patient or their guardian.

2 initial assessment

An initial assessment will be conducted to evaluate the patient’s health status and eligibility. This includes reviewing the patient’s medical history and current condition related to chronic graft versus host disease (cGVHD).

3 medication administration

The patient will receive Ibrutinib and Prednisone as part of the treatment. Ibrutinib is administered orally in the form of film-coated tablets or capsules. Prednisone is also administered orally in tablet form.

The dosage and frequency of administration will be determined based on the patient’s specific needs and the study’s protocol. The treatment duration will be specified by the study guidelines.

4 monitoring and follow-up

Regular monitoring will be conducted to assess the patient’s response to the treatment and to ensure safety. This includes checking for any side effects or changes in the patient’s condition.

Follow-up visits will be scheduled to evaluate the effectiveness of the treatment and to make any necessary adjustments.

5 completion of the study

Upon completion of the study, a final assessment will be conducted to evaluate the overall outcomes and any long-term effects of the treatment.

The patient will be informed about the results and any further steps if needed.

Who Can Join the Study?

Participants must have moderate or severe chronic Graft Versus Host Disease (cGVHD), which means the condition is affecting them significantly.
Participants should have tried at least one other treatment for cGVHD that did not work.
For Part B, participants can also be newly diagnosed with moderate or severe cGVHD and need treatment that affects the whole body (systemic immunosuppression).
Newly diagnosed participants should not have had previous treatments for cGVHD, except for corticosteroids (a type of medication) taken within 72 hours before agreeing to join the study.
Participants may be taking other medications to prevent or treat a different condition called acute GVHD, but if they are taking prednisone (a type of corticosteroid), it must be at a low dose (0.5 mg per kg of body weight per day or less) when they join the study.
Participants must have a history of receiving a stem cell transplant from another person (allogeneic transplantation).
For Part A, participants must be between 1 and less than 12 years old. For Part B, participants must be between 1 and less than 22 years old.
Participants or their parents/guardians must provide written consent, which means they agree to join the study after understanding what it involves.
Participants or their parents/guardians must understand the purpose and risks of the study and agree to share health information as required by privacy laws.
Children who are able to understand should also agree to participate in the study.

Who Cannot Join the Study?

Patients who are not diagnosed with Chronic Graft Versus Host Disease (cGVHD) cannot participate. This is a condition that can occur after a bone marrow or stem cell transplant, where the donated cells attack the recipient’s body.
Patients who are younger than 1 year old or older than 22 years old are not eligible to join the study.
Patients who do not meet the specific health criteria set by the study cannot participate. These criteria are based on guidelines from the National Institutes of Health (NIH).
Patients who are not able to safely take the study medication, ibrutinib, due to other health conditions or medications they are taking, will be excluded.
Patients who are pregnant or breastfeeding cannot participate in the study.
Patients who are unable to follow the study procedures or attend the required visits will not be eligible.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Robert Debre University Hospital	Paris	France
Prsldndr Mgflfp Cspceq Umppkze	Utrecht	The Netherlands
Fkvyqwujiy Mowal	Monza	Italy
Fkxgvebss Pnaj Ld Iuobcpaplyvlb Brolevzeb Dzj Hbxdxlvo Uffifrfguppnw Lg Pzs	Madrid	Spain
Htemnskw Vnqj dahbooci	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
France	Not recruiting	30.11.2018
Germany	Not recruiting	30.11.2018
Italy	Not recruiting	30.11.2018
Spain	Not recruiting	30.11.2018
The Netherlands	Not recruiting	30.11.2018

Trial locations

Investigated drugs:

Ibrutinib is a medication being studied for its potential use in children with chronic graft versus host disease (cGVHD). This trial aims to find the right dose for children and to understand how the body processes the medication. The study also looks at the safety of using this medication in young patients.

Investigated diseases:

Chronic graft versus host disease

Chronic Graft Versus Host Disease (cGVHD) – This condition occurs when donor cells attack the recipient’s body after a stem cell or bone marrow transplant. It is a long-term complication that can affect various organs, including the skin, liver, and digestive tract. Symptoms may include skin rashes, dry eyes, and mouth, as well as liver dysfunction. The disease can progress slowly, with symptoms appearing months or even years after the transplant. Over time, it can lead to chronic inflammation and fibrosis in affected tissues. The severity and progression of symptoms can vary widely among individuals.

Trial ID:

2023-507330-24-00

Protocol code:

PCYC-1146-IM

NCT ID:

NCT03790332

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Safety and Effectiveness of Ibrutinib for Children and Young Adults with Chronic Graft Versus Host Disease

What is this study about?

Who Can Join the Study?

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