Phase 2 Peripheral Neuropathy 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:

• The patient, parent, or guardian must have signed a consent form before any trial-related screening takes place
• People aged 2 to 40 years old at the time of signing consent
• People diagnosed with MCAP (a condition involving overgrowth and brain blood vessel changes) who also have neurodevelopmental difficulties, ranging from specific learning disorders to severe intellectual disability
• People with confirmed genetic testing results showing a specific mutation in the PIK3CA gene, done at a local laboratory using an approved DNA-based test
• People whose blood tests during screening show that their bone marrow, kidneys, liver, blood sugar, and other body systems are working at an adequate level (the trial site will check these results)
• People who are able to swallow the study medication in the appropriate form for their age — either as tablets, a drinkable liquid, or granules
• Women who could become pregnant must have a negative pregnancy test at the screening visit
• Male participants whose sexual partners are pregnant, possibly pregnant, or could become pregnant must agree to use condoms throughout the study and for one week after stopping the study drug
• For an optional part of the study only: people who have separately signed consent for a lumbar puncture (spinal fluid sample)

Who may not be able to join:

• People who have previously been treated with the study drug, alpelisib
• People who have a digestive or gut condition that could significantly affect how the study drug is absorbed (such as ulcerative diseases, uncontrolled nausea, vomiting, diarrhoea, malabsorption, or previous removal of part of the small bowel)
• People with uncontrolled diabetes (Type 1 or Type 2)
• People with a known allergy or hypersensitivity to alpelisib or similar drugs in its class (PI3K inhibitors)
• People with other severe or uncontrolled medical conditions that the treating doctor considers would make taking alpelisib unsafe (such as serious active infections, chronic active hepatitis, or severely reduced liver function)
• Female participants who could become pregnant, and male participants, who are not willing to use an approved, highly effective method of contraception (or remain abstinent) for the full duration of the study and for one week after stopping the study drug (confirm acceptable contraception methods with the trial site)
• People who have taken an mTOR or PI3K-AKT pathway inhibitor (a type of targeted medication) within one month before joining the trial
• People with a history of cancer in the past five years, with some specific exceptions for certain early-stage or fully treated cancers (confirm with trial site)
• People currently taking certain medications that strongly affect a liver enzyme called CYP3A4 or a protein called BCRP, if those medications cannot be stopped at least one week before screening
• People who have had major surgery, including debulking surgery, within three months before joining the trial
• People with a known history of severe skin reactions such as Stevens-Johnson syndrome, erythema multiforme, or toxic epidermal necrolysis
• People aged 6 and over who have documented lung inflammation (pneumonitis or interstitial lung disease) and significantly reduced lung function, where the cause is not related to their underlying MCAP-related condition
• Children aged 2 to 5 years who have documented or suspected lung inflammation based on MRI imaging
• People with a history of acute pancreatitis (sudden pancreas inflammation) within the past year, or a history of chronic pancreatitis
• People with significant heart conditions, including severe heart failure, uncontrolled irregular heart rhythms, long QT syndrome or a family history of it, an abnormal heart electrical reading at screening, or reduced kidney filtering function linked to heart assessment (confirm specific measurements with trial site)
• People currently enrolled in another interventional clinical trial, or who have been in one within the three months before joining
• People not affiliated with a national health insurance scheme (confirm with trial site, as this may be country-specific)
• Patients, parents, or legal representatives who are not able to give informed consent
• People who would be unable to attend all required trial visits
• For the optional lumbar puncture consent only: people with raised pressure in the skull, an infection at the puncture site, known blood clotting disorders, or a very low platelet count

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