Phase 2 Interstitial Lung Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Children and young people aged 180 days (about 6 months) up to under 22 years old at the time of joining the study
• People with a body surface area (a measure of body size based on height and weight) of at least 0.3 m²
• People who have been diagnosed with Langerhans cell histiocytosis (LCH) — a rare disease affecting certain immune cells — where the disease has come back, gotten worse, or has not responded to treatment, and where the disease can be measured on scans or tests
• People whose LCH diagnosis has been confirmed by a tissue sample (biopsy), either from the original diagnosis or from a later biopsy
• People whose LCH has a specific genetic change (mutation) detected in certain genes — including genes in the RAS, RAF, or related pathways, or in genes called CSFR1, ERBB3, or ALK — confirmed by an approved genetic test (note: a certain type of staining test called immunohistochemistry alone is not enough to confirm this)
• People who have already had at least one previous treatment for LCH that has not worked or has stopped working
• People who are able to take medication by mouth or through a feeding tube (such as a nasogastric, gastric, or jejunostomy tube), since the study medication comes as a liquid suspension or tablet only
• People who are considered well enough to take part, based on standard medical performance scales (scoring at least 50 on the Karnofsky or Lansky scale, depending on age)
• People whose blood cell counts meet minimum levels required by the trial (specific thresholds for white blood cells, platelets, and haemoglobin will be checked by the trial team)
• People whose kidney function meets the minimum levels required (checked through blood or urine tests — estimated kidney function tests alone are not accepted)
• People whose liver blood test results are within acceptable ranges for the trial (bilirubin, ALT, and albumin levels will be checked)
• People with liver disease caused by their LCH may still be considered even if some liver test results are abnormal, provided this is documented (confirm with trial site)
• People whose heart function meets the minimum required level, confirmed by a heart scan (echocardiogram or MUGA scan) within 28 days before joining
• People who do not have breathlessness at rest or difficulty with physical activity, and whose blood oxygen level is above 94% (unless breathing issues are caused by LCH in the lungs)
• People who have a seizure disorder that is well controlled may still be considered
• People living with HIV who are on effective treatment with an undetectable viral level in the 6 months before joining may be considered, provided their HIV medications do not interact with the study drug in a way that cannot be safely managed
• People who have had certain other previous treatments must have waited a minimum period before joining — for example, at least 14 days since chemotherapy or other cancer therapy, and at least 2 weeks since radiation therapy
• People must have fully recovered from any prior surgery and from side effects of previous treatments before joining
• People taking steroid medications may be considered if the dose is at or below a specified low level averaged over the month before joining
• All participants, and/or their parents or legal guardians, must sign a written consent form agreeing to take part

Who may not be able to join:

• People whose LCH occurs alongside another blood cancer (such as leukaemia), or who have a history of any non-histiocytic cancer
• People whose LCH affects only the skin
• People whose LCH affects only the gastrointestinal (digestive) tract in a way that can only be detected by endoscopic biopsy
• People with a condition called LCH-associated neurodegeneration (a type of nervous system complication of LCH) who do not also have other measurable disease in the brain tissue or elsewhere in the body
• People whose LCH has a specific genetic mutation called MAP2K1, as the study drug is not designed to target this mutation
• People who have severe, uncontrolled nausea and vomiting, a condition affecting how the gut absorbs food and medicine, or a bile drainage tube outside the body, if these would prevent the study medication from being properly absorbed
• People with an active, uncontrolled infection (bacterial, viral, or fungal)
• People who have had a major operation or serious physical injury within 14 days before joining, or who are expected to need major surgery during the study (minor procedures such as placement of a vascular access device are generally acceptable if the wound has healed)
• People who have had a significant portion of their bowel removed, or who have another condition that seriously affects how the gut absorbs nutrients and medications
• People with known serious eye conditions or known risk factors for certain eye problems (retinal vein occlusion or central serous retinopathy)
• People who have previously had a solid organ transplant or a bone marrow transplant
• People with significant active heart disease, a history of heart attack, blood clots in the legs or lungs within the past 6 months, ongoing heart muscle disease, or an abnormal heart electrical reading (QT interval greater than 440 ms)
• People with a history of a significant bleed in the brain (Grade 2 or higher), or any brain bleed within 28 days before joining
• People who have previously had a severe skin or immune reaction to medications (such as DRESS syndrome or Stevens-Johnson syndrome), or who are known to be allergic to the study drug or any of its ingredients
• People with a severely elevated level of a muscle enzyme called creatine kinase (CPK) causing significant symptoms
• People who are pregnant
• People who are breastfeeding and plan to continue doing so during the study
• People of reproductive age who are sexually active and have not agreed to use effective contraception for the duration of the study and for a specified period after the last dose of study medication (hormonal contraception is not permitted during treatment for people who can become pregnant; males must also use contraception and must not father a child during treatment and for 14 days after the last dose)
• People currently taking certain medications that strongly affect how the body processes the study drug (known as strong CYP2C8 inducers or inhibitors), or certain other medications with a narrow safe dose range (BCRP substrates), unless these can be safely stopped before and during the study

Important: Always verify eligibility with the trial site directly before applying.