Study of Elenestinib for Patients with Indolent Systemic Mastocytosis

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What is this study about?

This clinical trial is focused on studying a condition called Indolent Systemic Mastocytosis, which is a type of disease where certain cells in the body, known as mast cells, grow uncontrollably and cause various symptoms. The trial will test a new treatment called ELENESTINIB, also known by its code name BLU-263. This medication is taken as a film-coated tablet and is being studied to see if it can help manage the symptoms of this condition.

The purpose of the study is to determine the right dose of ELENESTINIB for treating Indolent Systemic Mastocytosis. Participants in the study will be randomly assigned to receive either the medication or a placebo, which looks like the medication but does not contain the active ingredient. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure the results are unbiased. The study will last for several weeks, during which participants will take the medication or placebo and have regular check-ups to monitor their health and any changes in their symptoms.

Throughout the study, the safety and tolerability of ELENESTINIB will be closely monitored by checking for any side effects or changes in health indicators like vital signs and laboratory test results. The effectiveness of the treatment will be assessed by measuring changes in symptoms and other health markers. The study aims to provide valuable information on whether ELENESTINIB can be a safe and effective treatment option for people with Indolent Systemic Mastocytosis.

1 joining the study

Upon joining the study, the patient will be informed about the trial’s purpose and procedures. The patient must meet specific criteria, such as age and health conditions, to participate.

The patient will provide informed consent, acknowledging understanding of the study and agreeing to participate.

2 screening period

During the screening period, the patient’s health status and eligibility will be assessed. This includes confirming the diagnosis of indolent systemic mastocytosis (ISM) or smoldering systemic mastocytosis (SSM) through medical tests.

The patient must have stable symptom management for at least 14 days before starting the trial.

3 randomization and treatment assignment

The patient will be randomly assigned to receive either the study medication, elenestinib, or a placebo. This process is double-blind, meaning neither the patient nor the researchers know which treatment is being administered.

The medication is provided in the form of film-coated tablets for oral use.

4 treatment phase

The patient will take the assigned medication daily. The dosage and specific instructions will be provided by the study team.

The treatment phase is divided into parts, with assessments at various intervals to monitor the patient’s response and any side effects.

5 monitoring and assessments

Regular monitoring will occur throughout the study to evaluate the safety and effectiveness of the treatment. This includes checking vital signs, conducting laboratory tests, and completing symptom assessments.

The primary goal is to observe changes in symptoms and any adverse effects.

6 end of treatment and follow-up

At the end of the treatment period, the patient will undergo final assessments to determine the overall impact of the medication.

Follow-up visits may be scheduled to ensure the patient’s well-being and to gather additional data on long-term effects.

Who Can Join the Study?

Patient must be at least 16 years old at the time of signing the consent form. In some countries like France, Sweden, Germany, and Spain, patients must be at least 18 years old.
Patient must have a specific genetic marker called KIT D816V in their blood or bone marrow, or have a type of cell called CD25+ Mast cells in their bone marrow.
Patient must have symptoms that match those of mast cell activation, affecting at least two body systems. These symptoms can include skin flushing, fast heartbeat, fainting, low blood pressure, diarrhea, nausea, vomiting, and stomach cramps. They must also have a blood test result called sBT above 8 ng/mL, or have severe and repeated allergic reactions known as anaphylaxis.
Patient must have a confirmed diagnosis of SSM (Smoldering Systemic Mastocytosis) through a bone marrow biopsy reviewed by a central pathology team, following specific diagnostic criteria.
Patient must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2, which is a scale used to assess how well a patient can perform daily activities.
Patient must have moderate-to-severe symptoms based on a symptom score from a specific assessment form over a 14-day period.
Patient must have a confirmed diagnosis of ISM (Indolent Systemic Mastocytosis) through a bone marrow biopsy reviewed by a central pathology team, following specific diagnostic criteria.
Patient must have tried and not achieved adequate symptom control with at least two different types of treatments, such as antihistamines, acid reducers, or other medications.
Patient must have their current symptom management stabilized for at least 14 days before starting the screening process.
If the patient is taking corticosteroids, the dose must be 20 mg or less per day of prednisone or an equivalent medication, and the dose must be stable for at least 14 days.
Patient must have a condition called mMCAS (mast cell activation syndrome) confirmed by a bone marrow biopsy reviewed by a central pathology team.
Patient must have a blood test result called tryptase that is less than 20 ng/mL.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Azienda Ospedaliero Universitaria Careggi	Florence	Italy
Oslo Universitetssykehus HF	Oslo	Norway
Hopital Necker Enfants Malades	Paris	France
University Hospital Maastricht	Maastricht	The Netherlands
Centro Hospitalar Universitario Sao Joao E.P.E.	Porto	Portugal
Oncopole Claudius Regaud	Toulouse	France
Katholieke Universiteit te Leuven	Leuven	Belgium
CHU Grenoble Alpes	La Tronche	France

Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)	Munich	Germany
Centre Hospitalier Universitaire De Poitiers	Poitiers	France
Universitair Ziekenhuis Gent	Gent	Belgium
Universitaetsklinikum Aachen AöR	Aachen	Germany
Geniko Nosokomeio Thessalonikis George Papanikolaou	Thessaloniki	Greece
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d’Aragona	Salerno	Italy
Unidade Local De Saude De Matosinhos E.P.E.	Senhora Da Hora	Portugal
Universitaetsklinikum Schleswig-Holstein AöR	Kiel	Germany
Odense University Hospital	Odense	Denmark
Universitaetsklinikum Erlangen AöR	Erlangen	Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Fondazione IRCCS Policlinico San Matteo	Pavia	Italy
Universita’ Degli Studi Di Verona	Verona	Italy
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Fakultni Nemocnice Brno	Brno	Czechia
Karolinska University Hospital	Solna	Sweden
Universitaetsklinikum Mannheim GmbH	Mannheim	Germany
Fakultni Nemocnice Kralovske Vinohrady	Prague	Czechia
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie	Cracow	Poland
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre hospitalier universitaire de Tivoli Institut medical des Mutualites socialistes	La Louviere	Belgium
Centre Hospitalier Universitaire Amiens Picardie	Amiens	France
Centro Hospitalar Universitario De Santo Antonio E.P.E.	Porto	Portugal
Hopital Beaujon	Clichy	France
Centre Hospitalier Universitaire De Caen Normandie	Caen	France
Kepler Universitätsklinikum Linz – Med Campus III	Linz	Austria
Uuniftsrts Mquoovu Cooioc Hllrysgrknbghbxau	Hamburg	Germany
Upqhpnmepscb Mijrqbi Cihdpbb Guaokpree	Groningen	The Netherlands
Cnjt Ukuojrtblo Hhhqvifu	Cork	Ireland
Czteim Hllpgirvwvs Ev Usbmnlzkcimqz Dh Lrpjjzj	Limoges	France
Upzljza Umwurruubz Hyowejaa	Uppsala	Sweden
Bisjwifs Usgstedauz Hiyxeicm Crszxe	Besançon	France
Evkyfwl Uqsqmlgbrirx Msjedlz Cgiitwn Rebbcbgwp (ypvalwm Mio	Rotterdam	The Netherlands
Kuzxmrun dyj Uzzmsvuquaii Mhhgfuqs All	Munich	Germany
Arsqzsu Uzygc Sjipvyhme Lvzhyo De Beoordx	Bologna	Italy
Ufcfdbomjcfqtf Ctrqmbd Kacgaupsy	Gdansk	Poland
Uciccqrtiz Ou Atcybjg	Edegem	Belgium
Hcduhzlg Vxrt djumjoso	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Austria	Recruiting	06.01.2022
Belgium	Recruiting	06.01.2022
Czechia	Recruiting	06.01.2022
Denmark	Recruiting	06.01.2022
France	Recruiting	06.01.2022
Germany	Recruiting	06.01.2022
Greece	Recruiting	06.01.2022
Ireland	Recruiting	06.01.2022
Italy	Recruiting	06.01.2022
Norway	Recruiting	06.01.2022
Poland	Recruiting	06.01.2022
Portugal	Recruiting	06.01.2022
Spain	Recruiting	06.01.2022
Sweden	Recruiting	06.01.2022
The Netherlands	Recruiting	06.01.2022

Trial locations

Investigated drugs:

Elenestinib Phosphate

BLU-263 is a medication being studied for its potential to treat indolent systemic mastocytosis. This condition involves an abnormal accumulation of mast cells in the body, which can lead to various symptoms. The trial aims to evaluate the effectiveness and safety of BLU-263 in managing these symptoms and improving the quality of life for patients with this condition.

Investigated diseases:

Indolent systemic mastocytosis

Indolent Systemic Mastocytosis – This is a condition where there is an abnormal accumulation of mast cells in various tissues of the body. These mast cells release substances that can cause symptoms like skin rashes, itching, and abdominal discomfort. The disease progresses slowly and is often characterized by mild symptoms. Over time, patients may experience more frequent or severe episodes of symptoms. The condition is considered a rare disease and can vary greatly in how it affects individuals.

Smoldering Systemic Mastocytosis – This is a subtype of systemic mastocytosis that is more advanced than indolent systemic mastocytosis but not as severe as aggressive forms. It involves a higher burden of mast cells in the body, which can lead to more pronounced symptoms. Patients may experience symptoms such as fatigue, bone pain, and gastrointestinal issues. The progression of the disease can lead to an increase in symptom severity and frequency. It is also classified as a rare disease and requires careful monitoring.

Trial ID:

2024-516728-32-00

Protocol code:

BLU-263-1201

NCT ID:

NCT04910685

Trial Phase:

Therapeutic use (Phase IV)

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Study of Elenestinib for Patients with Indolent Systemic Mastocytosis

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