Compassionate use access to molibresib/GSK525762 for eligible participant with NUT Midline Carcinoma; indication is a seriously debilitating or life-threatening disease.
Investigational Product GSK525762 is an orally bioavailable small molecule that is a potent inhibitor of the binding of BET proteins to acetylated histones. Binding of GSK525762 induces squamous differentiation and inhibits proliferation of patient-derived NMC cell lines both in culture and in murine xenografts. Compassionate use access to molibresib is restricted to patients with confirmed diagnosis of NUT Midline Carcinoma.
molibresib 20 mg will be supplied for compassionate use for the identified individual subject.
1. Participants meeting all three of the following criteria will be eligible: - Diagnosis of NUT Midline Carcinoma based on ectopic expression of NUT protein as determined by IHC and/or detection of NUT gene translocation as determined by FISH. - there is no satisfactory alternative treatment; and - there is reason to believe that the benefit:risk ratio for molibresib in the indication is positive.
2. Written informed consent can be obtained from the participant or legally authorised representative.
3. Participant must be ≥16 years of age, at the time of signing the informed consent.
4. Demonstrate adequate organ function defined as :Hematological Absolute neutrophil count (ANC) ≥1.5 X 109/liter (L) ; Platelets ≥100 X 109/L ; Hemoglobin≥9.0 g/deciliter ; total bilirubin ≤ 1.5 x X ULN (upper limit of normal)(isolated bilirubin >1.5 X ULN is acceptable if bilirubin is fractionated and direct bilirubin
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