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.
Drug: molibresib
molibresib 20 mg will be supplied for compassionate use for the identified individual subject.
Inclusion Criteria
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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